Pieris slammed with FDA hold; Bio­gen adds PhIV for Spin­raza

→ Boston-based Pieris $PIRS has been forced to hit the brakes on a slate of Phase I stud­ies for its 4-1BB/HER2 fu­sion pro­tein drug PRS-343 af­ter the FDA hit the com­pa­ny with a par­tial hold. The biotech can con­tin­ue to dose pa­tients al­ready in the stud­ies, but can’t add pa­tients un­til af­ter it con­ducts “an ad­di­tion­al in-use and com­pat­i­bil­i­ty study of PRS-343 with var­i­ous in­fu­sion ma­te­ri­als un­der spe­cif­ic con­di­tions to con­firm suit­abil­i­ty of PRS-343 for ad­min­is­tra­tion in clin­i­cal set­tings.” The biotech says no ad­verse events were cit­ed and still plans to launch their Phase II pro­gram for the drug lat­er this year — pro­vid­ed the FDA can be sat­is­fied.  “We al­so re­main on track to present com­pre­hen­sive da­ta from both the monother­a­py and ate­zolizum­ab com­bi­na­tion phase 1 stud­ies at a med­ical con­fer­ence lat­er this year,” says CEO Stephen Yo­der. Their stock was down about 6% in morn­ing trad­ing Tues­day.

Bio­gen wants to see ex­act­ly what ben­e­fits its big SMA block­buster Spin­raza can have on pa­tients who don’t re­spond suf­fi­cient­ly well to No­var­tis’ gene ther­a­py ri­val Zol­gens­ma. The big biotech says the Phase IV study was in­spired by the sto­ries of some pa­tients who went on Spin­raza af­ter the gene ther­a­py failed to prove a once-and-done treat­ment. “Avail­able da­ta now show that some pa­tients in the long-term study of Zol­gens­ma have moved on to treat­ment with Spin­raza. We be­lieve that, for cer­tain pa­tients, mo­tor neu­rons may be in­suf­fi­cient­ly treat­ed by this gene ther­a­py, and we plan to ini­ti­ate this study to un­der­stand the ex­tent to which Spin­raza may po­ten­tial­ly im­prove out­comes,” said Ma­ha Rad­hakr­ish­nan, the CMO at Bio­gen.

→ Myokar­dia is jump­ing on board Ful­crum‘s dis­cov­ery plat­form in search of new drugs to treat ge­net­ic car­diomy­opathies. The deal starts with a pay­ment of $12.5 mil­lion, with a lit­tle more than $300 mil­lion on the ta­ble for the first drug and up to $150 mil­lion more for each ad­di­tion­al pro­gram. “We have been im­pressed by Ful­crum’s abil­i­ty to dis­cov­er new bi­ol­o­gy around ge­net­ic mus­cle dis­or­ders,” not­ed Myokar­dia CSO Robert Mc­Dow­ell.

→ UPMC En­ter­pris­es, Cas­din Cap­i­tal and Dol­by Fam­i­ly Ven­tures have stepped up with an ex­tra $20 mil­lion to add to Cere­vance‘s lat­est round, bring­ing the to­tal to $65 mil­lion. Fore­site Cap­i­tal al­so chipped in some ex­tra cash on the round, which al­ready in­clud­ed GV (for­mer­ly Google Ven­tures), Bill Gates, Take­da Ven­tures, Inc., the De­men­tia Dis­cov­ery Fund and Light­stone Ven­tures. The biotech is work­ing on a pipeline of drugs aimed at a range of CNS dis­eases.

→ Ger­many’s Tubu­lis has raised €10.7 mil­lion in Se­ries A cash to back its work on an­ti­body-drug con­ju­gates. Bio­Med­Part­ners and High-Tech Grün­der­fonds co-led the launch round, with con­tri­bu­tions from some in­di­vid­ual in­vestors, the founders as well as Sev­en­ture Part­ners, co­par­i­on, Bay­ern Kap­i­tal, and Oc­ci­dent. “Tubu­lis’ ob­jec­tive is to use our dual plat­form ap­proach to gen­er­ate unique­ly matched and dis­ease-spe­cif­ic AD­Cs that com­bine se­lec­tive an­ti­bod­ies with ef­fec­tive pay­loads,” said Do­minik Schu­mach­er, CEO and co-founder of Tubu­lis.

