Steve Davis, Acadia CEO

In $900M+ deal, rare dis­ease biotechs team up on RNA ther­a­pies for CNS de­vel­op­men­tal dis­or­ders

Two biotechs that have built their rep­u­ta­tions in rare dis­ease re­search signed a new col­lab­o­ra­tion Mon­day morn­ing aim­ing to de­vel­op three new RNA-based ther­a­pies. And the deal could be worth near­ly $1 bil­lion in biobucks if every mile­stone is met.

Aca­dia Phar­ma­ceu­ti­cals and Stoke Ther­a­peu­tics will team up on the treat­ments in rare ge­net­ic neu­rode­vel­op­men­tal dis­eases of the CNS, the com­pa­nies an­nounced. Up first will be the re­cent­ly dis­cov­ered SYN­GAP1 syn­drome, fol­lowed by Rett syn­drome (MECP2) and an undis­closed tar­get the biotechs say is of mu­tu­al in­ter­est.

“Com­bin­ing Stoke’s ca­pa­bil­i­ties with Aca­dia’s ex­ten­sive ex­per­tise in neu­ro­science drug de­vel­op­ment and com­mer­cial­iza­tion en­ables us to push hard­er and faster in ex­plor­ing some of the new fron­tiers in rare cen­tral ner­vous sys­tem dis­or­ders,” Aca­dia CEO Steve Davis said in a state­ment.

Stoke will net a $60 mil­lion up­front pay­ment from Aca­dia to kick things off and is el­i­gi­ble to re­ceive up to $907 mil­lion in mile­stones.

The com­pa­nies will split R&D costs and prof­its even­ly for the SYN­GAP1 can­di­date, ac­cord­ing to the press re­lease. Stoke will take the lead in pre­clin­i­cal de­vel­op­ment for the oth­er two pro­grams — ef­forts that will be “ful­ly fund­ed” by Aca­dia — af­ter which Aca­dia will head up clin­i­cal stud­ies and com­mer­cial­iza­tion.

SYN­GAP1 syn­drome was first iden­ti­fied in 2009, the com­pa­nies say, and can be char­ac­ter­ized by de­vel­op­men­tal de­lay or in­tel­lec­tu­al dis­abil­i­ty, gen­er­al­ized epilep­sy and autism spec­trum dis­or­der, among oth­er things, pre­sent­ing in ear­ly child­hood. The con­di­tion is caused by mu­ta­tions in the SYN­GAP1 gene and is said to ac­count for “1% to 2%” of all in­tel­lec­tu­al dis­abil­i­ty cas­es.

Rett syn­drome, mean­while, typ­i­cal­ly oc­curs in young girls due to a mu­ta­tion of a gene on the X chro­mo­some. It’s a con­di­tion in which Aca­dia is work­ing on an­oth­er pro­gram, hav­ing re­vealed pos­i­tive topline da­ta in a Phase III study last month show­ing the can­di­date, known as trofine­tide, beat place­bo on two co-pri­ma­ry end­points.

Though many col­lab­o­ra­tions in the space are signed be­tween a Big Phar­ma com­pa­ny and a small­er biotech, Mon­day’s deal comes from two biotechs with his­to­ries of de­vel­op­ing rare dis­ease and CNS ther­a­pies. Aca­dia’s Nu­plazid was the first drug ap­proved to treat hal­lu­ci­na­tions and psy­chosis re­lat­ed to Parkin­son’s dis­ease, while Stoke re­vealed the first clin­i­cal da­ta for its lead Dravet syn­drome pro­gram last month.

Aca­dia has run in­to some trou­ble more re­cent­ly in try­ing to ex­pand Nu­plazid’s in­di­ca­tions, how­ev­er, as the FDA in April is­sued a CRL for de­men­tia-re­lat­ed psy­chosis. The biotech said a few weeks ago it’s plan­ning to re­sub­mit the sN­DA with a fo­cus on Alzheimer’s in­duced psy­chosis some­time this quar­ter.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Tim Walbert, Horizon Therapeutics CEO (via YouTube)

And then there were two: Janssen bows out of Hori­zon takeover ne­go­ti­a­tions

Horizon Therapeutics announced last week that it was in talks with three pharmaceutical giants that could take over the company. You can now remove one of them from the equation.

J&J’s Janssen, after Horizon reported its initial involvement in early discussions to acquire the rare disease biotech, issued a statement Saturday that said Janssen “does not intend to make an offer for Horizon,” and that Janssen is bound by restrictions set in Rule 2.8 of the Irish Takeover Rules. These rules are in place for any company interested in taking over Irish companies, with Horizon Therapeutics currently based in Dublin.

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Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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Yuling Li, Innoforce CEO

In­no­force opens new man­u­fac­tur­ing site in Chi­na

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.