Phase­Bio rais­es $34M to test or­phan dis­ease strat­e­gy — fo­cus­ing first on As­traZeneca drug

Af­ter spend­ing the past three years repo­si­tion­ing it­self as an or­phan dis­ease-fo­cused biotech, Phase­Bio is ready to roll with a fresh $34 mil­lion raise.

The Malvern, PA-based com­pa­ny owes its name to its elastin-like polypep­tides plat­form tech­nol­o­gy, which cre­ates ther­a­peu­tic fu­sion pro­teins that un­der­go a ful­ly re­versible phase tran­si­tion — there­by sig­nif­i­cant­ly ex­tend­ing their half lives.

Jonathan Mow

Phase­Bio once saw an ap­pli­ca­tion for that tech in di­a­betes as well as car­dio, at­tract­ing phar­ma gi­ants As­traZeneca and John­son & John­son’s ven­ture arm, as well as VC play­ers New En­ter­prise As­so­ci­ates, Hat­teras Ven­ture Part­ners and Fletch­er Spaght Ven­tures for its 2015 Se­ries C, in which it got $40 mil­lion to, among oth­er things, ad­vance a once-week­ly, long-act­ing basal in­sulin.

But that drug, like its long-act­ing GLP-1 that de­liv­ered seem­ing­ly ap­prov­able but not great Phase IIb re­sults, has now been set aside.

“For type 2 di­a­betes, if you’re not gonna be first in class or best in class, it’s chal­leng­ing, es­pe­cial­ly as a small biotech com­pa­ny de­vel­op­ing as­sets for that space,” CEO Jonathan Mow tells me.

In­stead, Mow’s team of 20 has now found a new lead drug in PB2452, a re­ver­sal agent for the blood thin­ner Bril­in­ta (tica­grelor) for use in acute sit­u­a­tions where pa­tients are ex­pe­ri­enc­ing ac­tive bleed­ing or re­quire ur­gent surgery.

“It’s very anal­o­gous to what Por­to­la did with An­dexxa, al­though An­dexxa is in a dif­fer­ent class of agents — it’s re­vers­ing fac­tor Xa, and we are re­vers­ing tica­grelor which is P2Y12 an­tag­o­nist,” Mow says.

John Sharp

While Phase­Bio on­ly li­censed the drug from As­traZeneca — which mar­kets Bril­in­ta — late last year and is still in the mid­dle of a proof-of-con­cept study, Mow sees his team wrap­ping up a Phase II and ini­ti­ate a Phase III around the end of 2019.

“PB2452, can be de­vel­oped in a much short­er time pe­ri­od, and it will pay for us to de­vel­op PB1046,” their in-house as­set for pul­monary ar­te­r­i­al hy­per­ten­sion that’s been in the pipeline for a while, he says. “So even though it’s on­ly in Phase I, we will get to the lat­er stages of de­vel­op­ment much more quick­ly than we will with PB1046.”

That com­bi­na­tion of a short de­vel­op­ment time­line and a plat­form tech is what drew their new in­vestors — Cor­morant As­set Man­age­ment, Rock Springs Cap­i­tal and Moun­tain Group Part­ners — to par­tic­i­pate in the round, CFO John Sharp says. Old sup­port­ers al­so came back for more.

The fund­ing will al­so pay for a Phase II with PB1046 in PAH. It’s not an easy mar­ket to break in­to, but Mow says their ana­log va­soac­tive in­testi­nal pep­tide — with its 60 hour half life and nov­el mech­a­nism of ac­tion — will make room for it­self along­side the likes of Unit­ed Ther­a­peu­tics, Acte­lion and Gilead.

George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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Douglas Love, Annexon CEO (Annexon)

IPO bound? A Bay Area biotech grabs a mega-round on the road to a piv­otal neu­rode­gen­er­a­tion pro­gram

South San Francisco-based Annexon has added $100 million to its cash reserves, along with a new roster of marquee investors backing their play on the classical complement pathway involved in neurodegeneration. And that may well fit the profile for an IPO — though right now everything seems to be working on that score.

Eighteen months after Bain and their syndicate partners put up $75 million to fuel clinical work, Annexon is back at the trough. And this time they’re adding Redmile Group for the lead role, with supporting investments from these new arrivals: BlackRock, Deerfield Management Company, Eventide Asset Management, Farallon Capital Management, Janus Henderson Investors and Logos Capital.

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No­var­tis los­es biosim­i­lar ap­peal as court up­holds a 31-year mo­nop­oly by Am­gen's En­brel

A new court ruling has strengthened Amgen’s grip on the IP estate around Enbrel, keeping biosimilars of the autoimmune and inflammatory drug at bay until 2029.

Novartis, the patent challenger, isn’t throwing in the towel yet. In a statement noting the failed appeal, its generics division Sandoz noted its reviewing options, “including potential appeal to US Supreme Court.”

It’s been almost four years since the FDA approved Erelzi, Sandoz’s copycat version of Enbrel. While sales of the Pfizer-partnered drug in the US — the market Amgen is in charge of — have dipped slightly during that time, it remains a solid megablockbuster with 2019 revenue slightly above $5 billion.

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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Randy Schatzman, Bolt CEO (Bolt Biotherapeutics)

Bolt Bio­ther­a­peu­tics nabs $93.5M to push Provenge in­ven­tor's new idea deep­er in the clin­ic

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotech’s platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Josh Cohen, Justin Klee

Armed with pos­i­tive ALS da­ta, Amy­lyx scores $30M in fresh fund­ing to com­plete Alzheimer's PhII

Four years after announcing themselves to the biotech world with a new idea for drugging neurodegeneration, backing by the late Henri Termeer and $5 million from Morningside Venture, the young entrepreneurs at Amylyx are back for round 2.

Morningside continued to lead the $30 million Series B, with participation from Termeer’s widow, Belinda, and other unnamed investors. Having celebrated a topline Phase II win for its lead program in amyotrophic lateral sclerosis, Amylyx expects the cash to fund talks with regulators as well as a separate trial for the same drug in Alzheimer’s — for which they had just finished enrolling.

Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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