Fol­low­ing US, Chi­na hos­pi­tal ef­forts, Gilead plots its own PhI­II tri­als for close­ly watched Covid-19 drug

Gilead is launch­ing its own Phase III tri­als of remde­sivir, the re­pur­posed an­tivi­ral that a WHO of­fi­cial called the “one drug right now we think may have re­al ef­fi­ca­cy” against Covid-19 as the nov­el coro­n­avirus orig­i­nat­ing from Wuhan, Chi­na rav­ages the world.

Mer­dad Parsey

An­nounced just a day af­ter the NIH and the Uni­ver­si­ty of Ne­bras­ka Med­ical Cen­ter reg­is­tered their US-based tri­al on­line, Gilead’s pro­gram will com­prise two stud­ies en­rolling around 1,000 pa­tients be­gin­ning in March. They will re­cruit pri­mar­i­ly in Asian coun­tries but will al­so in­clude pa­tients from oth­er lo­ca­tions with “high num­bers of di­ag­nosed cas­es,” the com­pa­ny said.

The news al­so came on the day that the num­ber of new cas­es re­port­ed out­side Chi­na of­fi­cial­ly sur­passed that in­side. The virus dubbed SARS-CoV-2 has now spread to at least 44 oth­er coun­tries, in­clud­ing Brazil and Al­ge­ria.

Since Gilead dust­ed it off in late Jan­u­ary, remde­sivir — a drug orig­i­nal­ly de­vel­oped for Ebo­la — quick­ly emerged as a fron­trun­ner in the race to find an ef­fec­tive treat­ment for res­pi­ra­to­ry dis­ease caused by SARS-CoV-2. Af­ter re­port­ing promis­ing in vit­ro da­ta, sci­en­tists from the Wuhan In­sti­tute of Vi­rol­o­gy even tried to patent the drug while Bright­Gene said it’s mass pro­duc­ing the ac­tive phar­ma­ceu­ti­cal in­gre­di­ent.

Chi­nese of­fi­cials an­nounced on Thurs­day that Gilead has been grant­ed three of the eight patents it’s filed for since 2011, though the one spec­i­fy­ing the use in coro­n­avirus­es is still un­der re­view.

Sev­er­al hos­pi­tals in Chi­na have ini­ti­at­ed clin­i­cal stud­ies with do­nat­ed drug and sci­en­tif­ic in­put from Gilead. The first re­sults are ex­pect­ed in April, de­spite strin­gent re­quire­ments that re­port­ed­ly slowed down en­roll­ment.

“Gilead’s pri­ma­ry fo­cus is on rapid­ly de­ter­min­ing the safe­ty and ef­fi­ca­cy of remde­sivir as a po­ten­tial treat­ment for COVID-19, and this com­ple­men­tary ar­ray of stud­ies helps to give us a more ex­pan­sive breadth of da­ta glob­al­ly on the drug’s pro­file in a short amount of time,” Gilead CMO Mer­dad Parsey said in a state­ment.

The Gilead stud­ies will eval­u­ate two dos­ing du­ra­tions — 5 or 10 days — of remde­sivir. One tri­al is de­signed for 600 pa­tients with se­vere clin­i­cal man­i­fes­ta­tions, who will be giv­en the drug in ad­di­tion to stan­dard of care; the oth­er will in­volve 400 mod­er­ate pa­tients and com­pare the treat­ment to stan­dard of care. Nor­mal­iza­tion of fever and oxy­gen sat­u­ra­tion will be the key met­ric for the first while the sec­ond will pri­mar­i­ly mea­sure pro­por­tion of pa­tients dis­charged.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

The mi­cro-cap that tapped a mask-skep­tic con­gress­man for their Covid DSMB is ap­ply­ing for an EUA. Their ev­i­dence? 21 pa­tients

NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.

Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to discuss their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.

J&J re­leas­es PhI­II safe­ty blue­print for Covid-19 vac­cine tri­al. How does it stack up to Mod­er­na, Pfiz­er and As­traZeneca?

Along with the initiation of its Phase III Covid-19 vaccine study announced Wednesday morning, Johnson & Johnson also released its trial protocol, giving an inside look at how the company is conducting its late-stage research.

The move comes after the other three companies conducting Phase III’s in the US — Moderna, Pfizer and AstraZeneca — each disclosed their own trial blueprints within the last week. Though the release of such protocols is typically done after trials have been completed, drug developers had come under intense pressure after a brief safety scare in an AstraZeneca trial and amid growing concern of a politically motivated vaccine authorization.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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