Neil Desai, Aadi Bioscience CEO (via YouTube)

FDA ap­proves pricey new treat­ment for ul­tra-rare and ag­gres­sive form of sar­co­ma

The FDA on Tues­day ap­proved Aa­di Bio­science’s first drug and the first treat­ment ap­proved specif­i­cal­ly for pa­tients with an ul­tra-rare and ag­gres­sive form of sar­co­ma that oc­curs most­ly in women.

The ap­proval of the drug, known as Fyarro, is for those with lo­cal­ly ad­vanced un­re­sectable or metasta­t­ic ma­lig­nant perivas­cu­lar ep­ithe­lioid cell tu­mor (PECo­ma), and is based on a Phase II tri­al.

Re­sults showed an over­all re­sponse rate as as­sessed by in­de­pen­dent re­view of 39% (12/31), with two pa­tients achiev­ing a com­plete re­sponse af­ter pro­longed fol­low up, Aa­di said. The com­pa­ny al­so said that among re­spon­ders, 92% had a re­sponse last­ing greater than or equal to six months; 67% had a re­sponse last­ing greater than or equal to 12 months; and 58% had a re­sponse last­ing greater than or equal to two years.

Bren­dan De­laney

The com­pa­ny said the drug will be priced at a WAC of about $39,000 per month, or $468,000 per year.

Aa­di COO Bren­dan De­laney said on Tues­day’s in­vestor call, “FYARRO’s whole­sale ac­qui­si­tion cost for a sin­gle-use 100 mil­ligram vial is $6,785.00, fac­tor­ing in the vari­abil­i­ty of pa­tient body sur­face area and dose mod­i­fi­ca­tions, we project the whole­sale ac­qui­si­tion cost for a month of FYARRO ther­a­py to be ap­prox­i­mate­ly $39,000.  Statu­to­ry gov­ern­ment dis­counts such as Med­ic­aid re­bates, sales to 340B cov­ered en­ti­ties and VA fa­cil­i­ties among oth­ers will re­duce the net price that Aa­di Bio­science will re­ceive by ap­prox­i­mate­ly 15% to 20%.”

De­laney added in an emailed state­ment to End­points News, “These pric­ing es­ti­mates are ap­prox­i­mate and will vary based on pa­tient and oth­er fac­tors (body weight, etc). We think this pric­ing is ap­pro­pri­ate giv­en the great need in PECo­ma, the small pa­tient pop­u­la­tion and over­all clin­i­cal pro­file of FYARRO.”

The rec­om­mend­ed dosage, ac­cord­ing to the FDA, is 100 mg/m2 ad­min­is­tered as an in­fu­sion “over 30 min­utes on days 1 and 8 of each 21-day cy­cle un­til dis­ease pro­gres­sion or un­ac­cept­able tox­i­c­i­ty.”

An­drew Wag­n­er, a se­nior on­col­o­gist at Dana-Far­ber Can­cer In­sti­tute and the prin­ci­pal in­ves­ti­ga­tor of the piv­otal tri­al, said in a state­ment that Fyarro will pro­vide physi­cians “with a new weapon for treat­ing pa­tients with this rare dis­ease.”

Shares of the com­pa­ny’s stock rose more than 25% in pre-mar­ket trad­ing Tues­day, but then fell to about a 6% spike by morn­ing.

An­drew Wag­n­er

Fyarro was cre­at­ed by Aa­di founder and CEO Neil De­sai, the for­mer glob­al R&D chief at Abrax­is, who tried to de­vel­op a sim­i­lar ap­proach to the one he and Patrick Soon-Sh­iong used to cre­ate block­buster can­cer drug Abrax­ane, which is now in short­age. Abrax­ane was a decades-old chemother­a­py, en­cased in an al­bu­min coat that tricks tu­mor cells in­to ab­sorb­ing the tu­mor-killing agent.

With Fyarro, De­sai took a decades-old im­mune-mod­u­lat­ing drug, ra­pamycin, and coat­ed it in a sim­i­lar al­bu­min coat. Once tak­en up by the tu­mor, it knocks down mTOR, a pro­tein that has long been linked to tu­mor pro­lif­er­a­tion, among oth­er things.

Aa­di in May merged with the strug­gling pub­lic biotech Aer­pio Phar­ma­ceu­ti­cals and raised $155 mil­lion from pri­vate in­vestors to com­mer­cial­ize Fyarro.

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.

The slate of products we’re offering here at Endpoints is continuing to grow, and it’s not just limited to editorial. If you haven’t, do visit your reader profile to see if there are any other weekly newsletters you’re interested in — as each comes with its own exclusive content. And don’t miss the publisher’s note from Arsalan Arif on Endpoints Studio, our latest avenue for advertising on Endpoints.

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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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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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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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Richard Pazdur (via AACR)

Ac­cel­er­at­ed ap­proval re­forms need mean­ing­ful con­fir­ma­to­ry tri­al im­prove­ments, pro­fes­sors write in Sci­ence

Outside of Covid-19, 2021 has been the year of the accelerated approval.

Beginning last spring, FDA openly challenged six “dangling” accelerated approvals (hadn’t confirmed their clinical benefit yet), three of which were later pulled by the companies.

Then in June, FDA pulled out the accelerated approval pathway, seemingly out of nowhere, to sign off on Biogen’s controversial Alzheimer’s drug Aduhelm. It hadn’t even been mentioned at the drug’s adcomm.

Common performs onstage, December 2020 (Getty Images)

Com­mon, Jamie Foxx among celebs stand­ing up for clin­i­cal tri­als in star-stud­ded can­cer group's pan­dem­ic push

Healthcare screenings and clinical trial enrollment were battered by the pandemic. But the well-known celebrity-backed Stand Up To Cancer non-profit, along with pharma and advocacy partners, has been working to reverse that and make up lost ground, by stepping up awareness campaigns.

Twelve campaigns launched in 2020 and another five in 2021 amplify the need for cancer screening and care, especially for underserved communities. While pharma companies have long been donors to the cancer research group, Covid brought new support — and increased awareness efforts.

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UP­DAT­ED: Am­gen halts some drug dis­counts for safe­ty net hos­pi­tals as SCO­TUS takes on 340B case

Amgen will soon be the 10th biopharma company to pull back on offering drug discounts to contract pharmacies of safety-net hospitals under a federal program. Like its peers, Amgen argues that the growth of these contract pharmacies has ballooned in recent years and needs to be reigned in.

Beginning Jan. 3, 2022, Amgen’s policy will only allow 340B covered hospitals to designate a single pharmacy location, with the exception of federal grantees and contract pharmacies wholly owned by a 340B hospital, or that have common ownership with a health system.