Amid monkeypox fears, biotechs spring to action; Moderna’s CFO trouble; Cuts, cuts everywhere; Crafting the right proteins; and more
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It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.
Amid monkeypox fears, biotechs spring to action
With a rapid rise of monkeypox cases causing concerns around the world, vaccine procurement is weighing on the mind of government officials. Bavarian Nordic landed contracts with both the US’ BARDA and an unnamed European country to supply its smallpox vaccine, Jynneos. While the BARDA’s $119 million option has a longer term timeframe, the Europe purchase highlights an “emergency situation.” At the same time, Emergent BioSolutions acquired Tembexa, a smallpox treatment, from Chimerix for $225 million in cash. Experts reckon that smallpox vaccines should offer some protection against monkeypox, and previous vaccinations, coupled with the right public health response, should stem the current outbreak.
Crafting the right proteins
For decades, protein design was a biological borderland. But in the last few years, a series of breakthroughs has suddenly turned an academic curiosity into a viable way of developing new drugs, including ones that are more accurate, more complex, or can be developed faster than medicines made through other methods. In his final Endpoints In Focus, Jason Mast takes us inside the lab of David Baker — once among the field’s long rangers — at the University of Washington’s Institute for Protein Design to take a peek at how design-based approaches are transforming drug development.
Cuts, cuts everywhere
Mauled by the bear market, a multitude of biotechs continue to feel the heat — and the list might surprise you. Agios is shaking things up as 50 exploratory research employees will be let go and CSO Bruce Car will depart the company at the end of July. With pivotal data still years away, Scholar Rock, the darkhorse SMA player, plans to lay off 39 people while CMO Yung Chyung steps down. French biotech Nanobiotix, which IPO’d during the pandemic boom, is chopping preclinical research, amending some ongoing studies and moving some employees fully remote to stay afloat. San Francisco’s Applied Molecular Transport will cut its workforce by 40% on top rolling back programs, and among those leaving is its CSO. Small cap biotech Aquestive, meanwhile, didn’t provide a reason for why longtime CEO Keith Kendall is handing the baton to COO Daniel Barber.
Janet Woodcock has a new role
Longtime CDER director Janet Woodcock, who’s now serving as FDA commissioner Rob Califf’s principal deputy, would take on more of a role as “a strategic thought partner, with decision-making authorities” at several different offices within the agency, according to a new memo. Insiders said the announcement effectively means she won’t be returning to or stepping on the toes of anyone serving in the various roles she held previously.
Moderna’s CFO trouble
Days after Moderna sent new CFO Jorge Gomez packing — after just one day on the job — over an investigation launched by his former employer, the mRNA biotech said it was blindsided by the probe. Although dental equipment company Dentsply began investigating Gomez in early March, Moderna claimed it was unaware until the public disclosure on May 10. It decided to part ways with Gomez that same day. And Flagship’s Noubar Afeyan, who’s also chair of the Moderna board, defended the vetting and hiring process.
Toward more diverse clinical trials
While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report that suggests swaths of patients in minority groups continue to be left out. Pfizer is delving into the issue through a new partnership with Headlands Research that aims to initiate research sites in areas with diverse populations, while J&J launched its “Depression Looks Like Me” campaign in hopes of normalizing the discussion around depression — especially for people in the LGBTQ+ community.
- Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago.
- AbbVie is joining its fellow Big Pharma competitors and jumping into the Treg space. In a new collaboration, it will be teaming up with private biotech Cugene to advance a single Treg program targeting IL-2. AbbVie is shelling out $48.5 million upfront to collaborate and will pay an undisclosed sum of milestones and royalties.
- As Big Pharma slowly but surely warms up to the potential of digital therapeutics, Pfizer took another step in its partnership with Icelandic biotech Sidekick Health to launch a product specifically geared for atopic dermatitis. The two have co-launched other products before and will work together to figure out how best to offer patients “personalized support around care and management of symptoms.”
