The FDA is working to reapprove GSK’s long-dormant drug Wellcovorin (leucovorin) for cerebral folate deficiency, which the agency linked to “developmental delays with autistic features.” This immediately followed a much-anticipated press conference in which President Donald Trump, flanked by Health Secretary Robert F. Kennedy Jr and other healthcare administrators, linked the use of Tylenol during pregnancy to rising rates of autism.
Meanwhile, Pfizer woke us all up Monday with the news that it had acquired breakout obesity rockstar Metsera for $4.9B. The deal should pump new life into Pfizer’s portfolio, which over the last two years has suffered three discontinued assets. Bite-sized deals—or those at or below the $5 billion mark—have defined biopharma recently, with Roche picking up metabolic dysfunction-associated steatohepatitis biotech 89bio for a potential $3.5 billion last week and Novartis putting another $5.7 billion on the line with partner Monte Rosa Therapeutics in a second molecular glue agreement.
Another therapeutic space primed for M&A action is psychedelics. After AbbVie bought Gilgamesh Pharmaceuticals’ lead depression asset for $1.2B last month, BioSpace spoke sought opinions from experts on who might be next to take the plunge. A few potential names included Eli Lilly, Bristol Myers Squibb and Merck.
On the policy front, the CDC’s revamped vaccine advisory committee convened for their first meeting to discuss COVID-19, MMRV and hepatitis B vaccine schedules. Industry watchers who spoke to BioSpace commented on the “lack of knowledge” and dearth of previous experience on the committee. And while the advisors ultimately voted to change the schedule for the MMRV vaccine, it appears unlikely to significantly affect manufacturers’ bottom lines.
Finally, in rare disease, Stealth BioTherapeutics secured its long-sought approval for elamipretide—now Forzinity—in Barth syndrome—a disorder that would fall under the purview of the FDA’s new Rare Disease Evidence Principles framework for ultra rare diseases affecting less than 1,000 people in the U.S. And we said “Bye Bye Bluebird,” as the famed gene therapy biotech—which was recently bought out by two private equity firms—returned to its original moniker, Genetix Biotherapeutics.
Lastly, make sure to sign up for Biopharm Executive here for access to a special deep dive into China biopharma.
In this episode, Lori Ellis and Colin Zick spend a little time further discussing some of the points brought up in the Bioprocessing Summit last month. AI is a tool, a powerful one but a tool. Understanding this, they explore the connections between hammers, AI, The Planet of the Apes and monoliths.
In the end, it is all about compliance.
Host
Lori Ellis, Head of Insights, BioSpace
Guest
Colin Zick, Partner, Foley Hoag LLP
Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.
A global shift may be underway in biopharma as the White House prepares legislation that would would place restrictions on drugs brought to the U.S. from China, and pharma companies exit the U.K. in droves. President Donald Trump is reportedly writing an executive order that would clamp down on the pharmaceutical industry’s ability to buy new molecules from biotechs based in China, while Sanofi, Merck and more have canceled or suspended investments in the U.K. following a sizeable increase in a mandatory levy in the region.
In other business news, Novo Nordisk’s newly appointed CEO Maziar Mike Doustdar hit the ground running, cutting around 9,000 employees and informing those who remained that they would need to return to the office. Novo’s headcount had climbed 81% in five years as its revenue soared—and then fell. The obesity juggernaut has been a key presence at the European Association for the Study of Diabetes’ annual meeting this week, announcing that it would seek FDA approval for a high-dose formulation of Wegovy, and presenting new data for long-acting amylin analog cagrilintide. Meanwhile, Reuters reported that Eli Lilly’s orforglipron could potentially qualify for the FDA’s recently launched Commissioner’s Priority Voucher, which could see the oral obesity candidate approved this year.
