BioSpace

Lori Ellis, head of insights at BioSpace, discusses some of the recent events and topics that are buzzing around BIO and DIA in 2025 with Rich Daly, CEO of Catalyst Pharmaceuticals, Peter Ronco, CEO of Emmes Corporation, and Phil Vanek, founder of Redline Bio Advisors. They address funding, the partnering market, AI, and also the recent FDA and CGT roundtable discussion.HostLori Ellis, Head of Insights, BioSpaceGuests⁠⁠Peter Ronco⁠⁠, CEO, Emmes⁠Phil Vanek⁠⁠, Founder, Redline Bio Advisors; Chief Commercial Officer, ISCT; Entrepreneur in Residence, Georgetown University School of MedicineRichard Daly, President & Chief Executive Officer, Catalyst PharmaceuticalsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

This week, BioSpace is at 50% power as Heather McKenzie and Jef Akst are off attending this year's BIO Conference in Boston. The half-team discusses this week's biggest news: the death of another patient who took Sarepta's Duchenne muscular dystrophy gene therapy Elevidys. The patient was a non-ambulatory teenager who experienced acute liver failure after receiving the gene therapy, which is the same cause of death for an Elevidys patient reported in March. Sarepta announced that it was halting treatments to non-ambulatory patients and on a media call discussed new steps in its therapeutic protocol for preventing further liver injuries.  Elsewhere, mergers and acquisitions are surging across biopharma. Eli Lilly picked up the gene editing company Verve Therapeutics for $1.3 billion, which helped bolster the gene editing space —particularly after Sarepta's report of the death. Supernus bought Sage Therapeutics for $795 million, five months after Sage rejected a smaller offer from Biogen.  BioNTech also got in on the dealmaking, buying its German rival—scientifically and in the courtroom—CureVac for about $1.25 billion. The deal seemed focused mostly on CureVac's early-stage cancer immunotherapy pipeline, but analysts were otherwise left scratching their heads on what BioNTech was getting for its money.  Last week on The Weekly the team discussed the sudden dismissal of the CDC's entire ACIP committee, and this week we have a new slate of members. The eight people replacing the 17 members that were removed last week include allies of HHS Secretary Robert F. Kennedy Jr., many of whom are vaccine skeptics who seem to share his skeptical view of vaccination in general.  

This week the BioSpace team was sent into an after-hours scramble by the news that Health and Human Services Secretary Robert F. Kennedy Jr. had fired the remaining members of the CDC's Advisory Committee on Immunization Practices. The secretary argued in an op-ed that accompanied the announcement that the “clean sweep” was necessary to “reestablish public confidence in vaccine science.” The move came amid heightening rhetoric from Kennedy about the safety of vaccines and sparked concerns that the Secretary will replace the committee with people who sympathize with his anti-vaccine positions. On the drug development front, Metsera once again made waves with new data for an amylin drug candidate showing weight loss of 8.4% at just 36 days. The drug could be a major competitor for Novo Nordisk's Cagrisema, which has failed to meet the sky-high expectations of investors despite being the leading amylin candidate in the space. And cell and gene therapy was back in the spotlight last week when the FDA held a round table discussion with industry leaders aimed at eliminating barriers to approval for these complex medicines. This support from the FDA came as Sarepta became the first company to receive a platform technology designation intended to streamline future gene therapy reviews and create predictability for new drug applications created using the same technologies.Over in Biopharm Executive, BioSpace looks at right of first negotiation deals in light of Sanofi's deal to buy Vigil Neuroscience. How often do these agreements turn into M&A? After some analysis, Jefferies found that a ROFN is not a golden ticket to a buyout.

In a dynamic regulatory environment, IQVIA's Michelle Gyzen suggests that AI may be the best and only way to keep with changes that are happening daily–and sometimes hourly.In this discussion Lori and Michelle touch on the governance frameworks for cybersecurity, risk, and how AI transformation and integration is evolving to help regulatory professionals navigate the speed and complexity of global requirements.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestMichelle Gyzen, Sr. Director, Strategic Regulatory Solutions; Head of Regulatory Services Innovation & Technology, IQVIA

The words of the week so far in biopharma are “deals” and “cancer”—or, more specifically, money being invested in cancer and other key therapeutic areas. With the American Society of Clinical Oncology's annual conference underway in Chicago, Bristol Myers Squibb got in the PD-1/PD-L1xVEGF game, paying potentially more than $11 billion to co-develop BioNTech's solid tumor bispecific BNT327. Elsewhere, Sanofi nabbed the year's second-biggest buyout, picking up Blueprint for $9.5 billion, expanding its rare disease portfolio. And Regeneron plunked down up to $2 billion to license a dual GLP-1/GIP receptor agonist from Chinese biopharma Hansoh Pharmaceuticals Group.  Back in Chicago, presentations by AstraZeneca, Gilead and Amgen drew rave reviews from investor analysts, while Pfizer and Arvinas elaborated on mixed data from a PROTAC that showed positive results in only a subsection of breast cancer patients, failing to impress Wall Street. Meanwhile, Bicara's solid survival stats in head and neck cancer weren't enough to clear the high bar set by rival Merus. At the meeting, BioSpace's own Dan Samorodnitsky sat down with Jazz Pharmaceuticals' CMO Rob Iannone to discuss the company's recently acquired pediatric glioma drug, and talked AI strategy with AstraZeneca's head of U.S. oncology for lung cancer Arun Krishna. Dan recaps his ASCO experience here.  Speaking of buzzy therapeutic spaces, there was more action on the vaccines front last week as Health and Human Services Secretary RFK Jr. announced that healthy children and healthy pregnant women would no longer be advised to get vaccinated against COVID-19. However, as of publication, the CDC still recommends a COVID vaccine for healthy children but instead of a universal recommendation advises that the decision should be made between parents and healthcare providers. Against this backdrop, the FDA signed off on Moderna's next-gen COVID-19 vaccine, mNEXSPIKE, for a limited population in line with its new guidelines. This was a much-needed win for Moderna, which last week had a $760 million-plus government contract for its mRNA-based bird flu vaccine terminated.  Also on the policy front, the Trump administration released its Make America Healthy Again report last week to much scrutiny after reports found studies and references that did not exist.  

The name of the biopharma game this season is vaccines—and RFK Jr. wasted no time returning from Memorial Day Weekend before making news on this front, removing the COVID-19 vaccine from the list of recommended immunizations for healthy kids and pregnant women on Tuesday. This follows a rash of recent moves, including a new risk-based strategy for the approval of new COVID vaccines focused on adults over 65 and high-risk individuals six months to 64 years of age and a request that Moderna and partners Pfizer and BioNTech update the myocarditis risk on their vaccines' labels.This increased vaccine scrutiny by the FDA and Department of Health and Human Services is having a significant impact on biopharma companies, several of whom have received stop-work orders on next-gen COVID vaccines. Meanwhile, Moderna last week pulled the biological license application for its combination COVID-19/flu vaccine, anticipating a request for additional data on flu shot efficacy from the FDA, as mRNA technology continues to be scrutinized after playing the hero during the pandemic. Also last week, the White House released its Make America Healthy Again (MAHA) report, which took aim at vaccines, along with pharma lobbying and GLP-1s.Also on the policy front, a California judge issued an order Thursday that indefinitely stops HHS' goal of reducing its divisions from 28 to 15 and firing upwards of 10,000 employees, among other reorganization and mass layoff plans. And speaking of government plays that could receive judicial pushback, we received a couple of new updates on the Most Favored Nation (MFN) front: first, President Donald Trump appointed his CMS chief Mehmet Oz as the leader of drug pricing negotiations, calling him “one tough hombre,” and second, HHS provided new guidance for streamlining the process for states to import drugs from Canada.In the obesity realm, Eli Lilly is calling out the Centers for Medicare and Medicaid Services. In an open letter last week, Lilly voiced its displeasure with the agency for a final ruling that left its Zepbound and Novo Nordisk's Wegovy out of Medicare Advantage and Part D coverage in 2026.And in the R&D realm, we returned from the long weekend to sad news from Rocket Pharmaceuticals, as the company reported that its pivotal Danon disease trial is on hold after the death of a young patient. The death—extremely sad on a human level—is also a setback for the gene therapy space, which had been buoyed earlier this month by the success of a personalized CRISPR treatment received by baby KJ.Finally, BioSpace looks ahead to ASCO#25 where Dan Samorodnitsky will be on the ground in Chicago.