- All Blue’s bid to take over a badly battered Zymeworks — bolstered by its appointment of celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to the proposed board — officially turned hostile as Zymeworks’ board rejected the unsolicited, $733 million offer.
- Six years after Otsuka first signed a licensing and co-development agreement for vadadustat, Akebia’s drug candidate for renal anemia related to chronic kidney disease, the whole deal is off. Otsuka had originally nabbed co-commercialization rights in the US in addition to other large markets but decided to pull out in the wake of a complete response letter.
- Drawn in by a cell pouch device, Evotec is taking a minority stake as it strikes a partnership with Sernova in hopes of creating a functional cure for diabetes. While Sernova’s current therapy makes use of donor cells, it can now use Evotec’s stem cell-derived, insulin-producing beta cells to make a cell replacement therapy.
- Shionogi, an active player in the arid field of antimicrobials, has now set its eye on the adjacent sphere of antifungals. The Japan-based pharma is paying $100 million upfront to partner with F2G to push olorofim through clinical trials and regulatory action in Europe and Asia. The drug is being tested to treat a rare mold infection that can impact immuno-suppresed people.
- Two months ago, Orphazyme enlisted the help of the Danish courts to help oversee a restructuring in the wake of trial failures, C-suite shakeups and regulatory rejections. The resolution of the court-run efforts are coming into view now as KemPharm agrees to acquire Orphazyme and its once-hyped “pipeline-in-a-product” for $12.8 million in cash, while also assuming some liabilities.
CELL & GENE TX
- Even Peter Marks, the director of the FDA’s CBER, is worried about the commercial viability of cell and gene therapies. In a rare, direct address to the “800 pound gorilla in the room” that is reimbursement, he talked at a conference about sponsors are starting to see indications where there may only be several dozen patients worldwide as nonviable.
- Pfizer, Sarepta, Solid Biosciences and Genethon believe they have found a reason for the safety issues that have plagued gene therapies for Duchenne muscular dystrophy. The four companies suggested there may be a “class effect,” and that more research is needed to find out the root cause.
- Ahead of the FDA, the EMA is giving its thumbs up for PTC Therapeutics’ gene therapy for aromatic amino acid decarboxylase deficiency, an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs.
- SQZ Biotech showcased new research suggesting that its unorthodox, prototype point-of-care cell therapy manufacturing tech could lead to comparable or even better performance than a conventional clean-room-based manufacturing process. The first generation is expected to enter the clinic in 2023.
- Dark Horse Consulting walked away from the FDA’s two-day adcomm on gene therapy last September feeling it was a “missed opportunity.” Now, the firm is stepping to the plate with its own recommendations for AAVs — submitting those ideas in the form of a draft guidance. The 10-page guidance hones in on the role that empty AAV vector capsids can play in product impurity, and looks to establish release criterion for full to empty capsid ratios.
- Fargo-based DNA and mRNA producer Aldevron has licensed the rights to manufacture and commercialize the Eureca-V Nuclease, a wild-type MAD7 CRISPR Type-V nuclease, from Inscripta. The partners will aim to offer this nuclease as a standard research-grade and GMP catalog item.
- Merck quietly inked a deal to pick up a certain “large molecule” cancer drug candidate from China’s Kelun Pharmaceutical. Covering all territories outside mainland China, Taiwan, Hong Kong and Macau, the deal starts at $47 million upfront but milestones can add up to $1.3 billion.
- Green Valley Pharma, the Shanghai drugmaker that scored a stunning approval to market an Alzheimer’s drug in China two years ago, is shuttering a Phase III trial that would’ve paved its way to the FDA. A lack of funding had forced its hand, according to the biotech, which said a recent Covid-19 outbreak put it in a cash crunch and it wasn’t able to raise funding in time.
- In an unusual arrangement, Chinese oncology giant Jiangsu Hengrui launched Luzsana Biotechnology to serve as an arm to develop and deliver its therapeutics outside of the Chinese market. The biotech already has locations in Tokyo, Basel, Switzerland and Princeton, New Jersey, with more than 120 employees. CEO Scott Filosi was a top commercial exec at Merck KGaA.
