Neil Desai, Aadi Bioscience CEO (via YouTube)
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. 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.’
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. 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.
The company did not respond to a request for comment on the price of the new treatment.
Aadi in May merged with the struggling public biotech Aerpio Pharmaceuticals and raised $155 million from private investors to commercialize Fyarro.
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The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.
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Joan Perelló, Sanifit CEO
Joan Perelló beat all the odds with his little Spanish biotech startup Sanifit.
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Supply chain issues and an inspection backlog have hindered manufacturing’s seemingly endless boom, following an influx of money. But a new threat looms over the industry: a strain of Windows malware.
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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)
As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.
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Pascal Soriot, AstraZeneca CEO (Chris Ratcliffe/Bloomberg via Getty Images)
While Europe is in the midst of a massive fourth Covid-19 surge, the UK has largely avoided the high levels of hospitalizations and deaths seen on the rest of the continent. And AstraZeneca CEO Pascal Soriot suggested Tuesday the reason for Britain’s better fortunes could be his company’s vaccine.
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Dragonfly Therapeutics is doing better and better in getting Big Pharma on its TriNKET platform. First Celgene, then BMS, and now Merck — again.
After Merck licensed its first candidate from the platform back in November 2020, the pharma giant is back for seconds as part of a 2018 collaboration between the two companies, which was initially focused on a number of solid tumor targets.
The companies expanded their collaboration last year with an agreement to develop and commercialize NK cell engaging immunotherapies for multiple targets in oncology, infectious diseases and immune disorders.
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The WHO’s Covid-19 Technology Access Pool (C-TAP) and the Medicines Patent Pool (MPP) finalized the deal with the Spanish National Research Council (CSIC), a public research institute, on Tuesday. It’s the first test license signed by the MPP, a United Nations-backed public health organization, and included in the WHO pool, according to the organizations. It covers all related patents and biological material necessary for manufacturing the tests, and CSIC has agreed to provide know-how and training.
https://endpts.com/fda-approves-pricey-new-treatment-for-ultra-rare-and-aggressive-form-of-sarcoma/
