John Maraganore joins up with Pro­QR as strate­gic ad­vi­sor; Servi­er nabs pri­or­i­ty re­view for Tib­so­vo sN­DA

Af­ter a month-long break from ad­vi­so­ry ap­point­ments, John Maraganore is once again be­ing tapped to join an­oth­er biotech.

The RNA-fo­cused start­up an­nounced Maraganore’s ap­point­ment this morn­ing, with the Al­ny­lam found­ing CEO join­ing up as a strate­gic ad­vi­sor to Pro­QR’s su­per­vi­so­ry board.

Mon­day’s move comes a few weeks af­ter the biotech’s lead can­di­date for the rare ge­net­ic eye dis­ease, Leber con­gen­i­tal amau­ro­sis, had shown no dif­fer­ence be­tween place­bo and any oth­er ef­fi­ca­cy mea­sures. Maraganore said in a state­ment that he first met Pro­QR CEO Daniel de Boer more than a decade ago through for­mer Gen­zyme CEO Hen­ri Ter­meer, be­fore his pass­ing in 2017.

With the new gig at Pro­QR, it brings Maraganore up to 11 po­si­tions across biotech and VC since he an­nounced last year he would be leav­ing the com­pa­ny he found­ed. — Paul Schloess­er

FDA grants pri­or­i­ty re­view for Servi­er’s plans to move Tib­so­vo up the lines

Servi­er is one step clos­er to the bright fu­ture it en­vi­sions for Tib­so­vo.

The FDA has grant­ed pri­or­i­ty re­view for its sN­DA tar­get­ing pa­tients with pre­vi­ous­ly un­treat­ed IDH1-mu­tat­ed acute myeloid leukemia, the French drug­mak­er said.

First de­vel­oped by Agios and even­tu­al­ly sold to Servi­er as part of an on­col­o­gy busi­ness in a $2 bil­lion-plus deal, Tib­so­vo tar­gets can­cer me­tab­o­lism by hit­ting mu­tat­ed IDH1 en­zymes. It’s cur­rent­ly ap­proved for that par­tic­u­lar sub­set of re­lapsed or re­frac­to­ry AML — as well as new­ly di­ag­nosed IDH-1 mu­tat­ed AML pa­tients who are old­er than 75 or have co­mor­bidi­ties that make in­ten­sive chemother­a­py a bad idea. More re­cent­ly, Servi­er nabbed an OK to treat IDH1-mu­tat­ed cholan­gio­car­ci­no­ma.

In a Phase III study fo­cus­ing on pre­vi­ous­ly un­treat­ed AML pa­tients, Agios and Serv­er showed that Tib­so­vo not on­ly im­proved event-free sur­vival, but al­so helped pa­tients live longer when giv­en in com­bi­na­tion with chemother­a­py. — Am­ber Tong

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Monopar puts a stop to its chemo com­pli­ca­tion drug; Ex­i­cure ex­plores fu­ture trans­ac­tions

The biotech Monopar Therapeutics is putting a kibosh on a study for its drug to prevent severe oral mucositis (SOM) in patients who are going through chemoradiotherapy for oropharyngeal cancer.

The interim analysis for Monopar’s Phase IIb/III had around 50% of the planned patients enrolled. An independent data safety monitoring board let Monopar know that the trial did not meet the threshold for efficacy. The trial was looking for a 15% of absolute difference in SOM prevention between its drug, Validive, and placebo. Monopar will now discontinue the study as well as the development of Validive.

FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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