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.

“This in­ser­tion leaves suf­fi­cient clot­ting ac­tiv­i­ty, whilst in­tro­duc­ing vir­tu­al­ly com­plete in­sen­si­tiv­i­ty to fac­tor Xa DOACS,” he added, re­fer­ring to oral fac­tor Xa in­hibitors.

Un­like Por­to­la’s An­dexxa — which is on­ly ap­proved to quick­ly dis­arm Eliquis and Xarel­to — Var­mX’s drug promis­es to counter the ef­fect of any DOAC. The biotech said it’s al­so eas­i­er to ad­min­is­ter and doesn’t lead to pro-throm­bot­ic risk.

Re­ports sug­gest that 2% to 3% of the 10 mil­lion pa­tients tak­ing FXa DOACs ex­pe­ri­ence spon­ta­neous se­vere bleed­ing, Vos said, which could be life-threat­en­ing. And there are al­ways pa­tients on an­ti­co­ag­u­lants who need to re­store some blood clot­ting func­tions for an emer­gency surgery.

The Se­ries B is de­signed to put the drug through clin­i­cal proof-of-con­cept, all the way up to the thresh­old of reg­is­tra­tional stud­ies for both se­vere bleed­ing and emer­gency surgery.

At that point — and there’s no guar­an­tee they will get there — Var­mX will find it­self fac­ing off with Alex­ion, which bagged Por­to­la and An­dexxa in a $1.4 bil­lion buy­out in May. The ac­qui­si­tion val­ued Por­to­la at $18 per share, a frac­tion of where it was at peak.

“The da­ta gen­er­at­ed so far sug­gests that VMX-C001 can be­come a dif­fer­en­ti­at­ed treat­ment so­lu­tion in an ever grow­ing mar­ket,” Guillem La­por­ta, whose Ys­ios Cap­i­tal co-led the fi­nanc­ing with INKEF Cap­i­tal, said in a state­ment.

Lund­beck­fonden Ven­tures joined the round, to­geth­er with ex­ist­ing in­vestors Bio­Gen­er­a­tion Ven­tures and In­no­va­tion­Quar­ter.

In ad­di­tion to the lead pro­gram, the lean team of 5 at Var­mX is de­vel­op­ing re­ver­sal agents for fu­ture class­es of an­ti­co­ag­u­lants, such as those tar­get­ing fac­tor XI(a).

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.

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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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.

The IPO queue adds 5 more biotechs hop­ing to ring in 2021 by blitz­ing Nas­daq

Following a record year for IPOs — in terms of both proceeds and count — there’s already a long lineup of biotechs ready to jump onto Nasdaq in the new year. The companies are likely looking for much higher raises than they initially projected on their S-1s. Now it’s time to see if investors are still hungry for another round of biotech stocks.

Sana helped set the pace early on, as its founders look to divvy up a fortune from their IPO. And late last week 5 more biotechs crowded in, looking to pick up the pace where 2020 left off. Here they are:

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Dan Skovronsky, Eli Lilly CSO (Lilly via Facebook)

Eli Lil­ly tees up dis­cov­ery pact worth more than $1.6B with Merus for T cell-fo­cused bis­pe­cif­ic an­ti­bod­ies

Under science chief Dan Skovronsky, Eli Lilly has taken some big swings at next-gen therapies, including trying to find the next big thing in oncology. Now, after one early failure in the field, Lilly is going back to the bispecific antibody well with a new deal with a Dutch biotech.

Lilly will pay $40 million upfront with an additional $20 million equity stake in Merus NV to identify and develop three bispecific antibodies looking to engage the CD3 antigen on T cells and redirect immune cells, the Indianapolis pharma giant said Tuesday.

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Sekar Kathiresan (Verve)

'Bluest of blue-chip in­vestors' pump $94M in­to Sekar Kathire­san's quest to bring CRISPR to the heart

Going into his first JP Morgan conference, Sekar Kathiresan had some major news to share. His ambitious cardiovascular-focused startup, Verve Therapeutics, had designated the PCSK9 base editor as its lead program, homed in on severe heterozygous familial hypercholesterolemia as the first indication, and released animal data suggesting the in vivo edits to the monkey livers last up to six months. For a company that had just been in the public for less than a year, “it was wonderful.”

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CEO Marshall Fordyce (Vera)

UP­DAT­ED: An am­bi­tious GV-backed gene edit­ing up­start re-emerges as a one-drug kid­ney com­pa­ny

In September 2019, Trucode Gene Repair launched, after two-plus years of early development, with $34 million from GV and Kleiner Perkins and ambitious plans to take on the raft of CRISPR companies with a new form of gene editing.

Those plans, though, would only last a few months. By February, company leadership was already pivoting, filing a trademark and domain name for a new company, called Vera Therapeutics. On Monday, they completed the switch, “launching” with an $80 million Series C round led by Abingworth and plans to focus their efforts on atacicept, a 20-year-old molecule now in development for kidney disease.

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A car­dio­vas­cu­lar cell ther­a­py play­er grabs $54M for a new be­gin­ning — where next-gen CAR-T ap­proach fea­tures promi­nent­ly

Before the advent of CAR-T, the term cell therapy conjured up very different images. Sure, there were cancer immunotherapies like Dendreon’s Provenge, but more common were the various flavors of stem cell therapies and cell transplantation.

None of that has gone away, even if they’ve been nudged out of the spotlight — and a little biotech has garnered $54 million (£40 million) to show that both the old and new ideas of cell (and gene) therapy can mesh together to form a special breed of platform company.

Andrew Allen (Gritstone)

As coro­n­avirus vari­ants trig­ger new alarms, the NIH is putting an un­der-the-radar ‘next-gen’ vac­cine in­to PhI

Over the past year, the world has been transfixed by the development of new vaccines to fight SARS-CoV-2. In a frenzy of activity, the new mRNA approach has delivered pioneering emergency approvals in record time. And with some setbacks, the more traditional big players are coming along with added jabs as the most affluent nations in the world begin to vaccinate large portions of their populations.

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