Payback? Analysts say Sarepta was blindsided by an FDA rejection driven by regulatory revenge
John Carroll
Editor & Founder
In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.
In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.
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.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.
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.
Premium subscription required
Unlock this article along with other benefits by subscribing to one of our paid plans.
Covid-19 roundup: Left out no longer, Novavax secures largest Warp Speed deal yet: $1.6B
Jason Mast
Associate Editor
It looks like Novavax won’t be left out of Operation Warp Speed after all.
A month after the Gaithersburg, MD biotech saw its shares tumble when it was left off the first reported list of finalists for the White House’s Covid-19 vaccine accelerator, HHS and the Department of Defense have announced a $1.6 billion deal to scale up their Covid-19 candidate. It is the largest deal HHS has announced yet, eclipsing the $1.2 billion deal the administration reached with AstraZeneca in May.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.
Investors give ailing Unum a lease on life and a whole new suite of experimental cancer drugs
Jason Mast
Associate Editor
Investors, it seems, are willing to give Unum Therapeutics one last shot — or at least one last shot to a company of that name.
The ailing cancer biotech, beset by a series of clinical holds and multiple failed lead programs, announced today that they’ve acquired Kiq LLC and that investors are putting in $104 million to advance Kiq’s pipeline of kinase inhibitors. Unum shareholders will now own only 16.2% of the company and CEO Chuck Wilson indicated that the cell therapies the biotech has worked on since its founding may be on their way out, saying Unum will “explore strategic options” for those products.
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.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.
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.
Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)
Second PhIII study for Idorsia's sleep drug returns positive results, but also raises new questions
Max Gelman
Following a successful Phase III study in April showcasing the safety and potential of its new sleep drug, Idorsia posted some mixed news in the second Phase III study, but that won’t stop a planned filing aimed at regulatory approval.
The drug, a dual orexin receptor antagonist (DORA) called daridorexant, was found to significantly improve sleep maintenance and subjective total sleep time in 25 mg doses, replicating results from the first Phase III study. However, improvements in sleep onset and daytime functioning narrowly missed statistical significance, despite numerical consistency with the April study.
UPDATED: Immunomedics spells out PFS benefit of Trodelvy in mTNBC, hunting a full OK just weeks after accelerated approval
Amber Tong
Editor
By the time the FDA finally granted an accelerated OK for Immunomedics’ Trodelvy, we already got a very strong hint that their confirmatory Phase III study in metastatic triple-negative breast cancer was a success.
That’s because the independent data safety monitoring committee recommended that the trial be stopped early. But just what pointed them to the conclusion was still unclear.
“We do not know the totality of their decision other than it’s pretty evident that the primary endpoint was met; otherwise they could not request to halt the study,” Behzad Aghazadeh, the executive chairman, told Endpoints Newsat the time.
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.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.
