UPDATED: Cytokinetics is getting its shot at a major PhIII showdown with Bristol Myers after posting positive data for a rare cardio disease. Shares rocket up
John Carroll
Editor & Founder
After losing a whole lot of market confidence in their lead drug, little Cytokinetics is now lining up for a crucial Phase III pivotal challenge for the next cardio drug in the pipeline. And that will put them toe-to-toe against powerhouse rivals at Bristol Myers Squibb, which just 9 months ago put down a $13 billion wager on what will likely be the first drug approved for the same rare cardio condition the biotech is contending for.
Premium subscription required
Unlock this article along with other benefits by subscribing to one of our paid plans.
SPONSORED
January 17, 2022 06:00 AM EST
ZS Perspective: 3 Predictions on the Future of Cell & Gene Therapies
Ben Hohn
The field of cell and gene therapies (C>s) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C>s that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.
Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.
Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.
'Altos was really a once-in-a-lifetime opportunity': Hal Barron reflects on his big move
By all accounts, Hal Barron had one of the best jobs in Big Pharma R&D. He made more than $11 million in 2020, once again reaping more than his boss, Emma Walmsley, who always championed him at every opportunity. And he oversaw a global R&D effort that struck a variety of big-dollar deals for oncology, neurodegeneration and more.
Sure, the critics never let up about what they saw as a rather uninspiring late-stage pipeline, where the rubber hits the road in the Big Pharma world’s hunt for the next big near-term blockbuster, but the in-house reviews were stellar. And Barron was firmly focused on bringing up the success rate in clinical trials, holding out for the big rewards of moving the dial from an average 10% success rate to 20%.
Premium subscription required
Unlock this article along with other benefits by subscribing to one of our paid plans.
Zymeworks replaces half its C-suite, aims to lay off 25% of total workforce as new CEO takes over
Max Gelman
Editor
New Zymeworks CEO Kenneth Galbraith is aiming to hit the ground running when his tenure officially begins next month, but he’ll be doing so with a much different looking team.
In a lengthy press release outlining the biotech’s 2022 goals, Galbraith said Zymeworks will be laying off at least 25% of its staff over the course of the year. Half of its C-suite will also be replaced immediately as Galbraith looks to remake the company in his image after Ali Tehrani, Zymeworks’ founder and CEO since 2003, stepped down two weeks ago.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 129,500+ biopharma pros reading Endpoints daily — and it's free.
European Parliament signs off on strengthening drug regulator's ability to tackle shortages
Zachary Brennan
Senior Editor
The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.
By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 129,500+ biopharma pros reading Endpoints daily — and it's free.
FDA+ roundup: FDA's neuroscience deputy departs amid ongoing Aduhelm investigations; Califf on the ropes?
Zachary Brennan
Senior Editor
Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.
Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.
Premium subscription required
Unlock this article along with other benefits by subscribing to one of our paid plans.
What kind of biotech startup wins a $3B syndicate, woos a gallery of marquee scientists and recruits GSK's Hal Barron as CEO in a stunner? Let Rick Klausner explain
John Carroll
Editor & Founder
It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.
At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.
Milner’s question on one of those mornings on foot: “What do you want to do?”
Premium subscription required
Unlock this article along with other benefits by subscribing to one of our paid plans.
Critics push back on Alzheimer’s Association ad blitz to get Medicare to change its Aduhelm ruling: 'Dead wrong'
Beth Snyder Bulik
Senior Editor
The latest Alzheimer’s Association advertising campaign encourages people to fight.
Not against the disease or for more research or treatments, but against the Centers for Medicare and Medicaid Services. More specifically, CMS’ recent reimbursement decision to only pay for Biogen and Eisai’s controversial Alzheimer’s drug Aduhelm for patients in clinical trials.
With CMS’ preliminary decision now in a 30-day comment period, patient advocates’ goal is to convince CMS to reverse its decision with a marketing blitz and public pressure.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 129,500+ biopharma pros reading Endpoints daily — and it's free.
Failing to confirm clinical benefit, Gilead pulls 2 accelerated approval indications for cancer drug
Zachary Brennan
Senior Editor
Gilead recently decided to pull two indications for its cancer drug Zydelig — in relapsed follicular B-cell non-Hodgkin lymphoma (FL) and relapsed small lymphocytic leukemia (SLL) — after failing to complete the confirmatory trials required as part of the accelerated approvals from 2014.
“As the treatment landscape for FL and SLL has evolved, enrollment into the confirmatory study has been an ongoing challenge,” Gilead said in a statement, noting it formally notified the FDA of its decision to voluntarily withdraw these indications.
Time limits on accelerated approvals? FDA's oncology chief Rick Pazdur eyes potential reforms via international approaches
Zachary Brennan
Senior Editor
The spotlight on the accelerated approval pathway continues to shine bright, with the FDA’s top oncology official writing in an opinion that the pathway may be strengthened with bits and pieces of what other regulators in Europe and elsewhere have done with their expedited approval pathways, such as adding expiration dates for these faster approvals to ensure they confirm clinical benefit in a timely manner.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 129,500+ biopharma pros reading Endpoints daily — and it's free.
