Al Sandrock, Voyager Therapeutics CEO

Af­ter 12 months of dig­ging, Pfiz­er opts for one of two Voy­ager cap­sids for gene ther­a­py

The path at Voy­ager Ther­a­peu­tics keeps wind­ing, and at the 12-month dead­line, Pfiz­er has elect­ed to on­ly move for­ward with one of two cap­sids out of the pre­clin­i­cal biotech for the Big Phar­ma’s AAV gene ther­a­pies.

Last Oc­to­ber, the Big Phar­ma and biotech linked arms on a deal that gave Pfiz­er the abil­i­ty to hit the gas ped­al on two cap­sids: one for a car­diac tar­get and the oth­er for a rare neu­ro­log­ic dis­ease tar­get.

Pfiz­er is on­ly go­ing with the lat­ter one, the biotech said Tues­day morn­ing, trig­ger­ing a $10 mil­lion pay­ment to Voy­ager af­ter last year’s ini­tial $30 mil­lion up­front. With that, the Cam­bridge, MA-based biotech’s shot at $580 mil­lion in back­loaded pay­ments has shrunk in half.

Voy­ager’s shares $VY­GR quick­ly rose about 12% af­ter Tues­day’s open­ing bell.

Al San­drock, Voy­ager CEO and famed for­mer head of R&D at Bio­gen, said in an emailed state­ment to End­points News that the biotech “can­not com­ment on Pfiz­er’s in­ter­nal pro­gram de­ci­sions” with re­gard to the choice to not move for­ward on the car­diac front. End­points has reached out to Pfiz­er for com­ment and will up­date ac­cord­ing­ly.

San­drock elab­o­rat­ed that Voy­ager’s ini­tial fo­cus on its so-called TRAC­ER cap­sid dis­cov­ery work has been around cen­tral ner­vous sys­tem dis­eases.

“This is where we have gath­ered the most da­ta and es­tab­lished clear dif­fer­en­ti­a­tion,” the CEO wrote in his state­ment. “We are ex­cit­ed about the po­ten­tial of the plat­form to tar­get oth­er tis­sues, such as car­diac and skele­tal mus­cle, and we will con­tin­ue to gath­er da­ta in these ar­eas.”

Since hop­ping aboard the biotech’s ship in the spring, San­drock has course-cor­rect­ed — af­ter set­backs pri­or to his tenure squashed hopes in Parkin­son’s and Hunt­ing­ton’s treat­ments — and added to his lead­er­ship team.

Pe­ter Pfre­und­schuh

In ear­ly Au­gust, the biotech fine-tuned its pre­clin­i­cal pipeline to fo­cus on GBA1 Parkin­son’s, SOD1 ALS (a rare form of the dis­ease for which Bio­gen awaits a drug ap­proval de­ci­sion by Jan. 25, 2023) and Alzheimer’s, the mem­o­ry-rob­bing dis­ease that com­prised a bulk of San­drock’s work at his for­mer em­ploy­er, which last week tout­ed Phase III da­ta on an Aduhelm fol­low-up.

In the in­ter­ven­ing weeks, Voy­ager pro­mot­ed re­search SVP Todd Carter to CSO — orig­i­nal­ly join­ing in 2016 af­ter serv­ing as sci­ence ad­vi­sor to the Broad In­sti­tute’s di­rec­tor — and hired CFO Pe­ter Pfre­und­schuh, com­ing from the same post at Fre­quen­cy Ther­a­peu­tics.

Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

Up­dat­ed: As­traZeneca jumps deep­er in­to cell ther­a­py 2.0 space with $320M biotech M&A

Right from the start, the execs at Neogene had some lofty goals in mind when they decided to try their hand at a cell therapy that could tackle solid tumors.

Its founders have helped hone a new approach that would pack in multiple neoantigen targets to create a personalized TCR treatment that would not just make the leap from blood to solid tumors, but do it with durability. And they managed to make their way rapidly to the clinic, unveiling their first Phase I program for advanced tumors just last May.

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Emily Leproust, Twist Bioscience CEO

Twist Bio­science’s 'fac­to­ry of the fu­ture' in Ore­gon could de­liv­er with com­pet­i­tive pric­ing, SVB Se­cu­ri­ties says

The synthetic DNA manufacturer Twist Bioscience has given a peek behind the curtain to several analysts into its “factory of the future” as well as insight into the cost structure, workflow and technology at the site.

The 110,000-square-foot manufacturing site in the city of Wilsonville, OR, just south of Portland, which was announced back in 2020, will double Twist’s production capacity and bring around 400 jobs to the area.

Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting:

Uğur Şahin, BioNTech CEO (ddp images/Sipa USA/Sipa via AP Images)

BioN­Tech bets on dif­fi­cult STING field via small mol­e­cule pact with a Pol­ish biotech

BioNTech is beefing up its relatively thin small molecule pipeline by adding weight to a clinically difficult corner of oncology R&D: STING agonists. To do so, BioNTech is teaming up with a 15-year-old Polish biotech and doling out €40 million, about $41.5 million, to start.

The deal is broken into two parts: First, BioNTech obtains an exclusive global license to develop and market Ryvu Therapeutics’ STING agonist portfolio as small molecules, whether alone or in combination with other agents.

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Tim Walbert, Horizon Therapeutics CEO (via YouTube)

Hori­zon Ther­a­peu­tics in takeover talks with Am­gen, J&J, Sanofi as po­ten­tial buy­ers

Amgen, J&J’s Janssen and Sanofi are all in talks to acquire Horizon Therapeutics, the rare disease biotech disclosed late Tuesday.

Horizon confirmed “highly preliminary discussions” with those companies regarding a potential buyout offer after the Wall Street Journal reported takeover interest.

Although the company — which commands a market cap of close to $18 billion — emphasized that “there can be no certainty that any offer will be made for the Company,” shares $HZNP still surged 31% in after-hours trading to near $103, bringing it to the point where it started the year.

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Lex­i­con slams FDA over hear­ing de­nial fol­low­ing a CRL for its SGLT2 in­hibitor can­di­date

Lexicon Pharmaceutical is not giving up on its Type I diabetes candidate, despite FDA’s repeated rejections. This week the company laid out is argument again for a hearing on sotagliflozin in response to the FDA’s most recent denial.

The issue goes back to March 2019 when the FDA made very clear to Lexicon and its now departed partner Sanofi that it would not approve their application for a potential Type I diabetes drug because it does not appear to be safe.

Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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