The gene therapy space was hit with more bad news as Capsida Biotherapeutics reported the death of a patient being treated with its investigational gene therapy for epileptic disorders. This follows an unfortunate trend in 2025 that has also seen deaths attributed to Sarepta’s approved Duchenne muscular dystrophy gene therapy Elevidys and a monoclonal antibody used for lymphodepletion in a study of Allogene’s CAR T cell therapy cema-cell. According to a new analyst survey, however, doctors are still prescribing Elevidys to ambulatory patients.
Meanwhile, on the regulatory front, FDA Center for Drug Evaluation and Research Director George Tidmarsh reportedly told two separate groups last week that he “would like to get away” from advisory committee meetings, but quickly appeared to walk the comments back in a statement to Endpoints News.
Finally, in BioPharm Executive, BioSpace takes a deep dive into the FDA’s new crackdown on pharmaceutical drug ads, and spotlights Akeso CEO Michelle Xia who built the biotech from a $3 million angel fundraising round to its current $15 billion valuation.
The Make America Healthy Again Commission released itssecond report Tuesday, recommending, among other efforts, an investigation into a possible link between vaccines and the uptick in chronic disease. At a livestreamed MAHA commission meeting, Health Secretary Robert F. Kennedy painted a dire picture of the country’s health, saying the U.S. now has “the highest chronic disease burden of any country in the world.”
Looking back to last week, all eyes were on HealthSecretary Robert F. Kennedy Jr.’s appearance before the Senate Finance Committee. The combative showdown amounted to little more than political theater, according to industry watchers, with Kennedy accusing former CDC Director Susan Monarez of lying in an op-ed published in the Wall Street Journal about his alleged request that she approve vaccine advisors’ recommendations in advance of their meeting later this month.
Over at the FDA, BioSpace combed through the latest cache of publicized complete response letters (CRLs), including one for Lykos’ MDMA-based therapeutic for post-traumatic stress disorder. Going forward, the agency has promised to release CRLs in real time. The greater transparency could help companies spinning on a carousel of confusion caused by all the recent regulatory change. In other FDA news, we take a deep dive into new expert panels, which some commentators view as one-sided, and into the new rare disease approval framework, which one critic called “all wrapper and no gift.” And in the weight loss space, the FDA debuted a consumer “green list” for GLP-1 ingredients.
Meanwhile, at the World Conference on Lung Cancer inBarcelona, several data readouts caught our attention. In particular, Summit Therapeutics released disappointing data for its PD-L1/VEGFa bispecific antibody ivonescimab in Western populations—a finding some analysts said could have readthroughs to Bristol Myers Squibb/BioNTech’s first ever global data readout for its L1/VEGFa bispecific.
Finally, in Biopharm Executive this week, check out features on contingent value rights, which have been getting tacked on to biopharma deals more and more, Amgen’s pipeline beyond the obesity drug MariTide and Novartis’ recent deal spree, which included the $1.4B acquisition of Tourmaline on Tuesday.
In this episode, Mike Garrett, CEO of Taconic Biosciences, discusses how preclinical research companies are helping drug developers navigate the current challenging funding environment. He explains that investors now demand more robust translational data showing real potential to impact human health, rather than just basic in vitro results, forcing researchers to generate better evidence packages earlier in development.
This episode is presented in partnership with Taconic Biosciences.
Host
Lori Ellis, Head of Insights, BioSpace
Guest
Mike Garret, CEO, Taconic Biosciences
Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.
We returned from the Labor Day holiday to a spate of intriguing deals, including two that could surpass $2 billion: Vertex’s new pact with Enlaza for autoimmune disease—which the Casgevy maker hopes could ease conditioning for the sickle cell/beta thalassemia gene therapy—and Novartis’ agreement with Arrowhead for neurodegenerative diseases such as Parkinson’s.
But as we look ahead, Thursday’s Senate Finance Committee will be the focus this week, as Health and Human Services Secretary Robert F. Kennedy Jr. will answer questions after the ousting of CDC Director Susan Monarez. Her departure is reportedly linked to changes to the regulation of COVID-19 vaccines, for which the FDA last week issued restricted approvals to Moderna, Pfizer/BioNTech and Novavax and rescinded the emergency use authorizations. The next ACIP meeting—where COVID-19 vaccines will be on the agenda—is set for Sept. 18 and 19.
