IPOs abound in the time of coro­n­avirus, as For­ma Ther­a­peu­tics pen­cils in $150M Nas­daq de­but

The IPO en­gine is thriv­ing, nev­er mind the ram­page of the coro­n­avirus cri­sis on R&D time­lines.

On Fri­day, along with syn­thet­ic lethal­i­ty-fo­cused biotech Re­pare Ther­a­peu­tics, an­oth­er Bris­tol My­ers part­ner For­ma Ther­a­peu­tics al­so un­veiled its plans to vault on to the Nas­daq — pen­cil­ing in a tar­get of $150 mil­lion.

Frank Lee

The Wa­ter­town, Mass­a­chu­setts-based com­pa­ny — which poached se­nior Genen­tech ex­ec­u­tive Frank Lee to take over the reins last year af­ter more than a decade un­der founder Steve Tre­gay — raised a plump $100 mil­lion late last year, while shep­herd­ing its sick­le cell dis­ease (SCD) drug through an ear­ly-stage tri­al.

Last No­vem­ber, the FDA ush­ered in the ap­proval of two SCD ther­a­pies, in­ject­ing some op­ti­mism in­to an oth­er­wise bar­ren treat­ment land­scape for pa­tients with the blood dis­or­der that is char­ac­ter­ized by atyp­i­cal he­mo­glo­bin mol­e­cules, which can dis­tort red blood cells in­to a sick­le, or cres­cent, shape. No­var­tis’ ther­a­py, Adakveo, is de­signed to pre­vent pe­ri­od­ic episodes of sear­ing pain called va­so-oc­clu­sive crises (VOCs) that de­prive the de­prive the body of oxy­gen-rich blood, while Glob­al Blood Ther­a­peu­tics’ vox­elo­tor is de­signed to work by in­creas­ing he­mo­glo­bin’s affin­i­ty for oxy­gen.

In­stead of ad­dress­ing symp­toms of SCD, For­ma’s lead ther­a­py, FT-4202, is de­signed to change the course of the dis­ease as an ac­ti­va­tor of the en­zyme pyru­vate ki­nase-R (PKR) to im­prove red blood cell me­tab­o­lism, func­tion and sur­vival, po­ten­tial­ly re­sult­ing in both in­creased he­mo­glo­bin lev­els and few­er VOCs. For­ma is in a crowd­ed field of ther­a­pies in de­vel­op­ment, with oth­er drug­mak­ers in­clud­ing blue­bird bio, Imara, and part­ners CRISPR Ther­a­peu­tics and Ver­tex, al­so work­ing on their own drugs.

Mean­while, For­ma al­so plans to eval­u­ate the use of FT-4202 in be­ta tha­lassemia. The com­pa­ny al­so has a brim­ming pipeline, in­clud­ing an AML drug and a NASH ther­a­py in mid-stage de­vel­op­ment, as well as ear­li­er-stage com­pounds for NASH, NHL (part­nered with Bris­tol My­ers Squibb)  and sol­id tu­mors (in col­lab­o­ra­tion with Boehringer In­gel­heim).

There has been a flur­ry of bio­phar­ma IPO’s in re­cent­ly — Gen­er­a­tion Bio, Avid­i­ty and Vax­cyte set their sights on a com­bined $325 mil­lion and the week be­fore ADC Ther­a­peu­tics raked in $233 mil­lion in an up­sized of­fer­ing — de­spite the gen­er­al pan­de­mo­ni­um on Wall Street due to Covid-19. But the life sci­ences sec­tor has emerged large­ly im­mune from the rout.

Swe­den’s Cal­lid­i­tas, which last week said it was eye­ing a $75 mil­lion raise (a mod­est sum com­pared to some of the splashier pub­lic de­buts seen in re­cent weeks), on Mon­day in­di­cat­ed it was com­menc­ing an in­vestor road­show. The com­pa­ny, which is de­vel­op­ing a ther­a­py for an or­phan kid­ney dis­ease, al­ready has a Swedish list­ing.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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Sin­gu­lar fo­cus on ROR1 earns Velos­Bio $137M to fund PhI ADC and oth­er pro­grams

Years after selling Acerta to AstraZeneca for $7 billion, largely on the promise of its BTK inhibitor, Dave Johnson has once again gathered hefty financial support behind a new cancer target.

