Daniel O'Day speaks during a meeting with President Donald Trump in the Oval Office of the White House (AP Images)

FDA cheers on Gilead­'s remde­sivir with a snap emer­gency OK, giv­ing bio­phar­ma a shot at re­demp­tion

The FDA didn’t leave much time to as­sess the pos­i­tive na­ture of the first cut of da­ta from a con­trolled study of remde­sivir in se­vere­ly af­flict­ed coro­n­avirus cas­es. On Fri­day the agency stamped their ap­proval on Gilead’s drug, which NI­AID di­rec­tor An­tho­ny Fau­ci has al­ready de­ter­mined should be­come the new stan­dard of care in treat­ing hos­pi­tal­ized pa­tients fight­ing off Covid-19.

Then on Sun­day, Gilead CEO Dan O’Day took to “Face the Na­tion” to promise that the first 1.5 mil­lion dos­es of this drug — enough to treat any­where from 100,000 to 200,000 pa­tients — would be rushed, free of charge, to US cities where it is need­ed the most.

“(W)e in­tend to get that to pa­tients in the ear­ly part of this next week, be­gin­ning to work with the gov­ern­ment, which will de­ter­mine which cities are most vul­ner­a­ble and- and where the pa­tients are that need this med­i­cine.”

While Wall Street an­a­lysts have been dis­ap­point­ed by O’Day’s lack of clar­i­ty on what it plans to charge for this drug, the com­pa­ny finds it­self in a unique po­si­tion to re­frame Amer­i­cans’ at­ti­tude to­ward drug­mak­ers. Be­fore the pan­dem­ic, phar­ma ex­ecs were held in low­er es­teem than law­mak­ers, rest­ing at the bot­tom of the totem pole in terms of pop­u­lar­i­ty. Now the com­pa­ny that shocked pay­ers in­to ac­tion on high hep C drug prices is in the dri­ver’s seat on Covid-19. If they pass muster on their price now, Gilead and the Roche vet who now helms the com­pa­ny will set the pace for the en­tire in­dus­try and go a long way for a pub­lic reap­praisal of the in­dus­try.

Any mis­han­dling of this, how­ev­er, could be a dis­as­ter.

Gilead has raised geopo­lit­i­cal con­cerns by hand­ing the ini­tial drug sup­ply over to the US gov­ern­ment. The whole world is scram­bling for ther­a­peu­tic in­ter­ven­tions, and giv­ing every­thing to Amer­i­cans won’t sit well abroad.

Af­ter re­cal­i­brat­ing the pri­ma­ry end­point, re­searchers de­ter­mined that the drug cut the av­er­age time to re­cov­ery to 11 days in the drug arm, com­pared to 15 in the con­trol group. The drug arm mor­tal­i­ty rate hit 8%, beat­ing out the con­trol group at 11%, but failed to come in at a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment on the first cut of the da­ta.

The ap­proval comes just 2 months af­ter the out­break tru­ly be­gan to rat­tle the US, and 3 months af­ter the Wuhan out­break trig­gered a pan­dem­ic that has killed peo­ple and shat­tered economies around the globe, sick­en­ing mil­lions. The FDA’s ac­tion fol­lows an emer­gency use au­tho­riza­tion for hy­drox­y­chloro­quine and chloro­quine, malar­ia meds that have al­so raised safe­ty alarms on their side ef­fects.

Stephen Hahn

As FDA com­mis­sion­er Stephen Hahn ob­served, the time it took to ramp up the tri­al and gain emer­gency au­tho­riza­tion stretched over 90 days — vir­tu­al­ly light speed in drug R&D and reg­u­la­to­ry terms. The FDA has made it clear that reg­u­la­tors will move first and ask com­plex ques­tions lat­er. And in this case, there’s still much left to learn about remde­sivir, in­clud­ing the best dose and its im­pact on dif­fer­ent pa­tient groups.