In the weight loss arena, Novo Nordisk presented results from a real-world study this weekend at the European Society of Cardiology Congress in Madrid showing that Wegovy cut the risk of heart attack, stroke or death by 57% compared to Eli Lilly’s tirzepatide in people with obesity and cardiovascular disease. The company also continues to throw money into the space, last week inking a $550 million deal with Replicate for RNA-based treatments for obesity and diabetes. Meanwhile, Lilly is dropping studies of one oral obesity candidate as another nears a regulatory filing. Finally, the FDA greenlit the first GLP-1 generic for obesity.
We also discuss reactions to the FDA’s new guidance on radiopharma drug development, four recent approvals for rare diseases, and everything you ever wanted to know about SPACs.
Eli Lilly posted data Tuesday from a second Phase III trial of its oral weight loss therapy orforglipron, providing the company with all it needs to head to the FDA with a new drug application. For more in-depth discussion on the oral weight-loss space, check out a special episode of The Weekly. And stay tuned to BioSpace for more unique coverage of this market as we learn which investigational assets will make it across the regulatory finish line and which will join the weight-loss wasteland.
On the business side of biopharma, AbbVie turned back to neuropsychiatric therapies this week, acquiring partner Gilgamesh Pharmaceuticals’ lead depression candidate for $1.2 billion. The deal is a display of resilience for AbbVie in the neuropsychiatric space as well, after the stunning failure last November of schizophrenia asset emraclidine—picked up in its nearly $9 billion acquisition of Cerevel Therapeutics. And is the cooler late summer weather thawing the IPO market? Neuropsych-focused LB Pharma revealed in an SEC filing on Friday that it plans to take the plunge, ending a six-month stalemate in biotech IPOs.
Finally, the Department of Health and Human Services (HHS) has been busy this past week. On Monday, the CDC named Retsef Levi—a known vaccine critic—to head the agency’s COVID-19 immunization working group, just as reports surfaced that the Trump administration could be “within months” of banning the COVID-19 vaccine in the U.S. Meanwhile, the FDA issued new draft guidance for cancer drug developers that prioritizes the use of survival data, and the agency began publishing drug-related adverse event reports daily.
HHS itself has also been the subject of recent headlines, announcing that it will no longer recognize employee unions, and last week, hundreds of HHS staffers penned an open letter calling on Secretary Kennedy to tone down what they called “dangerous and deceitful statements” that have fostered distrust against federal health workers, exposing them to physical harm. This followed the Aug. 8 shooting at CDC headquarters in Atlanta.
Finally, multinational pharmaceutical companies spent more than $48 billion on partnerships with China in the first half of 2025 alone, according to a new report from IQVIA—more than in all of 2024.
Oral therapies are projected to account for 25% of the anti-obesity medication market by 2030—but first returns have largely disappointed. This month, shares of both Eli Lilly and Viking Therapeutics took a hit as investors reacted negatively to highly anticipated Phase III and Phase II results for their respective candidates.
While the 9.1% placebo-adjusted weight loss generated by Lilly’s orforglipron over 72 weeks was an efficacy miss by most analyst accounts, tolerability tripped up oral VK2735’s otherwise best-case efficacy scenario—10.9% weight loss after just 13 weeks. These murky results have left observers wondering, just how game-changing these pills will be and which ones will be most effective?
In such a hot space, Lilly’s and Viking’s results—which follow Novo Nordisk’s new drug application for an oral form of Wegovy in May—are only the tip of the iceberg. Stay tuned to BioSpace for further in-depth coverage of the space as we learn which investigational assets will make it across the regulatory finish line and which will join the weight-loss wasteland.
While AI represents a significant advancement in efficiency for early-stage drug discovery, it won't dramatically change the 90% clinical trial failure rate. Most failures stem from fundamental gaps in biological understanding rather then the processes where AI is able to have the most impact.
