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.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

Alessandro Maselli, Catalent CEO

Catal­ent ac­quires North Car­oli­na CD­MO for $475M, boost­ing oral solids work

As Catalent has been expanding its reach in the US this year, as well as recently completing a C-suite shuffle, the company announced last night that it has acquired the CDMO Metrics Contract Services for $475 million from Mayne Pharma Group.

The acquisition will increase Catalent’s capabilities in oral solid formulation development, manufacturing and packaging as well as expand its capacity to handle more highly potent compounds.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

Craig Thompson, Cerevance CEO

UP­DAT­ED: Mer­ck makes first big splash for Alzheimer’s drug R&D since 2017 fail, ink­ing re­search pact with Cere­vance

For the first time since discontinuing its late-stage Alzheimer’s program, Merck has found promise on the path forward in the memory-robbing disease.

After a Phase III flop of its drug verubecestat, the New Jersey Big Pharma axed the study in early 2018. More than four years later, the company is ready to sign up for another pact to test the waters of the befuddling disease.

This time, there’s $1.1 billion in biobucks on the line and a target that its partner says no other biopharma is looking at en route to finding the next treatment for Alzheimer’s, a neuroscience field that has hit hurdle after hurdle for decades.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.

Horizon's back-to-school campaign for children with cystinosis includes an all about me poster as part of a care package box.

Hori­zon read­ies kids and fam­i­lies for back to school with week­long ac­tiv­i­ties around rare dis­ease cysti­nosis

Going back to school is usually a bumpy readjustment from summer freedom for all kids, but especially for kids with chronic health conditions. Horizon Therapeutics is hoping to help smooth the way for some who have the rare disease cystinosis. Cystinosis is a genetic disease that causes the amino acid cystine to build up in different tissues and organs.

The “Gear Up” for school campaign is running all week with different online and at-home events and activities for families and children with cystinosis. Each family who signed up receives a care package mailed to their home including an activity coloring book “Michael’s Show-and-Tell.” The book tells Michael’s story about living with cystinosis while offering kids matching, coloring and finding object games along with information.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

Phar­mas spend mil­lions on di­a­betes ad­ver­tis­ing, but few pa­tients can re­call brand names — sur­vey

While many Big Pharma diabetes brands spend millions of dollars on TV ads every year, most people with type 2 diabetes don’t recognize specific drug brand names, according to a new study.

No brand garnered more than 30% recognition in Phreesia Life Science’s latest in-office patient survey of more than 4,000 adults with type 2 diabetes. Eli Lilly’s Trulicity topped the list as the most recognized brand with 29% of those surveyed recalling it, followed by Boehringer Ingelheim and Lilly’s Jardiance at 27% and Merck’s Januvia and Novo Nordisk tying for the third spot with 24%. Meanwhile, 76% of the patients surveyed were familiar with the generic treatment metformin.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

Faced with thou­sands of opi­oid law­suits, En­do says it will like­ly file for bank­rupt­cy 'im­mi­nent­ly'

Endo International will likely be the next pharma company to file for bankruptcy under a mountain of opioid lawsuits.

The Dublin, Ireland-based company revealed in its Q2 results on Tuesday that it’s in talks with first lien creditors, and that “these negotiations will likely result in a pre-arranged filing under Chapter 11 of the U.S. Bankruptcy Code by Endo International plc and substantially all of its subsidiaries, which could occur imminently.”