An­oth­er mys­tery is how Gilead plans to make mon­ey on the drug — and how much. An­a­lysts grilled CEO O’Day on that score on Thurs­day evening, dur­ing their Q1 call, but to no avail. All that has yet to play out, as Gilead of­fers the first 1.5 mil­lion dos­es for free.

O’Day’s em­pha­sis on re­spon­si­bil­i­ty first and prof­its sec­ond, though, has some dis­count­ing any up­side for the com­pa­ny.

Evan Seiger­man at Cred­it Su­isse was one of the first an­a­lysts to make a com­ment, not­ing:

We do not ex­pect RDV to be a sig­nif­i­cant com. biz for GILD & do not mod­el any sales.

Gilead’s stock, which has surged dra­mat­i­cal­ly from the start of the year, was down close to 5% by the clos­ing bell on Fri­day.

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

CEO Stephen Yoder (Pieris)

Pieris fi­nal­ly vaults FDA hold on next-gen sol­id tu­mor hunter, clear­ing the path for mid-stage tri­al

Finally freed from the restraints of a partial FDA clinical hold on its lead HER2-positive solid tumor candidate, Pieris Pharmaceuticals is now racing toward Phase II.

The FDA slapped a partial hold on Pieris’ PRS-343 back in July, restricting the biotech from enrolling new patients in a Phase I trial. While Pieris was allowed to continue dosing patients who were already enrolled, the agency requested they conduct an additional “in-use and compatibility study” before recruiting any more.

Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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FDA hits the brakes on His­to­gen's knee car­ti­lage ther­a­py, ask­ing for more in­fo on man­u­fac­tur­ing process

A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

Andrew Allen, Gritstone CEO (Gritstone via website)

Grit­stone con­tin­ues Covid-19 push with deal to de­vel­op 'self-am­pli­fy­ing RNA' vac­cines, as shares con­tin­ue bal­loon­ing

Gritstone Oncology has had a big week, and it’s only Wednesday.

On Tuesday, the biotech revealed plans to start clinical testing of an experimental Covid-19 vaccine — in tandem with NIAID — that can also target other coronaviruses, with the goal of preventing future pandemics should SARS-CoV-2 prove difficult to cure with current vaccines. Then, on Wednesday morning, Gritstone licensed lipid nanoparticle technology from Genevant Sciences to develop what it’s calling “self-amplifying RNA vaccines” against Covid-19.

Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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Ugur Sahin, BioNTech CEO (AP Images)

Covid-19 roundup: BioN­Tech of­fers da­ta show­ing Pfiz­er-part­nered vac­cine pro­tects against vari­ant; No­vavax at­trib­ut­es re­spon­si­bil­i­ty for PhI­II de­lay to OWS

Ugur Sahin and his team at BioNTech have proffered more evidence that their Pfizer-partnered Covid-19 vaccine can protect people from a much-feared variant of SARS-CoV-2.

Colloquially known as the UK variant, the B.1.1.7 lineage triggered alarms because it appeared more transmissible. Among a series of mutations on its spike protein — the key antigen that all frontrunners in the vaccine race targeted — N501Y was of particular concern because it’s located on the receptor-binding site.

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Cardiovascular chief Joerg Koglin (Merck)

Mer­ck makes good on big CV gam­ble with veri­ciguat nod — but com­pe­ti­tion will be fierce

The results for Merck’s leading heart drug fell short of what some cardiologists had hoped for, but they proved more than enough to convince the FDA, which handed down an approval Wednesday.

The agency OK’d vericiguat, now marketed as Verquvo, for patients with heart failure, offering one of the first new pharmaceutical tools to tackle a hard-to-treat group of patients at high risk for what remains the leading cause of death in the US and Europe. It’s also a notable win for Merck, who rolled the dice on the molecule in 2016, paying Bayer $1 billion for US rights to the then-experimental drug and ex-US rights for the approved CV drug Adempas.

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