The discussion highlights AI's strengths and ability to reduce preclinical costs. However, they caution that AI faces significant limitations in predicting complex biological properties like toxicity due to insufficient data, and regulatory acceptance of AI-only safety assessments remains unlikely, meaning traditional clinical trials will continue to be necessary.
This episode is presented in partnership with Cresset.
Host
Lori Ellis, Head of Insights, BioSpace
Guests
Mutlu Dogruel, VP of AI, Cresset
Mark Mackey, CSO, Cresset
Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.
Three months after taking heat over an error-riddled first report, Health Secretary Robert F. Kennedy’s Make America Healthy Again Commission was due to submit a new strategy report to President Donald Trump last week. While the report is delayed—for disputed reasons— Politico obtained a draft copy, which homes in on creating a new vaccine framework and streamlining access to investigational drugs, specifically for children. The new strategy also touches on pharma lobbying, with plans to address alleged conflicts of interest within HHS—which, incidentally, a new JAMA study found have dropped dramatically over the past 25 years. In other HHS news, Kennedy is reviving the Task Force on Safer Childhood Vaccines—which has been dormant for 30 years, and the secretary voiced his support of mRNA vaccines for cancer.
Meanwhile, weight loss continues to coral the headlines. Viking Therapeutics reported greater than 12% weight loss after 13 weeks for its obesity pill VK2735, but tolerability tanked the biotech’s stock. As Viking, Eli Lilly and Novo Nordisk begin to report data from these oral options, manufacturing comes into focus, and Novo’s pending approval of an oral form of Wegovy specifically puts the spotlight on the variability of high-dose peptides.
Speaking of Novo, the Danish pharma appears to be sucking in some oxygen after a difficult run. Last week, the FDA approved Wegovy as just the second drug for metabolic dysfunction-associated steatohepatitis (MASH), and on Monday, Novo teamed up with GoodRx to offer Wegovy and Ozempic and $499 per month if patients skip insurance. Not to be outdone, fellow weight loss warrior Lilly announced a $1.3 billion partnership with AI/ML startup Superluminal Medicines to advance new small molecules for cardiometabolic diseases and obesity. Lilly has also been active on the policy front, saying that it will soon announce higher prices for its drugs in markets like Europe in an effort to “align prices across developed countries.”
Always a contentious topic, pricing plays a key role in why the pharmaceutical industry fails to command the same respect as steelworkers or other all-American pursuits. Also in BioPharm Executive this week, BioSpace takes a deep dive into how the Trump administration is using Most Favored Nation pricing to target pharma companies and pursue a broader trade war.
In this Denatured discussion, the conversation revolves around unpacking the patent policy changes that could make or break biotech companies. Guests Aaron Cummings and Anne Li of Brownstein Hyatt Farber Schreck highlight critical issues posed by the Patent Office including recent changes to the Inter Partes Review (IPR) such as discretionary denial and a proposed patent property tax.
Cummings and Li also discuss how biopharma can work with the administration as changes are being proposed and evaluated and stress the importance of individual advocacy and engagement.
Host
Lori Ellis, Head of Insights, BioSpace
Guests
Aaron Cummings
Anne Elise Herold Li, Shareholder, Brownstein Hyatt Farber Schreck
Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.
Vinay Prasad is back at the FDA as chief of the FDA’s Center for Biologics Evaluation and Research. Prasad’s return—which hit the news wires Saturday morning—came just 10 days after his unexpected exit on July 29, following blowback over the saga involving Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy Elevidys, and a campaign by conversative personalities to oust the outspoken physician.
Elsewhere in the government, Gray Delany, former head of HHS’ and President Donald Trump’s Make America Healthy Again agenda, has been fired—days after Health and Human Services Secretary Robert F. Kennedy Jr. axed 22 mRNA vaccine contracts under the Biomedical Advanced Research and Development Authority. Delany’s ouster came after he allegedly butted heads with other agency officials over how announcements were made, according to reporting from Endpoints News. The mRNA contract cuts, meanwhile, have sparked criticism from the scientific community and concern that the growing anti-mRNA sentiment could also have a negative effect on research for cancer therapeutics.
