Espero Biopharma

Jan 20, 2023

Seagen just scored an­oth­er ap­proval for one of its lead drugs. Tukysa won ac­cel­er­at­ed ap­proval from the FDA on Thurs­day to be used in com­bi­na­tion with Roche’s Her­ceptin for pa­tients with a spe­cif­ic type of HER2-pos­i­tive col­orec­tal can­cer. This is the first FDA-ap­proved treat­ment in HER2-pos­i­tive metasta­t­ic col­orec­tal can­cer, Seagen said in a state­ment. FDA, which had giv­en Tukysa (tu­ca­tinib) break­through ther­a­py des­ig­na­tion and pri­or­i­ty re­view in this in­di­ca­tion, said that it was ap­proved based on a read­out from a Phase II tri­al that saw a 38% over­all re­sponse rate in 84 pa­tients, with three com­plete re­spons­es and the me­di­an du­ra­tion of re­sponse be­ing 12.4 months. Tukysa was first  ap­proved back in 2020 — in com­bi­na­tion with Her­ceptin and capecitabine — for HER2-pos­i­tive breast can­cer. Thanks to the ac­cel­er­at­ed ap­proval path­way, con­tin­ued ap­proval is still con­tin­gent on da­ta from con­fir­ma­to­ry tri­als. — Paul Schloess­er RNA drug de­vel­op­er snags fresh cap­i­tal ADARx Phar­ma­ceu­ti­cals said it has se­cured an­oth­er $46 mil­lion to take its first three RNA drugs in­to the clin­ic. The San Diego biotech start­ed test­ing its siR­NA du­plex oligonu­cleotide drug ADX-324 last month. The Phase I study is ex­pect­ed to en­roll 53 pa­tients, in­clud­ing healthy vol­un­teers and an ex­pan­sion co­hort in peo­ple with hered­i­tary an­gioede­ma, or se­vere swelling on dif­fer­ent parts of the body. The start­up’s RNA plat­form is look­ing at in­hibit­ing, de­grad­ing and edit­ing ap­pli­ca­tions, with new oligonu­cleotide de­liv­ery tech, the com­pa­ny said. ADARx has nine ac­tive pro­grams across ge­net­ic, car­diometa­bol­ic and cen­tral ner­vous sys­tem dis­eases, ac­cord­ing to the biotech’s web­site. On the RNA edit­ing front, ADARx says its first pro­gram, which aims to ed­it a sin­gle base, will go af­ter al­pha-1 an­tit­rypsin de­fi­cien­cy, an in­her­it­ed dis­or­der that can lead to liv­er or lung dis­ease. As­cen­ta Cap­i­tal led the Se­ries B-1, along with Or­biMed, SR One Cap­i­tal Man­age­ment, Lil­ly Asia Ven­tures and Sirona Cap­i­tal. The biotech pre­vi­ous­ly raised a $75 mil­lion Se­ries B , dis­closed in the fall of 2021. — Kyle LaHu­cik Car­dio biotech rais­es mi­nus­cule IPO for PhI­II A new Flori­da biotech leaped on­to Nas­daq on Fri­day morn­ing less than a year af­ter form­ing to take on blood clots. Ca­dren­al Ther­a­peu­tics raised about $7 mil­lion by pric­ing its shares $CVKD at $5 a piece. The stock price was down near­ly 10% af­ter the open­ing bell. The biotech se­cured the rights to or­phan drug-des­ig­nat­ed tecar­farin last April. Ca­dren­al bought the rights from a se­cured lender of Es­pero Bio­Phar­ma, which had tak­en the an­ti­co­ag­u­lant through mul­ti­ple mid-stage tests. Es­pero had sub­mit­ted a Phase III de­sign to the FDA in 2019 for test­ing the drug as a pre­ven­ta­tive tool for blood clots of car­diac ori­gin in pa­tients with end-stage re­nal dis­ease and atri­al fib­ril­la­tion. Ca­dren­al, in an SEC fil­ing , said it will sub­mit its Phase III de­sign to the agency us­ing the same pro­to­col as its pre­de­ces­sor. The biotech hopes to start the po­ten­tial­ly piv­otal tri­al in the sec­ond half of this year, but the drug de­vel­op­er said in its IPO pa­per­work that it would like­ly need to raise an­oth­er $40 mil­lion to com­plete the late-stage study. The rel­a­tive­ly small Nas­daq en­try comes af­ter a dras­tic drop in Wall Street de­buts for biotech star­tups over the past year, fol­low­ing an in­dus­try boon dur­ing the ear­ly days of the Covid-19 pan­dem­ic. Hy­per­ten­sion drug­mak­er Min­eralys lined up its IPO am­bi­tions yes­ter­day af­ter a few oth­er biotechs in re­cent months. — Kyle LaHu­cik AUTHORS Kyle LaHucik Associate Editor The FDA flashed a red light on Eli Lilly’s investigational Alzheimer’s drug donanemab, spurning hopes of two new medications for the disease this month. The Big Pharma put out word after the markets closed Thursday saying the US regulator denied the accelerated approval “based on limited number of patients with 12-month drug exposure data” in the submission. Patients in the trial were allowed to wean off treatment once a certain amount of amyloid protein was cleared from their brains, meaning some patients did not receive the study drug for 12 months. “No other deficiencies were identified,” Lilly said in a statement. Keep reading Endpoints with a free subscription Unlock this story instantly and join 156,500+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Kyle LaHucik Associate Editor Ipsen’s cancer drug Onivyde, when combined with chemotherapy, performed better than standard of care at extending the lives of patients with a certain pancreatic cancer. The biopharma said the trial results mark the first time an investigational drug has shown better overall survival and progression-free survival as a first-line treatment than standard of care for patients with metastatic pancreatic ductal adenocarcinoma (mPDAC). This year, about 64,000 people in the US will be diagnosed with pancreatic cancer, which has the worst five-year survival rate, according to the American Cancer Society. Keep reading Endpoints with a free subscription Unlock this story instantly and join 156,500+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Katherine Lewin News Reporter Hematology-focused Disc Medicine has agreed to pay a $10 million down payment to Mabwell Therapeutics to develop and commercialize a portfolio of monoclonal antibodies for blood disorders. It’s the first deal Disc has made since it went public in August. The initial $10 million is a small piece of a potential $412.5 million of milestone payments and royalties of “close to two digit percentage” of net sales. Disc will have rights in the US, Europe and other unnamed territories outside of China and Southeast Asia. The monoclonal antibodies target transmembrane serine protease 6. Read More