Lori Ellis, head of insights and Ian Fisher, head of development of analytics at IQVIA, discusses the critical importance of Target Product Profiles (TPPs) for life sciences companies, especially during uncertain times with funding challenges. Fisher emphasizes that TPPs serve as strategic guiding light which help companies articulate their development goals and demonstrate value to potential investors and partners.This episode is presented in partnership with IQVIA.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestIan Fisher, Head of Development Analytics, IQVIA

Pfizer stole the headlines this week with a pact worth up to $6 billion with Chinese biotech 3SBio for a PD-1/VEGF candidate just three months after inking a clinical trials collab for a similar drug with Summit Therapeutics. It's the largest Chinese licensing deal in recent memory, as pharmas continue to turn to the country to fill their pipelines.  Also on Tuesday, the Department of Health and Human Services offered a smidge more detail on President Donald Trump's Most Favored Nation executive order. A press release explained that drug prices will be tied to the lowest price in certain countries with a GDP at least 60% that of the U.S. and that the effort will focus on branded drugs.   Over at the FDA, the strategy around COVID-19 vaccines is evolving. According to an editorial published in the New England Journal of Medicine Tuesday by FDA Commissioner Marty Makary and CBER director Vinay Prasad, future COVID-19 approvals will focus on adults over 65 and high-risk individuals six months to 64 years old—a strategy they say will better align the U.S. with other high-income nations. This is also in sync with the Novavax approval, which came in Friday after a few delays.   Meanwhile, the FDA's Oncologic Drugs Advisory Committee (ODAC) is currently in the midst of a two-day meeting, but acquiring the necessary expertise was “absolute chaos,” according to an agency insider who spoke with BioSpace. This is partly due to the decimation of an FDA office that includes staff responsible for screening scientific and therapeutic area experts for conflicts of interest.  Going back to last week—which seems like a year ago at this point—we were somewhat shocked to learn that the CEO of one of the world's most valuable pharma companies is on his way out. Friday morning, Novo Nordisk announced that CEO Lars Fruergaard Jørgensen will be leaving the company after eight years at the helm. After peaking last June at about $155 apiece, the obesity leader's shares are currently worth just $68. Novo said the decision for Jorgensen to leave was mutual, but Jorgensen was not made available to speak on a call following the announcement.  Finally, a couple of milestones are worth noting: First, the Alzheimer's space got a big win on Friday when the FDA approved the first blood-based test for the disease—news that could be a boon to Eli Lilly's Kisunla and Biogen & Eisai's Leqembi. And second, a nine-month-old boy named KJ with a disease called CPS1 deficiency that affects just 1 in 1.3 million U.S. babies was treated with a single-use CRISPR treatment created just for him. It's an incredible story that highlights just how far gene editing has come, but it also highlights a rare disease crisis, with these sorts of ultra- and nanorare diseases lacking the necessary financial incentive to motivate biopharma's focus.   

President Donald Trump unveiled a sweeping drug pricing policy this week, seeking to lower drug prices in the U.S. by up to 80% through a reprisal of the Most Favored Nation rule he attempted to introduce in his first term. The rule would essentially link U.S. prices to those paid in other nations where medications are cheaper. Biopharma reaction was one of tentative relief, with BMO Capital Markets analysts suggesting the executive order had “more bark than bite.” Meanwhile, the Centers for Medicare and Medicaid Services announced that among the next 15 drugs to undergo IRA-prescripted price negotiations could be drugs payable through Medicare Part B, and not just Part D, where the first two rounds have applied.  Into all of this action steps Vinay Prasad, the outspoken oncologist and hematologist who was named last week as the next director of the FDA's Center for Biologics Evaluation and Research. While the S&P Biotech ETF fell by more than 5% upon the news, overall reaction was fairly measured, with cell and gene therapy executive Audrey Greenberg summing up Prasad's selection as “anything but a status quo appointment.”  Over in the weight loss and obesity space, Eli Lilly can't seem to lose. This weekend, Lilly announced full data from a head-to-head trial showing a “superior benefit-to-risk ratio” for its Zepbound over Novo Nordisk's Wegovy. And last week, the Indiana-based pharma won a court battle against compounders when a judge sided with the FDA, stating that tirzepatide—the active ingredient in both Zepbound and diabetes sister drug Mounjaro—was no longer in shortage. Add on a presidential shoutout during Trump's Monday press conference for its U.S. manufacturing investments, and it really was Lilly's week.  Flying less high are some 2,000 Bayer employees who lost their jobs in the first quarter of 2025 as part of the company's new operating model, which is intended to make Bayer “much more agile.” On a less direct flight is Galapagos, which reversed course on plans to spin out a portion of the company and find a new CEO. Instead, CEO Paul Stoffels will make a quicker exit and the Belgian biotech could sell off its cell therapy assets as it looks to build up a new pipeline in house, having abandoned the spinout idea altogether. Stay tuned.  Finally, in ClinicaSpace this week, we took a deep dive into the HIV treatment space, where companies like Gilead and Immunocore are targeting a cure, while the Trump administration slashes funding for HIV research.  

Paul Offit, MD, Director of the Vaccine Education Center at the Children's Hospital of Philadelphia and the Maurice R. Hilleman Professor of Vaccinology, Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania, identifies himself as a "vaccine skeptic"–someone who demands data and evidence–which he believes is the appropriate stance for medical professionals and regulators.After the Cutter incident in 1955, which resulted in 250 cases of polio caused by batches of polio vaccine containing live polio virus given to the public, the FDA took up this mantle in evaluating vaccines. He warns that public discourse has moved dangerously past healthy skepticism into harmful cynicism, particularly through conspiracy theorists who dismiss scientific evidence by claiming researchers are "in the pocket of Big Pharma." This cynicism threatens public health as vaccine-preventable diseases like measles and pertussis are re-emerging due to declining vaccination rates, with polio potentially following if immunization continues to decrease.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestsPaul Offit, MD, Director of the Vaccine Education Center, Children's Hospital of Philadelphia; Maurice R. Hilleman Professor of Vaccinology and a Professor of Pediatrics, Perelman School of Medicine, University of PennsylvaniaDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

