Benvitimod

BASEL, Switzerland and LONDON and NEW YORK, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Roivant (Nasdaq: ROIV) today reported its financial results for the second quarter ended September 30, 2024, and provided a business update. Brepocitinib 52-week data from the Phase 2 NEPTUNE study in non-infectious uveitis (NIU) showed potential best-in-indication efficacy sustained to one year; first patients enrolled in Phase 3 NIU program IMVT-1402 cleared five Investigational New Drug (IND) applications across a range of therapeutic areas and FDA divisions, including the potentially registrational trial for difficult-to-treat rheumatoid arthritis (D2T RA) expected to initiate by March 31, 2025Batoclimab proof of concept data in Graves’ disease (GD) demonstrate potential of deeper IgG reduction with potent FcRn inhibition to transform treatment for GD patients who are not well controlled on antithyroid drugs (ATDs); initiation of potentially registrational trial to evaluate IMVT-1402 in GD expected by year endMosliciguat, a once-daily inhaled soluble guanylate cyclase (sGC) activator, unveiled as new pipeline program. Mosliciguat Phase 1b data in pulmonary hypertension (PH) patients demonstrated some of the highest pulmonary vascular resistance (PVR) reductions (~38%) in PH trials to date. The global Phase 2 PHocus trial for mosliciguat has been initiated in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD)Roivant continued to return capital through share repurchases with $106M purchased for the quarter, resulting in $754M cumulative share repurchases (inclusive of Sumitomo) through September 30, 2024Dermavant transaction with Organon closed on October 28, 2024. At closing, Roivant received $184M in cash, and Organon took on all of Dermavant’s remaining outstanding long-term debt, which, inclusive of Dermavant’s senior credit facility repaid at closing, had a carrying value of $336M as of September 30, 2024. In addition, Organon will pay Roivant a $75M milestone upon FDA approval for VTAMA in atopic dermatitis, with a target action date in Q1 2025Roivant reported consolidated cash, cash equivalents and marketable securities of approximately $5.4B at September 30, 2024 “I am pleased to finish out another quarter with continued clinical execution, including positive data in Graves’ Disease and FDA’s clearance of INDs in 5 indications at Immunovant,” said Matt Gline, CEO of Roivant. “I am also excited today to present the 52-week data from our Phase 2 study of brepocitinib in NIU. The sustained treatment benefits observed further our belief that brepocitinib is a potentially compelling and durable agent for a disease that is poorly treated today. We have a busy year ahead with major data expected in 2025 from Immunovant and Priovant, along with continued execution across other programs.” Recent Developments Immunovant: Endocrinology ProgramIn September 2024, Immunovant reported additional positive results from the Phase 2a trial of batoclimab in Graves’ Disease. Participants in the trial received 12 weeks of high dose batoclimab, 680 mg weekly by subcutaneous injection (SC) followed by 12 weeks of lower dose batoclimab, 340 mg weekly SC. At the end of the first 12 weeks, participants experienced a mean IgG reduction of 77% leading to a 76% Response rate. In addition, by the end of 12 weeks of higher dose batoclimab, 56% achieved an ATD-Free Response. During Weeks 13 to 24, the lower 340mg dose of batoclimab resulted in mean IgG reduction of 65% (vs. 77% on 680mg dose) with a correspondingly lower responder rate of 68%. In addition, a lower ATD-Free Response rate of 36% was also observed in the second 12 weeks. Patients who achieved at least a 70% IgG reduction at the end of the trial had nearly a threefold higher ATD-Free Response rate than those who did not (60% vs. 23%). Batoclimab was well tolerated with no new safety signals identified.In November 2024, additional data on the efficacy and safety of batoclimab in Graves’ thyroidal and extrathyroidal disease were presented in an oral presentation at the American Thyroid Association (ATA) 2024 Annual Meeting. These data showed that a 60% response rate (defined as T3 and T4 falling below the upper limit of normal (ULN) without increasing the ATD dose) was achieved by Week 2, demonstrating the rapidity of response to batoclimab 680mg dosed weekly. Meaningful improvements in proptosis and lid aperture were also observed at both Week 12 and Week 24. Pronounced improvements in multiple Thyroid-Related Patient-Reported Outcomes (ThyPRO-39) measurement scales were also observed, with ATD-Free Responders (defined as T3 and T4 falling below the ULN and ceasing all ATD medications) reporting greater improvements than other participants.Neurology ProgramIn November 2024, Immunovant announced completion of enrollment for