The final hours and days are ticking away on a $6B bet as deadline looms on a suspenseful FDA decision
The clock is ticking down to the year-end deadline for an FDA approval of liso-cel, and the suspense is killing investors.
You’ll recall that Bristol Myers CEO Giovanni Caforio insisted at the last minute of negotiations with Celgene execs that they had to replace cash on the table with a CVR $BMYRT tied to 3 key drug approvals by a certain deadline.
That’s all 3. Miss one, and anyone holding on to the $9 CVR would get nothing. For Bristol Myers, it’s a $6 billion play.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 107,000+ biopharma pros reading Endpoints daily — and it's free.
SPONSORED
April 12, 2021 06:00 AM EDT
BYOD Best Practices: How Mobile Device Strategy Leads to More Patient-Centric Clinical Trials
Jonathan Andrus
Chief Business Officer, Clinical Ink
Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.
In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?
Eli Lilly asks FDA to revoke EUA for Covid-19 treatment
Zachary Brennan
Senior Editor
Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.
“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 107,000+ biopharma pros reading Endpoints daily — and it's free.
J&J faces CDC advisory committee again next week to weigh Covid-19 vaccine risks
Zachary Brennan
Senior Editor
The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.
The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 107,000+ biopharma pros reading Endpoints daily — and it's free.
Osman Kibar lays down his hand at Samumed, stepping away from CEO role as his once-heralded anti-aging biotech rebrands
Nicole DeFeudis
Associate Editor
Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.
Why the sudden rebrand?
“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 107,000+ biopharma pros reading Endpoints daily — and it's free.
Exclusive interview: Peter Marks on why full Covid-19 vaccine approvals could be just months away
Zachary Brennan
Senior Editor
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.
Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.
Premium subscription required
Unlock this article along with other benefits by subscribing to one of our paid plans.
Nearly a year after Audentes' gene therapy deaths, the trial continues. What happened remains a mystery
Jason Mast
Editor
Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.
Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.
The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.
“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”
Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.
Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.
Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”
Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.
“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.
Premium subscription required
Unlock this article along with other benefits by subscribing to one of our paid plans.
After being goaded to sell the company, Alexion's CEO set some ambitious new goals for investors. Then Pascal Soriot came calling
John Carroll
Editor & Founder
Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.
In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 107,000+ biopharma pros reading Endpoints daily — and it's free.
Activist investor grabs a multibillion-pound stake in GlaxoSmithKline, triggering buzz about a looming showdown at the listless giant
Murmurs of investors’ growing unease about GlaxoSmithKline’s struggling revival strategy spilled out into the public on Thursday as the Financial Times reported that activist hedge fund Elliott Management has built up a multibillion-pound stake in the pharma giant, portending a high profile showdown over CEO Emma Walmsley’s reign.
Shares had been down 14% since Walmsley became CEO and brought in Hal Barron to spur a makeover in the pipeline. Now, the FT reports, some of the unhappy investors are making unfavorable comparisons between Walmsley and Pascal Soriot, the CEO credited with a major turnaround at that other UK pharma giant — AstraZeneca.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 107,000+ biopharma pros reading Endpoints daily — and it's free.
GlaxoSmithKline’s head of vaccine R&D is jumping to a wellness company concentrating on the microbiome as reports of an exodus start to spread
John Carroll
Editor & Founder
Back in the fall of 2019, GlaxoSmithKline vaccine R&D chief Emmanuel Hanon had plenty of good things to say about a wellness company called Viome and CEO Naveen Jain. He was particularly interested in Viome’s technology for analyzing the gut microbiome and how that could intersect with new vaccine research.
Today, Hanon is jumping ship to join his collaborator as R&D chief as reports circulate of an exodus at GSK’s big vaccine group.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 107,000+ biopharma pros reading Endpoints daily — and it's free.
