Plot­ting clin­i­cal en­try, James Wilson's gene ther­a­py start­up brings in bil­lion­aire for $110M Se­ries B

James Wil­son

The gene ther­a­py biotech that James Wil­son helped found to take some rare dis­ease pro­grams out of Penn all the way to an ap­proval has scored $110 mil­lion in its lat­est fi­nanc­ing.

Pas­sage Bio is get­ting a boost just sev­en months af­ter launch­ing with an­oth­er megaround. Ac­cess Biotech­nol­o­gy — the ther­a­peu­tic-fo­cused in­vest­ment arm of bil­lion­aire Len Blavat­nik’s con­glom­er­ate — is lead­ing the Se­ries B, which al­so in­clud­ed all the mar­quee in­vestors who pro­vid­ed the ini­tial $115 mil­lion: Or­biMed, Fra­zier Health­care Part­ners, Ver­sant Ven­tures, Lily Asia Ven­tures, New Leaf Ven­ture Part­ners and Vi­vo Cap­i­tal. New back­ers in­clude Box­er Cap­i­tal of Tavi­s­tock Group, High­line Cap­i­tal Man­age­ment, Lo­gos Cap­i­tal and Sphera Funds Man­age­ment.

Stephen Squin­to

The com­pa­ny is on track to ini­ti­ate clin­i­cal tri­als for its two lead pro­grams in the first half of 2020 as planned. True to its stat­ed mis­sion of tack­ling mono­genic CNS dis­ease, the ther­a­pies would treat GM1 gan­gliosi­do­sis, a neu­ron-de­stroy­ing dis­or­der most com­mon in in­fants, and fron­totem­po­ral de­men­tia, which over­whelm­ing­ly af­fects the el­der­ly, re­spec­tive­ly.

A third pro­gram in Krabbe dis­ease — in­tend­ed to fix a ge­net­ic de­fect caus­ing a short­age of galac­to­syl­ce­rami­dase, an im­por­tant en­zyme for mak­ing myelin — is ex­pect­ed to fol­low lat­er in the year.

Keep­ing the stream of pro­grams flow­ing, Pas­sage Bio dis­closed that it’s li­censed two ad­di­tion­al in­di­ca­tions from the Uni­ver­si­ty of Penn­syl­va­nia while keep­ing an op­tion to grab sev­en more.

Tachi Ya­ma­da

Once Penn’s Gene Ther­a­py Pro­gram — where Wil­son is di­rec­tor — com­pletes the IND-en­abling pre­clin­i­cal work with the help of the Or­phan Dis­ease Cen­ter, the ba­ton is passed to Pas­sage Bio for all clin­i­cal de­vel­op­ment, reg­u­la­to­ry strat­e­gy and even­tu­al com­mer­cial­iza­tion.

“As we con­tin­ue to work to­ward ad­vanc­ing our three lead pro­grams in­to the clin­ic over the com­ing year, we are fo­cused on our mis­sion to serve pa­tients by of­fer­ing best-in-class, life-trans­form­ing ther­a­pies,” said Stephen Squin­to, the Alex­ion co-founder and Or­biMed part­ner who is run­ning the shop as in­ter­im CEO, in a state­ment.

Liam Rat­cliffe, the new­ly mint­ed head of Ac­cess Biotech­nol­o­gy, is join­ing the board chaired by Tachi Ya­ma­da.

Ya­ma­da, a VC at Fra­zier for­mer­ly of Glax­o­SmithK­line and Take­da, was an old men­tor of Wil­son’s who of­fered cru­cial sup­port in the im­me­di­ate af­ter­math of a clin­i­cal tri­al that led to the death of a teenag­er and sent the gene ther­a­py field in­to a harsh win­ter.

FDA hands Mor­phoSys and In­cyte a quick OK on their po­ten­tial block­buster CAR-T al­ter­na­tive

Nearly three years after okaying the CAR-Ts Yescarta and Kymriah, the FDA has approved a new CD19 therapy.

MorphoSys’ Monjuvi, or tafasitamab-cxix, was cleared Friday for use in refractory diffuse large B-cell lymphoma (DBLCL). The approval sets up both MorphoSys and their commercial partner Incyte to compete with Gilead and Novartis in the ultra-competitive indication, where similar trial results and far easier delivery could allow them to cut a fair share of the market.

