Steve Davis, Acadia Pharmaceuticals CEO

Up­dat­ed: Aca­dia wins first FDA ap­proval for Rett syn­drome drug

The FDA on Fri­day ap­proved Aca­dia Phar­ma­ceu­ti­cals’ treat­ment for Rett syn­drome, a rare neu­rode­vel­op­men­tal dis­or­der, which will be sold un­der the brand name Day­bue (trofine­tide), the com­pa­ny an­nounced.

The av­er­age list price for the drug will be about $575,000 to $595,000 a year, CEO Steve Davis said on a con­fer­ence call Mon­day morn­ing. That comes from a $21.10 per mil­li­liter price that’s used for weight-based dos­ing. The com­pa­ny’s “net re­al­ized cost” will be ap­prox­i­mate­ly $375,000, he said.

Wall Street an­a­lysts have been await­ing word on how the com­pa­ny would price the drug, and Mizuho an­a­lyst Uy Ear said over the week­end that the price of the drug will be a ma­jor fac­tor in its com­mer­cial suc­cess.

“At cur­rent lev­el, we be­lieve the stock’s val­ue in­cor­po­rates a list price of ~$400K per year at launch,” Ear said in a note to clients.

Shares of $ACAD fell 7.2% Mon­day morn­ing af­ter the call.

There are about 4,500 di­ag­nosed Rett syn­drome pa­tients in the US, ac­cord­ing to Aca­dia. Caused by a ge­net­ic mu­ta­tion, the pa­tients — typ­i­cal­ly girls — de­vel­op nor­mal­ly in in­fan­cy and then be­gin to de­te­ri­o­rate, los­ing their speech, mo­tor con­trol of their hands and oth­er func­tions.

Aca­dia added that it be­lieves there are be­tween six and nine thou­sand pa­tients in the US with the dis­ease.

The com­pa­ny said Day­bue will be avail­able for those two years old and old­er by the end of April. It’s the first ap­proved US treat­ment for the dis­ease, and Aca­dia will get a pri­or­i­ty re­view vouch­er from the FDA, which could bring the com­pa­ny about $100 mil­lion if it de­cides to sell the vouch­er.

Mark Schney­er

CFO Mark Schney­er said on Mon­day’s call that it has to pay mile­stones to Neuren Phar­ma­ceu­ti­cals, the com­pa­ny from which Aca­dia in-li­censed Day­bue in 2018 to get US rights. Neuren re­tains all rights to Day­bue out­side of the US.

Aca­dia owes Neuren $40 mil­lion with­in 60 days of Day­bue’s first com­mer­cial sale. If the com­pa­ny us­es or sells its pri­or­i­ty re­view vouch­er, Neuren gets one-third of the vouch­er’s val­ue, Schney­er said — on top of roy­al­ties and sales mile­stones.

Aca­dia’s chief com­mer­cial of­fi­cer Bren­dan Tee­han told End­points News the sales force will con­sist of 50 peo­ple who will be spread out among cen­ters of ex­cel­lence, “high-vol­ume in­sti­tu­tions,” and then out in the com­mu­ni­ty.

Bren­dan Tee­han

“We just got the la­bel on Fri­day. So we will train the field force, but they will be out in full ef­fect in mid-April,” Tee­han said, not­ing that the drug will be avail­able be­fore the end of April.

As for Aca­dia’s plans to reach the un­di­ag­nosed pop­u­la­tion, Tee­han said that in­tro­duc­ing a ther­a­peu­tic op­tion could dri­ve peo­ple to get a di­ag­no­sis.

“If you didn’t think you had ther­a­peu­tic op­tions, then know­ing more about a dis­ease isn’t ter­ri­bly help­ful. But if you do know that there are ther­a­peu­tic op­tions, peo­ple tend to go and get a con­fir­ma­to­ry di­ag­no­sis, which we think is like­ly to oc­cur,” the ex­ec added.

Kathie Bish­op

Davis, along­side Aca­dia’s head of rare dis­ease Kathie Bish­op, ref­er­enced a col­lab­o­ra­tion with Stoke Ther­a­peu­tics on the con­fer­ence call, which was ce­ment­ed last year in a $900 mil­lion plus deal for SYN­GAP1 syn­drome, fol­lowed by Rett syn­drome (MECP2) and an­oth­er undis­closed pro­gram.

