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.

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.

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.

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.