New gene therapy treatments could add $45 billion to the cost of health care over the next five years, according to a new report from CVS Health Corp. While the number is staggering, without knowing the price of the currently unapproved therapies, how many patients will seek treatment and the likelihood of approval, the pharmacy benefit manager's estimate is basically an educated guess. "Estimating the precise cost impact of gene therapies is very complicated and challenging," the report cautions.
To get to the $45 billion cost, CVS assumes the treatments will cost $2 million each, and 40% of potential patients will be treated with the ex vivo therapies while 60% of potential patients will be treated with the therapies that are delivered in vivo.
On the regulatory front, with four gene therapies already approved and more than 900 in development, the FDA has finalized six guidances and issued a draft guidance to clarify the rules of the road for developing and manufacturing the treatments. The draft focuses on how the FDA will evaluate differences between gene therapies intended to treat the same disease, filling in a regulatory gap by explaining how the agency will determine “sameness” when it comes to applying the seven years of orphan drug exclusivity to eligible gene therapies.
More investments needed to stem AMR
Adding further evidence about the global threat of the increase of antimicrobial-resistant (AMR) infections and dearth of new antibiotics to treat those conditions, Thomas Cueni, chair of the AMR Industry Alliance, said the findings from the alliance’s newly released report are “a wake-up call” as they estimate current investments in AMR-relevant R&D are not enough to sustain a viable pipeline that will be needed to combat infectious diseases globally. If AMR remains unchecked, the annual death toll could climb from 700,000 each year to 10 million by 2050, and the economic impacts could be on par with those of the 2008 financial crisis, the report notes.
Deals this week
Eyepoint Pharmaceuticals Inc. and Ocumension Therapeutics Ltd. have established an exclusive license agreement for the development and commercialization of Dexycu (dexamethasone intraocular suspension) 9% to treat postoperative inflammation following ocular surgery in mainland China, Hong Kong, Macau and Taiwan.
Oncoceutics Inc. said the National Brain Tumor Society has committed to providing more than $200,000 in funding, with the potential for multiple years, to support a phase II trial of ONC-201, the first imipridone dopamine receptor D2 antagonist (DRD2), in a molecular subset of high-grade gliomas. Curevac AG and the Coalition for Epidemic Preparedness Innovations (CEPI) are collaborating to develop an mRNA-based vaccine against coronavirus nCoV-2019. CEPI is contributing $8.3 million for development, manufacturing and clinical trials. Sorrento Therapeutics Inc. and Celularity Inc. created a clinical and manufacturing collaboration to treat and prevent the Wuhan coronavirus, 2019-nCoV, by expanding the use of Celularity’s CYNK-001, an allogeneic, off-the-shelf, placental-derived natural killer cell therapy.
Three biopharmas completed IPOs last week: Black Diamond Therapeutics Inc. priced an upsized offering for gross proceeds of about $201 million. The company’s shares (NASDAQ:BDTX) closed 108% higher at $39.48 on its debut. Annovis Bio Inc. closed its IPO, raising about $13.8 million. Arcutis Biotherapeutics Inc., which is developing topical therapies for dermatologic disorders, priced its IPO for gross proceeds of about $159.4 million.
Public offerings: Applied Therapeutics Inc. raised about $143.4 million; Autolus Therapeutics plc raised gross proceeds of approximately $80 million; Biohaven Pharmaceutical Holding Co. Ltd. priced its underwritten public offering for gross proceeds of about $250 million; Concert Pharmaceuticals Inc. priced an underwritten public offering with gross proceeds expected to be approximately $65 million; Denali Therapeutics Inc. generated gross proceeds of about $180 million; Immunogen Inc. raised gross proceeds of $104.2 million; Trillium Therapeutics Inc. closed its public offering for gross proceeds of about $117 million; Xenon Pharmaceuticals Inc. raised gross proceeds of $60 million; and Zymeworks Inc. closed its underwritten public offering for gross proceeds of $320.8 million.
Bavarian Nordic A/S completed the sale of its priority review voucher (PRV) to an undisclosed buyer and will receive $95 million. The company was awarded the PRV in September upon FDA approval of Jynneos (MVA-BN) to prevent smallpox and monkeypox.
Bergenbio ASA raised NOK219.9 million (US$23.9 million) in a private placement. The fresh injection of cash will enable the firm to expand its broad development program for lead drug candidate bemcentinib (BGB-324), a first-in-class Axl inhibitor, which is currently in phase II development in acute myeloid leukemia (AML), non-small-cell lung cancer (NSCLC), triple-negative breast cancer and melanoma.
Private transactions: IDbyDNA Inc., a metagenomics technology company, received $20 million in series B financing in a round led by Artis Ventures. Genesys Capital and others participated in the round. The company plans to use the money to accelerate uptake of its Explify software platform and to continue enhancing and expanding the platform. Lyra Therapeutics Inc. completed a $30 million series C financing and Vizgen Inc. closed a $14 million series A financing.
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The FDA finalized its 2018 draft guidance to help sponsors use minimal residual disease (MRD) as a biomarker in clinical trials or to support marketing approval of products for treating specific hematologic malignancies. Using MRD as a biomarker in drug development is distinct from the agency’s requirement for investigating, clearing or approving an in vitro diagnostic device for clinical use in measuring MRD, the agency said. Sponsors interested in developing a specific MRD assay for clinical use should consult the Office of In Vitro Diagnostics and Radiological Health in the Center for Devices and Radiological Health. The final guidance made clarifications throughout the document and added some definitions.
Word on the Street: BioWorld's favorite quotes of the week
"As long as we continue to have this sort of short-termism view of the word, a lot of these very useful, very productive interventions are generally going to be frowned on because the big costs are incurred now but the benefits accrue over a patient's lifetime."
Kenneth Thorpe, chairman of the Partnership to Fight Chronic Disease, who believes companies need to help payers and the public see the overall benefits of the potentially curative gene therapy treatments
"As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field."
Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, on the agency’s approach on clarifying the rules of the road for developing and manufacturing gene therapies
“Most assessments of the current clinical pipeline of antibiotics find it to be fragile and inadequate for the unmet medical need. Improving the future clinical pipeline will require more generous funding for R&D to advance these preclinical products, including more substantial private investment, which has decreased in the past few years. Private capital appears to be waiting on the sidelines for changes in antibiotics reimbursement, as the current systems disincentivizes innovation.”
Kevin Outterson, founding executive director of CARB-X, commenting on the findings in the latest AMR Industry Alliance report