Legendary biotech leader passes away, renewed tension around aducanumab, Merck's rare defeat, and more
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Legendary Gilead CEO dies
John C. Martin, who led Gilead to greatness in developing a hugely profitable HIV drug franchise, has died at the age of 69. Martin helmed the company for a 20-year stretch just six years after joining as VP of R&D back in 1990, during which Gilead built a rep as a fearsome competitor in HIV, fielding drug cocktails that kept the deadly AIDS at bay for millions — while also being targeted by critics for being tone deaf on pricing controversies. Daniel O’Day, who took over as Martin and his close colleague and successor John Milligan both left a couple of years ago, noted his legacy “will be felt for generations to come.”
Aducanumab drama escalates
The review of Biogen’s Alzheimer’s drug aducanumab has been anything but typical. And the tension is growing even more as three members of the FDA’s advisory committee — who have already offered their perspective in a meeting — laid out their case for why the agency should reject aducanumab in an op-ed in JAMA. The editorial brings the committee’s concerns back to the fore two months before the deadline for regulators to make a decision. “The FDA needs to get the science right,” an author told Endpoints News in an interview. “They usually do and I believe in this instance they will as well.”
Rare snub for Merck
After getting a unanimous thumbs down from the adcomm, Merck has been handed an unsurprising CRL over its application to market Keytruda for high-risk triple-negative breast cancer. The expert panel had voted no after the FDA cancer czar, Richard Pazdur, personally took the stage to admonish Merck for rushing the filing before the data were there to support it — a rare and stinging rebuke for a pharma giant used to cruising its PD-1 toward new destinations with grace.
At long last, a BCMA CAR-T
It took much longer than Bristol Myers Squibb had hoped, but it’s finally clinched an approval for ide-cel, the BCMA CAR-T therapy for multiple myeloma it got from Celgene. The drug, now dubbed Abecma, will sport a list price of $419,500. Once a closely-watched event because of its weight in a $6 billion CVR, the approval in multiple myeloma received a relatively muted welcome as it was largely expected, even though certain details on the label still surprised some analysts. While Bristol Myers can now claim the honor as the first company to have two approved CAR-Ts in its fold, rivals like J&J may be catching up soon.
Bristol Myers Squibb’s tax trouble
Bristol Myers Squibb could be on the hook for $1.4 billion in back taxes according to an accidental disclosure from the IRS, New York Times reported. The US government has battled Big Pharma for years over offshore patent transfer schemes. At the center of this particular case is Bristol Myers’ effort in 2012 to establish an Irish subsidiary as a vehicle to shuttle US drug patent rights over to a country with lower corporate tax rates. For its part, the company says it’s in compliance with applicable regulations and is working to resolve the matter.
Gene editing run-in
On the surface, Intellia was giving a pretty routine — if exciting — presentation at a Cold Spring Harbor Lab conference about a new approach to gene editing. There was just one problem: They didn’t credit or acknowledge the scientists behind the early papers detailing the first base editing systems, from which the CRISPR biotech said they have differentiated. It seemed “there was a calculated intent to present these as new,” said an attendee. Jason Mast spoke with David Liu and Alexis Komor, two key figures in the pioneering work, to explore why the pair feared that Intellia was rewriting history in a way that could be detrimental to young researchers.
Lilly sexual harassment
A month after ousting CFO Josh Smiley for inappropriate conduct, Eli Lilly is facing new allegations that an executive and a senior manager at its Washington, DC office sexually discriminated against and harassed multiple women. Sonya Elling, a Lilly lobbyist of 16 years, accused her supervisor Leigh Ann Pusey of creating a hostile environment and being a “workplace bully.” Shawn O’Neail, another supervisor hired by Pusey, allegedly made “sexually aggressive gestures” toward the employees.
BridgeBio is wrapping a busy, buzzy month with a $2.4 billion alliance to roll out their next drug in the US — infigratinib — which looks to be in the label-writing stage at the FDA as regulators set up a chip shot on a near-term approval. Teaming up with a well-connected and influential Swiss company, Neil Kumar is giving away some commercialization rights in exchange for Helsinn shouldering a chunk of global development costs as they look to score a series of downstream approvals. “It’s the better-owner hypothesis,” he tells John Carroll.
As the FDA outlines plans to update the disease progression models and how they’re used in drug development, Novartis, Genentech as well as BIO spelled out suggestions on what to focus on and raised questions on how the agency might use these models to inform the agency’s decision making.
- With the clock ticking on Amgen’s patents on blockbuster immunology meds, Bob Bradway is taking a far-looking bet on a new pathway to treat immune system diseases. Seattle-based private biotech Rodeo Therapeutics is getting $55 million upfront and $666 million in future milestones hinged largely on the success of small molecule drugs targeting an enzyme known as 15-PGDH.