patients included in Period 1 of the Phase 2b trial of batoclimab in CIDP, with data expected by March 31, 2025, to inform the trial design for a potentially registrational program with IMVT-1402.Rheumatology ProgramIn November 2024, Immunovant also announced FDA clearance of the IND for IMVT-1402 in D2T RA and expects to initiate a potentially registrational trial by March 31, 2025. Priovant: In September 2024, Priovant announced receipt of Fast Track designation from FDA for brepocitinib in NIU and enrolled the first patients in the Phase 3 program. New 52-week data from the Phase 2 NEPTUNE study of brepocitinib in NIU showed potential best-in-indication efficacy sustained to one year. Treatment failure rate in the 45 mg dose arm was 35% at week 52 vs. 29% at week 24. Treatment failure rate in the 15 mg dose arm was 56% vs. 44% at week 24. In each treatment arm only one additional patient failed from week 24 to 52. Other important efficacy measurements at week 52 were consistent with the week 24 data, including measurements of retinal vascular leakage and prevention and treatment of macular edema. Safety and tolerability were consistent with prior clinical studies of brepocitinib, with no new safety or tolerability signals identified. Brepocitinib has been dosed in over 1,400 subjects and patients with a safety profile that appears consistent with approved and widely prescribed JAK inhibitors. Pulmovant: In September 2024, Roivant unveiled mosliciguat, a potential first-in-class and best-in-category inhaled once-daily sGC activator. Mosliciguat is being developed for PH-ILD, which affects ~200,000 patients in the U.S. and Europe with limited or no treatment options. In September 2024, Pulmovant also presented data from the Phase 1b ATMOS study showing a single dose of inhaled mosliciguat in PH patients (N=38) led to sustained, clinically meaningful mean-max reductions in PVR of up to ~38%, one of the highest reductions seen in PH trials to date. Mosliciguat was generally well-tolerated, with low rates of treatment-emergent adverse events (TEAEs).Pulmovant initiated the global Phase 2 PHocus trial of mosliciguat in patients with PH-ILD. Roivant: In October 2024, Roivant reported the close of Organon’s acquisition of Dermavant. At closing, Roivant received $184M in cash and Organon took on all of Dermavant’s remaining outstanding long-term debt, which, inclusive of Dermavant’s senior credit facility repaid at closing, had a carrying value of $336M as of September 30, 2024. In addition, Organon will pay Roivant a $75M milestone upon FDA approval for VTAMA in atopic dermatitis, with a target action date in the first quarter of calendar year 2025. The transaction also includes payments of up to $950 million for the achievements of certain commercial milestones, in addition to the tiered royalties on net sales that Organon will pay Dermavant shareholders.Roivant continued to return capital through share repurchases with $106M purchased for the quarter ending September 30, 2024, resulting in $754M cumulative share repurchases (inclusive of the repurchase of Sumitomo’s stake in April 2024) through September 30, 2024.Roivant reported consolidated cash, cash equivalents and marketable securities of approximately $5.4B at September 30, 2024. Major Upcoming Milestones Kinevant plans to report topline data from the ongoing Phase 2 trial of namilumab for the treatment of sarcoidosis in the fourth quarter of calendar year 2024. Immunovant plans to have initiated 4-5 potentially registrational programs by March 31, 2025, and plans to have initiated studies in a total of 10 indications by March 31, 2026, for IMVT-1402. In pursuit of this goal, Immunovant now has active INDs in Graves’ Disease and difficult-to-treat rheumatoid arthritis and expects to initiate potentially registrational trials in these indications by December 31, 2024 and March 31, 2025 respectively. Topline data from the batoclimab trial in MG is expected by March 31, 2025. Results from this trial are expected to inform a decision regarding next steps for batoclimab in MG and the design of the MG program for IMVT-1402, which is expected to initiate by March 31, 2025. Data from the batoclimab trial in CIDP is expected by March 31, 2025 and will be used to inform the trial design for a potentially registrational program for IMVT-1402. Topline data from the current pivotal program evaluating batoclimab in thyroid eye disease (TED) now expected in the second half of calendar year 2025. Priovant plans to report topline data from the ongoing Phase 3 trial of brepocitinib in DM in the second half of calendar year 2025.Genevant Markman hearing in Pfizer / BioNTech action scheduled for December 2024. Summary judgment phase of Moderna action scheduled for second and third quarter of calendar year 2025; Moderna trial scheduled