→ In more emerg­ing-from-stealth news, Bethes­da, MD-based Gain Ther­a­peu­tics re­ports that the com­pa­ny bagged a $10 mil­lion Se­ries B for their work tar­get­ing “nov­el al­losteric bind­ing sites on en­zymes for the treat­ment of rare ge­net­ic and CNS dis­eases.” They’re set­ting up 2 IND-en­abling stud­ies for their lead pro­grams. “Our pro­pri­etary and patent­ed plat­form of­fers the abil­i­ty to dis­cov­er and val­i­date pre­vi­ous­ly uniden­ti­fied non-clas­si­cal bind­ing sites on en­zymes that can be reg­u­lat­ed by our nov­el first-in-class drug can­di­dates to re­store (or gain) en­zyme func­tion in rare and dev­as­tat­ing ge­net­ic dis­eases,” said Khalid Is­lam, the founder and chair­man of Gain.

→ A col­lab­o­ra­tion in­volv­ing phar­ma gi­ants and a trio of tech trans­fer of­fices at top UK uni­ver­si­ties has out-li­censed a gene ther­a­py to Deer­field, which plans to hand it to re­searchers at their new­ly launched Cure build­ing in the heart of New York City. The pro­gram was ini­tial­ly de­vel­oped at Uni­ver­si­ty Col­lege Lon­don, which is part of the Apol­lo Ther­a­peu­tics al­liance.

→ There has been a slew of terms set for this week’s biotech IPOs. The on­col­o­gy biotech iTeos plans to raise around $151 mil­lion by pric­ing shares at $16 to $18. Re­nais­sance Cap­i­tal is al­so track­ing a $75 mil­lion IPO from In­ozyme. An­nex­on, mean­while, is scout­ing for $150 mil­lion to back its work in de­gen­er­a­tive dis­eases.

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.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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FDA ad­comm nar­row­ly votes in fa­vor of Mer­ck­'s an­tivi­ral for out­pa­tient Covid-19

With little explanation for why Merck’s potential Covid-19 antiviral was less effective in reducing Covid hospitalizations and deaths in a full analysis of a Phase III trial versus an interim look, the FDA’s antimicrobial drugs advisory committee on Tuesday voted 13-10 in favor of the pill’s benefits outweighing the risks for adults within 5 days of developing Covid symptoms.

Molnupiravir will likely be authorized by FDA in the coming days for adults with mild or moderate Covid-19. While Pfizer’s antiviral may prove to be more effective, Merck’s pill will be another weapon in the armamentarium of Covid-19 treatments for countries around the world, adding to the mAb treatments already in use in the outpatient space from Regeneron, Eli Lilly and Vir/GlaxoSmithKline.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Ap­peals court puts the fi­nal nail in the cof­fin for Tec­fidera patent, adding to Bio­gen's bur­geon­ing set­backs

In another setback for Biogen, the big biotech lost its appeal to revive a patent for the once-blockbuster drug Tecfidera, marking a likely conclusion to the case.

The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

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Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Mar­ket­ingRx roundup: Ab­b­Vie’s Hu­mi­ra TV turns fo­cus to HS skin con­di­tion; Sanofi amps par­ent­ing pol­i­cy

After years as the top spending pharma TV advertiser, AbbVie’s Humira brand finally downshifted earlier this year, ceding much of its marketing budget to up-and-coming sibling meds Skyrizi and Rinvoq. However, now Humira is back on TV with ads for another condition — Hidradenitis suppurativa (HS).

The chronic and painful skin condition results in lumps and abscesses caused by inflammation or infection of sweat glands, most often in the armpits or groin. Humira was first approved to treat HS in 2015 and remains the only FDA-approved drug for the condition. Two TV ads both note more than 30,000 people with HS have been prescribed Humira.