Sharon Cunningham, Shorla Pharma CEO

Shorla Phar­ma scores an ex­pe­dit­ed re­view for its mys­tery can­di­date to treat T-cell ma­lig­nan­cies

Since launch­ing Shorla Phar­ma three years ago to de­sign bet­ter ver­sions of ex­ist­ing can­cer drugs, co-founders Sharon Cun­ning­ham and Or­laith Ryan have of­fered scarce de­tails on what it is they’re work­ing on. But the FDA clear­ly likes what it sees.

Or­laith Ryan

The agency has grant­ed pri­or­i­ty re­view to SH-111, Shorla’s mys­tery can­di­date for the third-line treat­ment of adults and chil­dren with T-cell acute lym­phoblas­tic leukemia (T-ALL) and T-cell lym­phoblas­tic lym­phoma (T-LBL), the com­pa­ny said on Fri­day.

Ryan, Shorla’s CTO, told End­points News that the can­di­date is a new take on a stan­dard-of-care treat­ment which she says is in short sup­ply, but de­clined to com­ment any fur­ther. While most leukemias tar­get old­er peo­ple, T-cell leukemia is an ag­gres­sive blood and bone mar­row can­cer that’s most com­mon among chil­dren.

“In this in­stance it’s re­al­ly a treat­ment that has fre­quent­ly been in short­age,” Ryan said, adding that man­u­fac­tur­ing has posed a chal­lenge. “Giv­en our ex­per­tise and our re­source we were in a po­si­tion to, ba­si­cal­ly, bring a scal­able and se­cure source of the drug to mar­ket.”

She and Cun­ning­ham both hail from Ire­land’s Eir­Gen Phar­ma, which was bought out by Mi­a­mi-based Op­ko Health in 2015. The move in­spired them to launch Shorla in 2018 — a com­bi­na­tion of their first names — to “reimag­ine” med­i­cines for in­di­ca­tions where ex­ist­ing treat­ments are lim­it­ed, or in­ad­e­quate, or not ef­fec­tive as they could be. Back in June, they nabbed an $8.3 mil­lion Se­ries A round led by Ser­o­ba Life Sci­ences.

“We de­cid­ed to re­al­ly fo­cus our at­ten­tion on bring­ing the prod­ucts to mar­ket in the US first, fol­lowed by Eu­rope and rest of world,” Cun­ning­ham, Shorla’s CEO said.

The Clon­mel, Ire­land-based biotech has two oth­er pro­grams — one for breast and ovar­i­an can­cer, and an­oth­er for glioma. Cun­ning­ham and Ryan plan on ap­proach­ing reg­u­la­tors with the for­mer, SH-105, in Q1 of 2022. That treat­ment is a more eas­i­ly ad­min­is­tered ver­sion of a drug that was ap­proved as a freeze-dried pow­der in the 1950s. SH-110, a liq­uid ver­sion of a hard-to-swal­low pill used to treat glioma, is like­ly head­ed for the clin­ic at the end of the year.

All three pro­grams are based on es­tab­lished drugs, though they de­clined to say what those drugs are.

“We re­al­ly be­lieve in bring­ing af­ford­able and ac­ces­si­ble treat­ments to pa­tients, and that’s very much what we in­tend to do,” Ryan said.

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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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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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

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.

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.