“We be­lieve this will be the be­gin­ning of ad­di­tion­al ther­a­pies com­ing in due course. And we want to be at the fore­front of that,” Davis said.

“Those pro­grams are still pre­clin­i­cal, so we’re not talk­ing too much about them yet,” Bish­op added — not­ing that it rep­re­sents Aca­dia’s em­pha­sis on rare dis­eases and Rett in par­tic­u­lar.

The drug was ap­proved based on a Phase III tri­al of 187 fe­male pa­tients show­ing that they im­proved on two scor­ing sys­tems used to as­sess peo­ple with the dis­ease. Com­mon side ef­fects were di­ar­rhea and vom­it­ing.

The ap­proval is a wel­come dose of good news for Aca­dia, which has been go­ing back and forth with the FDA over its drug Nu­plazid, its main source of rev­enue, for use in Alzheimer’s-re­lat­ed psy­chosis. The agency re­ject­ed it for that use last year, rec­om­mend­ing an­oth­er tri­al.

Ed­i­tor’s note: This sto­ry has been up­dat­ed with con­text from Aca­dia’s con­fer­ence call Mon­day morn­ing, along­side an in­ter­view with Aca­dia’s chief com­mer­cial of­fi­cer Bren­dan Tee­han.

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.

Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Ivana Magovčević-Liebisch, Vigil Neuroscience CEO

FDA lifts par­tial clin­i­cal hold on Vig­il Neu­ro­science's TREM2 an­ti­body, re­mov­ing dos­ing cap

When Vigil Neuroscience filed its IPO papers in late 2021, the biotech revealed that the FDA had just cleared its Phase I trial — but with a partial clinical hold that limited dosing to under a certain level.

More than a year later, the FDA has lifted the hold.

Vigil is now free to dose VGL101, an antibody targeting TREM2, at levels higher than 20 mg/kg in its ongoing and future clinical trials in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), an inherited condition that affects the brain and spinal cord.

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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Man­u­fac­tur­ing roundup: Catal­ent to pro­duce low-cost ver­sion of nalox­one; CSL opens R&D site

Catalent will be manufacturing a low-cost version of the opioid overdose treatment naloxone as part of a contract with Harm Reduction Therapeutics.

Catalent plans to manufacture the treatment at its facility in Morrisville, NC. No financial details on the deal were disclosed.

Harm Reduction was granted priority review status for the NDA on its spray last year. The company has been working on a naloxone product since 2017. It is anticipating approval in July of this year and a US launch in early 2024.

As­pen looks to re­bound in pro­duc­tion and rev­enue af­ter Covid-19

Last year, South African-based vaccine manufacturer Aspen Pharmacare was facing reports that it had not received a single order for its manufactured Covid-19 shots and that manufacturing lines were sitting idle. But now the vaccine producer is looking to turn things around.

Aspen’s disclosure of its financial results in March unveiled that manufacturing revenue had decreased by 12% to R 603 million ($33.8 million), which Lorraine Hill, Aspen Group’s COO, said is attributable to lower Covid vaccine sales.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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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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Ribbon cutting ceremony for Thermo Fisher's new cell therapy manufacturing site in San Francisco

Ther­mo Fish­er moves on cam­pus with new cell man­u­fac­tur­ing site in San Fran­cis­co

Thermo Fisher Scientific is putting down more roots in the Bay Area.

The manufacturer opened the doors to a new cell therapy manufacturing facility next to the University of California-San Francisco Medical Center’s Mission Bay campus and on the university’s campus.

UCSF and Thermo Fisher have had a partnership since 2021, with the new site focusing on manufacturing cell therapeutics for certain cancers, including glioblastoma and multiple myeloma. The new site plans to use Thermo Fisher’s expertise in manufacturing services to help UCSF accelerate the development of cell therapies and eventually get them into the clinic, said Dan Herring, the general manager of cell therapy services at Thermo Fisher, in an interview with Endpoints News.