- Already a major player in radiopharmaceuticals, Novartis is doubling down by acquiring rights to two new therapies from Switzerland’s iTheranostics. The drugs are designed to bind to fibroblast activation proteins on the surface of tumor cells and bring a radioactive particle there — shining a light on a new target just days after declaring a prostate cancer win.
- Merck is lining up a buyout deal to get its women’s health spinout started, setting aside up to $240 million to acquire Alydia Health, a medical device company working on technology to control and treat abnormal postpartum uterine bleeding or hemorrhage.
- On a spree for discovery deals, Takeda is taking a chance on a low-profile player that promises to deploy its covalent small molecule “fishing” platform against difficult targets, developed by a protégé of KRAS expert Kevan Shokat. The upfront and biobucks add up to $500 million over the next two years.
- In a continued campaign to crack down on big biopharma M&A, the FTC is trying to block Illumina’s proposed acquisition of Grail and its cancer-detecting blood test, for which the sequencing giant had agreed to pay $8 billion. Illumina vowed to “vigorously defend” its buyout — although it’s already lost to the antitrust watchdog once.
- Eli Casdin and Keith Meister have swooped in with a merger for their second SPAC, sending proteomics player SomaLogic to Nasdaq with a $1.23 billion valuation. Coming just three months after the company’s Series A extension, the company says the deal would allow it to double down on its suite of testing tools.
- As President Joe Biden unveiled his $2 trillion infrastructure plan, the White House also detailed measures to make it much harder for large multinationals — biopharma included — to avoid or evade taxes by using tax havens abroad.
- The Biden administration is in no rush to nominate a permanent commissioner, perfectly happy for Janet Woodcock to run the show in the acting role, Zach Brennan reports. But former FDA officials and commissioners are pushing the Biden administration to nominate someone quickly for the role. Here’s why.
- Former FDA commissioner Rob Califf called out the agency’s “unrecognized ability to badger sponsors” on matters of clinical trial diversity as a panel of experts agreed they could play a more prominent role, listing possible follow-up actions to ensure studies are representative.
- House Democrats have kicked off a process to officially overturn the Trump administration’s last-minute attempt to tear down more than 95% of the FDA’s regulations, calling it a “malicious rule” that would cause chaos. Meanwhile, the latest semi-annual regulatory agenda for HHS is conspicuously light on biopharma-related rules, either proposed or final, for the FDA.
- In an effort to ease the process around pediatric cancer drug development, the FDA and EMA are offering a new template to provide companies with an easier way to seek scientific advice from both agencies, featuring more closely aligned timelines for submissions.
- The FDA is putting its foot down on Makena, the pre-term birth drug marketed by AMAG Pharmaceuticals (recently acquired by Covis Pharma). A recently published meta-analysis is not changing their decision to pull the accelerated approval, the agency said in a public rebuke, after the confirmatory study failed to show a benefit.
- Vaulting ahead on a post-Martin Shkreli turnaround, Humanigen announced that its antibody helped keep patients with Covid-19 off ventilation in a Phase III trial. Despite a data discrepancy, the biotech says it’s filing a EUA as soon as possible, potentially giving doctors just their second proven tool for helping patients with severe disease.
- BioNTech, Pfizer’s relatively quiet German biotech partner for its Covid-19 vaccine, made noise with its earnings report as it projected about €10 billion ($11.5 billion) in vaccine revenue. The two companies have been racking up supply contracts, just as a new real-world study from the CDC confirms the high efficacy of mRNA vaccines from them and Moderna.
- As variants thwart the solo use of Eli Lilly’s Covid-19 antibody bamlanivimab, the pharma giant has declared interim success at pairing it with a rival treatment from GlaxoSmithKline and Vir. Just what the results on viral loads mean on the respective antibodies, though, remain up for interpretation.
- The Government Accountability Office concluded in a report that the US federal government’s contribution to research on Gilead’s antiviral remdesivir did not result in any patents, settling a longstanding dispute over whether the government can lower the drug’s price by claiming co-inventor rights.
- Although AstraZeneca’s Covid-19 vaccine is safe and effective, new data show that younger women are more frequently seeing the very rare cases of sometimes fatal blood clots when compared to the general population, the EMA says. More women received the vaccine than men, and regulators warn it’s premature to draw any conclusions.
- Oncology dominated the venture rounds announced this week, with a range of flavors on the menu: Genentech vets going head-to-head with Merck in I/O ($73 million); targeted therapies that don’t give in easily to tumor resistance ($106 million); a Flagship startup unleashing its epigenomic controllers against c-myc ($126 million); Pfizer’s ADC platform in the hands of an alum ($152 million), and so on. But neuromuscular player Entrada also squeezed in a $116 million crossover round, while Jennifer Doudna’s newest CRISPR startup nabbed $100 million and a vocal investor.
