Sarepta Announces Voluntary Pause of Elevidys Shipments in US
The Food and Drug Administration asked Sarepta to pause shipments of the drug on Friday following news reports of a patient death in a clinical trial for another Sarepta therapy that works in a similar way. The company initially refused, which drew backlash.
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Yahoo
10 minutes ago
- Yahoo
Zymeworks Announces FDA Clearance of Investigational New Drug Application for ZW251, a Novel Glypican 3-Targeted Topoisomerase 1 Inhibitor Antibody-Drug Conjugate
Second antibody-drug conjugate (ADC) to progress into clinical development utilizing our proprietary payload and optimized antibody Preclinical results demonstrate strong anti-tumor activity and favorable tolerability profile Phase 1 clinical trial evaluating ZW251 in hepatocellular carcinoma (HCC) expected to be initiated in 2025 VANCOUVER, British Columbia, July 28, 2025 (GLOBE NEWSWIRE) -- Zymeworks Inc. (Nasdaq: ZYME), a clinical-stage biotechnology company developing a diverse pipeline of novel, multifunctional biotherapeutics to improve the standard of care for difficult-to-treat diseases, including cancer, inflammation, and autoimmune disease, today announced the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for ZW251, a novel glypican-3 (GPC3)-targeted ADC incorporating the company's proprietary topoisomerase 1 inhibitor (TOPO1i) payload, ZD06519, for the treatment of HCC. HCC is the most common type of primary liver cancer, with GPC3 expressed in over 75% of cases1. ZW251 is a potential first-in-class ADC engineered to selectively target GPC3. It is composed of a humanized IgG1 antibody conjugated to a novel camptothecin-based TOPO1i using a validated peptide cleavable linker. A drug-antibody-ratio (DAR) of four was selected for ZW251 as a lower DAR potentially could unlock a broader range of dose levels, a potential benefit as HCC patients are commonly challenged by impairment of liver function as a result of chronic liver disease and cirrhosis. In preclinical studies, ZW251 demonstrated strong activity in a range of HCC models, including a range of patient derived xenografts exhibiting a breadth of GPC3 expression and noteworthy tolerability in non-human primate toxicology studies at doses up to 120 mg/kg. 'This advancement marks the second ADC from our wholly-owned pipeline, utilizing our proprietary TOPO1i payload, to progress into clinical development, reinforcing confidence in our approach,' said Paul Moore, Ph.D., Chief Scientific Officer of Zymeworks. 'Like ZW191, which is currently in clinical trials, ZW251 utilizes the same payload paired with an optimized antibody. Our observations with ZW191 in the clinic to date provide a strong foundation as we initiate clinical development of this second ADC. With its novel design, unique mechanism of action, and promising preclinical activity, ZW251 offers the potential to meaningfully improve upon the current standard of care for HCC either as a monotherapy or in combination.' We plan to commence Phase 1 clinical studies for ZW251 in 2025. About Zymeworks Inc. Zymeworks is a global clinical-stage biotechnology company committed to the discovery, development, and commercialization of novel, multifunctional biotherapeutics. Zymeworks' mission is to make a meaningful difference in the lives of people impacted by difficult-to-treat conditions such as cancer, inflammation, and autoimmune disease. The Company's complementary therapeutic platforms and fully integrated drug development engine provide the flexibility and compatibility to precisely engineer and develop highly differentiated antibody-based therapeutic candidates. Zymeworks engineered and developed zanidatamab, a HER2-targeted bispecific antibody using the Company's proprietary Azymetric™ technology. Zymeworks has entered into separate agreements with BeOne Medicines Ltd. (formerly BeiGene, Ltd.) and Jazz Pharmaceuticals Ireland Limited, granting each exclusive rights to develop and commercialize zanidatamab in different territories. The U.S. FDA granted accelerated approval and China' s NMPA granted conditional approval for zanidatamab to treat adults with previously-treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer. The European Commission (EC) has granted conditional marketing authorization for Ziihera® as monotherapy for the treatment of adults with unresectable locally advanced or metastatic HER2-positive (IHC 3+) biliary tract cancer previously treated with at least one prior line of systemic therapy. Zanidatamab is the first and only dual HER2-targeted bispecific antibody approved for HER2-positive biliary tract cancer in the U.S., Europe, and China. In addition, zanidatamab is being evaluated in multiple global clinical