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.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists

President Donald Trump, who seems intent on announcing a COVID-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

News brief­ing: Bausch Health clos­ing in on deal to ac­quire Al­le­gro as­sets; PharmAbcine strikes deal with Sam­sung Bi­o­log­ics to de­vel­op an­ti­body pro­gram

Bausch Health is closing in on a deal that would allow it to buy out all of Allegro Ophthalmics’ eye-related assets — including the rights to lead candidate risuteganib — for $50 million.

The payment would be made in two tranches: $10 million at signing, and $40 million in 2021.

Risuteganib is in clinical development for intermediate dry Age-related Macular Degeneration (AMD). It’s expected to enter two concurrent Phase III trials for that indication in the next year. The drug is also being tested in patients with diabetic macular edema (DME), and last year met the primary endpoint in a Phase II study, with 48% of patients gaining 8 or more letters in visual acuity from baseline at week 28, compared to 7% in the control group at week 12.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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Anthony Fauci (AP Images)

A press of­fi­cer at An­tho­ny Fau­ci’s NI­AID was un­masked as a hard-right Covid troll. He just re­tired to­day

William B Crews had been a public affairs specialist at the NIH’s National Institute of Allergy and Infectious Diseases.

That ended today when he informed the agency of his decision to retire, after he was identified as the managing editor at RedState, a prominent Trump loyalist website.

Crews’ RedState duties are performed under the alias streiff. While enjoying the benefits of pseudonymity, he disparaged and worked against NIAID with an incendiary level of rhetoric in the midst of a pandemic.

Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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