- The Jenner Institute, the Oxford group behind AstraZeneca’s Covid-19 vaccine, just got funding from CARB-X to pursue a different kind of vaccine for an infection that’s not quite as pressing, but nevertheless on the rise: gonorrhea.
- We now know where Mike Nally is headed after leaving the chief marketing post at Merck. Reportedly passed up for the top job at Merck, Nally will become Flagship Pioneering’s latest CEO-partner and take the helm at machine learning-focused Generate Biomedicines.
- Longtime Koch Institute director Tyler Jacks can finally wrap his 19-year tenure after locking in Matthew Vander Heiden, an MIT biology professor and cancer cell metabolism researcher, as his successor. Jacks’ next move? Return “full time to the excitement of the lab” while Vander Heiden steers the center toward machine learning and AI.
- Brian Kaspar is ready to leave the Zolgensma data manipulation scandal behind. The founding CSO of AveXis is taking a board role at Endsulin, a small gene therapy player, with an old buddy from the pioneering biotech. The goal? To take a shot at curing type 1 diabetes.
- Since the FDA slapped a clinical hold on its lead gene therapy in December, uniQure has been working with experts to sort out what led to the single liver cancer case causing concern. The biotech now says it’s “highly unlikely” that its hemophilia B drug was to blame — with “multiple analyses” they will now take to regulators.
- Overflowing with ideas to shift the cell therapy paradigm, Rick Klausner’s Lyell has spun out some of its protein engineering research into a new biotech called Outpace Bio, with a goal to “make step change in how we can program cells to be more efficacious,” Max Gelman reports.
- Wave’s closely-watched antisense oligonucleotide program became the latest casualty in the crusade against Huntington’s disease, forcing the biotech to scrap two drug candidates and turn to a back-up approach — a second time.
- Danish biotech Orphazyme’s lead drug arimoclomol — a supposedly “pipeline in a product” molecule — has whiffed in a second Phase III rare disease trial. It couldn’t prevent progression of inclusion body myositis in a 150-person study. An FDA decision, though, still awaits for a different indication based on a secondary endpoint.
- Ionis says one of the mid-stage, wholly-owned antisense drugs for rare, non-neurological diseases that CEO Brett Monia boasted about at JP Morgan earlier this year has delivered a solid slate of positive Phase II results, potentially setting it up for a blockbuster showdown with Takeda.
- Sanofi has taken home a second approval for Sarclisa, the key CD38 drug that it’s taking into a major rivalry with J&J. It’s now OK’d for use in combination with Amgen’s Kyprolis and the steroid dexamethasone for adults with relapsed or refractory multiple myeloma who have received between one and three previous lines of treatment.
- In search of cash to finance the spinout of its Pfizer-partnered consumer health division, GlaxoSmithKline has made the unorthodox call to sell its royalty rights for Exelixis drugs Cabometyx and Cometriq for $342 upfront — and promises of up to $50 million more from Royalty Pharma.
- A booming market for inflammatory bowel disease drugs could be just the ticket for AbbVie’s new immunology launches to fill Humira’s shoes, according to SVB Leerink analyst Geoffrey Porges. But there’s plenty of meat left on the $32 billion bone.
- Putting a cap to one of the country’s biggest health scandals, a French court found pharma company Servier guilty of deception and manslaughter around its diabetes drug Mediator. France’s health medicines agency was fined alongside Servier.
- The US Office of Special Counsel has unveiled the details of a whistleblower complaint from a former FDA inspector who alleged numerous violations at a Merck manufacturing plant in North Carolina that’s now being readied to make the J&J Covid-19 vaccine. Among the allegations: the agency had learned of these issues as early as 2018 but failed to act on them.
- When — not if — a flu pandemic comes around, Sanofi wants to be prepared for it. The French drugmaker, which produces the world’s bestselling flu vaccine, is dropping more than $703 million to build a new manufacturing facility and increase the supply of Fluzone.
- With its own vaccine efforts lagging behind, GlaxoSmithKline has signed another deal with Novavax pledging fill-finish capacity for 60 million doses of the recombinant nanoparticle shots designated for the UK.
- Theories abound on why virtually all Alzheimer’s drugs of the past two decades have failed. Stuart Lipton, a leading dementia expert who helped develop the last FDA-approved Alzheimer’s drug, has proposed an alternative explanation: Perhaps the amyloid-clearing antibodies were “paradoxically inducing more inflammation in the brain.” His lab is working on a way to fix that.
- With the barriers of entry to traditional biotech hubs like Boston and San Francisco getting ever high, is it time for companies to take R&D homes elsewhere? A new report by JLL suggests so, with Seattle topping the list for growth in life sciences employment.
- With plenty of giants romping and stomping in the AI drug discovery landscape these days, startups must find a niche to break in. RA Capital is seeding one such biotech, using AI to learn physics and redoing algorithms in a way that founder Virginia Burger says will open up the notorious class of intrinsically disordered proteins.