- Suzhou, China-based Starna Therapeutics emerged with about $24 million to take on two difficult areas in biotech R&D: RSV and idiopathic pulmonary fibrosis. The Series A will support preclinical work for its mRNA vaccine candidate in RSV, which is based on tech from its co-founder, a PI at Peking University.
- As the US transitions away from Covid-19 vaccine science into more therapeutic work, the NIH awarded $577 million to nine academic groups in an effort to drive antiviral development for Covid and a slew of other viruses. The grants will create nine antiviral drug discovery (AViDD) centers, whose main goals are to do the preclinical discovery work for drugs against viruses with pandemic potential.
- Two years after a bitter setback at the FDA, with its cash reserves ebbing away, Tricida is executing a high-stakes gamble on winning a swift approval at the FDA by putting an “administrative stop” to a pivotal trial. A win would put them back in play with a new drug to commercialize. A loss, as one Wall Street analyst recently assessed, would take the stock to zero.
- Athersys’ stem cell therapy has failed yet again. In a 206-person trial conducted in Japan, Athersys’ experimental treatment for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores. Then, as it’s done in the past, the company pivoted to a post hoc analysis, focusing on a younger subset of patients.
- A Phase IIb flop is casting a shadow on Enanta Pharmaceuticals’ efforts to take on RSV, a notoriously tough field strewn with Big Pharma rivals. Although its antiviral failed to reduce symptoms for otherwise healthy adults, the biotech is holding out hope for a narrower group of patients who would need a treatment the most.
- AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition. Its NDA was for ABBV-951, a solution of levodopa and carbidopa prodrugs administered through a 24-hour infusion. It’s designed for advanced Parkinson’s patients who don’t respond well to oral therapy.
- Rocket Pharmaceuticals’ latest cut of gene therapy data has at least one analyst wondering about the curative potential. Reporting topline results from a Phase II pivotal trial involving patients with severe leukocyte adhesion deficiency-I (LAD-1), Rocket said among nine participants, the overall survival at one year is 100% — and there were also improvements in symptoms, paving the way for a regulatory filing early next year.
- When Zegalogue, also known as dasiglucagon, got approved by the FDA early last year for severe hypoglycemia in diabetes patients, Zealand Pharma said that wasn’t the end of its vision for the drug. It’s now delivered on a Phase III trial for a new indication — a genetic condition called congenital hyperinsulinism — with positive results that could pave the way for a filing.
- Moderna and nonprofit research group IAVI (International AIDS Vaccine Initiative) are jumping into the next stage of their collaboration around an mRNA anti-HIV vaccine. IAVI will sponsor a new Phase I trial dubbed IAVI G003, which will take place in Rwanda and South Africa to see if earlier results in the US will translate into a different population.
- Months after touting some fuzzy topline readouts, AstraZeneca and its biotech partner Avillion detailed the data they’re taking to regulators for their asthma drug, PT027. The inhaled, fixed-dose rescue medication, which combines two commonly used drugs, cut the risk of severe exacerbation by 27% (p<0.001) compared to albuterol rescue alone.
- While Seagen is still investigating claims of domestic violence against Clay Siegall, the company and its longtime CEO agreed that it’s better he resign now. Since the biotech revealed that Siegall was on leave, more details have emerged, including the fact that he was arrested and placed under a temporary restraining order from his wife. He denied the allegations.
- Moderna’s rapid fire development of its hugely successful mRNA vaccine for Covid saved lives, changed the vaccine industry forever and made CEO Stéphane Bancel a billionaire. But perhaps the sweetest reward came this week, when Bancel was named a Chevalier — basically knighted — by the president of France.
- Ready to transform itself into a serious mid- to late-stage cancer player that could take on rivals as big as AstraZeneca, Pfizer-partnered biotech Zentalis appointed Eli Lilly vet Kim Blackwell to succeed Anthony Sun as CEO. To fuel the development of its two lead candidates, Zentalis raised an additional $200 million from an offering.
