John Crowley, Amicus CEO

Am­i­cus spins out gene ther­a­py unit in SPAC deal as it piv­ots ef­forts to­ward Pompe dis­ease pro­gram

A rare dis­ease biotech out of Philadel­phia is spin­ning out its gene ther­a­py unit to re­verse merge with a blue-chip SPAC.

Am­i­cus Ther­a­peu­tics will send off its gene ther­a­py com­po­nent in a new com­pa­ny called Car­i­tas Ther­a­peu­tics, which will part­ner with Per­cep­tive’s fourth blank-check out­fit and head to Nas­daq, the biotech an­nounced Wednes­day morn­ing. Car­i­tas will get about $400 mil­lion over­all in the deal, in­clud­ing ap­prox­i­mate­ly $150 mil­lion held in ARYA Sci­ences Ac­qui­si­tion Corp IV, a $200 mil­lion PIPE in­vest­ment and a $50 mil­lion cash in­vest­ment from Am­i­cus.

“This is a big, bold vi­sion and a mas­sive step for­ward for next gen­er­a­tion biotech­nolo­gies for peo­ple world­wide liv­ing with some of the most dev­as­tat­ing rare dis­eases,” Am­i­cus CEO John Crow­ley said in a state­ment. “In a sin­gle stroke with the for­ma­tion and fund­ing of Car­i­tas we will cre­ate what will be one of the world’s pre­em­i­nent next-gen­er­a­tion ge­net­ic med­i­cines com­pa­nies.”

The deal is ex­pect­ed to close some­time in the fourth quar­ter this year, or ear­ly next year.

As Car­i­tas gets things go­ing, Am­i­cus will fo­cus on com­mer­cial­iz­ing its Galafold drug for Fab­ry dis­ease, as well as push­ing for­ward on a la­bel ex­pan­sion. The biotech is al­so re­dou­bling its ef­forts to get its Pompe dis­ease can­di­date, dubbed AT-GAA, ap­proved and hopes to roll it out glob­al­ly. On Wednes­day, the FDA ac­cept­ed Am­i­cus’ BLA and NDA for both com­po­nents of the pro­gram, set­ting PDU­FAs dates of May 29, 2022 and Ju­ly 29, 2022, re­spec­tive­ly.

The Pompe dis­ease pro­gram will like­ly run in­to head­winds with reg­u­la­tors, how­ev­er, as a Phase III study showed AT-GAA did not pass a head-to-head test against Sanofi’s Lu­mizyme in the 6-minute walk test, the gold stan­dard for demon­strat­ing ef­fi­ca­cy. Re­searchers saw a p-val­ue of p=0.072 in pa­tients who were both switched from ERT ther­a­py or ERT naïve.

When look­ing at those pop­u­la­tions sep­a­rate­ly, AT-GAA did hit nom­i­nal sta­tis­ti­cal sig­nif­i­cance in those who orig­i­nal­ly switched from Lu­mizyme at a p-val­ue of p=0.046. But in pa­tients who had nev­er re­ceived treat­ment be­fore, AT-GAA land­ed with a thud, fail­ing to show su­pe­ri­or­i­ty on the walk test and hit­ting a p-val­ue of p=0.57 on forced vi­tal ca­pac­i­ty.

Car­i­tas will kick things off with a pipeline of two clin­i­cal pro­grams in Bat­ten dis­ease, with the com­pa­ny say­ing it’s achieved proof of con­cept in CLN6 and CLN3. There will al­so be six ac­tive pre­clin­i­cal can­di­dates span­ning a wide range of rare ge­net­ic dis­eases like Fab­ry dis­ease, Pompe dis­ease and CD­KL5 de­fi­cien­cy dis­or­der.

The spin­out is al­so get­ting ex­clu­sive glob­al rights for about rough­ly 50 more dis­eases through a col­lab­o­ra­tion with gene edit­ing pi­o­neer Jim Wil­son and his team at the Uni­ver­si­ty of Penn­syl­va­nia, the an­nounce­ment said. Car­i­tas will start with a team of about 115 em­ploy­ees, in­di­cat­ing Am­i­cus ex­pects the new biotech to hit the ground run­ning.

Am­i­cus will be the largest share­hold­er in Car­i­tas once the SPAC deal clos­es, own­ing about 36% of shares. It will al­so re­tain co-de­vel­op­ment and com­mer­cial­iza­tion rights to the Fab­ry and Pompe gene ther­a­py pro­grams.

In con­junc­tion with the spin­out, Am­i­cus al­so raised $200 mil­lion in a pri­vate place­ment from Red­mile Group, Avoro Cap­i­tal Ad­vi­sors, Per­cep­tive Ad­vi­sors, In­vus, Sphera Health­care, and Janus Hen­der­son In­vestors.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; 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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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.

Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

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