Matrix Capital Management and Surveyor Capital are leading a $137 million round for VelosBio, which has recently begun a Phase I study for its lead antibody-drug conjugate targeted against ROR1. Johnson took up the CEO post in October 2018.

Alexander Vos, VarmX CEO

'Fun­da­men­tal­ly dif­fer­en­t' from Por­to­la, Dutch biotech lands €32M to steer an­ti-an­ti­co­ag­u­lant through the clin­ic

Portola may not have had much success proving the commercial value of an anti-anticoagulant, but that’s not stopping European investors from pouring $36.2 million (€32 million) into what they see as a superior approach put forth by a Dutch biotech.

VarmX’s blood thinner reversal agent stems from research done by founder and CSO Pieter Reitsma at Leiden University Medical Center. A modified recombinant form of factor X, VMX-C001 “has an insertion of 16 amino acids that replaces a stretch of 7 amino acids in the so-called serine protease domain” compared to the native coagulation factor, CEO Alexander Vos told Endpoints News.

FDA bars the door — for now — against Mer­ck’s star can­cer drug af­ter Roche beat them to the punch

Merck has been handed a rare setback at the FDA.

After filing for the accelerated approval of a combination of their star PD-1 drug Keytruda with Eisai’s Lenvima as a first-line treatment for unresectable hepatocellular carcinoma, the FDA nixed the move, handing out a CRL because Roche beat them to the punch on the same indication by a matter of weeks.

According to Merck:

Ahead of the Prescription Drug User Fee Act action dates of Merck’s and Eisai’s applications, another combination therapy was approved based on a randomized, controlled trial that demonstrated overall survival. Consequently, the CRL stated that Merck’s and Eisai’s applications do not provide evidence that Keytruda in combination with Lenvima represents a meaningful advantage over available therapies for the treatment of unresectable or metastatic HCC with no prior systemic therapy for advanced disease. Since the applications for KEYNOTE-524/Study 116 no longer meet the criteria for accelerated approval, both companies plan to work with the FDA to take appropriate next steps, which include conducting a well-controlled clinical trial that demonstrates substantial evidence of effectiveness and the clinical benefit of the combination.

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Covid-19 roundup: Mod­er­na sticks to Ju­ly for its Phase III as ru­mors swirl; Fol­low­ing US lead, EU buys up Covid-19 treat­ments

The Phase III might be delayed from its original early July goal, but Moderna says it will still kick off the pivotal study for what could ultimately be the first Covid-19 vaccine before the end of the month.

A day after Reuters reported that squabbling between the Cambridge biotech and government regulators had held up the trial by about two weeks, Moderna released a statement saying that they had completed enrollment of their 650-person Phase II trial and were on track to begin Phase III by the end of the month. The protocol for that study, which is meant to prove whether or not the vaccine can prevent people from becoming sick, has been finalized, they said.

Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Stephen Hahn, AP

Trump and Navar­ro press again for hy­drox­y­chloro­quine. Can the FDA stay in­de­pen­dent?

Tuesday morning, economist and Trump advisor Peter Navarro walked onto the White House driveway and promptly brought a political cloud back onto the FDA.

Speaking to a White House pool reporter, Navarro said that four Detroit doctors were, based on a single disputed study, filing for the FDA to again issue an emergency authorization for hydroxychloroquine, the anti-malarial pill that President Trump hyped for months as a Covid-19 treatment over the objections of his own scientists. Then, while avoiding directly calling for the FDA to OK the drug, blasted the agency. He said its decision to pull an earlier authorization “was based on bad science” and “had a tremendously negative effect” on doctors and patients.

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