Al­ny­lam's new pipeline star gets on FDA fast track; ARCH-backed biotech nabs or­phan drug sta­tus

→ Just a day af­ter Black­stone and Al­ny­lam sketched out a R&D part­ner­ship as part of a sprawl­ing $2 bil­lion in­vest­ment plan, the biotech said it’s ob­tained fast track des­ig­na­tion for one of the drugs high­light­ed in the deal. The sta­tus was con­ferred to vutrisir­an in the treat­ment of polyneu­ropa­thy of hAT­TR amy­loi­do­sis — an in­di­ca­tion that Black­stone will help fund but not re­ceive roy­al­ties on. Al­ny­lam is now el­i­gi­ble to sub­mit a rolling NDA for the drug, for which Phase III re­sults are ex­pect­ed in ear­ly 2021.

Epir­i­um, the ARCH-backed biotech that un­veiled a $85 mil­lion round ded­i­cat­ed to its syn­thet­ic fla­vanol, has se­cured or­phan drug des­ig­na­tion for the drug as a treat­ment of Duchenne and Beck­er mus­cu­lar dy­s­tro­phy. “Our ini­tial em­pha­sis on Beck­er mus­cu­lar dy­s­tro­phy stems from our re­search demon­strat­ing that EB 002 may stim­u­late mi­to­chon­dr­i­al bio­gen­e­sis, lead­ing to the restora­tion of tis­sue bioen­er­get­ics, while up­reg­u­lat­ing pro­duc­tion of key pro­teins which may lead to im­proved mus­cle struc­ture and func­tion,” CMO Ran­si So­maratne said. It’s un­clear what stage the pro­gram is in.

→ Glob­al health-fo­cused VC Ad­ju­vant Cap­i­tal has led a $15 mil­lion Se­ries A round for 54gene, help­ing boot­strap a re­search, ser­vices and de­vel­op­ment ven­ture tap­ping in­to Africa’s ge­nom­ic datasets. Launched in Jan­u­ary 2019, the Nige­ria-based com­pa­ny aims to ad­dress the lack of African rep­re­sen­ta­tion in the da­ta com­mon­ly used in genome-wide as­so­ci­a­tion stud­ies (GWAS), with the goal of dis­cov­er­ing in­sights of use in ther­a­peu­tic de­vel­op­ment. No­var­tis, the Gates Foun­da­tion and oth­er in­vestors al­so par­tic­i­pat­ed.

Stan­ley Crooke has re­ceived an­oth­er $500,000 do­na­tion for the n-Lorem Foun­da­tion, sup­port­ing his non-prof­it’s ef­forts to pro­vide an­ti­sense treat­ments for pa­tients with ul­tra-rare, N of 1 dis­eases.

George Scangos (L) and Marianne De Backer

Pi­o­neer­ing biotech icon George Scan­gos hands in his re­tire­ment pa­pers — and this time it’s for re­al

George Scangos, one of the all-time great biotech CEOs, says the time has come to turn over the reins one last time.

The 74-year-old biotech legend spent close to three decades in a CEO post. The first was at Exelixis — which is still heavily focused on a drug Scangos advanced in the clinic. The second “retirement” was at Biogen, where he and his team were credited with a big turnaround with the now fading MS blockbuster Tecfidera. And the third comes at Vir, where he traded in his Big Biotech credentials for a marquee founder’s role back on the West Coast, hammering out a Covid-19 alliance with Hal Barron — then R&D chief at GSK — and breaking new ground on infectious diseases with some high-powered venture players.

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Jeanne Loring, director of the Center for Regenerative Medicine (Credit: Jamie Scott Lytle)

A stem cell pi­o­neer sent an ex­per­i­ment in­to space. Pa­tients are the next fron­tier

Last July, Jeanne Loring stood on a dirt road surrounded by Florida swampland and watched as a nearby SpaceX rocket blasted into the sky. The payload included a very personal belonging: cell clusters mimicking parts of her brain.

For more than two decades, Loring has been at the forefront of a stem cell field that always seems on the brink of becoming the next thing in medicine, but has been slow to lift off.

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FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

Evotec inks li­cense agree­ment with J&J; On­colyt­ic virus biotech prices $15M IPO

Drug discovery and development player Evotec entered another licensing deal with Big Pharma — this time with J&J’s Janssen.

The companies put out word that they entered into a strategic collaboration and license agreement with each other that focuses on targeted immune-based cancer therapies, to be commercialized by Janssen.

According to a statement, the collaboration will hinge on Evotec’s integrated drug discovery and manufacturing capabilities. During the pre-clinical R&D phase, the companies will collaborate closely — and then Janssen will take on full responsibility for both clinical development and commercialization.

In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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#JPM23: What's re­al­ly dri­ving the cost of health­care and drugs in 2023?

Executive Editor Drew Armstrong spoke with PhRMA CEO Steve Ubl, EmsanaRx CEO Greg Baker and ICER President Steve Pearson about how the debate over drug costs has changed (or not) in the last decade, the shifting payer landscape and why there seems to be so little movement on drug rebates. This transcript has been edited for brevity and clarity.

Drew Armstrong:

So first of all, thank you to everybody for being here and for our panel for being here. Incredibly excited to have this discussion on the cost of healthcare and drugs and what’s driving that. We’re here with Steve Ubl, the head of PhRMA. Thank you so much. Steve Pearson from ICER, and Greg Baker from EmsanaRx. I want to start this conversation with a little bit of a personal reminiscence. So about almost 10 years exactly. I was a reporter back in my previous job and I was covering drug pricing and Gilead had just launched their hepatitis C drug and I was having a conversation with another Steve over at Express Scripts and he made some comments essentially about how they intended to launch a price war over hepatitis C therapies.

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Pa­tient death spurs tri­al halt for Ma­gen­ta Ther­a­peu­tics

Magenta Therapeutics is pausing an early-stage clinical trial after a patient died. The death was deemed to be possibly related to its drug, MGTA-117.

The biotech said the pause of the Phase I/II trial is voluntary and gives it time to review all available data before deciding what to do next. It’s also reported the known information to the FDA.

The dose-escalation trial was designed to test whether MGTA-117, an antibody-drug conjugate, could serve as a more targeted alternative to high-intensity chemotherapy as a conditioning agent for cancer patients who are set to receive a stem cell transplant. It recruited patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.

In a win for Re­gen­eron, No­var­tis' sy­ringe for AMD drug de­clared 'un­patentable'

Regeneron has won a patent case against Swiss pharma giant Novartis over the delivery system for its eye drug Eylea.

The US Patent Trial and Appeal Board ruled that Novartis’ pre-filled syringe for injecting its eye medication Lucentis was “unpatentable” and handed the victory to Regeneron and its AMD drug Eylea.

In the initial complaint in 2020, Novartis alleged to the US International Trade Commission that certain pre-filled syringes for the intravitreal injection, and ultimately Regeneron’s delivery system for Eylea, were infringing on Novartis’ patent. Regeneron filed a petition to review Novartis’ claims in 2021.