- Richard “Ric” Kayne, the billionaire who mounted an activist attack at Alzheimer’s-focused Athira after it ousted founder Leen Kawas, is suspending his campaign to insert himself to the company’s board of directors. Though Kayne will no longer be emphasizing the need for change, Athira shareholders are still allowed to vote for his slate of nominations.
- Flagship crafted a new startup out of pieces from a pair of fledglings in the VC’s nest. And as Inzen and Cygnal merge into Sonata Therapeutics, Roche vet and Cygnal CEO Pearl Huang will depart in pursuit of new opportunities. Whereas Inzen was focused on the cellular microenvironment, Cygnal zeroed in on the peripheral nervous system.
- We’ve seen plenty of new companies focusing on CAR-Ts — or CAR-NKs and CAR-Ms. But Inceptor Bio wants to attempt all three at once. With $37 million, it hopes to flesh out its oncology programs while also building out its own manufacturing facility in Gainesville, FL. While acknowledging the challenges, the biotech hopes the approach yields more shots on goal.
- New York-based Cambrian Biopharma spun out a new startup with the idea of extending women’s “healthspans.” Focused on ovaries, Oviva is armed with $11.5 million from Cambrian and the scientific knowhow of two researchers at Mass General.
- Private equity is making more big inroads into the biotech scene. And Antoine Papiernik and the crew at Sofinnova Partners are getting a substantial boost in the money they have available to invest. The $32 billion private equity player Apollo Global Management has stepped up to take a minority stake in the life sci player, offering €1 billion of managed capital to its funds.
- Jeff Aronin’s cell and gene therapy biotech Castle Creek Biosciences has raised $112 million in equity, Endpoints News learned. After pulling IPO ambitions last December, the company secured money from 54 private investors to fund late-stage work on its ex vivo gene therapy for chronic wounds due to recessive dystrophic epidermolysis bullosa.
- Just a few short weeks after SwanBio laid off a quarter of its 60-person workforce, it has returned with $56 million in new financing. While scuttled fundraising plans apparently triggered the layoffs, at least two founding investors remain steadfast and confident in its plan to head toward the clinic.
- Basking in the spotlight of a $1 billion deal with J&J, Remix Therapeutics is back with $70 million in VC money as it continues to try and drug RNA processing. While the platform could be used for drug discovery across fields, Remix has primarily been focusing on “high value targets in oncology and neuroscience right now.”
- Shankar Ramaswamy doesn’t intend to repeat the Roivant playbook that his brother, Vivek, started eight years ago. Instead, he wants to consolidate the gene therapy process and be the go-to shop in a field that has ridden a rocky road for many years. With that vision for a “consolidated engine,” he and his 160-employee biotech have reeled in a $270 million Series C.
- Three years after Clover Health tapped ex-Pfizer exec Cheng Zhang to leverage the health insurer’s data in unearthing biomarkers and drug targets, Clover Therapeutics got a facelift. Now named Character Biosciences, it raised $18 million to carry on with the same goal, which is to discover potential therapies for diseases of aging.
- Working out of North Carolina’s research triangle, precision antibacterial drugmaker Locus Biosciences closed a $35 million Series B featuring some notable VC names. The BARDA-backed biotech’s lead candidate is a phage therapy targeting E. coli causing urinary tract infections.
- An Iowa seed supplier led a $29.5 million infusion into tiny, Rochester, MN-based biotech Vyriad, which is developing an oncolytic virus derived from the Vesicular stomatitis virus. The round is an extension to a Series B led by Regeneron, which is collaborating on a combo with the PD-1 drug Libtayo.
- Capping a years-long trademark case, Roche’s Spark Therapeutics and bluebird bio filed a joint motion to dismiss a trademark infringement suit over bluebird’s “Be the Spark” sickle cell campaign — which Roche claimed infringed on its Spark trademarks. But the actual settlement agreement remains confidential.
