No­vo Nordisk gets PhI­II he­mo­phil­ia tri­als for a Roche ri­val back on track af­ter com­plet­ing work-around on blood clot threat

Af­ter abrupt­ly shut­ting down three he­mo­phil­ia stud­ies back in March due to se­ri­ous safe­ty is­sues, No­vo Nordisk is get­ting things go­ing again.

The Dan­ish phar­ma gave the go-ahead to re­sume Phase III tri­als for its con­cizum­ab can­di­date, which are in­ves­ti­gat­ing sub­cu­ta­neous pro­phy­lax­is treat­ment in he­mo­phil­ia A and B pa­tients re­gard­less of in­hibitor sta­tus. No­vo Nordisk had halt­ed the stud­ies af­ter three pa­tients ex­pe­ri­enced non-fa­tal blood clots.

In a state­ment, the com­pa­ny said it had found a “new path for­ward” for con­cizum­ab. The FDA has agreed to new safe­ty mea­sures and guide­lines and lift­ed the clin­i­cal hold, No­vo Nordisk said.

Asked to ex­pand on these ad­di­tion­al mea­sures, a No­vo spokesper­son said they in­clude “spe­cif­ic rec­om­men­da­tions for the treat­ment of break­through bleed­ing episodes, both in terms of dos­ing and fre­quen­cy, man­dat­ing the use of low­er dos­es of ad­di­tion­al he­mo­sta­t­ic agents, as well as a new con­cizum­ab dos­ing reg­i­men, with an ini­tial dai­ly dose of 0.20 mg/kg con­cizum­ab for all pa­tients.”

These tri­als had ini­tial­ly be­gun in late 2019 af­ter No­vo had re­leased pos­i­tive proof-of-con­cept da­ta. Con­cizum­ab is an an­ti-tis­sue fac­tor path­way in­hibitor that tar­gets Fac­tor Xa, at­tempt­ing to lim­it its ac­ti­va­tion. If the can­di­date works, it could re­store the de­fi­cien­cies present in he­mo­phil­ia A (fac­tor VI­II) and he­mo­phil­ia B (fac­tor IX).

He­mo­phil­ia drugs are de­signed to pre­vent un­con­trolled bleed­ing in pa­tients and the cur­rent mar­ket leader is Roche’s Hem­li­bra, which No­vo had hoped to ri­val. So far in 2020, Hem­li­bra has net­ted Roche over $700 mil­lion in US sales and topped $1 bil­lion glob­al­ly. The drug ac­counts for 23% of US pa­tient share, ac­cord­ing to the phar­ma’s sec­ond quar­ter earn­ings re­port.

But the he­mo­phil­ia are­na has been trend­ing to­ward new­er gene ther­a­py treat­ments for some time. Roche’s own ther­a­py, com­ing out of the biotech Spark Ther­a­peu­tics, ran in­to some bumps back in 2017 for he­mo­phil­ia A and now ex­pects to start a Phase III some­time in 2021. That put it be­hind fron­trun­ner Bio­Marin, which ex­pects an FDA de­ci­sion on its he­mo­phil­ia A pro­gram be­fore the end of Au­gust.

Should Bio­Marin re­ceive ap­proval, it would be the world’s first gene ther­a­py pro­gram ap­proved for he­mo­phil­ia A. But it’s shown signs of trou­ble as well, as mean Fac­tor VI­II ac­tiv­i­ty lev­els have slid in each suc­ces­sive year. CEO JJ Bi­en­aimé has in­di­cat­ed the biotech in­tends to charge some­where in the $2 mil­lion to $3 mil­lion range, a price tag that could make the ther­a­py the most ex­pen­sive sin­gle-use ther­a­py.

Sang­amo and Pfiz­er are al­so in the he­mo­phil­ia gene ther­a­py game, and while they’re be­hind both Bio­Marin and and Spark, stronger da­ta on dura­bil­i­ty could pre­vent com­peti­tors from cap­tur­ing and keep­ing mar­ket share.

Biotech IPOs: New Steps to Suc­cess on the Road to Go­ing Pub­lic

By RBC Capital Markets

Key Points

2020 is on track to post the highest number of biotech IPOs in five years
COVID-19 has created a new roadmap for going public
Crossover investors are becoming even more active
Companies are going public with strong balance sheets

As the search for COVID-19 vaccines and therapeutics continues, investor interest in biotech IPOs has surged. 2020 is on track to post the highest number of biotech IPOs in five years.

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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The pri­ma­ry failed. The key sec­ondary failed. But this biotech still be­lieves it can win an FDA OK in ALS

Two years after the executive team at BrainStorm Cell Therapeutics decided to back off a controversial attempt to sell their stem cell therapy for ALS under the new ‘Right to Try’ legislation, the biotech is back with the top-line data from Phase III. And the data aren’t good.