In his effort to onshore manufacturing, President Donald Trump issued an executive order on Monday afternoon ordering the FDA to ease permitting processes for new and expanded U.S. facilities. The announcement comes as more and more Big Pharma companies commit billions to expanding their U.S. footprints. Bristol Myers Squibb CEO Christopher Boerner announced this week that the company will pump $40 billion into its stateside operations over the next five years, even as the pharma executes a massive cost-cutting effort that involves shaving $3.5 billion from expenses by 2027 and cutting thousands of jobs, including another 516 in New Jersey, according to a May WARN notice.  In other policy news, the Department of Health and Human Services on Wednesday said it will require all new vaccines to be tested in placebo-controlled trials to earn FDA approval but some vaccine experts have raised concerns about this approach. Meanwhile, turmoil still envelopes the FDA, with staff cuts and rehires continuing at a dizzying pace. On Monday, several states sued HHS, saying that the cuts offload critical functions and costs onto the states and impede public health efforts.  As Q1 earnings season for Big Pharma begins to wind down, there are still headlines coming from the biotech sector. Vertex revealed last week that it is abandoning all of its adeno-associated virus vector work, while BioNTech on Monday announced that tariffs could get in the way of its ambitious plans for a closely watched PD-L1-VEGF therapy. Moderna, meanwhile, continues its fall from COVID grace, missing Q1 revenue expectations and announcing plans to reduce operating expenses by around $1.5 billion by 2027.   In the weight loss space, Novo Nordisk announced on Friday that the FDA has accepted the application for a pill version of Wegovy, with a decision expected this fall. Novo has also struck partnerships with CVS and Hims & Hers pharmacies to market injectable Wegovy, drawing the attention of Eli Lilly CEO David Ricks.   Also this week, check out BioSpace's deep dives into advances in base editing—a technology that's been touted as a “safer” CRISPR—and Summit Therapeutics' push to bring closely watched PD-1/VEGF immunotherapy ivonescimab to the U.S. market after its recent approval in China. 

Policy issues—particularly tariffs—loom large as Q1 2025 earnings season rolls on, with Pfizer , Novartis, AstraZeneca and many more all reporting this week. On Pfizer's call, CEO Albert Bourla called the Trump administration's national security concerns “legitimate,” but objected to the proposed tariffs in general. Meanwhile, Novartis CEO Vas Narasimhan brushed off the tariff risk but expressed concern over President Donald Trump's desire to bring back the ‘Most Favored Nations' rule.  Meanwhile, cancer conference season is in full swing, with the American Association for Cancer Research's annual event continuing in Chicago. Merck, GSK, Boehringer Ingelheim, Roche and others have presented highly anticipated data from some of their cornerstone cancer drugs and other candidates.  Turning to the latest news out of the FDA, newly minted Commissioner Marty Makary gave his first two big press interviews last week, making statements that both jibed with and contradicted recent reporting. And in the midst of the ongoing Novavax vaccine saga, many have raised concerns that the FDA could become politicized under the watch of HHS Secretary RFK Jr.  Finally, check out this week's BioPharm Executive stories, including a deep dive into the trend toward mining innovative therapies from China and BioSpace's annual report on pharma CEO-to-employee pay ratios. 

This discussion features BioSpace's head of insights Lori Ellis, Kearney partner Martin Hadosi, and Melissa Laitner, director of strategic initiatives at the National Academy of Medicine emphasizing the need for collaboration to improve women's health.This discussion features Lori Ellis, Martin Hadosi, and Melissa Laitner, who emphasize the need for collaborative effort across multiple sectors including industry, healthcare organizations, researchers, and patient advocacy groups. Regarding investment challenges, they acknowledge the current difficult economic environment affects all biomedical research, not just women's health specifically.This episode is presented in partnership with ⁠DIA⁠, in support of their ⁠2025 Global Annual Meeting⁠ taking place June 15-19 in Washington DC.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuests⁠Martin Hodosi⁠, Partner, Kearney⁠Melissa Laitner⁠, Director of Strategic Initiatives, National Academy of MedicineDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

While much of the country was off celebrating Easter and Passover this weekend, the FDA—and its new leader Marty Makary—were busy making news. Makary, who was confirmed as FDA commissioner last month, gave his first major interview, where he unveiled a new regulatory pathway for rare disease therapies and addressed the “epidemic of distrust” he feels is imperiling the agency. Makary also shared his thoughts on the conflicts of interest he sees between the FDA and pharma industry, highlighting a new directive to ban industry representatives from serving on the agency's advisory committees—a move experts believe will have minimal impact in reducing COIs.Makary also touched on vaccines and what he believes to be the cause of autism. On this, he was more restrained than his boss, HHS Secretary Robert F. Kennedy Jr., who continues to insist on re-investigating what he believes is a link between autism and vaccines. The interview comes as more former FDA officials sound the alarm about the changes happening within the agency.Elsewhere on the policy front, President Donald Trump continues to threaten tariffs on pharma products. In response, two more companies, Roche and Regeneron, are committing billions of dollars to enhance their U.S. footprints. They join Novartis, J&J and Eli Lilly in this regard, with the latter additionally promising to manufacture its investigational oral obesity drug orforglipron in the U.S.That announcement followed the news that orforglipron generated “injectable-like efficacy” in a Phase III diabetes trial. Lilly intends to submit for approval of the pill for weight management by the end of this year and diabetes in 2026. Not to be outdone, chief rival Novo Nordisk revealed Saturday that it has already filed for FDA approval of an oral formulation of its own blockbuster, semaglutide, for overweight and obesity.Finally, make sure to check out this week's edition of BioPharm Executive where we take a deep dive into another sizzling space—transthyretin amyloid cardiomyopathy (ATTR-CM)—and find out which pharma CEO has the biggest golden parachute.

It's all about tariffs again this week. No sooner had Donald Trump announced that “major” tariffs on the pharmaceutical industry would be coming “very shortly”—sending stocks spiraling—the president announced a 90-day pause on most taxes for imports from countries not named China. But this respite was not to last. On Sunday, Secretary of Commerce Howard Lutnick told ABC News that tariffs would be coming for pharma in the next month or two, according to Leerink.  A Federal Register Notice revealing an investigation into pharmaceutical product imports and the associated potential national security threats additionally signaled that tariffs might be imminent. Accordingly, tariffs were a major theme as Johnson & Johnson kicked off Q1 earnings season on Tuesday, where CEO Joaquin Duato urged President Donald Trump to consider tax policy changes instead. Last week, Novartis joined J&J and Eli Lilly in preparing for said tariffs with a $23 billion pledge to expand its own U.S. R&D and manufacturing. And amid all of these trade tensions, the National Security Commission on Emerging Biotechnology released a report warning that China is “dangerously close” to overtaking U.S. biotech.  Meanwhile, after unprecedented cuts to its workforce, the FDA is looking toward a future of drug review delays and increased executive oversight, according to experts. Industry watchers have also expressed concern that the cuts could trigger a little-known mechanism built into the Prescription Drug User Fee Act that could potentially cost the FDA nearly half of its funding and set the agency—and U.S. patients—back 35 years.  Also fighting for headlines on Monday morning was Pfizer's announcement that it is ending development of its lead obesity candidate danuglipron after detecting a potential case of drug-induced liver injury. Pfizer's loss could be Viking Therapeutics' gain, however, as analysts immediately floated the idea of a marriage between the two companies. Viking has long been a top takeover target, and the biotech's shares rose 13% upon Pfizer's announcement.  Finally, it's that time of year again: In BioPharm Executive this week, BioSpace looked at the top paid CEOs in biopharma. Who is this year's highest paid CEO? Click here to find out.  

In this bonus episode, BioSpace's vice president of marketing ⁠Chantal Dresner⁠ and careers editor ⁠Angela Gabriel⁠ take a look at job market performance in the first quarter of 2025. They discuss job posting trends, application rates and layoffs. Also discussed are recent decisions of the Trump administration and how they are impacting the workforces of the private sector, HHS and academia.Want to receive our latest quarterly job market reports? ⁠Subscribe⁠ to Career Insider for our job market updates, job trends, career advice and more.

