Alon Seri-Levy, Sol-Gel Technologies CEO

Bridge­Bio com­pa­ny sells off rare dis­ease can­di­date to Gal­der­ma part­ner

Is­raeli biotech Sol-Gel Tech­nolo­gies an­nounced Fri­day that it got its hands on a rare dis­ease drug can­di­date from Pelle­Pharm for al­most $75 mil­lion, amid claims that the drug has the po­ten­tial to reach a $300 mil­lion mar­ket.

Ex­ecs said on a con­fer­ence call Fri­day morn­ing that patideg­ib, a hedge­hog sig­nal­ing path­way block­er, is be­ing in­ves­ti­gat­ed to treat Gor­lin syn­drome, a rare ge­net­ic dis­or­der that in­creas­es the risk of de­vel­op­ing cer­tain kinds of can­cer such as basal cell skin can­cer and medul­loblas­toma, a type of brain can­cer. The dis­ease af­fects around one in every 31,000 peo­ple, and an es­ti­mat­ed 70,000 peo­ple world­wide.

The biotech said that it will be pay­ing Pelle­Pharm $4.7 mil­lion up­front with an ad­di­tion­al $70 mil­lion in de­vel­op­ment, NDA and com­mer­cial mile­stones, plus sin­gle-dig­it roy­al­ties. The deal is ex­pect­ed to close on Jan. 30.

Sol-Gel, which al­ready has two FDA-ap­proved der­ma­tol­ogy treat­ments with part­ner Gal­der­ma, said that this new ac­qui­si­tion re­flects a shift in fo­cus for the com­pa­ny. Per board chair­man Mori Arkin Fri­day morn­ing, the com­pa­ny is piv­ot­ing from der­ma­to­log­i­cal prod­ucts in a “very crowd­ed mar­ket” to in­stead push­ing “for more unique prod­ucts, for more lu­cra­tive prod­ucts that cater to rare dis­ease and to re­al prob­lems.”

“This will be al­so our strat­e­gy for the fu­ture,” Arkin re­it­er­at­ed.

A query to Sol-Gel by End­points News was not im­me­di­ate­ly re­turned.

Pelle­Pharm is a Bridge­Bio com­pa­ny that has been qui­et in re­cent years, with its last press re­lease hav­ing been pub­lished in Oc­to­ber of 2020. Back in 2018, Leo Phar­ma took a mi­nor­i­ty stake in Pelle­Pharm, agree­ing to pro­vide R&D sup­port to the com­pa­ny that was plan­ning on test­ing patideg­ib in a Phase III.

Ac­cord­ing to the com­pa­ny’s LinkedIn page, on­ly five em­ploy­ees re­main at Pelle­Pharm.

Sol-Gel is plan­ning its own Phase III tri­al, which will in­clude se­lect­ing pa­tients that have a spe­cif­ic mu­ta­tion. Sol-Gel’s study is ex­pect­ed to be­gin in the sec­ond half of this year, with re­sults ex­pect­ed by the end of 2025.

Sol-Gel added that the drug, al­ready hav­ing or­phan drug des­ig­na­tion from both the FDA and EMA, on top of FDA’s break­through ther­a­py des­ig­na­tion, may have enough sup­port for ap­proval from a sin­gle piv­otal tri­al, ac­cord­ing to the agen­cies.

Sol-Gel al­so is­sued a sec­ond press re­lease Fri­day, where it an­nounced it was rais­ing $22.8 mil­lion via con­cur­rent di­rect and pri­vate place­ment of­fer­ings. In­vestors in­clud­ed Armistice Cap­i­tal and M. Arkin Der­ma­tol­ogy Ltd., an en­ti­ty whol­ly-owned by Arkin.

“I’m very hap­py to pro­vide my own cap­i­tal to this project, and to con­tin­ue to sup­port the com­pa­ny all the way un­til the suc­cess of this project,” Arkin added on the con­fer­ence call.

Shares of Sol-Gel $SLGL went down slight­ly af­ter the mar­ket opened Fri­day.

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.

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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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.

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.

Geoff McDonough, Generation Bio president and CEO

Mod­er­na part­ners on non-vi­ral gene ther­a­py with Gen­er­a­tion Bio af­ter swing­ing gene edit­ing deals

Moderna has inked a five-year partnership with gene therapy biotech Generation Bio, it announced Thursday morning, wading deeper into the genetic medicines space as it navigates beyond its vaccine work.

Moderna will pay Generation Bio $40 million upfront and invest another $36 million into the gene therapy biotech. In exchange, Moderna can license Generation Bio’s non-viral gene therapy platforms for two immune cell programs and two liver programs, with an option for a fifth program. Moderna will fund all the research work under the partnership, and could be on the hook for milestone, fee and royalty payments totaling up to $1.8 billion, a company spokesperson tells Endpoints News.

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Steven James, Pionyr Immunotherapeutics CEO

Gilead pass­es on ful­ly ac­quir­ing Pi­o­nyr, as eyes now turn to Tizona, a fel­low sum­mer 2020 buy­out op­tion

Gilead and Pionyr Immunotherapeutics, a biotech trying to follow up on the first generation of checkpoint inhibitors, have “mutually agreed” on a rewrite to their 2020 terms, with Gilead deciding not to buy out the company.

The California biopharma waived its option to acquire the remaining 50.1% of Pionyr, which would have triggered a $315 million upfront payment and up to $1.15 billion down the road. Had Gilead waited to decide, the drugmaker would have had a potential payment to make in the near term under their agreement, a spokesperson said in an email to Endpoints News.

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