Polyneuropathies

Alnylam CEO Yvonne Greenstreet, M.D., characterizes Amvuttra's entrance in the suddenly competitive ATTR-CM market as a "category growth story." With attention focused on their recently approved drugs for transthyretin amyloid cardiomyopathy (ATTR-CM), there was a serendipitous timeliness to Alnylam and BridgeBio presenting their quarterly updates on consecutive days.While BridgeBio blew away analyst projections on Wednesday, reporting sales of $37 million for Attruby in its first full quarter on the market, Alnylam countered with positive news as well for Amvuttra on Thursday.Since Amvuttra earned its FDA label expansion to treat patients with ATTR-CM so late in the first quarter on March 20, there were no significant sales to report for the indication. But the company is pointing to encouraging signs in its uptake.“Our first priority out of the gate was the access setup and we’re pleased to say it’s progressing exactly to plan, enabling the second half growth story that we have portrayed,” Tolga Tanguler, Alnylam’s chief commercial officer, said during a conference call.Tanguler explained that 80% of TTR volume in the U.S. flows through 170 health systems, and that the company had gained formulary placements of Amvutta within four weeks of the label expansion at more than half of the systems. He added that a “majority” have initiated Amvuttra treatment for ATTR-CM patients.Tanguler also reported early feedback from patients and doctors indicates “excitement for a new mechanism of action in ATTR-CM.”“What we’re hearing from prescribers is that the Amvuttra’s disruptive value proposition resonates and is enabling this early encouraging launch momentum,” Tangular said.Alnylam and BridgeBio are challenging powerhouse Pfizer, which has dominated the market with its booming Vyndaqel franchise. In 2024, the Pfizer treatments achieved sales of $5.4 billion, which was up from $3.3 billion in 2023.Alnylam CEO Yvonne Greenstreet, M.D., said there is plenty of room for new options for ATTR-CM patients.“We really are in a category growth story,” Greenstreet said. “What we’re seeing is continued evidence of a large, growing, and frankly, unsatisfied market and I think we’re very excited to be able to participate.” Alnylam points to its experience with treating patients with polyneuropathy caused by hereditary transthyretin amyloidosis (hATTR-PN). The company earned approvals for Onpattro (2018) and Amvuttra (2022) to treat the condition.“We are finding that in categories where the majority of patients remain untreated, more treatment options mean more voices helping to raise disease awareness ultimately accelerating category growth and increasing the number of patients on therapy,” Tanguler said. “We believe about 80% of the addressable patient population remains untreated. This bodes well for the opportunity ahead in ATTR-CM.” In the first quarter, sales for Amvuttra and Onpattro came in at $310 million and $49 million, which were up a combined 37% year over year and 4% sequentially. The combined $359 million TTR franchise sales were in line with the analysts' forecast of $355 million.In 2024, sales of the two treatments were at $1.22 billion. With the new indication for Amvuttra, the company is sticking to its previously announced guidance for the pair to generate between $1.6 billion and $1.725 billion in 2025.For the company overall, revenue was up in the quarter 20% to $594 million. Alnylam is guiding to overall sales of between $2.05 billion and $2.25 billion, which would be a 31% increase year over year at midpoint.

Moderna\'s R&D costs fell 19% to $856 million in the first quarter.\n Moderna is deprioritizing its flu/COVID combination vaccine in younger adults amid a push to cut around $1.5 billion from its operating expenses. The biotech disclosed the changes alongside news of a delay to the expected approval of the vaccine in older adults. Massachusetts-based Moderna filed for FDA approval of its mRNA-1083 combination shot last year. As of February, the mRNA specialist was aiming to win approval in adults aged 50 years and older this year and expand the label to cover younger adults in 2027. Moderna axed both targets in its first-quarter results Thursday.The biotech has deprioritized the development of mRNA-1083 in people aged 18 to 49 years. Moderna is still working to get the vaccine to older adults, but the process is taking longer than hoped. Having filed for approval using immunogenicity data, Moderna has learned the FDA wants to see phase 3 efficacy data before signing off on the vaccine. The request pushes the targeted approval date back into 2026.Uptake of standalone COVID-19 and flu vaccines is lowest in the demographic deprioritized by Moderna. As of last month, the Centers for Disease Control and Prevention (CDC) estimated 36.2% of people aged 18 to 49 years in the U.S. had received a flu vaccine for the 2024 to 2025 season. Uptake rose to 48.9% in people aged 50 to 64 years and 72.0% in seniors.  The COVID-19 vaccination data follow the same pattern. Around 14% of people aged 18 to 49 years have received a 2024 to 2025 COVID-19 vaccine, according to the CDC, compared to 44.2% of people aged 65 years and older. Stephen Hoge, M.D., Moderna’s president, talked up the prospects of the combination shot at an event run by TD Cowen in March. Hoge said Moderna’s market research found around two-thirds of people are likely to prefer the combination product over monotherapy vaccines. “We do believe that the combination product is the majority, for convenience, and ultimately on healthcare systems is much less of a burden. You can do twice as many injections and take half as much time, half the infrastructure, much more efficient from a storage perspective, all of the above,” Hoge said. “So, for all those reasons, we really do believe the combo is the best.”Moderna used its first-quarter results to outline its ambition to reduce its operating expenses by up to $1.7 billion by 2027. R&D costs fell 19% to $856 million in the first quarter. Moderna attributed the drop to “lower clinical development costs across respiratory programs due to timing of trial activities and program wind-downs.”The decline in spending on respiratory programs was partly offset by increased investment in the individualized neoantigen therapy mRNA-4157 and norovirus programs. Moderna said the FDA has lifted the hold it put on its norovirus program in response to a case of Guillain-Barré syndrome. The timing of the phase 3 readout will depend on case accruals. 