In somewhat related news, the Annals of Internal Medicine is refusing to retract a large-scale study it ran in July that pointed to the lack of an association between childhood aluminum exposure through vaccination and chronic conditions, despite Kennedy’s criticism. In an op-ed published earlier this month, the Health Secretary took issue with the study’s design.
In obesity news, biopharma darling Eli Lilly suffered a rare chink in its normally impenetrable GLP-1 armor. The first Phase III trial for oral weight loss therapy orforglipron read out last week, and the results were underwhelming, at least to Wall Street. Truist Securities wrote that the data support approval but “leaves room for competition.” This is good news for a whole host of companies, including Roche, Viking, Terns and Rhythm Pharmaceuticals, who all have weight loss pills at various stages of development.
And in gene therapy, the hits just keep coming. The FDA has limited the use of bluebird bio’s gene therapy Skysona to patients with cerebral adrenoleukodystrophy for whom no other therapies or stem cell donors are available, due to a heightened risk of blood cancers. This follows safety issues in gene therapy trials experienced by Allogene, and, of course, Sarepta Therapeutics. This week in ClinicaSpace, Dan Samorodnitsky explored the future of AAV technology. And in BioPharm Executive, Annalee Armstrong sat down with Chris Anzalone, CEO of Arrowhead Pharmaceuticals—Sarepta’s key strategic partner—to learn how his company has been weathering the storm.
In this thought-provoking episode, Cresset Group’s CSO, Mark Mackey, and VP of AI, Mutlu Dogruel, dissect the emerging geopolitical battle for AI supremacy, revealing how China's DeepSeek model fundamentally disrupted the AI landscape by achieving GPT-4 level performance for just $6 million versus OpenAI's reported $100+ million investment.
The discussion exposes a critical divide in global AI governance: Europe's "regulatory fortress" approach with the AI Act creates strict but vague rules that leave companies navigating a complex maze, while the US employs a "Wild West" philosophy of sector-specific oversight and red-teaming strategies. In the race for global AI dominance, China is leaping ahead of the US and the EU.
This episode is presented in partnership with Cresset.
Host
Lori Ellis, Head of Insights, BioSpace
Guests
Mutlu Dogruel, VP of AI, Cresset
Mark Mackey, CSO, Cresset
Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.
Center for Drug Evaluation and Research Head George Tidmarsh will oversee the Center for Biologics Evaluation and Research on an acting basis after Vinay Prasad’s abrupt departure hours after the recording of last week’s episode of The Weekly. While the situation is being billed as temporary, rumors are swirling that structural changes may be afoot at the FDA with Commissioner Marty Makary looking for better drug, biologic alignment.
Meanwhile, the agency has come under criticism for another recent decision—one apparently driven by CDER’s Oncology Center of Excellence director Richard Pazdur: the rejection of Replimmune’s advanced melanoma drug, RP1. According to reporting by multiple outlets, Pazdur opposed the consensus opinion of CBER staff to approve the drug. The research team behind Replimmune’s Phase III study penned an open letter to the FDA on Friday responding to the issues outlined in the agency’s complete response letter. Meanwhile, the FDA’s Sarepta saga continues, highlighting “unprecedented” FDA leaks and a veritable communications disaster.
On the business side of biopharma, Q2 earnings continue to unfurl, with Pfizer, Vertex, BioNTech, Merck and Moderna all reporting this past week. Merck’s $3 billion savings push has claimed 6,000 jobs, contributing to a brutal July that saw the entire biopharma industry axe 7,900 employees, a 487% year-over-year increase, based on BioSpace tallies. Meanwhile, Pfizer CEO Albert Bourla confirmed he has been in direct contact with President Donald Trump to negotiate a path forward on Most Favored Nation drug pricing after the president sent letters to 17 Big Pharma companies—and posted on his Truth Social platform—asking them to comply with the policy within 60 days or face potential unspecified consequences.
