Pfiz­er boosts its gene ther­a­py work, bags up­start Bam­boo in $645M buy­out deal

Bam­boo Ther­a­peu­tics and its nascent gene ther­a­py pipeline is be­ing bought out by Pfiz­er, just five months af­ter the biotech raised close to $50 mil­lion. The phar­ma gi­ant, al­ready a mi­nor­i­ty in­vestor in Bam­boo, is pay­ing $150 mil­lion up­front for the rest of the eq­ui­ty as it sets its sights on be­com­ing a leader in gene ther­a­py de­vel­op­ment. And there’s an­oth­er $495 mil­lion in mile­stones on the ta­ble for Bam­boo’s in­vestors.

Pfiz­er had al­ready paid $43 mil­lion to buy its first stake in Bam­boo, bring­ing its to­tal bet on the com­pa­ny to up to $688 mil­lion.

Pfiz­er is land­ing a top man­u­fac­tur­ing fa­cil­i­ty for gene ther­a­pies in the deal for the UNC spin­out. It’s al­so gain­ing a pipeline of gene ther­a­pies for rare neu­ro­log­ic dis­eases, in­clud­ing Gi­ant ax­on­al neu­ropa­thy (GAN, now in Phase I/II), Cana­van dis­ease, Friedre­ich’s atax­ia and Duchenne mus­cu­lar dy­s­tro­phy.

Jude Samul­s­ki

Gene ther­a­py ex­pert Jude Samul­s­ki, who head­ed up UNC’s Gene Ther­a­py Cen­ter, will now join the phar­ma gi­ant’s ranks.

Samul­s­ki has more than 25 years ex­pe­ri­ence de­vel­op­ing AAV vec­tors for gene ther­a­pies, us­ing a be­nign virus to de­liv­er ge­net­ic ma­te­r­i­al for a po­ten­tial once-and-done fix for de­fec­tive genes that can trig­ger dis­ease.

Samul­s­ki will be join­ing a group al­ready not­ed for its chief, Michael Lin­den, who has been steadi­ly ramp­ing up work in the R&D are­na. Pfiz­er is al­so work­ing with Spark on a gene ther­a­py for he­mo­phil­ia B. And it’s col­lab­o­rat­ing with 4D Mol­e­c­u­lar Ther­a­peu­tics on car­dio reme­dies.

Gene ther­a­pies have been mak­ing a dra­mat­ic come­back in re­cent years. Blue­bird, Spark and oth­ers have been hur­ry­ing new pro­grams along and rolling out IPOs to help sup­port their work. And just a few days ago Bio­Marin added some con­sid­er­able ex­cite­ment to the field with stel­lar da­ta from a small tri­al of its he­mo­phil­ia A drug.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

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When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

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First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

Mallinck­rodt, once the na­tion’s largest oxy­codone pro­duc­er, an­nounces ten­ta­tive $1.6B set­tle­ment

Three years after it first paid out fines for its role in the US opioid abuse epidemic, Mallinckrodt has announced an agreement-in-principle that will see the company pay out $1.6 billion and place its generics unit in bankruptcy.

The tentative deal would settle hundreds of lawsuits from state and local governments over Mallinckrodt’s role in the epidemic, while also helping address the company’s increasingly mountainous debt. Although Purdue Pharma has drawn the bulk of both public and legal acrimony for opioid sales, documents made public earlier this year showed that Mallinckrodt subsidiary SpecGx, along with the generic subsidiaries of Teva and Endo Pharmaceuticals, accounted for the vast majority of the 76 billion opioid pills distributed from 2006 to 2012. Mallinckrodt was at the top of that list.