Sanofi Gen­zyme deserts gene ther­a­py de­vel­op­er Voy­ager Ther­a­peu­tics

While gene ther­a­py com­pa­nies re­joice as the sec­tor gains trac­tion with ap­provals and a flur­ry of M&A ac­tiv­i­ty, one play­er is feel­ing the heat.

Back in 2015, Voy­ager Ther­a­peu­tics joined forces with Sanofi Gen­zyme in a deal worth up to $845 mil­lion ($100 mil­lion up­front + a po­ten­tial $745 mil­lion in mile­stones) to co-de­vel­op gene ther­a­pies for se­vere cen­tral ner­vous sys­tem dis­or­ders. But two years lat­er, the French drug­mak­er re­treat­ed, elect­ing to not pick up the op­tion to work on Voy­ager’s Parkin­son’s dis­ease pro­gram. (Last year, the FDA dis­ap­point­ed Voy­ager, telling the com­pa­ny that it was not open to an ac­cel­er­at­ed fil­ing on the Parkin­son’s drug on the ba­sis of Phase II da­ta — in­stead re­quir­ing an ad­di­tion­al piv­otal study.)

Sanofi Gen­zyme’s re­treat now re­sem­bles a de­ser­tion.

On Mon­day, Voy­ager $VY­GR said Sanofi Gen­zyme is walk­ing away from op­tions to ac­quire US co-com­mer­cial­iza­tion rights and ex-US de­vel­op­ment and com­mer­cial­iza­tion rights to ex­per­i­men­tal Hunt­ing­ton’s dis­ease drug VY-HTT01 and to Friedre­ich’s atax­ia pro­gram VY-FXN01; and a fu­ture Voy­ager CNS or­phan pro­gram. In ad­di­tion, Voy­ager and Sanofi Gen­zyme’s al­liance on SMA has al­so been ter­mi­nat­ed —  the in­tel­lec­tu­al prop­er­ty rights of the pro­gram are be­ing re­turned/ex­clu­sive­ly li­censed to Sanofi Gen­zyme.

Con­se­quent­ly, Voy­ager has agreed to give $10 mil­lion up­front to Sanofi Gen­zyme, and an ad­di­tion­al $10 mil­lion mile­stone pay­ment up­on the po­ten­tial fil­ing of an ap­pli­ca­tion to test in hu­mans the Hunt­ing­ton’s dis­ease drug, VY-HTT01 or, cer­tain back­up com­pounds, as well as po­ten­tial roy­al­ties. In ad­di­tion, Voy­ager is al­so of­fer­ing Sanofi Gen­zyme ex­clu­sive dibs to se­lect AAV cap­sids from its ar­se­nal of drugs in de­vel­op­ment in up to two non-CNS in­di­ca­tions.

Re­cent­ly, Voy­ager has been busy shop­ping for oth­er part­ners. It has inked deals with Ab­b­Vie and Neu­ro­crine this year.

On Mon­day, Voy­ager trans­ferred the ex-US rights to the Sanofi-aban­doned Friedre­ich’s atax­ia pro­gram to Neu­ro­crine. The com­pa­ny al­so sig­naled that it plans to part­ner out its ALS gene ther­a­py, VY-SOD102, (and no longer plans to file an IND for it this year), to fo­cus its re­sources on Hunt­ing­ton’s.

“While it is un­clear ex­act­ly who might be look­ing to in-li­cense this pro­gram, Bio­gen might serve as an in­ter­est­ing can­di­date giv­en their ex­ist­ing work on SOD1 with their an­ti­sense oligonu­cleotide (ASO), tofersen, and close prox­im­i­ty giv­en both com­pa­nies are based in Cam­bridge. How­ev­er, if Bio­gen were to do this deal it might al­so be an ear­ly sig­nal to in­vestors that they view gene ther­a­py as strong al­ter­na­tive to their ASO port­fo­lio, po­ten­tial­ly ac­cept­ing that Zol­gens­ma is go­ing to be a strong com­peti­tor in SMA,” Baird’s Bri­an Sko­r­ney wrote in a note.

So­cial im­age: Shut­ter­stock

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.

Photo: Shutterstock

Bio­phar­ma's suc­cess rate in bring­ing drugs to mar­ket has long been abysmal. Can new tools help rewrite that trou­bled past?

In 2011, a team of researchers at British drugmaker AstraZeneca had a problem they were looking to solve.

For years, drug discovery and development were a wasteland for innovation. Novel drugs largely fell into one of two categories — monoclonal antibodies and small molecules — and new therapeutic modalities were hard to come by. After a rush of promising approvals in the late 1990s — including then-Biogen’s CD20 targeting antibody breakthrough Rituxan — the field stagnated and attrition rates stayed sky-high. What exactly is the industry doing wrong? AstraZeneca asked itself.

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