- Two years ago, Novartis got slammed with hundreds of millions in fines from the US Department of Justice for engaging in bribery and misconduct in order to boost sales in different countries. Now, one of those countries, Greece, is on the offensive — issuing a statement that the government is committed to claiming compensation from the Swiss mega-pharma.
- Sanofi has faced a slew of allegations over the last several years that its epilepsy drug Depakine causes birth defects when taken during pregnancy. In a setback, a court in Nanterre — a city several miles outside of Paris — ruled that Sanofi must pay up to €450,000 in damages to a family whose child suffered a form of autism after exposure to Depakine.
- Henrietta Lacks’ family’s fight for justice faces an uncertain fate as a Baltimore federal judge considers tossing the case. Judge Deborah Boardman is weighing Thermo Fisher’s motion to dismiss the claims against it on the grounds that the statute of limitations has passed, and the continuing harm doctrine does not apply.
- Pune, India-based generic drug manufacturer Emcure Pharmaceuticals is pressing a court in Washington to toss out a suit brought on by HDT Bio over a vaccine to prevent Covid-19. Calling its former partner’s demand for $950 million in damages “bloated,” Emcure slams the suit as “a made-for-headlines hit piece.”
- AstraZeneca is doubling down on developing antibodies for Covid-19. Building on the long-acting antibody combo now known as Evusheld, AstraZeneca this time is betting on newly-launched RQ Biotechnology, a UK-based outfit with roots at the University of Oxford and medical research charity LifeArc. The $157 million deal brings in a suite of early-stage antibodies targeting SARS-CoV-2.
- Amid rising concerns of potential development of resistance to Pfizer’s Covid-19 pill Paxlovid, the company reportedly declined to supply researchers with the antiviral for use in combination studies. For its part, Pfizer said it’s evaluating a “multi-pronged strategy” that includes thinking ahead to second-generation antivirals and combination therapies, but it hasn’t begun any trials.
- The latest wave of the pandemic — marked by Omicron and its sub-variants — has seen higher hospitalization rates for young children, health agencies have observed. That’s part of the reason why the FDA is authorizing a single booster shot of Pfizer and BioNTech’s mRNA vaccine for kids between 5 and 11 years old.
- The FDA rejected University of Minnesota ID doc David Boulware’s EUA to use fluvoxamine, a generic SSRI already approved to treat OCD, as an outpatient treatment for those testing positive for Covid-19. According to regulators, submitted data on “clinically meaningful outcomes” were “not persuasive.”
- With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen. The FDA approved the drug to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus, which Patel called the first in a “second wave” of indications.
- Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint. It plans to build a $595 million center in the Seaport area to accommodate growing R&D as well as manufacturing needs, especially in its cell and gene therapies program.
- The European Medicines Agency is recommending the suspension of about 100 drugs after finding data integrity issues at an India-based CRO that ran tests for the generic drugs. The CRO in question, Synchron Research Services, isn’t the first to come under the microscope for data failings. As with those cases, regulators are requiring the drug sponsors to repeat their bioequivalence/bioavailability studies at an acceptable alternate study site.
- Industry lobbying group PhRMA lodged a win against commercial insurers taking advantage of financial assistance programs designed to shoulder the high costs of drugs. A new court ruling sided with PhRMA, which sued the federal government over a 2020 rule that would’ve required drug manufacturers to ensure their assistance went to patients.
- A team of researchers dug through all the patent and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020 and found that they often rewarded incremental adjustments to older products, especially on the delivery device. The new paper, published in Health Affairs, argues that this system enabled companies to skirt around cheaper competition.
- You probably already call it this way already, but GlaxoSmithKline finally officially changed its name to GSK. Folding the history of Glaxo Wellcome and SmithKline Beecham into three simple letters, the name change comes weeks ahead of GSK’s anticipated consumer healthcare spinout.
- The Senate voted along party lines, 51-50, with Vice President Kamala Harris casting the tie-breaker, to make President Biden’s appointee Alvaro Bedoya the deciding vote on a split 2-2 Federal Trade Commission. His appointment could not only spell more trouble for biopharma M&A activity, but it may also mean that FTC Chair Lina Khan has what she needs to move forward on a study around the pharma middlemen known as pharmacy benefit managers. And a bipartisan trio of senators didn’t waste any time calling on Khan to move forward with the investigation.