18 presentations across five cancer types include new insights on the potential of checkpoint inhibitors and bispecific antibodies TARRYTOWN, N.Y. , May 01, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced new and updated data from its oncology and hematology portfolio will be shared at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place from May 30 to June 3 in Chicago, IL. Eighteen presentations will share the latest insights from ongoing research of approved and investigational treatment regimens across a range of difficult-to-treat cancers including non-melanoma and melanoma skin cancer, lung cancer, lymphoma and multiple myeloma. “Our broad oncology and hematology programs are uniquely designed to investigate regimens that could provide meaningful impact for people living with difficult-to-treat cancers across all stages of the treatment paradigm,” said George D. Yancopoulos , M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron . “Our PD-1 inhibitor Libtayo is the standard of care in advanced cutaneous squamous cell carcinoma, and in clinical trials, our investigational BCMAxCD3 bispecific antibody linvoseltamab has demonstrated a compelling profile in relapsed or refractory multiple myeloma. At ASCO, our presentations showcase how we are seeking to further transform the treatment of these diseases with updates from two key programs – our Phase 3 trial exploring adjuvant Libtayo in high-risk cutaneous squamous cell carcinoma and early data from investigational combinations of linvoseltamab and different proteasome inhibitors in third-line or higher multiple myeloma.” Notable presentations at ASCO on Regeneron’s oncology pipeline include detailed efficacy and safety findings from the Phase 3 C-POST trial evaluating the adjuvant use of the PD-1 inhibitor Libtayo in post-surgical high-risk cutaneous squamous cell carcinoma (CSCC). The results will be presented in an oral session on Saturday, May 31 . In hematology, Regeneron will debut results from two cohorts of the LINKER-MM2 trial, which is exploring combinations of linvoseltamab, Regeneron’s investigational BCMAxCD3 bispecific antibody. These include combinations of linvoseltamab with carfilzomib or bortezomib in relapsed/refractory (R/R) multiple myeloma (MM) after at least two lines of therapy, which will be featured in two rapid oral presentations on Monday, June 2 . In addition, the results of a cooperative group study reporting on the primary analysis of a randomized Phase 2 trial of vidutolimod in combination with an anti-PD-1 versus anti-PD-1 as neoadjuvant therapy in stage 3 resectable melanoma will be presented in an oral session on Tuesday, June 3 . Vidutolimod is a toll like receptor 9 antagonist that was acquired by Regeneron in 2022. The full list of Regeneron presentations at ASCO includes: The potential uses of Libtayo in adjuvant CSCC, fianlimab, REGN7075, vidutolimod, and the combinations with linvoseltamab described above are investigational, and their safety and efficacy in these uses have not been fully evaluated by any regulatory authority. Fianlimab, REGN7075 and vidutolimod are not currently approved for use in any indication. Odronextamab is conditionally approved as Ordspono™ in the European Union for the treatment of R/R follicular lymphoma (FL) or diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy, although its safety and efficacy have not been fully evaluated by any other regulatory authority. Linvoseltamab is conditionally approved as Lynozyfic™ in the European Union for the treatment of adult patients with R/R multiple myeloma who have received at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy, although its safety and efficacy have not been fully evaluated by any other regulatory authority. The U.S. Food and Drug Administration (FDA) accepted for review the Biologics License Applications for linvoseltamab and odronextamab with respective target action dates for FDA decisions of July 10, 2025 and July 30, 2025 . About Regeneron in Cancer We aspire to turn revolutionary discoveries into medicines that can transform the lives of those impacted by cancer. Our team around the world is driven to solve the needs and challenges of those affected by one of the most serious diseases of our time. Backed by our legacy of scientific innovation and a deep understanding of biology, genetics and the immune system, we’re pursuing potential therapies across more than 30 types of solid tumors and blood cancers. Our cancer strategy is powered by cutting-edge technologies and therapies that can be flexibly combined to investigate potentially transformative treatments for patients. Oncology assets in clinical development comprise nearly half of Regeneron’s pipeline, and include checkpoint inhibitors, bispecific antibodies and costimulatory bispecific antibodies. Our approved PD-1 inhibitor Libtayo serves as the backbone of many of our investigational combinations. To complement our extensive in-house capabilities, we collaborate with patients, healthcare providers, governments, biopharma companies and each other to further our shared goals. Together, we are united in the mission to serve as a beacon of transformation in cancer care. Libtayo U.S. FDA-approved IndicationsLibtayo is a prescription medicine used to treat: People with a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) that has spread or cannot be cured by surgery or radiation. People with a type of skin cancer called basal cell carcinoma (BCC) when your BCC cannot be removed by surgery (locally advanced BCC) or when it has spread (metastatic BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with a HHI. Adults with a type of lung cancer called non-small cell lung cancer (NSCLC). LIBTAYO