This discussion features BioSpace's head of insights Lori Ellis, Kearney partner Martin Hadosi, and Melissa Laitner, director of strategic initiatives at the National Academy of Medicine examining the underrepresentation of women in clinical trials. They stress that rather than waiting for regulatory guidance, the industry should proactively improve trial accessibility and inclusivity, as there's mutual benefit in faster enrollment and more diverse participation.This episode is presented in partnership with ⁠DIA⁠, in support of their ⁠2025 Global Annual Meeting⁠ taking place June 15-19 in Washington DC.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuests⁠Martin Hodosi⁠, Partner, Kearney⁠Melissa Laitner⁠, Director of Strategic Initiatives, National Academy of MedicineDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Pushback against the Trump administration's massive government cuts exploded this weekend across America—and biopharma is no exception. Taking a deep dive into the leadership cuts, we found that more than half of the senior FDA leaders from six months ago are no longer there, and that's just the leadership. Across the Department of Health and Human Services, some 20,000 people will be leaving—of their own accord or otherwise—amounting to a 25% reduction in headcount that would leave HHS with around 62,000 employees. With many of these people likely destined for biopharma, does this pose an ethical problem?   Meanwhile, the escalating tariff war is beginning to ring alarm bells for the economy, with Goldman Sachs now predicting a 45% chance of a recession happening in the next 12 months. While pharma has historically been considered resistant to the challenges of a recession—people always need medicines—this time around could be different, in part because of the consumer-led obesity market. Thanks to the Trump administration's decision to nix a Biden era proposal to cover GLP-1s under Medicare Part D—at least for now—these drugs will continue to cost consumers a pretty penny.   Even without a recession being declared, Eli Lilly's and Novo Nordisk's stocks are falling significantly with the rest of the market. Lilly has lost more than $95 billion in market value in one month. Novo's value has declined $72 billion.   The drugmakers are also still battling makers of knockoff versions of their drugs. Most recently, Lilly filed a lawsuit against a med spa in Indianapolis for allegedly taking vials of tirzepatide and splitting them up into single doses that are sold to patients without the packaging inserts.  Of course, Lilly and Novo aren't the only pharma drug rivalry out there. There's also Keytruda vs. Opdivo, Leqembi vs. Kisunla and more.   Finally, we take a look at seven late-stage MASH candidates that could hit the market in the next few years, following the FDA's 2024 approval of Madrigal's Rezdiffra, the first treatment for the metabolic disorder. 

Biopharma's reaction to the forced resignation of venerable CBER director Peter Marks has been swift and furious, with former FDA Commissioner Robert Califf saying on LinkedIn that “the FDA as we've known it is finished.” Analysts, meanwhile, called Marks' exit “arguably biotech investors' greatest fear,” as company shares across the industry tumbled.   Marks' announcement added insult to injury for the agency, which was already reeling from the announced cuts of 10,000 employees across the Department of Health and Human Services, including 3,500 FDA staffers. Those layoffs began to roll out on Tuesday as some employees showed up to work only to discover they no longer had a job. Amid all this chaos, Cantor Fitzgerald analysts called for Kennedy himself to get the axe, saying in part that he was “undermining the trusted leadership of health care in this country.”    Despite the turmoil, drug development continues in the obesity space, with Novo Nordisk presenting mixed data from its semaglutide franchise at the American College of Cardiology's annual conference last weekend. While an oral version of the blockbuster drug showed cardiovascular benefits for some patients, it failed to change the trajectory of other major adverse cardiovascular events. Meanwhile, Novo continues its battle against compounding pharmacies manufacturing copycat versions of semaglutide—as multiple players scramble for a piece of this massive pie.    On the Alzheimer's front, Eli Lilly's Kisunla failed last week to win the recommendation of the EU's Committee for Medicinal Products for Human Use. This decision is consistent with CHMP's recent stance on anti-amyloid antibodies, as Biogen and Eisai have also struggled to get Leqembi approved in Europe.    On a positive note, pharma R&D returns grew again in 2024, but Deloitte warned that this progress is “fragile.” The firm urged companies to be bold and embrace cutting edge technology like gene therapy and AI. These returns can't help everyone, however, as the past week has seen an uptick in layoffs across biopharma, including at Carisma Therapeutics, Organon and Tenaya Therapeutics.   Finally, as April kicks off cancer conference season, BioSpace took a deep dive into the recent action in the always-hot in radiopharmaceuticals space.  

Martin Hodosi, partner at Kearney and Melissa Laitner, director of strategic initiatives, National Academy of Medicine, join Lori Ellis, head of insights, to discuss the evolution of women's inclusion in clinical trials. They note that while overall representation has improved, significant challenges remain. They highlight how industry mindset has evolved from being protectionist to inclusion.This episode is presented in partnership with DIA, in support of their 2025 Global Annual Meeting taking place June 15-19 in Washington DC.Host⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠, Head of Insights, BioSpaceGuestsMartin Hodosi, Partner, KearneyMelissa Laitner, Director of Strategic Initiatives, National Academy of MedicineDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

President Donald Trump doubled down on tariff threats targeting pharma, saying additional levies on pharmaceuticals will come “at some point,” per CNBC. Meanwhile, Johnson & Johnson became the latest big pharma to respond to Trump's warning of potential tariffs if companies don't reshore their manufacturing, announcing a massive $55 billion U.S. manufacturing and R&D investment. Not all companies are on board, however: AstraZeneca is looking eastward, pumping $2.5 billion into a new research facility in Beijing.    Also on the policy front, Trump nominated acting CDC director Susan Monarez for the top job after pulling his first nominee, Dave Weldon, days before his senate hearing was expected to begin. If confirmed, Monarez would be the first CDC director since 1953 to not have a medical degree; she holds a Ph.D. in microbiology and immunology from the University of Wisconsin.   In weight loss news, Novo Nordisk is paying China-based United Laboratories $200 million upfront to license a triple agonist of the GLP-1, GIP and glucagon receptors that could one day compete with Eli Lilly's retatrutide. And BioSpace examines the next great challenge for GLP-1s: oral formulation manufacturing.    Two more therapeutic spaces in focus last week are Duchenne muscular dystrophy and spinal muscular atrophy, where companies including Dyne Therapeutics, REGENXBIO and Novartis presented new data on their respective candidates. And the Duchenne community continued to react to news of the death of a patient taking Sarepta's approved gene therapy Elevidys.  In cardiovascular news, Alnylam won a much-anticipated approval for Amvuttra as the first RNAi silencer for transthyretin amyloid cardiomyopathy, setting up a three-way race with Pfizer's tafamidis—marketed as Vyndaqel and Vyndama—and BridgeBio's Attruby. Next up is Milestone Therapeutics' CARDAMYST in paroxysmal supraventricular tachycardia, which has a PDUFA date of March 27.   Finally, the saga of Cassava Sciences' Alzheimer's hopeful simufilam is over, as the company announced it has ended development of the controversial candidate.   

