Prax­is Pre­ci­sion Med­i­cines launch­es with $100M and bold sights on long-eva­sive neu­ro­log­i­cal dis­or­ders

It’s go­ing to be the era of neu­ro­science, Roche CEO Bill An­der­son de­clared on stage at the JP Mor­gan Health­care Con­fer­ence in Jan­u­ary. The field, he said, had “the po­ten­tial to be in the ‘20s what on­col­o­gy was for the last decade.”

Five months in­to that decade, a new biotech is emerg­ing from stealth mode with large in­vest­ments from Black­stone and two drugs al­ready in Phase II, one of them near­ing a piv­otal tri­al. Called Prax­is Pre­ci­sion Med­i­cines, since 2016 it’s raised $100 mil­lion — with No­vo Hold­ings, Vi­da Ven­tures and Even­tide al­so chip­ping in — to back a bet that, by find­ing the un­der­ly­ing cause of rare neu­ro­log­i­cal dis­eases, they could find and treat mech­a­nisms be­hind more com­mon ones.

For all An­der­son’s op­ti­mism about the fu­ture, though, Prax­is emerges at a tough time for neu­ro­science-fo­cused biotechs. Much of the rest of Big Phar­ma has all but aban­doned the field. The last ma­jor piece of neu­ro-news of the pre­vi­ous decade was the an­nounce­ment, in De­cem­ber, that Sage Ther­a­peu­tics’ vaunt­ed drug for ma­jor de­pres­sive dis­or­der had failed a large tri­al, a read­out that has since cost the com­pa­ny $6 bil­lion in mar­ket cap and in­duced them to cut more than half their staff. Prax­is’s lead drug goes af­ter the same in­di­ca­tion.

Mar­cio Souza

“It’s a chal­leng­ing field,” Prax­is CEO Mar­cio Souza ac­knowl­edged in an in­ter­view.

The com­pa­ny be­gan as an ef­fort to find de no­vo mu­ta­tions that caused epilep­sy — the spon­ta­neous ge­net­ic mal­func­tions that cause the dis­ease in pa­tients who did not in­her­it it from their par­ents. The search turned up, among oth­er things, a gene that af­fects cal­ci­um chan­nels in the brain. But in­stead of try­ing to fix that mu­ta­tion, they used that in­for­ma­tion to fig­ure out how that chan­nel — and how that chan­nel falling out of bal­ance, with neu­rons fir­ing too much or too lit­tle —played a role in oth­er dis­eases.

“What be­came quite clear is that when you were look­ing be­yond just the pure mu­ta­tions … we were all talk­ing about im­bal­ances in a giv­en part of the brain, and nor­mal­ly as it re­lates to a spe­cif­ic chan­nel,” Souza said. “A lot of peo­ple jumped in the past from mu­ta­tion — cor­rect­ing that mu­ta­tion, or cor­rect­ing the ge­net­ic de­fect. What we’re do­ing dif­fer­ent­ly is look­ing in­to how that man­i­fests and at­tempt­ing to cor­rect the ac­tu­al man­i­fes­ta­tion.”

The re­sult was PRAX-944, a T-type cal­ci­um chan­nel block­er that is in the ear­ly stages of de­vel­op­ment for rare forms of ge­net­ic epilep­sy, but which the com­pa­ny is more ag­gres­sive­ly tak­ing for­ward in es­sen­tial tremor, one of the more com­mon neu­ro­log­i­cal con­di­tions. They ex­pect to have Phase II proof-of-con­cept re­sults be­fore the end of the year.

The lead pro­gram, though, is PRAX-114, a de­pres­sion drug. The over­lap­ping in­di­ca­tions here are per­i­menopausal de­pres­sion, a rel­a­tive­ly rare con­di­tion com­pared with ma­jor de­pres­sive dis­or­der. De­spite the vast med­ical need and mar­ket, sci­en­tists have strug­gled for years lead­ing up to Sage’s flop to build bet­ter an­ti-de­pres­sants.

Prax­is hopes to have an an­swer on their drug soon, with plans to en­ter a piv­otal tri­al be­fore the end of the year. The drug works by al­loster­i­cal­ly tar­get­ing GABAa, the neu­ro­trans­mit­ter im­pli­cat­ed in a long list of dis­or­ders and tar­get­ed head-on by ben­zo­di­azepines. Souza said the new tri­al will try to track pa­tients as they would use it in their dai­ly lives, hop­ing to show what he says they saw in Phase II: a safe and quick drug.

“They have the right safe­ty pro­file,” Souza said. “Most of the is­sue is not on­ly with the ef­fi­ca­cy, but with the safe­ty of these com­pounds. And we think 114 has that bal­ance.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected this year.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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New safe­ty da­ta ex­pose po­ten­tial weak­ness as Pfiz­er's abroc­i­tinib takes on Dupix­ent in eczema

Last September, when Pfizer celebrated positive data from a second Phase III study of abrocitinib, many watchers applauded the efficacy but were still waiting to see whether the JAK1 inhibitor is “safe enough to be a formidable competitor to Dupixent,” the clear leader in the atopic dermatitis field. The full slate of safety data are now out and, according to one analyst, the answer is: probably not.