- The social part of Roche’s environmental, social and governance (ESG) strategy is centered on access to its drugs and diagnostics. The Roche inaugural Access to Healthcare investor call follows a general pharma industry trend in highlighting ESG, and during the call execs outlined their targets while also pointing out what they have accomplished.
- Staying in the backseat to Merck’s Keytruda, Bristol Myers Squibb reported another pivotal setback trying to move into a bigger position in frontline therapy. Its Opdivo/Yervoy combo flopped in a Phase III study of first-line therapy for previously untreated patients with the most common form of bladder cancer, failing to hit overall survival.
- It looks like Warren Buffett is rethinking his healthcare investments. Berkshire Hathaway backed out of two major holdings in the pharma industry, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb. The move comes after Berkshire abandoned its Teva shares just last quarter.
- In pharma public relations, every day feels like it might be a crisis. In fact, 76% of pharma communications leaders expect to face three or more crises this year — compared to less than half (49%) of their peers in other industries, according to a new survey. Endpoints MarketingRx editor Beth Bulik dives into the sentiment, how the Covid-19 pandemic amplified it and what PR experts are doing to prepare.
- What do physicians think about the emerging market for recurrent C. diff? That depends on which specialists you talk to. Fecal microbiota transplants, or FMT, are favored by gastroenterologists familiar with them, while infectious disease doctors prefer traditional small molecule therapies, according to a recent future market evaluation by Spherix Global Insights.
- Pharma ad spending is concentrated at the top. A handful of health conditions — along with a handful of brands inside each condition — dominate industry advertising, according to deep dive research from MediaRadar. Its latest report finds that five therapy areas accounted for almost half (43%) of all pharma DTC spending last year, with diabetes coming up on top, followed by psoriasis, arthritis, HIV/AIDs and breast cancer.
- GSK enlisted ’80s child star turned director, writer and mom Soleil Moon Frye, known for her “Punky Brewster” character, during the pandemic last year to spotlight meningitis B vaccinations for teens. At the time, the already low rate for MenB vaccinations was dipping even lower. Now the “Ask2BSure” campaign is re-upping with Frye and other parent influencers to continue to raise awareness and with the additional goal to emphasize the importance of parents’ shared decision-making with doctors.
- AbbVie’s Allergan Aesthetics is enlisting real people for the first time in a new campaign for CoolSculpting. Its latest push for the body fat-freezing system includes TV commercials, along with digital and social media, where people talk about having children or aging and their bodies changing.
- Hologic enlists longtime spokesperson and breast cancer survivor Sheryl Crow to talk about annual mammograms; Three-time Olympic medal winner Gail Devers stars in Horizon’s latest disease awareness campaign; J&J centers public health threats in its newest QuickFire challenge; catch up on these and snippets in this week’s MarketingRx roundup.
- After a patient died in an early-stage study, the FDA is apping the brakes on a Flagship-backed biotech’s R&D efforts to develop a new treatment for blood cancers. Foghorn’s partial clinical hold follows a similar incident at Kura Oncology, which was similarly conducting a study in acute myeloid leukemia. Foghorn said differentiation syndrome may be to blame.
- The FDA is dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D. In a rare statement that observers say amounts to lobbying, the FDA wrote that the ruling that it never should’ve approved a rare disease drug due to orphan drug exclusivity could have catastrophic consequences.
- The first version of the Senate health committee’s bill to reauthorize all the different FDA user fees contains only a few controversial items and does not address either accelerated approval reforms or clinical trial diversity. The House Energy and Commerce Committee, on the other hand, found bipartisan support for a bill that features accelerated approval reforms, manufacturing inspection tweaks at the FDA, and others.