trials as a potential best-in-class treatment for patients with multiple HER2-expressing cancers. Zymeworks is rapidly advancing a robust pipeline of wholly-owned product candidates, leveraging its expertise in both antibody-drug conjugates and multispecific antibody therapeutics targeting novel pathways in areas of significant unmet medical need. Phase 1 studies for ZW171 and ZW191 are actively recruiting and ZW251 is expected to enter clinical trials in 2025. In addition to Zymeworks' pipeline, its therapeutic platforms have been further leveraged through strategic partnerships with global biopharmaceutical companies. For information about Zymeworks, visit and follow @ZymeworksInc on X. Cautionary Note Regarding Forward-Looking StatementsThis press release includes 'forward-looking statements' or information within the meaning of the applicable securities legislation, including Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements in this press release include, but are not limited to, statements that relate to the efficacy and safety of zanidatamab and Zymeworks' product candidates; ongoing clinical studies and regulatory reviews; the potential addressable market of zanidatamab and Zymeworks' product candidates; the timing of and results of interactions with regulators; Zymeworks' clinical development of its product candidates and enrollment in its clinical trials; the timing and status of ongoing and future studies, clinical trials and the related data; expectations regarding future regulatory filings and approvals and the timing thereof; potential safety profile and therapeutic effects of zanidatamab and Zymeworks' product candidates; and the commercial potential of technology platforms and product candidates. When used herein, words such as 'plan', 'believe', 'expect', 'may', 'anticipate', 'potential', 'will', 'intend', 'continues', 'progress', and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon Zymeworks' current expectations and various assumptions. Zymeworks believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. Zymeworks may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various factors, including, without limitation: clinical trials, including any required confirmatory trials, may not demonstrate safety and efficacy of any of Zymeworks' or its collaborators' product candidates; any of Zymeworks' or its partners' product candidates may fail in development, may not receive required regulatory approvals, or may be delayed to a point where they are not commercially viable; conditional regulatory approval may be withdrawn or revoked if any of Zymeworks' or its partners' product candidates fail to satisfy the requirements of any such conditional regulatory approvals; regulatory agencies may impose additional requirements or delay the initiation of clinical trials; the impact of new or changing laws and regulations; market conditions, including the impact of tariffs; potential negative impacts of FDA regulatory delays and uncertainty and new policies implemented under the current administration, including executive orders, changes in the leadership of federal agencies such as the FDA, staff layoffs, budget cuts to agency programs and research, and changes in drug pricing controls; the impact of pandemics and other health crises on Zymeworks' business, research and clinical development plans and timelines and results of operations, including impact on its clinical trial sites, collaborators, and contractors who act for or on Zymeworks' behalf; zanidatamab and Zymeworks' product candidates may not be successfully commercialized; clinical trials and any future clinical trials may not demonstrate safety and efficacy of any of Zymeworks' or its collaborators' product candidates; inability to maintain or enter into new partnerships or strategic collaborations; and the factors described under 'Risk Factors' in Zymeworks' quarterly and annual reports filed with the Securities and Exchange Commission (copies of which may be obtained at and Although Zymeworks believes that such forward-looking statements are reasonable, there can be no assurance they will prove to be correct. Investors should not place undue reliance on forward-looking statements. The above assumptions, risks and uncertainties are not exhaustive. Forward-looking statements are made as of the date hereof and, except as may be required by law, Zymeworks undertakes no obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances, or to reflect the occurrences of unanticipated events. Contacts: Investor Inquiries:Shrinal InamdarSenior Director, Investor Relations(604) 678-1388ir@ Media Inquiries:Diana PapoveSenior Director, Corporate Communications(604) 678-1388media@ _______________________ 1 Wang HL et al., Arch Pathol Lab Med 2008.
