Sean Nolan, RA Session II

Crossover round? Check. Top team? Check. Taysha sails to an IPO with $100M ini­tial ask

On the same day No­var­tis erased the AveX­is brand from the com­pa­ny, the ex-AveX­is crew says their new gene ther­a­py start­up is ready for an IPO.

Taysha Gene Ther­a­pies has left enough bread crumbs for biotech sleuths to fig­ure out that it’s go­ing down this path. CEO RA Ses­sion II, who hap­pi­ly chat­ted to mul­ti­ple re­porters at launch, de­clined in­ter­views for the re­cent Se­ries B (and pre­sumed crossover round); the biotech brought in a gene ther­a­py pro­gram de­vel­oped by its co-founder Steven Gray from Abeona; and days ago it tapped the ex-CMO and ex-CFO of AveX­is to join the board, chaired by their for­mer boss Sean Nolan.

What’s still re­mark­able is the speed at which Dal­las-based Taysha has moved. It’s been just a year since the com­pa­ny was formed, a lit­tle over four months since Taysha launched with $30 mil­lion and a slate of 15 pro­grams, and bare­ly a month af­ter it closed a $95 mil­lion Se­ries B. The ac­cu­mu­lat­ed deficit so far is just $27.8 mil­lion.

That casts a unique spot­light on Taysha com­pared to the 48 play­ers that have rid­den to a pub­lic list­ing on the biotech boom so far this year. Even Pas­sage Bio, the Philadel­phia-based start­up that has a sim­i­lar pact with gene ther­a­py gu­ru Jim Wil­son, took a year from Se­ries A to go pub­lic.

For now Taysha is pen­cil­ing in a $100 mil­lion raise, but these days the place­hold­er fig­ure could mean lit­tle.

Oth­er than lay­ing out the clin­i­cal tri­al time­lines for the lead pro­grams — the first study, a Phase I/II of TSHA-101 for GM2 gan­gliosi­do­sis, is planned in Cana­da for lat­er this year — the S-1 re­veals some in­ter­est­ing de­tails about the part­ner­ships that are core to the biotech.

To get UT South­west­ern on board, it turned out Taysha didn’t pay a pen­ny. In­stead, it is­sued 2 mil­lion shares of its stock to the uni­ver­si­ty, with no fu­ture mile­stones or roy­al­ty promis­es at­tached oth­er than costs of main­tain­ing patents. Gray, an ex­pert in AAV-based gene ther­a­pies for CNS dis­or­ders, is lead­ing the col­lab­o­ra­tion along­side child neu­rol­o­gy di­vi­sion chief Berge Mi­nass­ian.

By the end of 2021, Taysha al­so ex­pects to file INDs in the US for four pro­grams, all pack­aged in an AAV9 vec­tor and span­ning the three pil­lars of neu­rode­gen­er­a­tive, neu­rode­vel­op­men­tal and epilep­tic dis­or­ders:

TSHA-101, a bi­cistron­ic HexBP2A-HexA trans­gene pack­aged in­to an AAV9 vec­tor un­der the con­trol of a CAG pro­mot­er de­signed for con­di­tions like Tay-Sachs and Sand­hoff dis­ease. TSHA-102, which com­bines a neu­ronal spe­cif­ic pro­mot­er, MeP426, with the min­iMECP2 trans­gene, plus a miR­NA-Re­spon­sive Au­to-Reg­u­la­to­ry El­e­ment for the treat­ment of Rett syn­drome. TSHA-103, which tar­gets a ge­net­ic form of epilep­sy caused by SLC6A1 hap­loin­suf­fi­cien­cy dis­or­der, is con­struct­ed from a codon-op­ti­mized ver­sion of the hu­man SLC6A1 gene un­der the con­trol of a JeT pro­mot­er. TSHA-104, which ad­dress­es SURF1 de­fi­cien­cy, adopts a ver­sion of the hu­man SURF1 gene un­der the con­trol of a mod­i­fied ver­sion of the pro­mot­er CBA hy­brid in­tron, or CBh.

With 18 pro­grams now in the pipeline re­flect­ing a port­fo­lio ap­proach, Taysha is en­ti­tled to an op­tion for four more pro­grams un­der the re­search pact with UT South­west­ern, which ex­pires in No­vem­ber 2021.

Then there’s TSHA-118, a po­ten­tial treat­ment of in­fan­tile Bat­ten dis­ease in­vent­ed by Gray that Taysha re­cent­ly li­censed from Abeona. Taysha is paid on­ly $7 mil­lion up­front to get its hands on the clin­ic-ready drug, then known as ABO-202, with $3 mil­lion in li­cense fees and $4 mil­lion for the in­ven­to­ry — in­clud­ing clin­i­cal-grade CLN1 plas­mid. Mile­stones add up to $56 mil­lion.

In its fil­ing, Taysha dis­closed that it’s al­so struck a deal with Queen’s Uni­ver­si­ty at Kingston to ac­cess some of its tech­nolo­gies, in­clud­ing plas­mid pro­duc­tion, for next-gen­er­a­tion gene ther­a­pies. In ad­di­tion to $3 mil­lion up­front, it re­im­bursed around $220,000 in costs and agreed to pay an­oth­er $20 mil­lion for mile­stones.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

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.