for September 2025. Second Quarter Ended September 30, 2024 Financial Summary Cash and Marketable Securities As of September 30, 2024, the Company had consolidated cash, cash equivalents, restricted cash and marketable securities of approximately $5.4 billion. Research and Development Expenses Research and development (R&D) expenses increased by $28.3 million to $143.1 million for the three months ended September 30, 2024, compared to $114.8 million for the three months ended September 30, 2023. This increase was primarily driven by increases in program-specific costs of $19.2 million, personnel-related expenses of $7.2 million, and share-based compensation of $1.6 million. Within program-specific costs, the increase of $19.2 million was primarily driven by an increase in expense of $34.2 million related to the anti-FcRn franchise, partially offset by a decrease in expense of $18.6 million related to RVT-3101, which was sold to Roche in December 2023. Non-GAAP R&D expenses were $132.4 million for the three months ended September 30, 2024, compared to $105.3 million for the three months ended September 30, 2023. General and Administrative Expenses General and administrative (G&A) expenses increased by $114.3 million to $202.9 million for the three months ended September 30, 2024, compared to $88.6 million for the three months ended September 30, 2023. This increase was primarily due to an increase in personnel-related expenses of $87.0 million, of which $79.1 million related to the one-time cash retention awards approved in July 2024 for each of Matthew Gline, Chief Executive Officer; Mayukh Sukhatme, President and Chief Investment Officer; and Eric Venker, President and Chief Operating Officer (the "2024 Senior Executive Compensation Program") and $6.6 million related to the special one-time cash retention bonus award granted to employees, following approval in December 2023. The increase was also driven by an increase in share-based compensation expense of $21.7 million, primarily due to the long-term equity incentive awards granted in July 2024 pursuant to the 2024 Senior Executive Compensation Program. Non-GAAP G&A expenses were $142.3 million for the three months ended September 30, 2024, compared to $49.6 million for the three months ended September 30, 2023. Loss from continuing operations, net of tax Loss from continuing operations, net of tax was $236.8 million for the three months ended September 30, 2024, compared to a loss from continuing operations, net of tax of $244.6 million for the three months ended September 30, 2023. On a basic and diluted per common share basis, loss from continuing operation was $0.25 and $0.28, respectively, for the three months ended September 30, 2024 and September 30, 2023. Non-GAAP loss from continuing operations, net of tax was $218.7 million for the three months ended September 30, 2024, compared to $154.8 million for the three months ended September 30, 2023. ROIVANT SCIENCES LTD.Selected Balance Sheet Data(unaudited, in thousands) September 30, 2024 March 31, 2024 Cash, cash equivalents and restricted cash$1,969,914 $6,506,189 Marketable securities 3,428,021 — Total assets 6,206,028 7,222,482 Total liabilities 625,986 773,953 Total shareholders’ equity 5,580,042 6,448,529 Total liabilities and shareholders’ equity 6,206,028 7,222,482 ROIVANT SCIENCES LTD.Condensed Consolidated Statements of Operations(unaudited, in thousands, except share and per share amounts) Three Months Ended September 30, Six Months Ended September 30, 2024 2023 2024 2023 Revenue, net$4,475 $3,648 $12,465 $8,131 Operating expenses: Cost of revenues 234 223 447 1,206 Research and development (includes $9,911 and $8,309 of share-based compensation expense for the three months ended September 30, 2024 and 2023 and $20,443 and $15,726 for the six months ended September 30, 2024 and 2023, respectively) 143,073 114,790 263,580 224,206 Acquired in-process research and development — 13,950 — 26,450 General and administrative (includes $59,443 and $37,755 of share-based compensation expense for the three months ended September 30, 2024 and 2023 and $96,284 and $76,472 for the six months ended September 30, 2024 and 2023, respectively) 202,881 88,576 302,773 179,858 Total operating expenses 346,188 217,539 566,800 431,720 Gain on sale of Telavant net assets — — 110,387 — Loss from operations (341,713) (213,891) (443,948) (423,589)Change in fair value of investments (48,375) 45,849 (63,601) 53,413 Change in fair value of liability instruments (635) 11,789 515 51,967 Gain on deconsolidation of subsidiaries — (17,354) — (17,354)Interest income (69,773) (14,299) (141,900) (31,014)Other expense, net 1,453 1,530 5,061 4,357 Loss from continuing operations before income taxes (224,383) (241,406) (244,023) (484,958)Income tax expense 12,458 3,236 24,421 4,911 Loss from continuing operations, net of tax (236,841) (244,642) (268,444) (489,869)(Loss) income from discontinued operations, net of tax (43,083) (86,476) 46,010 (169,094)Net loss (279,924) (331,118) (222,434) (658,963)Net loss attributable to noncontrolling interests (49,740) (26,791) (87,547) (62,820)Net loss attributable to Roivant Sciences Ltd.