A patient with Duchenne muscular dystrophy taking Sarepta's gene therapy Elevidys has died of acute liver failure, possibly related to a recent viral infection. Sarepta, which said it will update Elevidys' label to reflect the new safety signal, saw its shares drop 22% on the news but analysts still seem positive on the drug, as treatment options for Duchenne remain limited.Meanwhile, both AstraZeneca and Taiho Pharmaceuticals announced acquisitions worth up to $1 billion or more in two sizzling therapeutic spaces, cell therapy and antibody-drug conjugates, respectively.Despite canceling a vaccine advisory committee late last month, the FDA on Thursday selected flu strains to be targeted in the upcoming 2025-2026 flu season. And at another federal agency, the Centers for Disease Control and Prevention, employees will have to wait a bit longer to see who will take the helm under Donald Trump, as the president's nominee, Dave Weldon, was pulled hours before he was set to appear before a Senate committee on Thursday. Like HHS Secretary Robert F. Kennedy Jr., Weldon has expressed anti-vaccine views in the past, particularly his continued suggestion of the link between vaccines and autism. Guggenheim Partners called the move to revoke Weldon's nomination “a positive sign for reigning in vaccine criticism.”In the weight loss arena, BioSpace takes deep dives into the tendency for biopharma to develop fast-followers, or me-too drugs—following a pattern seen with PD-1 checkpoint inhibitors after the approvals of Merck's Keytruda and Bristol Myers Squibb's Opdivo. One key difference between these two markets, however, is that when it comes to GLP-1s for weight loss, patients are not staying on these medicines. Drug developers are trying several approaches to improve treatment persistence, including titration, combinations and even secondary drugs that address side effects. They're also making other moves to differentiate themselves, including focusing on overall health outcomes—in areas like cardiovascular, sleep apnea and kidney disease.Following on BioSpace's coverage of the major patent cliffs that many Big Pharma companies are facing in coming years, we also take a look back at some of the companies that have already weathered such loss of exclusivity. It's rarely a straightforward story of sales crashing off patent, as companies take various tacks to extend their blockbuster sales.Finally, the cardiovascular space is expecting some movement this week. First, Alnylam is anticipating a decision on its RNAi silencer Amvuttra in ATTR-CM. An approval—which is widely expected—would make three companies on the market in this rapidly expanding space after Pfizer's tafamidis was approved in May of 2019, and BridgeBio's Attruby got the greenlight in November last year. And second, Milestone Pharmaceutical has a PDUFA coming up for etripamil in paroxysmal supraventricular tachycardia.

On the sidelines of this year's J.P. Morgan Healthcare Conference in San Francisco, BioSpace Senior Editor Annalee Armstrong sat down with Mark McKenna, CEO of Mirador Therapeutics, a member of BioSpace's NextGen Class of 2025. Their discussion here focused on the company's two-pronged approach to developing therapies for inflammatory and fibrotic diseases, as well as the importance of operating under stealth at this time for the biotech.This is the second episode in a special series of The Weekly focused on how NextGen companies are navigating the current business environment.

Miruna Sasu, CEO of COTA, has been labeled as a disrupter in the industry. In this episode, she discusses the challenges of stringent inclusion and exclusion criteria of clinical trials. She also dives into her main takeaways from this year's JP Morgan Healthcare Conference and Scope Summit around exits and investment in women's health.Host⁠⁠⁠Lori Ellis⁠⁠⁠, Head of Insights, BioSpaceGuests⁠Miruna Sasu⁠, President and CEO, COTADisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

As we mark five years since the World Health Organization officially declared the COVID-19 pandemic, the BioSpace editorial team reflects on the health crisis of a generation and how it changed us—and the biopharma industry. While companies like Pfizer, BioNTech and Moderna raked in billions from their vaccines, J&J and Novavax struggled to capture a significant piece of the market.   Five years later, much has changed. A substantial number of us now work from the comfort of our homes—though that may be changing for some in the life sciences—and biopharma has a new obsession: obesity. In this space, Novo Nordisk has had a rough week, reporting lower-than-expected results from a second straight trial of its next-gen weight loss drug CagriSema. And in an effort to protect revenues from its blockbuster weight-loss drug Wegovy, Novo jumped into the legal battle between the FDA and compounding pharmacies over the regulator's decision to declare the shortage of Wegovy over. Viking Therapeutics had a better week, securing “multiple metric tons” of its investigational obesity medication VK2735 in a deal with CordenPharma. Meanwhile, companies continue to tackle adverse events associated with GLP-1s.  Another space that has seen its fair share of failures in the past couple of years is depression. The latest flop comes from J&J, which announced it would discontinue its a kappa opioid receptor blocker aticaprant after a disappointing Phase III readout. J&J joins Biogen and Sage Therapeutics, Relmada Therapeutics and Alto Neuroscience, all of whom have suffered regulatory misses or trial flops. Finally, two of Donald Trump's healthcare nominees, Marty Makary and Jay Bhattacharya, sailed through their confirmation hearings in the Senate last week. Both are up for confirmation on Thursday.   

Donald Trump's tariffs have headlined myriad news stories this week—including at BioSpace, where we reported Pfizer CEO Albert Bourla's claim that his company is prepared to reshore manufacturing if the president makes good on threats made last month. Eli Lilly also appears to be preparing, commiting $27 billion to boost its U.S. manufacturing capacity.   Meanwhile, another regulatory meeting has been canceled under new HHS Secretary Robert F. Kennedy Jr. Reuters revealed last week that an upcoming meeting of the FDA's external advisers for vaccine policy on March 13 has been canceled—just a week after the CDC Vaccine Advisory Board's first meeting of 2025 was postponed. Also on the policy front, BioSpace took a deep dive into priority review vouchers (PRVs) after Congress failed to reauthorize the rare pediatric disease PRV program at the end of 2024. Our reporting shows this will be painful for many biopharma companies who rely on funds from the sale of PRVs.   Speaking of money, AbbVie and Eli Lilly struck a pair of mid-size deals in hot spaces. AbbVie made a late obesity play this week, inking a licensing deal worth up to $2.2 billion with service provider Gubra to bring a long-acting amylin drug to the market, while Lilly hopped onto the hot molecular glue train, paying more than $1.2 billion in a licensing deal with Magnet Biomedicine.  Finally, we examined the somewhat lethargic immuno-oncology space, which has companies, including BMS, Roche, Summit Therapeutics and BeiGene, targeting TIGIT, VEGF, RAS and more in their quest to bring the next Keytruda—which led the way in 2024 as the world's best-selling drug—to the market.  

On the sidelines of this year's J.P. Morgan Healthcare Conference in San Francisco, BioSpace Senior Editor Annalee Armstrong sat down with Kevin Marks, CEO of Delphia Therapeutics, a member of BioSpace's ⁠NextGen Class of 2025⁠. Their discussion here focused on the company's novel science of activation lethality, which involves forcing cancer cells to overactivate and thereby overload cell stress pathways, as well as Delphia's relatively smooth path to a $67 million series A raise thanks to contributions from Google Ventures (GV), where Marks was previously employed.This is the second episode in a special series of The Weekly focused on how NextGen companies are navigating the current business environment.Host⁠Annalee Armstrong⁠, Senior Editor, BioSpaceGuest⁠Kevin Marks⁠, President and CEO, Delphia TherapeuticsFor a complete list of NextGen 2025 companies, ⁠click here⁠.

Miruna Sasu, CEO of COTA, has been labeled as a disrupter in the industry. In this discussion, she highlights the challenges of investing in the life sciences industry. She also suggests how the investor mindset needs to change as well as offer solutions that benefit both investors and developers seeking investment.Host⁠⁠Lori Ellis⁠⁠, Head of Insights, BioSpaceGuestsMiruna Sasu, President and CEO, COTADisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

There was a high point this week for employees riding the rollercoaster that is currently the FDA, as 300 recently fired employees are reportedly being asked to return. This could serve to boost spirits at the agency, which is reeling from unpredictable and seemingly ‘indiscriminate' layoffs—and low morale as a result.   Trump made additional waves over the weekend, threatening to enact tariffs on the largest pharmaceutical companies—unless they relocate their manufacturing operations to the U.S. And the early impacts of the new administration continue to be felt, as the CDC's vaccine advisory board postponed its first meeting of 2025—just a week after RFK Jr.'s confirmation as HHS secretary.   In weight loss news, the Outsourcing Facilities Association (OFA), which represents compounders, is suing the FDA after the agency declared an end to the shortage of semaglutide, marketed by Novo Nordisk as Ozempic and Wegovy. This followed a similar OFA lawsuit last fall, filed after the FDA removed Eli Lilly's tirzepatide— Zepbound and Mounjaro—from its shortage list. In a possible attempt to protect Zepbound from compounder competition, Lilly announced Tuesday that it will expand single dose vials options of the blockbuster weight loss drug at a reduced price. This all comes as a new study compares physician wishlists with investment in the obesity space—and reveals key indicators for drug developers.  And it's been a good news/bad news kind of week in gene therapy, as Pfizer discontinued hemophilia B drug Beqvez worldwide, Regeneron reported more positive data from its gene therapy for genetic deafness and, facing a cash crunch, bluebird bio elected to go private in an acquisition by global investment firms Carlyle and SK Capital Partners.   Finally, let us leave you with a ghost story—the story of so-called “ghost assets” that have found a new home, and a new purpose.  