Researchers say the drug — with a once-proposed price of $300,000 — failed the primary endpoint as well as the key secondaries on disease progression. But the executive crew still thinks it’s approvable. And in fact, the biotech also insists the FDA is eager to review it.

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Simeon George, SR One CEO (SR One)

Scoop: SR One crew com­pletes a com­pli­cat­ed spin­out from Glax­o­SmithK­line. And now they have a $500M fund to in­vest on their own

It’s taken close to 2 years, but Simeon George and his team at SR One have completed their spinout from GlaxoSmithKline, ending a saga as one of the longest running venture arms of Big Pharma as they go out on their own to forge the next chapter with a new and independent $500 million fund.

GSK is sticking with the spinout, this time as a minority investor — though a big one. And I’m told that the R&D group at GSK will remain involved in evaluating their new plays, helping with the scientific due diligence involved in scouting the world for new opportunities during a period of explosive growth in biotech investing.

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CEO Matthew Kane (Precision BioSciences)

In an ap­par­ent R&D about-face, Eli Lil­ly part­ners with Pre­ci­sion Bio­Sciences on genome edit­ing in a deal worth up to near­ly $2.7B

As a large multinational corporation, Eli Lilly has their hands in boundless projects, from cancer and immuno-oncology to diabetes, psoriasis and Crohn’s disease. But Friday they signaled a shift in their R&D focus toward genome editing, leaping into a cutting-edge field CEO Dave Ricks had shied away from as recently as January 2019.

The big pharma is ponying up $100 million upfront to partner with Precision BioSciences, focusing initially on Duchenne muscular dystrophy and two other undisclosed in vivo targets. Lilly is also acquiring $35 million worth of the biotech’s stock, and has the option to develop three additional in vivo therapies.

CEO Matt Kapusta (uniQure)

Look­ing for leg up in he­mo­phil­ia B gene ther­a­py race, uniQure flash­es da­ta that helped lead to $2B CSL deal

In the two-headed race to develop the first gene therapy for hemophilia B, uniQure just got a major leg up.

In a late-breaking abstract at ASH, the company released data from their pivotal 54-person Phase III trial, showing that it increased levels of Factor IX from below 2% of normal to 37% of normal after 26 weeks. Although the company will still need to collect 52-week data, the levels come in above what Pfizer has shown in early studies for their rival gene therapy and at or above what analysts suggested would be needed to show efficacy.

Albert Bourla, Pfizer CEO (Steven Ferdman/Getty Images)

It's of­fi­cial: Pfiz­er and BioN­Tech have sub­mit­ted their Covid-19 vac­cine to the FDA -- and the agency cir­cled a date for the ad­comm

Pfizer and BioNTech’s request for emergency authorization of its Covid-19 vaccine is now in the FDA’s hands. Regulators caught the application Friday afternoon in an open field dash to the goal line.

The agency immediately marked Dec 10 on the calendar for an advisory committee meeting to discuss the request. Pfizer and BioNTech first announced the submission was coming on Friday morning, and Pfizer CEO Albert Bourla confirmed the filing in a video statement later that afternoon. The “historic day,” as Bourla called it, came just 248 days after the pharma first announced plans to develop the candidate with BioNTech — a process which typically takes years.

Thomas Kuhn, Poxel

UP­DAT­ED: Vivek Ra­maswamy’s Meta­vant hits a brick wall, aban­don­ing a lead pro­gram for di­a­betes. And there’s noth­ing vis­i­ble left to see here

Just a few years after Vivek Ramaswamy’s epic Alzheimer’s fail, another one of his startups is cutting its losses because of a high-risk endeavor — this time on the diabetes front — didn’t pan out.

Metavant, Ramaswamy’s biotech #7, has decided not to advance its lead candidate imeglimin into Phase III, instead of looking for a quick sell-off. If it doesn’t secure a deal within 60 days, the rights go back to Merck KGaA spinout Poxel, which licensed the drug to Roivant — the parent company to Ramaswamy’s suite of “vants” —  back in 2018 for $50 million in cash and up to $600 million in milestones.

Jen­nifer Zi­olkows­ki re­signs from CFO post at Sol­id Bio­sciences; Sean Bo­hen's Ole­ma On­col­o­gy adds San­dra Horn­ing to board of di­rec­tors

Jennifer Ziolkowski has decided to step down from Ilan Ganot’s team at Solid Biosciences “to pursue a new career opportunity,” according to an SEC document filed Friday. Ziolkowski will continue to be Solid’s CFO until Jan. 29, 2021. The day after she announced her resignation at the company, the Duchenne muscular dystrophy biotech entered into a consulting agreement with Danforth Advisors, at which point Danforth managing partner Stephen DiPalma was named interim CFO. DiPalma has been a CFO before at Forum Pharmaceuticals and Aquila Biopharmaceuticals, and he founded Catalyst Oncology and Athena Diagnostics.

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