$(230,184) $(304,327) $(134,887) $(596,143)Amounts attributable to Roivant Sciences Ltd.: Loss from continuing operations, net of tax$(187,101) $(218,226) $(181,052) $(427,784)(Loss) income from discontinued operations, net of tax (43,083) (86,101) 46,165 (168,359)Net loss attributable to Roivant Sciences Ltd.$(230,184) $(304,327) $(134,887) $(596,143) Basic and diluted net (loss) income per common share:Basic and diluted loss from continuing operations$(0.25) $(0.28) $(0.25) $(0.56)Basic and diluted (loss) income from discontinued operations$(0.06) $(0.11) $0.06 $(0.22)Basic and diluted net loss per common share$(0.31) $(0.40) $(0.18) $(0.78) Weighted average shares outstanding: Basic 735,470,796 770,227,849 735,642,721 764,780,630 Diluted 735,470,796 770,227,849 735,642,721 764,780,630 ROIVANT SCIENCES LTD.Reconciliation of GAAP to Non-GAAP Financial Measures(unaudited, in thousands) Three Months Ended September 30, Six Months Ended September 30, Note 2024 2023 2024 2023 Loss from continuing operations, net of tax $(236,841) $(244,642) $(268,444) $(489,869)Adjustments: Research and development: Share-based compensation(1) 9,911 8,309 20,443 15,726 Depreciation and amortization(2) 724 1,205 1,419 2,694 General and administrative: Share-based compensation(1) 59,443 37,755 96,284 76,472 Depreciation and amortization(2) 1,094 1,235 2,184 2,485 Gain on sale of Telavant net assets(3) — — (110,387) — Other: Change in fair value of investments(4) (48,375) 45,849 (63,601) 53,413 Change in fair value of liability instruments(5) (635) 11,789 515 51,967 Gain on deconsolidation of subsidiaries(6) — (17,354) — (17,354)Estimated income tax impact from adjustments(7) (3,986) 1,100 (4,190) 369 Adjusted loss from continuing operations, net of tax (Non-GAAP) $(218,665) $(154,754) $(325,777) $(304,097) Three Months Ended September 30, Six Months Ended September 30, Note 2024 2023 2024 2023 Research and development expenses $143,073 $114,790 $263,580 $224,206 Adjustments: Share-based compensation(1) 9,911 8,309 20,443 15,726 Depreciation and amortization(2) 724 1,205 1,419 2,694 Adjusted research and development expenses (Non-GAAP) $132,438 $105,276 $241,718 $205,786 Three Months Ended September 30, Six Months Ended September 30, Note 2024 2023 2024 2023 General and administrative expenses $202,881 $88,576 $302,773 $179,858 Adjustments: Share-based compensation(1) 59,443 37,755 96,284 76,472 Depreciation and amortization(2) 1,094 1,235 2,184 2,485 Adjusted general and administrative expenses (Non-GAAP) $142,344 $49,586 $204,305 $100,901 Notes to non-GAAP financial measures:(1) Represents non-cash share-based compensation expense.(2) Represents non-cash depreciation and amortization expense.(3) Represents a gain on the sale of Telavant net assets to Roche due to achievement of a one-time milestone in June 2024.(4) Represents the unrealized (gain) loss on equity investments in unconsolidated entities that are accounted for at fair value with changes in value reported in earnings.(5) Represents the change in fair value of liability instruments, which is non-cash and primarily includes the unrealized (gain) loss relating to the measurement and recognition of fair value on a recurring basis of certain liabilities.(6) Represents the one-time gain on deconsolidation of subsidiaries.(7) Represents the estimated tax effect of the adjustments. Investor Conference Call Information Roivant will host a live conference call and webcast at 8:00 a.m. ET on Tuesday, November 12, 2024, to report its financial results for the second quarter ended September 30, 2024, and provide a corporate update. To access the conference call by phone, please register online using this registration link. The presentation and webcast details will also be available under “Events & Presentations” in the Investors section of the Roivant website at https://investor.roivant.com/news-events/events. The archived webcast will be available on Roivant’s website after the conference call. About Roivant Roivant is a biopharmaceutical company that aims to improve the lives of patients by accelerating the development and commercialization of medicines that matter. Roivant’s pipeline includes IMVT-1402 and batoclimab, fully human monoclonal antibodies targeting FcRn in development across several IgG-mediated autoimmune indications; brepocitinib, a potent small molecule inhibitor of TYK2 and JAK1 in development for the treatment of dermatomyositis and non-infectious uveitis; mosliciguat, an inhaled sGC activator