The End­points 11 cel­e­brates bio­phar­ma's most promis­ing star­tups. Live event on Sep­tem­ber 30

Next month John Car­roll and the End­points ed­i­to­r­i­al team con­tin­ue a proud tra­di­tion of pro­fil­ing a stand­out group of 11 star­tups that just might be head­ed for great­ness. And we’re adding a new, live el­e­ment that we want to share with all of you — and we’re ask­ing for just one hour of your time.

Please mark your cal­en­dars for a 1-hour main event on Sep­tem­ber 30 at 4pm ET. The agen­da will ex­pand in the com­ing weeks, and we’ll have more than one hour’s worth of con­tent for you. But the cen­ter­piece of the 2020 End­points 11 is a one-hour live-streamed event.

It will start at 4pm ET / 1pm PT, and you can sign up with this link.

Our goal is to cel­e­brate bold sci­ence in the pur­suit of new biotech drugs, and that’s a lot eas­i­er said than done. When it comes to pick­ing pri­vate com­pa­nies, we’re deal­ing with in­com­plete and im­per­fect in­for­ma­tion. And the lim­its of our cur­rent un­der­stand­ing of bi­ol­o­gy can frus­trate the most ex­pe­ri­enced sci­en­tif­ic teams armed with tremen­dous fi­nan­cial back­ing. Re­gard­less, we’re go­ing to make our picks and ar­gue our case for them. The com­pa­nies that are in our sight are all swing­ing for the fences. Some will fail, but they each rep­re­sent a trend in new biotech com­pa­ny cre­ation. And they emerge from a his­tor­i­cal back­drop, with the pan­dem­ic fo­cus­ing the world’s at­ten­tion on bio­phar­ma like nev­er be­fore.

This year we’re spon­sored by Catal­ent, a com­pa­ny that has sup­port­ed our in­de­pen­dent mis­sion at End­points from the start. This year, part of our agen­da fea­tures my co-founder and End­points ed­i­tor John Car­roll pre­sent­ing the awards live to the win­ners along­side Catal­ent CEO John Chimin­s­ki. Both “John C”s have a fun his­to­ry of this kind of work, which we last saw at JP Mor­gan this year in San Fran­cis­co. It’s go­ing to be a lot of fun and I hope you all can be a part of it.

Feel­ing Zoomed out? That’s un­der­stand­able, and we’ll be work­ing to keep your at­ten­tion. There is a gen­uine need to con­tin­ue the kind of cel­e­bra­to­ry events that would bring us to­geth­er in a nor­mal year — and we think this is worth your time. There’s more than one hour’s worth of con­tent to share with you all, but the ex­pe­ri­ence will be shaped in a way where the live show is the cen­ter­piece. Every­thing else is on-de­mand. And there’s no cost to at­tend. You can sign up here.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.