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.”

Eli Lilly CEO David Ricks at the Rose Garden, May 26, 2020 (Evan Vucci/AP Images)

Eli Lil­ly lines up a block­buster deal for Covid-19 an­ti­body, right af­ter it failed a NI­AID tri­al

Two days after Eli Lilly conceded that its antibody bamlanivimab was a flop in hospitalized Covid-19 patients, the US government is preparing to make it a blockbuster.

The pharma giant reported early Wednesday that it struck a deal to supply the feds with 300,000 vials of the drug at a cost of $375 million — once it gets an EUA stamp from the FDA. And once that 2-month supply deal is done, the government has an option on another 650,000 doses on the same terms — which could potentially add another $812 million.

En­her­tu picks up an­oth­er win for As­traZeneca and Dai­ichi Sankyo, join­ing the pri­or­i­ty re­view lane for gas­tric can­cer

Five months after Enhertu received twin breakthrough therapy designations, AstraZeneca and Daiichi Sankyo are one step closer to nabbing another approval for their potential blockbuster drug.

The companies announced Wednesday morning that their billion-dollar antibody-drug conjugate has received priority review for HER2 positive metastatic gastric cancer. Already approved in the US for third-line metastatic breast cancer patients that are HER2 positive, Enhertu’s gastric cancer PDUFA date is scheduled for the first quarter of 2021.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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Chi­nese rare dis­ease play­er inks first deal around nar­colep­sy drug Wak­ix af­ter grab­bing $80M to build an ecosys­tem

Two months ago, the narcolepsy therapy Wakix propelled Harmony Biosciences to a $128 million debut on Nasdaq. Now, the same drug is serving as the foundation for a Chinese biotech looking to pioneer a rare disease platform in the country.

Citrine Medicine — which closed $80 million in Series A funding in July — was incubated by F-Prime and Eight Roads, two VC funds affiliated with Fidelity Investments that saw an opening in China to replicate in the vibrant orphan drug landscape in the US (and to a lesser extent, Europe).

Sci­en­tists warn Amer­i­cans are ex­pect­ing too much from a coro­n­avirus vac­cine

The White House and many Americans have pinned their hopes for defeating the Covid-19 pandemic on a vaccine being developed at “warp speed.” But some scientific experts warn they’re all expecting too much, too soon.

“Everyone thinks COVID-19 will go away with a vaccine,” said William Haseltine, chair and president of Access Health International, a foundation that advocates for affordable care.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early- and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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