in development for pulmonary hypertension associated with interstitial lung disease; and namilumab, an anti-GM-CSF monoclonal antibody in development for the treatment of pulmonary sarcoidosis. We advance our pipeline by creating nimble subsidiaries or “Vants” to develop and commercialize our medicines and technologies. Beyond therapeutics, Roivant also incubates discovery-stage companies and health technology startups complementary to its biopharmaceutical business. For more information, www.roivant.com. Roivant Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “would” and variations of such words or similar expressions. The words may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. Our forward-looking statements include, but are not limited to, statements regarding our or our management team’s expectations, hopes, beliefs, intentions or strategies regarding the future, and statements that are not historical facts, including statements about the clinical and therapeutic potential of our product candidates, the availability and success of topline results from our ongoing clinical trials and any commercial potential of our product candidates following applicable regulatory approvals. In addition, any statements that refer to projections, forecasts or other characterizations of future events, results or circumstances, including any underlying assumptions, are forward-looking statements. Actual results may differ materially from those contemplated in these statements due to a variety of risks, uncertainties and other factors. Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, those risks set forth in the Risk Factors section of our filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Contacts:InvestorsKeyur Parekh keyur.parekh@roivant.com MediaStephanie Leestephanie.lee@roivant.com

A pharmaceutical ingredient that’s been a key component in nasal decongestants for decades is having its efficacy called into question, and the FDA is now weighing whether to change the compound’s regulatory status — potentially leading to many widely used over-the-counter cold and allergy products being pulled from pharmacy shelves. But before the FDA takes any action, it wants to hear what the public thinks. The ingredient, oral phenylephrine, by itself and in combination with other active ingredients, is in decongestants sold under numerous brand names, such as Sudafed and Mucinex. All products containing oral phenylephrine continue to be available to consumers for now. But on Thursday, the FDA proposed an order that would remove oral phenylephrine from its non-prescription drugs guidelines, called the OTC Monograph. The regulator emphasized that this proposed order is due to lack of efficacy, not because of safety. At one time, phenylephrine was rare in decongestants because pseudoephedrine was the preferred active ingredient. But that compound can be made into the methamphetamine. Efforts to curb meth production led to the Combat Methamphetamine Act of 2005, which moved pseudoephedrine-containing drugs behind pharmacy counters. Drugmakers responded by making more products with oral phenylephrine, which is not covered by this law. presented by Sponsored Post The Promise of Value-Based Care and MedTech Innovation Monica Vajani, Executive Director for mHUB’s MedTech Accelerator, discusses how mHUB is helping innovators transition healthcare towards value-based care. By Monica Vajani - mHUB Last year, an FDA advisory committee discussed phenylephrine and unanimously voted that current scientific data do not support the ingredient’s efficacy as a nasal decongestant. The committee raised no concerns about the safety of oral phenylephrine when used at the recommended dose. The FDA now says it has conducted a comprehensive review of all available safety and efficacy data for oral phenylephrine as a nasal decongestant, including historical data used to support the compound 30 years ago. Speaking during a conference call with journalists Thursday, Theresa Michele, director of the FDA’s office of nonprescription drugs, said new data had become available since the advisory committee meeting, which the agency also reviewed. “At this point, based on the available data, and taking into account the advice of the advisory committee, we are proposing that oral phenylephrine is not effective to relieve nasal congestion at the recommended dose set forth in the monograph,” Michele said. Phenylephrine is also an ingredient in some nasal spray decongestants. The FDA’s proposal does not extend to such products. The proposed FDA order can be found here. Public comments may be submitted here (use docket number FDA-2024-N-4734 ) through May 7, 2025. After considering the comments, the agency may issue a final order removing oral phenylephrine from its guidelines, which would mean nasal decongestants