Jan Møller Mikkelsen, Ascendis

As­cendis wins ap­proval for once-week­ly growth hor­mone drug as Pfiz­er, Op­ko Health keep up the pres­sure

Den­mark’s As­cendis Phar­ma won its first FDA ap­proval Wednes­day af­ter­noon, beat­ing out a ri­val pro­gram at Pfiz­er for pe­di­atric growth hor­mone de­fi­cien­cy.

As­cendis notched the OK for lon­apeg­so­ma­t­ropin, to be mar­ket­ed as Skytro­fa, as the first once-week­ly treat­ment for chil­dren aged one and old­er who don’t pro­duce enough growth hor­mone and weigh at least 11.5 kilo­grams, the biotech an­nounced. There’s no in­fo on the pric­ing yet and End­points News has reached out for com­ment, but CEO Jan Møller Mikkelsen said on a con­fer­ence call Wednes­day the cost will be “re­spon­si­ble.”

Pre­vi­ous GHD treat­ments re­quire dai­ly in­jec­tions of so­ma­t­ropin, a syn­thet­ic form of hu­man growth hor­mone, but As­cendis hopes the sus­tained re­lease over week­ly dos­ing will pro­vide pa­tients with a more con­ve­nient op­tion. The com­pa­ny said the ap­proval cov­ers not on­ly the drug, but car­tridges that can be stored at room tem­per­a­ture for up to six months.

“This once-week­ly treat­ment could re­duce treat­ment bur­den and po­ten­tial­ly re­place the dai­ly so­ma­t­ropin ther­a­pies, which have been the stan­dard of care for over 30 years,” As­cendis Phase III in­ves­ti­ga­tor Paul Thorn­ton said in a state­ment.

Wednes­day’s ap­proval was based on a Phase III tri­al that en­rolled 161 chil­dren who hadn’t tak­en any pri­or treat­ment. The study com­pared lon­apeg­so­ma­t­ropin to Genotropin dai­ly in­jec­tions, and demon­strat­ed As­cendis’ pro­gram was not just non-in­fe­ri­or to af­ter one year, but al­so su­pe­ri­or. Chil­dren tak­ing the As­cendis drug grew an av­er­age of 0.9 cm per year faster than those on Genotropin.

The FDA’s de­ci­sion al­so came ear­li­er than ex­pect­ed af­ter As­cendis’ PDU­FA date was pushed back in June. Orig­i­nal­ly slat­ed for a June de­ci­sion, reg­u­la­tors de­layed the PDU­FA to Sep­tem­ber 25 af­ter As­cendis sub­mit­ted ad­di­tion­al in­for­ma­tion as part of its ap­pli­ca­tion.

At least one an­a­lyst ap­peared con­fi­dent in Skytro­fa’s abil­i­ty to shake things up. SVB Leerink’s Joseph Schwartz wrote to in­vestors Wednes­day evening that, de­spite the lim­it­ed in­for­ma­tion re­vealed on the con­fer­ence call re­gard­ing pric­ing and oth­er mat­ters, As­cendis’ fo­cus on long-term gains rather than strict­ly sales num­bers is a pos­i­tive sign.

“Con­sid­er­ing the growth hor­mone mar­ket is frag­ment­ed with mul­ti­ple un­dif­fer­en­ti­at­ed prod­ucts that must be treat­ed dai­ly, [As­cendis] be­lieves that mar­ket is primed for dis­rup­tion,” Schwartz wrote. He added that he ex­pects the drug to be ap­proved in Eu­rope by the end of the year and that As­cendis is set­ting up ac­cess pro­grams to help pa­tients switch from dai­ly treat­ment.

As­cendis’ ap­proval beat out a can­di­date tout­ed by Pfiz­er and Op­ko Health, which re­vealed its lat­est Phase III da­ta in Oc­to­ber 2019 and Oc­to­ber 2020. In the first study, the pair’s so­ma­tro­gon pro­gram showed non-in­fe­ri­or­i­ty to Genotropin and that the drug arm saw pa­tients grow at an av­er­age of 0.33 cm per year faster than stan­dard of care.

The sec­ond tri­al, mean­while, ob­served im­prove­ment on the av­er­age score on a com­mon GHD test, known as the Life In­ter­fer­ence Ques­tion­naire, af­ter 12 weeks on each treat­ment.

Pfiz­er and Op­ko are ex­pect­ed to hear back from the FDA in Oc­to­ber and are al­so hop­ing their long-act­ing hor­mone treat­ment can shake up the mar­ket, par­tic­u­lar­ly af­ter a high-pro­file flop way back in 2016. But As­cendis beat them to the punch by a lit­tle over a month, giv­ing the biotech a po­ten­tial leg up on get­ting its drug to mar­ket.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The slate of products we’re offering here at Endpoints is continuing to grow, and it’s not just limited to editorial. If you haven’t, do visit your reader profile to see if there are any other weekly newsletters you’re interested in — as each comes with its own exclusive content. And don’t miss the publisher’s note from Arsalan Arif on Endpoints Studio, our latest avenue for advertising on Endpoints.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Richard Pazdur (via AACR)

Ac­cel­er­at­ed ap­proval re­forms need mean­ing­ful con­fir­ma­to­ry tri­al im­prove­ments, pro­fes­sors write in Sci­ence

Outside of Covid-19, 2021 has been the year of the accelerated approval.

Beginning last spring, FDA openly challenged six “dangling” accelerated approvals (hadn’t confirmed their clinical benefit yet), three of which were later pulled by the companies.

Then in June, FDA pulled out the accelerated approval pathway, seemingly out of nowhere, to sign off on Biogen’s controversial Alzheimer’s drug Aduhelm. It hadn’t even been mentioned at the drug’s adcomm.

Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.