Piv­otal fail­ure trig­gers job cuts at San Diego-based Toca­gen

Toca­gen is cir­cling the drain, fol­low­ing the crush­ing late-stage fail­ure of its ex­per­i­men­tal brain can­cer ther­a­py last month. On Thurs­day, the San Diego-based com­pa­ny slashed its work­force by 65%.

Its two-part ther­a­py was be­ing test­ed in pa­tients with re­cur­rent high-grade glioma un­der­go­ing re­sec­tion in the To­ca 5 study. On av­er­age, pa­tients on the Toca­gen reg­i­men ac­tu­al­ly did not live as long as those giv­en stan­dard treat­ment (11.1 months me­di­an com­pared to 12.2 months). In ad­di­tion, the com­pa­ny’s ther­a­py hit a haz­ard ra­tio of 1.06, re­flect­ing an in­creased risk to pa­tients, along with an un­sa­vory p-val­ue of 0.6154.

Toca­gen’s tech­nol­o­gy em­ploys retro­vi­ral repli­cat­ing vec­tors, or RRVs, which are en­gi­neered to se­lec­tive­ly de­liv­er ther­a­peu­tic genes in­to can­cer cells. The ex­per­i­men­tal brain can­cer ther­a­py in ques­tion com­bines an RRV, called To­ca 511, with an in­ves­ti­ga­tion­al small mol­e­cule, To­ca FC.

To­ca 511 is de­signed to iso­late can­cer cells and de­liv­er a gene for the en­zyme, cy­to­sine deam­i­nase (CD). On­ly in­fect­ed can­cer cells are meant to car­ry and spawn CD. When To­ca FC en­coun­ters CD, it is tai­lored to trans­form in­to the an­ti-can­cer drug 5-FU, and in turn to an­nex the tu­mor.

Fol­low­ing the lay­offs, Toca­gen — which made its pub­lic de­but in 2017 — is left with 30 em­ploy­ees. They will fo­cus on the Phase II/III tri­al of To­ca 511-To­ca FC in new­ly-di­ag­nosed glioblas­toma (HGG) pa­tients — as well as an ear­ly-stage study in blad­der can­cer.

“Giv­en the To­ca 5 miss on both pri­ma­ry and sec­ondary end­points (re­lat­ing to over­all sur­vival and dura­bil­i­ty of re­sponse, re­spec­tive­ly), we main­tain low ex­pec­ta­tions (0% POS) for the To­ca 511 & To­ca FC reg­i­men in HGG in the ab­sence of new in­for­ma­tion,” Chardan an­a­lysts wrote in a note on Thurs­day.

The com­pa­ny plans to present the com­plet­ed analy­sis of the To­ca 5 tri­al at the 2019 So­ci­ety for Neu­ro-On­col­o­gy an­nu­al meet­ing in No­vem­ber, it said.

Brain can­cer is no­to­ri­ous­ly hard to treat and re­mains the dead­liest of can­cers, de­spite decades of re­search. Tu­mors are of­ten lo­cat­ed be­yond the reach of even the most con­sum­mate neu­ro­sur­geon, and pro­tect­ed by the blood-brain bar­ri­er, there­by thwart­ing ex­po­sure to sys­temic chemother­a­py. In ad­di­tion, the par­tic­u­lar de­vel­op­men­tal, ge­net­ic, and mi­cro-en­vi­ron­men­tal ecosys­tem in the brain ren­ders tu­mors re­sis­tant to tra­di­tion­al weapons in the on­col­o­gist’s ar­ma­men­tar­i­um.

In its sec­ond quar­ter re­port — pub­lished in Au­gust — Toca­gen in­di­cat­ed it had cash, cash equiv­a­lents and mar­ketable se­cu­ri­ties of $68.3 mil­lion, as of June 30. Shares of the drug de­vel­op­er $TO­CA have been lan­guish­ing in pen­ny stock ter­ri­to­ry since its piv­otal fail­ure dis­clo­sure in Sep­tem­ber.

So­cial im­age: Shut­ter­stock

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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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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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.

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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Mark Womack, BioCina CEO

Q&A: BioCi­na’s new CEO Mark Wom­ack on the CD­MO he says is 'worth trav­el­ing over'

A handful of CDMOs have made changes at the top over the past few weeks, including Genezen and Curia.

That also includes Australian CDMO BioCina, which announced last week that Mark Womack would be taking the helm. Womack previously served as chief business officer at AGC Biologics, CEO of Indian manufacturer Stelis Biopharma and most recently, CEO at CDMO KBI Biopharma and Selexis SA.

BioCina completed the takeover of a Pfizer manufacturing facility in Adelaide in 2021 and is now prepping for wider growth. Endpoints News sat down with Womack to discuss his new role, plans for the future, and how to compete in the wider CDMO market. This interview has been edited for brevity and clarity.

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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