could no longer contain the compound. Michele said the FDA would then provide manufacturers with time to either reformulate their medicines or remove oral phenylephrine-containing products from the market. Don’t let the autumn sniffles get you down. Here’s a look back at other recent regulatory news, including U.S. and European actions on several novel medications (alas, no new decongestants): Exclusive Heard at HLTH 2024: Insights from Innovative Healthcare Executives Executives from Imagine360, Verily, BrightInsight, Lantern, and Rhapsody shared their approaches to reducing healthcare costs and facilitating digital transformation. By MedCity News Decisions for Drugs and Devices —The FDA needs more time to review Organon drug Vtama as a treatment for atopic dermatitis. The company said the regulator has pushed out the December target date for a decision by three months to March 12, 2025. Vtama is from Roivant Sciences subsidiary Dermavant, which Organon recently acquired for $175 million up front. Under Dermavant, the topical drug won FDA approval in 2022 as a treatment for plaque psoriasis. —Novartis cancer drug Scemblix expanded its FDA-approved uses to include the treatment of newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ PML). The tablet, part of a class of drugs called STAMP inhibitors, initially landed accelerated FDA approval in 2021 as a treatment for advanced cases of Ph+ PML. —The FDA approved Iterum Therapeutics antibiotic Orlynvah. The regulatory decision covers the treatment of uncomplicated urinary tract infections in women who have limited or no oral antibacterial treatment options. The drug, whose main ingredient is sulopenem, is taken as a tablet twice daily for five days. —Astellas Pharma landed FDA approval for zolbetuximab, brand name Vyloy, as a treatment for gastric or gastroesophageal junction adenocarcinoma. It’s the first FDA approval for a drug targeting the cancer protein claudin 18.2 (CLDN18.2). The regulatory decision specifically covers patients whose cancer is negative for a different cancer protein called HER2. Drugs are already available for HER2-positive cancers. —In other Astellas news, the Japanese pharma company withdrew its European Medicines Agency application for geographic atrophy drug avacincaptad pegol. The company said it made the decision after discussions with the agency’s Committee for Medicinal Products for Human Use. The drug, administered as a once-monthly eye injection, won FDA approval in geographic atrophy last year and is marketed in the U.S. under the brand name Izervay. Failure to secure approval in Europe will make it harder for Astellas to recoup the $5.9 billion it spent to acquire Iveric Bio, the company that developed the drug. —Pfizer respiratory syncytial virus vaccine Abrysvo expanded its FDA approval to include adults age 18 through 59, broadening the market for the product. The initial FDA approval of Abrysvo last year covered patients age 60 and older. It’s also approved for use by pregnant individuals as a maternal vaccine; antibodies produced by the mother confer protection against RSV intended to last from the baby’s birth up to six months of age. —Gilead Sciences’ Trodelvy will no longer be available in the U.S. for treating urothelial carcinoma, an aggressive bladder cancer. After discussions with the FDA, the company decided to voluntarily withdraw the product, which had won accelerated approval in this indication in 2021. In May, the company reported Trodelvy failed its confirmatory Phase 3 test in advanced urothelial carcinoma. The withdrawal in urothelial carcinoma does not affect drug’s approved uses in breast cancer. —After previous regulatory setbacks, AbbVie finally landed FDA approval for Vyalev, a Parkinson’s disease drug/device combination product that provides 24 hour subcutaneous infusion of the medications foscarbidopa and foslevodopa. Last year, the regulator turned down the product’s application, asking for more information about the pump in the device. The FDA did not raise any questions about safety or efficacy, nor did it ask for another clinical trial. This past June, the agency again turned down AbbVie’s application for issues at the product’s third-party manufacturer. —There’s a new Pfizer drug for both hemophilia A and B. The FDA approved the antibody drug marstacimab for preventing or reducing frequency of bleeding episodes in patients 12 and older. Pfizer will market the drug under the brand name Hympavzi. As a once-weekly injectable medication, Hympavzi will provide an alternative to IV-infused hemophilia therapies. —The FDA needs more time to review Intercept Pharmaceuticals’ application seeking full regulatory approval of Ocaliva, which had received accelerated approval in 2016 for the rare liver disease primary biliary cholangitis. Intercept, now a subsidiary of