At the 2025 National Biotechnology Conference, gene therapies, bispecific antibodies and other novel modalities—relative newcomers to medicine—will be much discussed. In this curtain raiser, BioSpace speaks with conference chair Prathap Nagaraja Shastri of J&J about these highly anticipated topics. HostJef Akst, Managing Editor, BioSpaceGuestPrathap Nagaraja Shastri, Scientific Director and Group Leader, Clinical Pharmacology and Pharmacometrics, Johnson and JohnsonBioSpace is a media partner of the National Biotechnology Conference.

President Trump has been on a staff cutting rampage since taking office, and this weekend, the hammer came down on an undisclosed number of FDA employees. The firings—which reportedly affected staffers within the FDA's centers for food, medical devices and tobacco products—came days after Robert F. Kennedy Jr. was confirmed as HHS secretary.One space that could have an ally in RFK Jr. is psychedelic therapies, which the new HHS boss has accused the FDA of “suppressing,” along with stem cell therapies. On the flip side, Kennedy is a well-known vaccine critic, though his confirmation didn't affect the approvals of a pair of vaccines—one for GSK's meningococcal shot and another for Bavarian Nordic's chikungunya jab.One notable vaccine maker, however, is struggling. Moderna reported Q4 and 2024 earnings last week, revealing full-year revenue of just over $3 billion—less than half of the nearly $7 billion the company brought in the year before. Adding insult to financial injury, Moderna is also laying off around 50 employees on its digital team.Moderna isn't the only company facing challenges. Several biopharmas, including Merck, Regeneron and Bristol Myers Squibb are facing patent headwinds that, according to William Blair, are the strongest they've been in nearly 15 years. It's also a difficult climate in which to stay alive as a biotech, to which the 13 bankruptcies in 2024 can attest.Finally, BioSpace launched a new NextGen podcast series this week, in which senior editor Annalee Armstrong speaks with execs from four of our NextGen Class of 2025 companies—starting with COUR Pharmaceuticals.

On the sidelines of this year's J.P. Morgan Healthcare Conference in San Francisco, BioSpace Senior Editor Annalee Armstrong sat down with Dannielle Appelhans, CEO of COUR, a member of BioSpace's NextGen Class of 2025. Their discussion here, focused on the company's long history leading up to its $105 million in series A funding at the start of 2024 and the possibility of an initial public offering in the future, among other things, represents the first of a special series of The Weekly focused on how NextGen companies are navigating the current business environment. HostAnnalee Armstrong, Senior Editor, BioSpaceGuestDannielle Appelhans, CEO, COURFor a complete list of NextGen 2025 companies, click here. The NextGen Class of 2025 is brought to you by Pliancy.

BioSpace's Head of Insights Lori Ellis and Tom Whitehead, co-founder of the Emily Whitehead Foundation discuss the challenges navigated by cancer patients, weaving in their personal experiences with treatment and how they would encourage those undergoing treatment to speak up and advocate for themselves.This episode is presented by the ⁠Genscript Biotech Global Forum 2025.⁠Host⁠⁠Lori Ellis⁠⁠, Head of Insights, BioSpaceGuests⁠⁠Tom Whitehead,⁠⁠ Author, Keynote speaker; Co-Founder, Emily Whitehead FoundationDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

We saw more action on the M&A front this week as Novartisbought back its blood thinner abelacimab and the rest of Anthos Therapeutics for up to $3.1 billion and still ahead, a potential buyout of SpringWorks Therapeutics by Merck KGaA, which confirmed it's in “advanced discussions” with the biotech. Meanwhile, Eli Lilly signed aglobal licensing deal for a MASH asset with South Korea's OliX Pharmaceuticals.As Q4 and full-year 2024 earnings continue to roll in, BMS, Eli Lilly,Novo Nordisk, AstraZeneca, Eisai, Vertex and Gilead all reported their results in the past week. Notably, BMS continues to reorganize, now targeting an additional $2 billion in savings through 2027, and AstraZeneca axed two rare disease drugs from its $39 billion acquisition of Alexion. When it comes to revenue growth, Novo's Wegovy and Lilly's Zepbound continue to climb at a striking pace—a phenomenon that has at times driven the weight loss drugs into shortage and spawning a controversialshadow market of off-brand versions.Speaking of controversy, Regeneron is suing its Dupixent partner Sanofi, claiming the French pharma has failed to provide it with adequate information regarding sales of the blockbuster anti-inflammatory drug—which missed analyst expectations in Q4.On the clinical side, the pulmonary fibrosis space continues to see positive results, with Boehringer Ingelheim notching its second Phase III win in six months for nerandomilast. It wasn't all good news, however, as Pliant Therapeutics suspended dosing and enrollment in a Phase IIb/III study of its idiopathic pulmonary fibrosis candidate, causing its stock to crash.Finally, BioSpace released a special edition of ClinicaSpace this week focused on the resurgent cardiovascular space—just in time for Valentine's Day! Sign uphere to receive your copy.

U.S. political maneuverings are keeping biopharma on its toes. Tuesday, Robert F. Kennedy, Jr. passed a major hurdle as the Senate Finance Committee voted to advance his nomination for HHS secretary to a floor vote of the entire Senate. If confirmed, RFK Jr. could have an impact on myriad regulatory issues, including PDUFA negotiations. In other political news, Donald Trump announced his promised tariffs on Canada, Mexico and China—the last of which could have particular implications for biopharma companies looking to China for promising new drug candidates. Both were subjects of conversation as leading pharma companies—including Pfizer, Merck and Regeneron—reported their Q4 and full-year 2024 earnings. Notably, Pfizer is eyeing deals between $10 billion and $15 billion, while Merck took a 12% stock hit on Gardasil challenges in China and Regeneron issued its first-ever dividends. On the regulatory front, the FDA greenlit Vertex's Jounavx last week as the first new drug for acute pain in more than two decades. Journavx leads a non-opioid pain space that is seeing significant momentum this year, with Tris Pharma, Algiax Pharamceuticals and SiteOne Therapeutics all announcing positive data in the past month. Meanwhile, in obesity news, the U.K.'s pharmacies regulator rolled out stricter guidelines for online pharmacies selling medicines including Novo Nordisk's Wegovy and Eli Lilly's Mounjaro, and an obesity-focused deal inked with Novo last year wasn't enough to keep Omega Therapeutics afloat as the Massachusetts-based biotech revealed it is heading toward bankruptcy. Finally, a new CEO will be taking the reins at Takeda in 2026—but the selection of Julie Kim is an exception in an industry still struggling for gender parity at the top leadership ranks.

