Novartis takes a hit as FDA orders a hold on Zolgensma trials in wake of fresh safety concerns
John Carroll
Editor & Founder
The FDA has ordered Novartis to halt enrollment and dosing in a set of trials for Zolgensma, their gene therapy for lethal cases of spinal muscular atrophy. And the latest setback triggered a frenzy of analyst notes as they sized up who among Novartis’ rivals in the field would benefit most.
The hold was implemented after Novartis’ subsidiary AveXis — still operating under the glare of an FDA investigation into an ethics scandal — informed regulators about concerns raised by an animal study pointing to “dorsal root ganglia (DRG) mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss.”
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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)
Which drug developers offer Trump a quick, game-changing ‘solution’ as the pandemic roars back? Eli Lilly and AbCellera look to break out of the pack
We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.
— Donald Trump, July 4
Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”
— NBC News, July 3
Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.
Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.
So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.
We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.
So what about a drug solution?
Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.
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Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)
OWS shifts spotlight to drugs to fight Covid-19, handing Regeneron $450M to begin large scale manufacturing in the US
John Carroll
Editor & Founder
The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.
The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.
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Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)
Flagship execs take a lesson from nature to master ‘gene writing,’ launching a star-studded biotech with big ambitions to cure disease
John Carroll
Editor & Founder
Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.
The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.
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Cellectis slammed after patient dies and FDA slaps a hold on their trial for an off-the-shelf CAR-T for multiple myeloma
John Carroll
Editor & Founder
Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.
The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.
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RA Capital doubles down on Siddhartha Mukherjee's vision for a new cell engineering approach, leading Vor's $110M Series B
Amber Tong
Editor
Vor Biopharma is muscling up.
CEO Robert Ang, who was reluctant to divulge the headcount when discussing his move from Neon Therapeutics to Vor last August, readily offered that the team has grown from 6 to 50 in less than a year. The biotech is moving to a larger office on Cambridge Parkway Drive in weeks, giving it more space to complete the IND-enabling work and manufacturing scale-up — conducted by a CDMO partner — in preparation for clinical trials planned for the first half of 2021.
Covid-19 roundup: Squabbles with government delay Moderna’s PhIII — reports; Novavax secures largest Warp Speed deal yet: $1.6B
Jason Mast
Associate Editor
Amber Tong
Editor
A much-anticipated Phase III trial for Moderna’s Covid-19 vaccine is being held up as the company delayed submitting trial protocols and sparred with government scientists on how to run the study and even what the benchmark for success should be, Reuters reported.
Moderna, the first US company to put their vaccine into human testing, was supposed to enter a 30,000-person study this month in partnership with the NIH to determine whether it can prevent infection. STAT reported last week that the trial was facing delays over the protocol, but that a July start was still possible. Neither the NIH nor Moderna ever disclosed a specific date the trial should start, but Reuters reported that the agency had hoped to begin on July 10.
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Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)
A little biotech slaps back at a 'criminal' short attack, vowing to pursue a prosecution of their case
John Carroll
Editor & Founder
As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.
But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.
Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.
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A booming Dragonfly is taking its TriNKETs to Copenhagen as the latest Bristol Myers pact spurs expansion plans — outside the US
John Carroll
Editor & Founder
Bristol Myers Squibb is making a habit out of collaborating with the crew at Dragonfly, adding their 3rd deal in a series that now will take them into newly charted R&D territory. And the fast-growing team at the Cambridge-based biotech is adding a facility in Copenhagen for its next growth spurt, where the government is making it easy to recruit scientists internationally as the U.S. throttles back.
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Boehringer Ingelheim ties the knot with Numab on new antibodies; Cabaletta inks pact with Artisan
John Carroll
Editor & Founder
→ Switzerland’s Numab Therapeutics has added Boehringer Ingelheim to its roster of collaborators. And they will start with two projects aiming at developing new drugs for difficult-to-treat lung and gastrointestinal cancers and patients with geographic atrophy. “Numab’s technology platform fits well with our internal antibody discovery and engineering capabilities and will enhance our efforts to deliver transformative antibody-based therapeutics to patients,” said Paige Mahaney, an SVP at Boehringer Ingelheim.