may be used in combination with chemotherapy that contains a platinum medicine as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor does not have an abnormal “EGFR,” “ALK,” or “ROS1” gene. LIBTAYO may be used alone as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor tests positive for high “PD-L1,” and your tumor does not have an abnormal “EGFR,” “ALK,” or “ROS1” gene. LIBTAYO may be used in combination with chemotherapy that contains a platinum medicine as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor does not have an abnormal “EGFR,” “ALK,” or “ROS1” gene. LIBTAYO may be used alone as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor tests positive for high “PD-L1,” and your tumor does not have an abnormal “EGFR,” “ALK,” or “ROS1” gene. It is not known if Libtayo is safe and effective in children. IMPORTANT SAFETY INFORMATION FOR U.S. PATIENTS What is the most important information I should know about LIBTAYO?LIBTAYO is a medicine that may treat certain cancers by working with your immune system. LIBTAYO can cause your immune system to attack normal organs and tissues in any area of your body and can affect the way they work. These problems can sometimes become severe or life-threatening and can lead to death. You can have more than one of these problems at the same time. These problems may happen anytime during treatment or even after your treatment has ended. Call or see your healthcare provider right away if you develop any new or worsening signs or symptoms, including: Lung problems: cough, shortness of breath, or chest pain Intestinal problems: diarrhea (loose stools) or more frequent bowel movements than usual, stools that are black, tarry, sticky or have blood or mucus, or severe stomach-area (abdomen) pain or tenderness Liver problems: yellowing of your skin or the whites of your eyes, severe nausea or vomiting, pain on the right side of your stomach-area (abdomen), dark urine (tea colored), or bleeding or bruising more easily than normal Hormone gland problems: headache that will not go away or unusual headaches, eye sensitivity to light, eye problems, rapid heartbeat, increased sweating, extreme tiredness, weight gain or weight loss, feeling more hungry or thirsty than usual, urinating more often than usual, hair loss, feeling cold, constipation, your voice gets deeper, dizziness or fainting, or changes in mood or behavior, such as decreased sex drive, irritability, or forgetfulness Kidney problems: decrease in your amount of urine, blood in your urine, swelling of your ankles, or loss of appetite Skin problems: rash, itching, skin blistering or peeling, painful sores or ulcers in mouth or nose, throat, or genital area, fever or flu-like symptoms, or swollen lymph nodes Problems can also happen in other organs and tissues. These are not all of the signs and symptoms of immune system problems that can happen with LIBTAYO. Call or see your healthcare provider right away for any new or worsening signs or symptoms, which may include: chest pain, irregular heartbeat, shortness of breath or swelling of ankles, confusion, sleepiness, memory problems, changes in mood or behavior, stiff neck, balance problems, tingling or numbness of the arms or legs, double vision, blurry vision, sensitivity to light, eye pain, changes in eyesight, persistent or severe muscle pain or weakness, muscle cramps, low red blood cells, or bruising Infusion reactions that can sometimes be severe or life-threatening. Signs and symptoms of infusion reactions may include: nausea, vomiting, chills or shaking, itching or rash, flushing, shortness of breath or wheezing, dizziness, feel like passing out, fever, back or neck pain, or facial swelling Rejection of a transplanted organ. Your healthcare provider should tell you what signs and symptoms you should report and monitor you, depending on the type of organ transplant that you have had Complications, including graft-versus-host disease (GVHD), in people who have received a bone marrow (stem cell) transplant that uses donor stem cells (allogeneic). These complications can be serious and can lead to death. These complications may happen if you underwent transplantation either before or after being treated with LIBTAYO. Your healthcare provider will monitor you for these complications Getting medical treatment right away may help keep these problems from becoming more serious. Your healthcare provider will check you for these problems during your treatment with LIBTAYO. Your healthcare provider may treat you with corticosteroid or hormone replacement medicines. Your healthcare provider may also need to delay or completely stop treatment with LIBTAYO if you have severe side effects. Before you receive LIBTAYO, tell your healthcare provider about all your medical conditions, including if you: have immune system problems such as Crohn’s disease, ulcerative colitis, or lupus have received an organ transplant have received or plan to receive a stem cell transplant that uses donor stem cells (allogeneic) have received radiation treatment to your chest area have a condition that affects your nervous system, such as myasthenia gravis or Guillain-Barré syndrome are pregnant or plan to become pregnant. LIBTAYO can harm your unborn baby Females who are able to become pregnant: Your healthcare provider will give you a pregnancy test before you start treatment You should use an effective method of birth control during your treatment and for at least 4 months after your last dose of LIBTAYO. Talk to your healthcare provider about birth control methods that you can use during this time Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with LIBTAYO are breastfeeding or plan to breastfeed. It is not known if LIBTAYO passes into your breast milk. Do not breastfeed during treatment and for at least 4 months after the last dose of LIBTAYO Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. The most common side effects of LIBTAYO when used alone include tiredness, muscle or bone pain, rash, diarrhea, and low levels of red blood cells (anemia). The most common side effects of LIBTAYO when used in combination with platinum-containing chemotherapy include hair loss, muscle or bone pain, nausea, tiredness, numbness, pain, tingling, or burning in your hands or feet, and decreased appetite. These are not all the possible side effects of LIBTAYO. