Taysha buys clin­ic-ready gene ther­a­py from Abeona, bring­ing Steven Gray's port­fo­lio back in one place

Less than four months af­ter Taysha launched with a suite of AAV gene ther­a­pies de­vel­oped at UT South­west­ern — and two weeks af­ter the ex-AveX­is team raised $95 mil­lion in fresh cash — the biotech is look­ing to its chief sci­en­tif­ic ad­vi­sor for an­oth­er as­set.

The new pro­gram is ABO-202, which is de­signed to treat CLN1 dis­ease (or in­fan­tile Bat­ten dis­ease) by de­liv­er­ing a cor­rect copy of the PPT1 gene with a mod­i­fied AAV9 vec­tor.

Steven Gray

Steven Gray de­vel­oped the ther­a­py while at UNC-Chapel Hill, where he had stud­ied un­der Jude Samul­s­ki years ago. Abeona li­censed it in 2016, built out the clin­i­cal tri­al pro­to­col and man­u­fac­tur­ing process, and has since had an IND for a Phase I/II clin­i­cal tri­al cleared with the FDA.

Taysha is pay­ing on­ly $7 mil­lion up­front to get its hands on ABO-202, with $3 mil­lion in li­cense fees and $4 mil­lion for the in­ven­to­ry — in­clud­ing clin­i­cal-grade CLN1 plas­mid. Abeona is el­i­gi­ble to re­ceive up to $56 mil­lion down the road for de­vel­op­ment and sales mile­stones, plus roy­al­ties.

The deal puts Taysha on track to be­gin two clin­i­cal stud­ies next year: both the CLN1 pro­gram and an­oth­er gene ther­a­py for Tay-Sachs dis­ease. Three more INDs are on the dock­et for 2021.

RA Ses­sion

“CLN1 is a pro­gres­sive mono­genic CNS dis­ease with sig­nif­i­cant un­met med­ical need, and we be­lieve the ABO-202 da­ta gen­er­at­ed thus far demon­strate great trans­la­tion­al po­ten­tial and of­fer hope to chil­dren suf­fer­ing from this dev­as­tat­ing dis­or­der,” CEO RA Ses­sion II said.

Gray, who now leads 50 trans­la­tion­al sci­en­tists and man­ages a GMP man­u­fac­tur­ing site at UT South­west­ern’s vi­ral vec­tor core, will con­tin­ue to ad­vise the com­pa­ny along­side Berge Mi­nass­ian, the di­vi­sion chief of child neu­rol­o­gy.

Rather than shep­herd­ing one as­set at a time like the first gen­er­a­tion of gene ther­a­py play­ers, Taysha had opt­ed for a port­fo­lio ap­proach to ad­vance mul­ti­ple gene ther­a­pies in par­al­lel. It’s look­ing to build three big fran­chis­es: ge­net­ic forms of epilep­sy, neu­rode­vel­op­men­tal dis­or­ders and neu­rode­gen­er­a­tive dis­or­ders. And al­though the crew has eyes on new­er tech­nolo­gies like re­dos­ing, bi­cistron­ic plas­mids, mi­croR­NA knock­down, hair­pin tech­nol­o­gy to turn on a silent al­lele and de­liv­ery through the va­gus nerve, the cor­ner­stone re­mains ade­no-as­so­ci­at­ed virus (AAV).

“If it’s not broke, you don’t fix it,” Ses­sion told End­points News back in April.

Sean Nolan

Taysha, which is chaired by for­mer AveX­is CEO Sean Nolan, has re­served rights to tap four more pro­grams out of Gray’s lab.

For Abeona, the deal with Taysha gives them time and some cash to fo­cus on its more ad­vanced pro­grams in re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa, San­fil­ip­po syn­drome type A and San­fil­ip­po syn­drome type B as it re­cov­ers from a some­what tu­mul­tuous 2019.

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

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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 clinicaltrials.gov, 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.