- Sanofi CEO Paul Hudson’s effort to develop new rare disease drugs and broaden his company’s offerings leaped forward with the EMA’s signing off on Sanofi’s — and the EU’s — first drug to treat the non-central nervous system manifestations of the rare and debilitating Niemann-Pick disease. The enzyme replacement therapy was first developed by Genzyme, and it’s not yet approved in the US.
- As the majority of drug shortages are still associated with manufacturing-related quality issues, the FDA published new draft guidance spelling out how to proactively assess risks to manufacturing processes and supply chains, while understanding the market’s vulnerabilities. According to the agency, three types of drugs will require risk management plans.
- An FDA-funded pilot program found that only about half of a select group of clinical trials could be well-emulated with available real world evidence. The program sought to emulate 30 randomized clinical trials using clinical practice data, which they were able to for 50% of them. But for others, substantive emulation challenges led to divergent results.
- Eli Lilly has clinched a key OK for tirzepatide, drawing the curtain on what it hopes will be a major cardiometabolic franchise and a serious rival to a megablockbuster from Novo Nordisk. With the FDA approval, Lilly will start marketing tirzepatide injection as Mounjaro to adults with type 2 diabetes, the first such treatment that’s also a dual GIP/GLP-1 receptor agonist.
- Back in late December, the FDA flagged concerns about the glass vials Gilead used to contain its experimental HIV therapy lenacapavir, citing risks of forming sub-visible glass particles in the drug solution. A complete response letter later, Gilead said it’s finally found an alternative glass vial that satisfies the FDA, which is lifting the clinical hold.
- The path Cytokinetics has plotted for its lead heart drug will face one more potential obstacle as it approaches its PDUFA date this fall: an adcomm. The biotech was informed that the FDA plans to convene its panel of experts to discuss the application for omecamtiv mecarbil in heart failure, setting up another possible speed bump following disappointing data and deal breakups.
- Resilience, the biomanufacturing shop co-founded by Bob Nelsen, found its next partner within the ARCH portfolio. In a collaboration built for the long haul, it will make Be Bio’s B cell therapies for rare diseases, shouldering the initial manufacturing costs in exchange for milestones and royalties down the road.
- Continuing its expansion spree, Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China. According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services.
- Even after Utah-based Clene failed to hit its primary endpoints for its ALS drug last year, the state of Maryland is putting its money at least behind Clene’s manufacturing facility. The Maryland Board of Public Works finalized a $3 million, 60-month loan facility with Clene that will go toward equipment purchases and support the expansion of Clene’s operations.
- Just a week after Taiwan-based CDMO Bora Pharmaceuticals acquired Eden Biologics, it established Bora Biologics, expanding into the biopharmaceutical market. With the buyout, it gained production capacity of cell lines as well as improved quality control and inspection specifications.
- Four years after Novartis first announced plans to close a site in Grimsby, UK — and following several delays — it finally sold the facility. As a result of the sale to Humber Industrials, more than 200 workers will face imminent layoffs.
- As Catalent ventures further into the world of cell and gene therapy manufacturing, the New Jersey-based CDMO has revealed its new adeno-associated viral vector platform. The UpTempo Virtuoso platform can streamline the AAV manufacturing process and it’s designed to produce products for the clinic in just nine months.
- Biotech is one of the smartest, best educated industries on the planet. And in these times, savvy, experienced leaders running biotech startups are being tested as never before, as even those with strong prospects are being cooked in the same kettle as the bad. So how do you respond to these extraordinary times? That’s what Endpoints’ John Carroll will be exploring during a live panel discussion at BIO in San Diego. He introduces his guests here.
- Martin Shkreli, the infamous biotech CEO who made headlines for his jeering assault on a legion of critics in and out of Congress, is back on the streets after four years inside federal prison. As his lawyer confirmed, the “pharma bro” had been transferred to a halfway house in New York with a few more months to go under federal custody, slated to end September 14.
- Gilead Sciences’ $100 million pledge to fight the HIV epidemic in the southern US states vaulted it to the top of an annual philanthropic giving list, ahead of the Bill & Melinda Gates Foundation for the first time since the Funders Concerned About AIDS began counting.