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Regeneron Pharmaceuticals at 1-877-542-8296. Please see full Prescribing Information, including Medication Guide. About Regeneron's VelocImmune Technology Regeneron 's VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved or authorized fully human monoclonal antibodies. This includes Dupixent® (dupilumab), Libtayo, Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb), Inmazeb® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz® (pozelimab-bbfg). In addition, REGEN-COV® (casirivimab and imdevimab) had been authorized by the FDA during the COVID-19 pandemic until 2024. About RegeneronRegeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases. Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite®, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases. For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X. Forward-Looking Statements and Use of Digital Media This press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, the nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Products”) and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Product Candidates”) and research and clinical programs now underway or planned, including without limitation Libtayo® (cemiplimab) and Regeneron’s Product Candidates discussed or referenced in this press release (such as linvoseltamab, REGN7075, fianlimab, and odronextamab); the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s Product Candidates and new indications for Regeneron’s Products, such as adjuvant Libtayo in high-risk cutaneous squamous cell carcinoma, linvoseltamab (as a monotherapy or in combination with other cancer treatments discussed or referenced in this press release) in relapsed/refractory (“R/R”) multiple myeloma, REGN7075 in combination with Libtayo and chemotherapy in non-small cell lung cancer (“NSCLC”), fianlimab in combination with Libtayo in perioperative melanoma or NSCLC, odronextamab in R/R follicular lymphoma, and the other clinical programs discussed or referenced in the press release; uncertainty of the utilization, market acceptance, and commercial success of Regeneron’s Products and Regeneron’s Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the studies discussed or referenced in this press release, on any of the foregoing or any potential regulatory approval of Regeneron’s Products and Regeneron’s Product Candidates (such as those referenced above); the ability of Regeneron’s collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and Regeneron’s Product Candidates; the ability of Regeneron to manage supply chains for multiple products and product candidates and risks associated with tariffs and other trade restrictions; safety issues resulting from the administration of Regeneron’s Products and Regeneron’s Product Candidates (such as those referenced above) in patients, including serious complications or side effects in connection with the use of Regeneron’s Products and Regeneron’s Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize Regeneron’s Products and Regeneron’s Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron’s Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement or copay assistance for Regeneron’s Products from third-party payors and other third parties, including private payer healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payors and other third parties and new policies and procedures adopted by such payors and other third parties; changes in laws, regulations, and policies affecting the healthcare industry; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron’s Products and Regeneron’s Product Candidates (including biosimilar versions of Regeneron’s Products); the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license, collaboration, or supply agreement, including Regeneron’s agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable) to be cancelled or terminated; the impact of public health outbreaks, epidemics, or pandemics on Regeneron 's business; and risks associated with litigation and other proceedings and government investigations relating to the Company and/or its operations (including the pending civil proceedings initiated or joined by the U.S. Department of Justice and the U.S. Attorney's Office for the District of Massachusetts ), risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA® (aflibercept) Injection), the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron’s business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron’s filings with the U.S. Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2024 and its Form 10-Q for the quarterly period ended March 31, 2025 . Any forward-looking statements are made based on management’s current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron . Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise. Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron 's media and investor relations website (https://investor.regeneron.com) and its LinkedIn page (https://www.linkedin.com/company/regeneron-pharmaceuticals). Source: Regeneron Pharmaceuticals, Inc.