Imetelstat

The year started well for the biotechnology industry. Steady dealmaking coupled with a rebound in initial public offerings helped lift investor confidence and, with it, drugmaker stocks.While that optimism has moderated somewhat since, a series of decisions by the Food and Drug Administration over the next three months could help the sector sustain its upturn. The agency may expand approvals for two cancer cell therapies and clear a third vaccine for respiratory syncytial virus. Sanofi and Regeneron await a decision on a lucrative new use for their top-selling Dupixent, while Geron could see decades of drug research finally pay off.Here are five FDA decisions to watch in the second quarter.CAR-T therapies for early multiple myeloma treatmentThe FDA this quarter could broaden the use of two cell therapies for multiple myeloma in decisions potentially worth billions of dollars to their respective developers.By April 5, the agency will decide whether to clear Johnson & Johnson and Legend Biotechs Carvykti for use in myeloma patients as early as after one relapse. That decision could be accompanied by a similar OK for Bristol Myers Squibbs Abecma and open up use of the therapies, which are currently only available in the U.S. for people with more advanced disease.European regulators have already endorsed earlier use of Abecma and Carvykti based on trial data showing they help people live longer without their disease progressing. But the FDA has appeared less supportive. The agency delayed a decision on Abecma last year and held a March advisory meeting to more closely scrutinize the data for it and Carvykti. In briefing documents, staff scientists flagged an apparent increase in the risk of death early on in the clinical trials of both therapies.The companies attributed that finding to limitations in the bridging therapies study participants could receive while waiting for CAR-T treatment. Panelists were convinced and backed both, agreeing that those issues likely wouldnt occur as frequently in practice.Nonetheless, the regulatory scrutiny suggests that the FDA might put new safety restrictions on Carvykti and Abecma if it chooses to clear them for earlier use. Jonathan GardnerModernas vaccine for RSVCOVID-19 vaccine makers Pfizer and Moderna have been facing the downside of their pandemic success, as falling sales have brought tough questions from investors.Moderna, for its part, aims to follow its COVID shot with several other messenger RNA-based vaccines, including one for respiratory syncytial virus thats now under FDA review.Respiratory syncytial virus, or RSV, can cause severe illnessin infants, older adults and people with compromised immune systems. Two vaccines, from GSK and Pfizer, are already approved to prevent RSV-related disease in adults over the age of 60. Sales of each have been strong, although GSK has established a clear edge.Moderna argues its vaccine, dubbed mRNA-1345, can compete. The shot is a pre-filled, single-dose vaccine, potentially offering more convenient administration.In Phase 3 testing, the vaccine was 84% effective at preventing infection with two or more symptoms in older adults. That efficacy does appear to wane over time, however.At a recent meeting, Centers for Disease Control and Prevention advisers reviewed known safety risks for GSKs and Pfizers shots, both of which were associated in very rare cases with Guillain-Barr syndrome, or GBS, a neurological condition affecting the nerves. Moderna has not reported any cases of GBS in Phase 3 testing of its shot.An FDA decision on mRNA-1345 is set for May 12. Delilah AlvaradoGerons imetelstat for myelodysplastic syndromesDrug development is a time-consuming, expensive process. Geron, a biotech founded 34 years ago to pursue stem cell research, knows this well.The companys long journey may finally reach an end by June 16, when the FDA will decide whether to approve a bone marrow disease drug Geron has been developing for nearly two decades. Called imetelstat, the drug treats myelodysplastic syndromes and some analysts believe it has blockbuster potential.Over the many years of Gerons research, imetelstat failed studies in multiple cancer types, and the company lost Johnson & Johnson as a partner along the way. But the drug finally succeeded in Phase 3 testing for myelodysplastic syndromes last year. And in March, it secured the endorsement of an FDA advisory panel despite skepticism from agency reviewers.Geron aims to challenge Bristol Myers Squibbs Reblozyl, first with a clearance in people with lower-risk disease and then in more people who have the condition. Stifel analyst Stephen Wiley estimated the drug could generate $2 billion in peak yearly sales, as it could be helpful in people who dont respond well to Reblozyl.Geron could be a buyout target, too, according to Wiley, though the company has said it plans to sell the drug on its own. Ben FidlerSanofi and Regenerons Dupixent for COPDDupixent is one of the pharmaceutical industrys most widely used and lucrative products. Approved for asthma, eczema and three other inflammatory conditions, the drug earned the companies more than $11 billion in sales last year.By June 27, the FDA could add another major indication to Dupixents list of approved uses. The regulator is currently reviewing the injectable medicine as a treatment for a type of chronic obstructive pulmonary disease, or COPD.If cleared by the FDA, Dupixent would be the first biologic drug for people with moderate-to-severe COPD who also have evidence of whats called Type 2 inflammation. A pair of late-stage studies, which enrolled nearly 1,900 people, showed Dupixent could cut the rate of COPD attacks by about one-third. Analysts therefore expect the FDA to issue an approval and forecast billions of dollars in additional yearly sales. Ned PagliaruloPfizers gene therapy