The triple crown in biotech: An all-or-nothing bet on an FDA approval of 3 drugs over 16 months starts today
John Carroll
Editor & Founder
Bristol-Myers Squibb’s $74 billion Celgene deal closed as expected Wednesday evening. And now a new clock has begun to tick down for Celgene shareholders who came away from the deal with CVRs — contingent value rights — worth $9 or nothing. Those CVRs start trading today as $BMYRT.
The new deadline they have is the end of March 2021, a little more than 16 months from now, when Bristol-Myers will need to gain approvals on 3 late-stage drugs it’s picking up in the buyout: Ozanimod and liso-cel (JCAR017) are due up at the end of 2020, with bb2121 deadlined at the end of Q1 in 2021.
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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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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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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.
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Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)
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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UPDATED: ObsEva’s second uterine fibroids PhIII comes through, sending some investors to the hills
Jason Mast
Associate Editor
In the three-company race to develop a new uterine fibroid treatment, ObsEva long lagged behind AbbVie and Myovant. Still, they hoped that better efficacy — including a 93.9% response rate in one Phase III trial — could ultimately deliver better sales.
Now, though, the second of those Phase III studies is out, and it has brought the Swedish biotech back to earth.
In the second of their Phase III trials, 75.5% of patients who took ObsEva’s experimental tablet linzagolix plus a hormone saw their bleeding reduced by at least 50% and at least 80 ml after 24 weeks. Pooling those results with new data from their first Phase III trial — which showed a 93.9% response rate at 24 weeks and a 91.6% response rate at 52 weeks — ObsEva calculated a collective 84.7% responder rate for patients taking their therapy and said the data “confirm potential best-in-class” status.
Covid-19 roundup: Teamed up with NIH, Regeneron launches PhIII prevention trial for antibody cocktail
Amber Tong
Editor
John Carroll
Editor & Founder
As Regeneron moves its antibody cocktail into Phase II/III trials testing REGN-COV2 as a treatment for both hospitalized and non-hospitalized patients with Covid-19, the biotech is also starting a Phase III in the prevention setting.
The National Institute of Allergy and Infectious Diseases — which orchestrated the large, randomized study for remdesivir that produced positive results — will jointly run the study.
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Bellus hit with a setback as its PhII chronic cough drug flops on all 4 doses -- shares cremated on Wall Street
John Carroll
Editor & Founder
Bellus Health says their Phase II study for a new drug to treat chronic cough failed, but they already lined up Plan B to help ease the sting with investors.
The biotech tested 4 different doses of BLU-5937 in the mid-stage study, all of which fell short of the mark for placebo-adjusted reductions in coughing — several by a wide margin. It also didn’t help that there was no clear dose response in the mix, with a jumble of outcomes to report. Bellus $BLU immediately tried to shift focus to a silver lining, noting that they hit statistical significance among a subgroup of “high cough count patients (all patients at or above the baseline median average of 32.4 coughs per hour).”