Alfasigma, said the agency did not provide a new date for a regulatory decision. —Alzheimer’s disease drug Leqembi hit a regulatory obstacle in Australia, where the Therapeutic Goods Administration decided against registering the medication. Eisai said it will request a reconsideration as permitted under Australian law. It’s the second setback for the drug in recent months. Over the summer, the drug received a negative opinion from a European Medicines Agency committee concerned about the risk of severe side effects and limited benefit. Leqembi has approvals in the U.S., Japan, China, and several other markets. —The FDA approved a Thermo Fisher Scientific companion diagnostic to Voranigo, a Servier Pharmaceuticals’ drug approved over the summer for treating patients whose glioma is driven by mutations to IDH1 or IDH2 enzymes. The diagnostic, called Ion Torrent Oncomine Dx Target Test, identifies patients whose cancer carries the genetic signature making them eligible for treatment with the Servier drug. —Advanced cases of non-small cell lung cancer can now be treated with a wearable device. The FDA approved Novocure’s Optune Lua, a wearable technology that kills cancer cells with electrical signals administered via electrodes placed on the skin. Novocure’s “tumor-treating field” technology was first approved in 2011 as a treatment for a recurrent type of brain cancer. —The FDA rejected Zealand Pharma’s dasiglucagon, a drug developed to prevent and treat hypoglycemia in children who have the rare disease congenital hyperinsulinism. According to the Copenhagen-based biotech, the regulator cited issues at a third-party manufacturer. The FDA did not raise any concerns about the drug’s clinical data or safety. Advisory Committee Outcomes —An FDA advisory committee voted 11 to 3 that the benefits of Lexicon Pharmaceuticals’ sotagliflozin do not outweigh its risks when used as an adjunct to insulin to treat adults with type 1 diabetes and chronic kidney disease. But some committee members expressed support for use of the drug in subpopulations of patients. Sotagliflozin, given the brand name Zynquista, is expected to receive an FDA decision by Dec. 20. Sotagliflozin was approved last year for the treatment of heart failure and is marketed as Inpefa in this indication. —The European Medicine Agency’s Committee for Medicinal Products for Human Use yet again recommended against renewing the marketing authorization for Translarna, a PTC Therapeutics drug for Duchenne muscular dystrophy. Translarna received conditional marketing authorization in 2014. After the drug failed its Phase 3 study, the committee twice recommended against renewing the authorization. The latest opinion comes after the EMA directed the committee to reconsider, taking into account “the totality of evidence.” The committee said its reassessment “concluded that the effectiveness of Translarna has not been confirmed.” —Stealth BioTherapeutics moved a step closer to potential approval of the first drug for Barth syndrome. An FDA advisory committee voted 10 to 6 that Stealth’s drug, elamipretide, was effective at treating the ultra-rare mitochondrial disorder. The committee vote follows several regulatory setbacks, including a 2021 refuse-to-file letter in which the FDA pointed out that elamipretide failed its clinical trial. Stealth maintains there was improvement in a subgroup of patients and the short duration of treatment is the reason for the trial failure. An FDA decision is expected by Jan. 29. What’s New in Covid-19 —The European Commission granted marketing authorization to Novavax’s updated Covid-19 vaccine, Nuvaxovid. Authorization for the protein-based vaccine covers its use in those age 12 and older. The FDA authorized the updated version of Novavax’s Covid-19 vaccine in September. —In other Novavax Covid-19 news, the FDA pressed pause on the company’s plans for late-stage testing of a combination flu/Covid-19 vaccine as well as a standalone influenza vaccine. According to Novavax, the clinical hold stems from a report of motor neuropathy in a single patient outside the U.S. who received the combination vaccine in a Phase 2 study. The trial was completed in July 2023 and the serious adverse event was reported in September. Novavax said previous Covid-19 and influenza trials showed no signs of motor neuropathy. The company’s standalone Covid-19 vaccine is unaffected by the clinical hold. —In Covid-19 and flu testing news, the FDA granted marketing authorization to a Healgen Scientific over-the-counter, at-home test for both pathogens. The OTC Healgen Rapid Check Covid-19/Flu A&B Antigen Test uses a sample from a nasal swab. The authorization covers use of the product by people age 14 and older within five days of the start of symptoms. For children as young as 2, the sample must be collected by an adult.