for hemophilia BSome time during the second quarter, the FDA will decide whether to clear a gene therapy from Pfizer for hemophilia B, the less common form of the blood clotting disorder.Reaching this point has been a long time coming for Pfizer, which licensed the therapy from Spark Therapeutics a decade ago. In subsequent testing, treatmentlowered bleeding rates and reduced the need for regular blood transfusions powerful benefits meant to last many years with a single infusion.But its unclear whether Pfizer will find a market if the FDA chooses to approve its therapy. A rival genetic medicine, from CSL Behring and UniQure, won U.S. approval for hemophilia B in November 2022. However, few patients have been treated to date, sluggish uptake that could reflect the breadth of effective options available.The story is similar with Roctavian, a gene therapy BioMarin Pharmaceutical sells for the more common A form of hemophilia. Only three patients received Roctavian last year: two in Germany and one in the U.S.Pfizers therapy, which in January won Canadian approval, could provide another data point. But the company seems to be downplaying its potential. In recent presentations for investors, a once-weekly shot called marstacimab has gotten higher billing. Ned Pagliarulo '

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced the pricing of an underwritten offering consisting of 41,999,998 shares of its common stock at a price of $3.00 per share and pre-funded warrants to purchase 8,002,668 shares of its common stock. The pre-funded warrants are being sold at a price of $2.999 per pre-funded warrant. All of the securities in the offering are to be sold by Geron. The offering included participation from RA Capital Management, Fairmount, OrbiMed, Farallon Capital Management, Adage Capital Partners, L.P., Boxer Capital, Vivo Capital, Deep Track Capital, and multiple large investment management firms, in addition to other new and existing investors. The offering is expected to close on or about March 21, 2024, subject to the satisfaction of customary closing conditions. The gross proceeds to Geron from this underwritten offering, before deducting the underwriting discount and other estimated offering expenses, are expected to be approximately $150.0 million. Geron currently intends to use the net proceeds from this offering, together with its existing cash, cash equivalents, and current and noncurrent marketable securities, to fund the potential commercialization of imetelstat in low or intermediate-1 risk myelodysplastic syndromes in the U.S, and potential launch and commercialization of imetelstat in low or intermediate-1 risk myelodysplastic syndromes in the EU, subject to receipt of regulatory approvals, as well as continued development and potential regulatory submissions for imetelstat in relapsed/refractory myelofibrosis. Geron intends to use the remaining proceeds, if any, for working capital and general corporate purposes. TD Cowen, Stifel and Barclays are acting as joint book-running managers for the offering. Wedbush PacGrow is acting as co-manager for the offering. An automatically effective shelf registration statement on Form S-3 relating to the offering of the shares of common stock and pre-funded warrants described above was filed with the Securities and Exchange Commission (SEC) on January 4, 2023. A prospectus supplement and accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at . When available, copies of the prospectus supplement and accompanying prospectus relating to the offering may be obtained from: Cowen and Company, LLC, 599 Lexington Avenue, New York, NY 10022, by email at Prospectus_ECM@cowen.com or by telephone at (833) 297-2926; and Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, by telephone at 415-364-2720 or by email at syndprospectus@stifel.com. This press release does not constitute an offer to sell or the solicitation of an offer to buy any of these securities, nor will there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. About Geron Geron is a late-stage biopharmaceutical company pursuing therapies with the potential to extend and enrich the lives of patients living with hematologic malignancies. Its investigational first-in-class telomerase inhibitor, imetelstat, harnesses Nobel Prize winning science in a treatment that may alter the underlying drivers of disease. Use of Forward-Looking Statements Investors are cautioned that statements in this press release regarding the anticipated gross proceeds from the offering, Geron’s intended use of the net proceeds from this offering and completion and timing of the offering constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks and uncertainties related to: market and other conditions; the satisfaction of customary closing conditions related to the offering and the impact of general economic, industry or political conditions in the United States or internationally and the current or evolving effects of macroeconomic conditions, such as civil or political unrest or military conflicts around the world, such as the military conflict between Ukraine and Russia and in Israel, inflation, rising interest rates or prospects of a recession, on Geron’s business operations and activities. There can be no assurance that Geron will be able to complete the offering on the anticipated terms, or at all. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on other potential factors that could affect Geron’s results and other risks and uncertainties can be found under the heading “Risk Factors” or other similar headings found in documents Geron files from time to time with the SEC, including Geron’s current report on Form 8-K filed with the SEC on March 19, 2024. Geron expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in its expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.