Yvonne Greenstreet, Alnylam CEO (Suzanne Kreiter/The Boston Globe via Getty Images)

Al­ny­lam ex­pands RNAi com­mer­cial­iza­tion deal in Eu­rope

Al­ny­lam is dou­bling down on a part­ner­ship to ex­pand its com­mer­cial pres­ence in Eu­rope, as it forges ahead on for­mer CEO John Maraganore’s mis­sion to be­come a top-five biotech by the mid­dle of the decade.

The com­pa­ny an­nounced on Thurs­day that it’s ex­pand­ing a five-year-run­ning com­mer­cial part­ner­ship with Medi­son Phar­ma. While the duo ini­tial­ly paired up in 2019 to com­mer­cial­ize Al­ny­lam’s RNA in­ter­fer­ence port­fo­lio in Is­rael, they’re now adding Poland, the Czech Re­pub­lic, Hun­gary, Slo­va­kia, Lithua­nia, Es­to­nia and Latvia to the list.

“The ex­pand­ed agree­ment with Medi­son is part of an ex­ist­ing mod­el im­ple­ment­ed in 2022 that fo­cus­es on how we sus­tain­ably and com­pli­ant­ly broad­en the reach of our com­mer­cial med­i­cines by work­ing close­ly along­side our Part­ners,” Nor­ton Oliveira, Al­ny­lam’s se­nior VP and head of part­ner and emerg­ing mar­kets, told End­points News via email.

The fi­nan­cial terms of the part­ner­ship were not dis­closed.

John Maraganore

Oliveira added that the move is in line with Al­ny­lam’s “P5x25 strat­e­gy,” which Maraganore es­tab­lished back in 2021 in the hopes of be­com­ing a top-five biotech by 2025. While Maraganore an­nounced his plans to re­tire lat­er that year, end­ing a 19-year ca­reer at the biotech he helped launch in 2002, his suc­ces­sor Yvonne Green­street con­tin­ues to push to­ward that goal.

Al­ny­lam boasts a hand­ful of RNAi prod­ucts, in­clud­ing On­pat­tro, Givlaari, Oxlu­mo and the close­ly watched Amvut­tra. Back in Oc­to­ber, the com­pa­ny point­ed to the In­fla­tion Re­duc­tion Act when it scrapped plans for a Phase III tri­al for Amvut­tra in Star­gardt dis­ease, a rare ge­net­ic eye con­di­tion. The drug is al­ready ap­proved by the FDA to treat hered­i­tary transthyretin-me­di­at­ed (hAT­TR) amy­loi­do­sis, and should pro­duce Phase III re­sults in transthyretin amy­loi­do­sis (AT­TR) with car­diomy­opa­thy next year.

Mean­while, On­pat­tro awaits an FDA ad­vi­so­ry com­mit­tee meet­ing to ex­pand its la­bel to treat pa­tients with AT­TR-CM, with a de­ci­sion ex­pect­ed by Oct. 8.

Al­ny­lam and Medi­son said in the news re­lease on Thurs­day that Givlaari and Oxlu­mo are ap­proved for re­im­burse­ment in Poland, and the teams will “work to­geth­er to ob­tain ad­di­tion­al re­im­burse­ments in the ter­ri­to­ries.”

“This suc­cess­ful col­lab­o­ra­tion led to a nat­ur­al choice of Medi­son as Al­ny­lam’s part­ner in ad­di­tion­al mar­kets in Cen­tral East­ern Eu­rope and go­ing for­ward Al­ny­lam will ben­e­fit from Medi­son’s full suite of ser­vices in this re­gion as well by lever­ag­ing Medi­son’s ex­ten­sive ex­ist­ing ca­pa­bil­i­ties on the ground,” Gil Gurfinkel, VP of cor­po­rate de­vel­op­ment at Medi­son, said in an email.

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.

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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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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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.”

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.

See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

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Hugo Peris, Spiral Therapeutics CEO

Hear­ing-fo­cused biotech grabs trio of pro­grams from Oton­o­my's fire sale

Otonomy may be shutting down, but the lessons learned there will live on at another biotech working on new treatments for hearing loss.

San Francisco-based Spiral Therapeutics has bought certain assets related to three of Otonomy’s programs, ranging from data, patent rights, and know-how to inventory. That includes data around Otonomy’s twice-failed lead program, OTO-104 (Otividex), a sustained-exposure formulation of dexamethasone.

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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