In other policy news, the Centers for Medicare and Medicaid Services is reportedly considering coverage of GLP-1 drugs for weight management and obesity—reviving a Biden era proposal the Trump admin scrapped earlier this year.
Finally, in BioPharm Executive this week, we have a special report on the situation in China as international drugmakers swoop into the region to find new drug candidates, while other companies build their therapeutic farm systems from incubators and venture arms. And check out BioSpace’s brand new Manufacturing Brief, where we bring you the latest news and analysis in the area of biopharma manufacturing, starting with a feature on how to make cell and gene therapies commercially viable.
While AI excels at repetitive tasks like triaging medical information calls, document retrieval, and adverse event detection, the industry must address valid concerns from physicians (41% are excited but concerned, according to AMA studies) and patients who distrust AI systems.
In this episode, IQVIA’s Louise Molloy advocates for complete transparency, including clear disclaimers when AI generates or supports responses, arguing that the industry serves as "custodians, not owners" of patient data with ethical responsibilities.
This episode is presented in partnership with IQVIA.
Host
Lori Ellis, Head of Insights, BioSpace
Guest
Louise Molloy, Associate Director Medical Information & Pharmacovigilance, IQVIA
Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.
The Sarepta saga continued into another week as the FDA recommended that the voluntary hold on the company’s Duchenne muscular dystrophy gene therapy be lifted for ambulatory patients, after determining that the death of an 8-year-old Brazilian Duchenne patient who had received Elevidys’ was not caused by the drug. Sarepta’s stock has swung wildly and its transparency questioned after it elected not to reveal the death of a third patient—a participant in a trial of a gene therapy for limb girdle muscular dystrophy—during a business update last week.
Speaking of entities—or individuals—who have trouble staying out of the news, Health Secretary Robert F. Kennedy Jr. plans to dissolve the U.S. Preventive Services Task Force because it is “too woke.” Also on Monday, Kennedy addressed what he called the “broken” vaccine injury compensation program. Without offering details, he vowed to “fix” the U.S.’s VICP and return it to its “original Congressional intent.”
On the business side of the biopharma house, Q2 earnings are in full swing, with AstraZeneca announcing estimate-beating numbers and CEO Pascal Soriot saying the world “needs to share” in global pharma R&D, while Merck cut $3 billion to support an aggressive launch schedule. Meanwhile, a week ahead of its own earnings report, Novo Nordisk named a new CEO and lowered its 2025 sales guidance for the second time this year.
In clinical development, the Alzheimer’s Association Annual Conference is underway in Toronto, with Roche’s trontinemab the standout so far. In a Phase Ib/IIa trial, the next-gen anti-amyloid antibody rapidly cleared amyloid from the brains of patients with Alzheimer’s disease after just seven months—besting the 18-month timeframe for Biogen and Eisai’s Leqembi and Eli Lilly’s Kisunla. While Leqembi and Kisunla have shown some progress is slowing down the progression of Alzheimer’s, their effect size is modest and they don’t work for all patients—leaving plenty of room for symptomatic treatments, such as those being developed by Bristol Myers Squibb and Acadia Pharmaceuticals. The space is gearing up for several readouts, for both symptomatic and disease-modifying therapies alike.
And in BioPharm Executive this week, we dig into the top VC rounds so far this year and highlight a few scrappy biotechs walking the solo road.
Just a few weeks ago, it seemed like Sarepta had weathered a spate of bad news, after two patients died from liver injuries from its Duchenne muscular dystrophy gene therapy Elevidys. Then came news of a third patient death. Last Wednesday, the company announced a major restructuring and 500-person layoff.
Then, in just a few days time, Sarepta Therapeutics went from enjoying a notable stock bump in response to that corporate update to its lowest price in nearly 10 years as it halts shipments of Elevidys. In addition to requesting the shipment hold, the FDA revoked the company’s technology platform designation and paused all clinical trials for Sarepta’s limb-girdle muscular dystrophy (LGMD) gene therapy. The turmoil was set in motion by media reports that a patient who received the LGMD treatment had died—a fact the company chose not to disclose during an investor call.