BioSpace's Head of Insights Lori Ellis and Tom Whitehead sit down and discuss cell and gene therapies from the perspectives of patients. What do caregivers and patients consider to be risks when lives are on the line? This episode is presented by the Genscript Biotech Global Forum 2025. Host ⁠Lori Ellis⁠, Head of Insights, BioSpace Guests ⁠Tom Whitehead,⁠ Author, Keynote speaker; Co-Founder, Emily Whitehead Foundation Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

In the week since Donald Trump took office, he's caused quite the stir with healthcare-centered moves that include ordering the Department of Health and Human Services to stop communications, hiring and travel and announcing that he would withdraw the U.S. from the World Health Organization. Wednesday, the U.S. Senate Finance Committee convenes to vote on the controversial nomination of Robert F. Kennedy, Jr. for health secretary—a vote that Jefferies analysts said they expect to be “close.” Biogen continues to grab headlines this month, as the latest chapter in the Sage saga saw the smaller biotech rejecting its neuro partner's unsolicited buyout offer. Meanwhile, Biogen laid off an undisclosed number of employees from its research unit, just as a higher dose of its Ionis-partnered spinal muscular atrophy therapy Spinraza was accepted for review by both the FDA and EMA. Elsewhere, the weight loss space continues to click on all cylinders, with Versant Ventures debuting its newest obesity biotech Helicore Biopharma on Tuesday with $65 million in series A funds, and two obesity-focused companies, Aardvark Therapeutics and Metsera, seeking entry to the public markets. These up-and-comers will have to compete against the likes of Eli Lilly and Novo Nordisk, the latter of which reported data last week showing that its next-gen obesity drug amycretin could elicit up to 22% weight-loss. And Veru announced that its enobosarm could significantly improve the quality of weight loss in seniors also taking Novo's Wegovy. Another busy therapeutic space is Duchenne muscular dystrophy, where analysts predict a lot of action in the next couple of years, with a number of data readouts and regulatory submissions. And finally, Annalee Armstrong caught up at JPM with Novavax CEO John Jacobs, who said the vaccine maker is at a pivot point.

Donald Trump was sworn in as the 47th president of the United States on Monday, bringing with him a host of healthcare nominees and potential changes to the FDA, M&A and drug pricing. The new administration was a key focus point at the 2025 J.P. Morgan Healthcare Conference last week, where Annalee Armstrong spoke to executives from several companies about what they're expecting from a second Trump term. Also at JPM, Mirador Therapeutics CEO Mark McKenna boldly predicted that 2025 would see the return of the megamerger. Speaking of M&A, Annalee spoke with leaders from Biogen, who declined to address the company's unsolicited takeover bid for Sage Therapeutics. For its part, Sage sued Biogen last week, “seeking preliminary injunctive relief to enforce a standstill agreement.” Meanwhile, Gilead CEO Daniel O'Day addressed the split of Galapagos—a company Gilead has poured more than $5 billion into since 2019. Check out more personal stories from JPM here. In the obesity space, Novo Nordisk reported data from a Phase III trial showing that a high dose of Wegovy elicited more weight loss than the approved regimen—but still fell short of results posted by Eli Lilly's Zepbound. Relatedly, Wegovy and sister drug Ozempic are both on the list of the next fifteen drugs whose prices could be negotiated under the Inflation Reduction Act. Finally, the FDA on Friday made its first high-profile decision of the year, greenlighting AstraZeneca and Daiichi Sankyo's Dato-DXd—now Datroway—to treat certain types of advanced breast cancer. The approval is the first for the highly touted antibody-drug conjugate, which is also under FDA review for EGFR-mutated non-small cell lung cacner. Analysts expect 2025 to be a pivotal year for the ADC.

In this bonus episode, BioSpace's vice president of marketing Chantal Dresner and careers editor Angela Gabriel take a look at Q4 job market performance and what we expect to see ahead. We dive into year-on-year data and recent job trends. Want to receive our latest quarterly job market reports? Subscribe to Career Insider for our job market updates, job trends, career advice and more.

In this episode of Denatured, Dr. Peter Marks M.D., PhD. gives his thoughts on the future of cell and gene therapies. He speaks candidly about opportunities and realities, manufacturing and its role in efficacy and the CGT runway.  Dr. Marks was keynote speaker at the GenScript Biotech Forum in San Francisco this week. Host ⁠Lori Ellis⁠, Head of Insights, BioSpace Guest ⁠Peter Marks M.D., PhD.⁠, Director, Center for Biologics Evaluation and Research (CBER)

The J.P. Morgan Healthcare Conference kicked off Monday with a flood of high-value deals, reinvigorating sentiment across the biopharma industry. Johnson & Johnson made the biggest splash, acquiring neurology leader Intra-Cellular Therapies for $14.6 billion, while GSK picked up precision therapy specialist IDRx for $1B upfront and Eli Lilly laid down up to $2.5 billion for Scorpion's PI3Kα inhibitor program. Meanwhile, the immunology and inflammation space continues to fire on all cylinders as Gilead invests up to $1.7 billion for LEO Pharma's preclinical oral small molecule STAT6 program. And those are only the deals accepted by both parties. Prior to the conference, Biogen offered to acquire its struggling neuro partner Sage Therapeutics for around $469M. The proposal follows a catastrophic run for Sage, which has seen its shares fall more than 90% in the past two years. 2024 sales and earnings forecasts have also generated attention this week, with Sarepta reporting that Duchenne muscular dystrophy gene therapy Elevidys beat analysts' expectations in the fourth quarter, and Eli Lilly projecting a full-year revenue miss driven largely by lower-than-expected sales of GLP-1 blockbusters Zepbound and Mounjaro. As expected, obesity has been a hot topic at JPM, with Pfizer CEO Albert Bourla announcing that his company is going “all in” in the space. This follows new FDA guidance revealed last week recommending a minimum weight loss threshold for drug developers. Among the many companies taking notice is newcomer Verdiva Bio, which launched last week with more than $410 million in opening funds. Also debuting last week was Kardigan, which raised $300 million to tackle heart disease. Kadigan joins a resurgent cardiovascular space, where several companies—including those developing gene therapies—are targeting myriad diseases. Finally, BioSpace senior editor Annalee Armstrong caught up with Daphne Zohar, CEO of BioSpace NextGen 2025 company Seaport Therapeutics Daphne Zohar, who offered her thoughts on the current state of the neuropsychiatric space.

TEASER: This episode is little sneak peak into upcoming podcasts as well as topics that will be discussed at the 2025 GenScript Biotech Global Forum on January 15 in San Francisco. Listen in as Peter Marks and Tom Whitehead share their thoughts on the CGT space for 2025. Register for the event here. Host Lori Ellis, Head of Insights, BioSpace Guests Peter Marks M.D., PhD., Director, Center for Biologics Evaluation and Research (CBER) Tom Whitehead, Author, Keynote speaker; Co-Founder, Emily Whitehead Foundation

Happy New Year! BioSpace released our NextGen: Class of 2025 this week, highlighting 25 biopharma startups to watch this year. The companies on this list are not afraid of a challenge, wading into some of biopharma's most competitive therapeutic spaces. As analysts predict an uptick in M&A in '25, could some of them be potential targets? One NextGen 2025 company that could already be fielding calls is Metsera, which reported promising Phase II data for its investigational subcutaneous GLP-1 therapy on Tuesday. The GLP-1 space continues to garner significant attention, with both Eli Lilly and Novo Nordisk seeking to protect their drugs against competition from compounders. This strategy makes a lot of sense as the sizzling class continues to expand into heart disease, sleep apnea and metabolic dysfunction-associated steatohepatitis, among other indications. On the regulatory side, the FDA approved 55 novel medicines in 2024—including a few inaugural company approvals—and is looking ahead to several key PDUFA dates in Q1, 2025. Meanwhile, the agency released three new guidance documents seeking to provide clarity around confirmatory trials supporting accelerated approvals, the use of AI in drug development and the use of tissue biopsies in clinical trials. Finally, BioSpace senior editor Annalee Armstrong heads to the J.P. Morgan Healthcare Conference next week as biopharma seeks an M&A spark to improve the currently a gloomy sentiment. Stay with us for all the biggest news coming out of JPM.