J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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Rich Heyman (ARCH)

Rich Hey­man joins PMV Phar­ma, a p53 biotech, as it adds $70 mil­lion in Se­ries D

Less than a year after pulling in an impressive $62 million Series C round, PMV Pharma is back at it again.

The Cranbury, NJ-based biotech announced Monday an additional $70 million in Series D financing as it seeks to develop cancer therapies targeting p53 mutations. Additionally, PMV also introduced longtime biotech entrepreneur Rich Heyman as chairman of the board of directors.

“This financing provides PMV Pharma with the resources to expand our pipeline and to potentially advance multiple p53 therapies into the clinic,” said PMV president and CEO David Mack in a statement.

Covid-19 roundup: Eli Lil­ly retro­fits RVs for first-of-its-kind an­ti­body tri­al with NIH; Am­gen, Ab­b­Vie, Take­da team on a drug

Eli Lilly and the NIH are about to start a first-of-its-kind trial that researchers and developers have talked about for months as a way of providing temporary immunity to the most at-risk populations.

Lilly announced this morning that it will start a 2,400-person trial with the National Institute for Allergy and Infectious Diseases to test whether its experimental Covid-19 neutralizing antibody can prevent people in nursing homes and assisted living facilities from developing the disease. The idea, known as passive immunity, is that rather than waiting on a vaccine to induce people to develop antibodies, doctors can give them lab-grown antibodies. Ideally, those antibodies will either attack the new SARS-CoV-2 infection, if the patient has recently been exposed, or persist in the blood for several weeks and prevent infection or disease for that period.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

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Sanofi un­der for­mal in­ves­ti­ga­tion for De­pakine al­le­ga­tions; Beam li­cens­es CAR-T tech from Ox­ford Bio­med­ica

Sanofi is facing a formal investigation on manslaughter charges, due to accusations that its epilepsy drug Depakine caused birth malfunctions and slow neurological development when taken during pregnancy.

The French pharma was formally charged in February, years after evidence surfaced that the drug, sodium valproate, posed neurodevelopmental risks. Sodium valproate first hit the market in 1967 for the treatment of epilepsy and bipolar disorder, and is currently prescribed in more than 100 countries.

Days af­ter seal­ing Sanofi pact, Kymera beats a path to the Nas­daq with $100M IPO pitch

Back in March, when Kymera Therapeutics closed $102 million in Series C funding led by Biotechnology Value Fund and Redmile Group, CEO Nello Mainolfi noted the protein degradation player was “at the cusp of transitioning” into a fully integrated R&D company. Five months and a major Sanofi pact later, he’s back asking for another little push to get there.

Kymera has penciled in $100 million in its first IPO pitch — although given the public market’s seemingly insatiable appetite for biotechs these days the final figure is anyone’s guess.

CymaBay flash­es pos­i­tive re­sults from the tri­al they have to re­launch

Two weeks after the FDA lifted its clinical hold on their lead drug, CymaBay said it showed positive results in an aborted Phase III trial.

The drug, a small molecule known as seladelpar, had been in development for three different liver conditions before an independent review of a NASH study last year showed that it might actually be damaging patient’s liver cells. The FDA slapped a clinical hold across all three trials, only lifting it last month when an FDA review determined that the drug hadn’t caused liver damage.

Tony Coles, Cerevel Therapeutics CEO

Adding $445M, Tony Coles and his big Pfiz­er neu­ro spin­out hitch a ride to Wall Street on Per­cep­tive’s SPAC

Two years ago, after Pfizer abruptly shut down its entire neuroscience division, Bain Capital bet $350 million that those assets were still worth something and packaged them into a new biotech: Cerevel Therapeutics. A year later, they got seasoned executive Tony Coles, who had recently jumped back into the C-suite of another neuroscience startup, to run the company.

Now Coles is steering Cerevel public, in what he says is the largest ever transaction of its kind. Cerevel has agreed to merge with Perceptive Advisors’ specialty acquisition company ARYA II. Between the roughly $125 million Perceptive raised through ARYA and an additional investment of $320 million Bain Capital, Perceptive and — yes, really — Pfizer, among others, Cerevel will now move forward with an added $445 million in its coffers.

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