In other news, the FDA’s Center for Drug Evaluation and Research gets a new director in biotech veteran George Tidmarsh, also an adjunct professor of pediatrics and neonatology at Stanford University’s School of Medicine. Tidmarsh enters the agency at a time of mass layoffs as well as voluntary departures.
Meanwhile, Replimmune and Roche suffer FDA rejections as therapies from Otsuka/Lundbeck and GSK fail to earn adcomm support, as the bar for acceptable controls and demonstrations of efficacy continue to change under FDA commissioner Marty Makary and CBER director Vinay Prasad.
Finally, Big Pharmas continue to pump billions into U.S. manufacturing, with Biogen and AstraZeneca joining the list of companies to have made such pledges, pledging $2 billion and $50 billion, respectively. These latest announcements come as President Donald Trump reiterates that pharma-specific tariffs of up to 200% could come as soon as Aug. 1.
This episode continues the discussion regarding the rapid evolution of mRNA technologies since COVID-19. Guests discuss the improvements that have occurred within just a few years, which are making these therapies more reliable, cost-effective and viable for personalized cancer, rare disease chronic disease treatment.
Today’s episode is sponsored by Eclipsebio. From AI-ready datasets to sequencing validation, they drive RNA success.
Explore their solutions at https://eclipsebio.com/.
Host
Lori Ellis, Head of Insights, BioSpace
Guests
Andy Geall, Co-founder and Chief Development Officer, Replicate Bioscience; Chair of the Board, Alliance for mRNA Medicines
Pad Chivukula, Co-founder, CSO & COO, Arcturus Therapeutics
Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.
This episode focuses on how AI is transforming pharmacovigilance (PV) on a global scale, particularly focusing on the evolving role of local qualified persons for pharmacovigilance in the EU.
This episode is presented in partnership with IQVIA.
Host
Lori Ellis, Head of Insights, BioSpace
Guest
Ana Pedro Jesuíno, Global Head Local QPPV Network, IQVIA
Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.
Up to 3,500 FDA staffers received their final walking papers Monday after the U.S. Supreme Court found last week that the government is “likely to succeed” in arguing that its overhaul of HHS is “lawful.” Meanwhile, FDA Commissioner Marty Makary floated policy changes for the agency, including a proposal to lower prescription drug user fees for the next iteration of the program, and one to offer speedier reviews to companies willing to lower the cost of their drugs.
Last week, the regulator opened its cache of complete response letters (CRLs), offering transparency into the rationale behind more than 200 recent rejections for ultimately approved therapies, including those for Eli Lilly’s Alzheimer’s drug Kisunla and Sarepta’s Duchenne muscular dystrophy (DMD) treatment Vyondys 53. The FDA did not, however, release the CRLs for two new rejections: those of therapies from Ultragenyx and Capricor Therapeutics in Sanfilippo syndrome type A and cardiomyopathy associated with DMD, respectively. It was an especially rough week for Ultragenyx, which also, along with partner Mereo BioPharma, released seemingly negative Phase II/III data for their osteogenesis imperfecta therapy.
On a more positive note, two bustling therapeutic spaces continue to see positive data. In obesity, Hengrui Pharma’s Kailera Therapeutics–partnered dual GLP-1/GIP receptor agonist elicited 17.7% average weight loss in a pivotal Chinese trial. And the psychedelic therapeutics space is again generating excitement with two recent positive readouts in treatment-resistant depression. BioSpace took a deep dive into the market reaction to these readouts for Compass Pathways and Beckley Psytech and atai Life Sciences, and what exactly investors are looking for in a successful psychedelic therapy.
Finally, we examine the progress of AI biotech unicorns and kick off our series on women in biopharma with profiles on Mayo Venture Partner Audrey Greenberg and the all-female CEO/R&D tandem at Acadia Pharmaceuticals.