This conversation focused on a few remaining topics discussed at length at Jefferies late last year, with the same topics anticipated to make an appearance at JPM. In this episode, our guests the industry's need to catch up with women's health issues and the innovative lead the APAC region has taken in clinical trials. Host ⁠⁠Lori Ellis⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Miguel Forte⁠⁠, President, International Society for Cell & Gene Therapy; Board Member, ARM; CEO and Board Member, Kiji Therapeutics ⁠⁠Ali Pashazadeh,⁠⁠ Founder, Treehill Partners

2025 is set to change the industry. The new administration is poised to challenge many existing processes. The question is whether they will be based on data-driven analysis or ideologies and beliefs. Our guests address this concern along with the existing challenges CEOs face, particularly manufacturing processes. Additionally, they discuss weight loss drugs, focusing on GLP-1s, with medical precision. Host ⁠Lori Ellis⁠, Head of Insights, BioSpace Guests ⁠Miguel Forte⁠, President, International Society for Cell & Gene Therapy; Board Member, ARM; CEO and Board Member, Kiji Therapeutics ⁠Ali Pashazadeh,⁠ Founder, Treehill Partners

Note: BioSpace is taking a break for the holidays. The next episode of The Weekly will air Jan. 8, 2025. Happy New Year! Novo dominated headlines over the last week, with a kidney disease label expansion for Novo Nordisk's Ozempic in Europe but a newly flagged safety risk for the drug, with a Danish regulator requesting that the EMA review reports of a rare eye disease in patients who took the GLP-1. Meanwhile, over the weekend Novo Nordisk's parent company, Novo Holdings, and CDMO Catalent announced that they had received all regulatory clearances to close the proposed $16.5 billion acquisition. The deal, which will see Novo Holdings sell three sites to Novo Nordisk, is the biggest manufacturing investment this year but certainly not the only, with supply chain becoming a focus for biotech and Big Pharmas alike. Meanwhile, 2024 proved to be a snacky year for M&A activity and a tough environment for IPOs, with this year's newly public biotechs mostly seeing their stock prices plummet after entering the public markets. Moderna, while not new to the markets, has similarly seen its stock tank. President-elect Donald Trump's nomination of well-known anti-vaccine advocate Robert F. Kennedy Jr. to head the Department of Health and Human Services certainly didn't help. On the clinical research side of the industry, 2024 saw its fair share of flops and victories. One of the most striking in each category was the failure of AbbVie's emraclidine. The Big Pharma acquired the schizophrenia hopeful with its nearly $9B Cerevel buy late last year. The flops came not long after the approval of BMS' Cobenfy, which came out of the even-bigger purchase of Karuna and was the first new schizophrenia medication in more than three decades.

This week, GSK and Gilead and Arcellx presented key data at the American Society of Hematology (ASH) annual meeting as they vie for a competitive advantage in multiple myeloma. Meanwhile, Vertex unveiled positive long-term data for its CRISPR Therapeutics–partnered gene therapy Casgevy in sickle cell disease—results BMO Capital Markets analysts said should help Casgevy keep its edge over bluebird bio's Lyfgenia. On that note, Casgevy and Lyfgenia have a new outcomes-based payment model after the Centers for Medicare and Medicaid Service (CMS) said both companies have agreed to participate in a voluntary program to improve access to the gene therapies. Speaking of access, Eli Lilly and Novo Nordisk both announced significant manufacturing investments aimed at shoring up production of their diabetes and weight loss blockbusters tirzepatide and semaglutide. And in related news, the European Commission gave its blessing to Novo Holdings' controversial acquisition of contract manufacturing firm Catalent. Elsewhere, AbbVie got a much-needed win for Cerevel-acquired Parkinson's disease therapy tavapadon—a month after the deal's cornerstone asset emraclidine failed in schizophrenia—while uniQure announced it has aligned with the FDA on “key elements” of the accelerated approval pathway for its investigational gene therapy for Huntington's, AMT-130.

“Cautious optimism” is circling once again as the industry approaches 2025. However, many companies are treading water, barely holding on as the market continues at a slow pace. Ali Pashazadeh and Miguel Forte have an open discussion about the state of the market. Miguel Forte President International Society for Cell & Gene Therapy; ARM Board Member; CEO and Board Member of Kiji Therapeutics Ali Pashazadeh Founder at Treehill Partners

It's officially holiday shopping season, and biopharma is getting into the spirit. Novartis expanded its presence in Huntington's, paying up to $2.9 billion to advance PTC Therapeutics' Phase II candidate, while as Gilead committed up to $415 million to Tubulis to develop novel ADCs for solid tumors and Roche's Genentech inked an autoimmune development deal with COUR Pharmaceuticals potentially worth more than $900 million. Finally, Takeda put up to $1.3 billion on the line for Keros' blood cancer anemia drug, a potential competitor to Bristol Myers Squibb's Reblozyl. On the regulatory front, Applied Therapeutics' stock crashed more than 80% after the FDA last week denied approval of govorestat in the rare metabolic disorder classic galactosemia. Also last week, the FDA announced it is looking into the safety of bluebird bio's gene therapy Skysona after new reports of secondary blood cancers in treated patients. On a more positive note this week, Intra-Cellular Therapies submitted an application for Caplyta in major depressive disorder, potentially opening up an additional $1 billion in revenue. Finally, R&D investment in glioblastoma is ticking up as Merck, Kazia Therapeutics and Black Diamond Therapeutics look to advance treatments for the rare but devastating brain tumor.

Biopharma had its collective deal-making hat on heading into the Thanksgiving holiday, with Roche buying Poseida for up to $1.5 billion, and Sarepta and Arrowhead tying up in a collaboration potentially worth up to $10 billion. These announcements followed last week's news from Novartis, which snatched up Kate Therapeutics for a little over $1 billion while promising more acquisitions below $5 billion. On the regulatory front, the FDA gave BridgeBio—and transthyretin amyloid cardiomyopathy (ATTR-CM) patients—something to be thankful for late last week with the approval of Attruby for the rare, cardiovascular disease. The nod sets up a potential three-way race with Pfizer's tafamidis and Alnylam's Amvuttra, the latter of which was accepted for FDA review in ATTR-CM on Monday.   On the opposite end of the clinical development spectrum, Cassava Sciences' controversial Alzheimer's drug failed to reduce cognitive or functional decline in a Phase III trial. And investors were unimpressed by the 20% weight loss generated by Amgen's MariTide in a much-anticipated Phase II trial, as the company's stock tumbled 11% Tuesday. Drawing much attention from the biopharma industry, President-elect Donald Trump continued with his nominees for top healthcare posts, announcing COVID-19 critic Marty Makary to lead the FDA and well-known vaccine skeptic Dave Weldon for CDC director. Meanwhile, the FDA is grappling with the loss of Chevron Deference pertaining to a legal challenge over Eli Lilly's GLP-1 shortages. Finally, the next generation of antibody-drug conjugates remains hot, with Danish biotech Adcendo reeling in $135M in a Series B financing round.

Women represent 51% of the population; not investing is a poor investment strategy. Incorporating women into leadership positions, into board rooms, is the only way to tap this market. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠Konstantina Katcheves⁠, Senior Vice President, Innovative Medicines Global Business Development and Acquisitions, Teva Pharmaceuticals ⁠Sanskriti Thakur⁠, Chairwoman, TOWER Capital Group