Bloomberg
12 minutes ago
- Bloomberg
How US Health Insurers Got 2025 Numbers So Wrong
Hi, it's John in New York. In the last few months, I've watched a set of insurers that reliably grew profits for years collapse with spectacular speed. We try to unpack what's going on with US health insurance below. But first ... Every year, number crunchers at US health insurance companies look at their enrolled populations and calculate how many of them will break their arms, require a heart stent or be diagnosed with cancer. They use these figures to determine how much to charge in premiums to be able to cover patients' medical issues and still make a profit.
Fox News
12 minutes ago
- Fox News
World's most premature baby defies all medical odds to reach 1st birthday
An Iowa family recently celebrated a major milestone for a very special baby. Mollie and Randall Keen welcomed their son, Nash Keen, on July 5, 2024. He was born 133 days early, at just 21 weeks gestation. Guinness World Records has officially recognized Nash as the world's most premature baby to survive. Earlier this month, Nash — affectionately nicknamed "Nash Potato" — turned 1 year old, defying all odds. When he was born at the University of Iowa Health Care Stead Family Children's Hospital, Nash weighed just 285 grams (10 ounces) at birth — less than a grapefruit — and measured 24 centimeters long, according to a press release from the hospital. Two years before Nash's premature birth, the Keens lost a baby girl, McKinley, at 18 weeks gestation. At that time, Mollie Keen was diagnosed with an incompetent cervix, which is when the lower part of the cervix begins to open (dilate) too early, typically in the second trimester, the release shared. She also suffers from polycystic ovary syndrome (PCOS), a hormonal disorder that can cause fertility difficulties. Six months after their loss, the Keens found out another baby was on the way. "When we went to our local doctor's office for the 20-week scan for Nash, I just had some concerns about how I was feeling, so I asked them to look at me closer — which they normally don't do at that appointment — and they found I was already 2 centimeters dilated," Mollie Keen said. A few days later, she began having contractions and was placed on bed rest. "We were devastated," she said. "We thought we were going through the exact same thing — we thought we were going to lose this baby." The medical team at Stead Family Children's Hospital's neonatal intensive care unit (NICU) provides life-saving care for babies born at 21 weeks gestation and later. Fortunately, Mollie's care team was able to delay labor until just 10 hours after Nash surpassed the 21-week mark. "We want what is best for patients, so we really try to convey that we do not know what the outcomes will be for these extremely premature births," said Malinda Schaefer, M.D., Ph.D., the high-risk obstetrician who delivered Nash. "It is important for parents to understand most survival rates are low, and if babies do survive, they have a very high risk of long-term complications, even at 22 weeks." The team quickly provided medicine to Nash to support his organ development and to reduce the risk of complications, according to the release. "Sometimes babies born at 21 weeks are just too small for even our tiniest breathing tubes and intravenous lines," said neonatologist Amy Stanford, who treated Nash. "Our NICU team assessed Nash, and I was able to place a breathing tube. Once we had the breathing tube in, his heart rate stabilized and his oxygen levels were good." Even so, Nash's chances were slim, as no baby that young had ever survived. Before Nash's birth, the most premature baby to survive was Curtis Zy-Keith Means, born to Michelle Butler on July 5, 2020, at the University of Alabama at Birmingham Hospital, according to Guinness World Records. He was born at a gestational age of 21 weeks and 1 day, which was 132 days premature. "We never want the parents to lose hope, but many of them are in an unreal situation, so we have to be very honest with them," said Patrick McNamara, M.D., division director of neonatology at Stead Family Children's Hospital. "I would have told his parents, 'The chance is zero, but I hope I'm wrong, and we will do everything we can to help him.'" "I want him to see his story as a source of strength." Around the one-month mark, Stanford said, the team began to "breathe a little easier." "While we knew Nash still had a long journey ahead, that was the point when we started to feel more confident that he had a real chance of going home." "It was a subtle but powerful shift – from day-to-day survival to long-term hope." Nash received ongoing care during his 198 days in the hospital, as the team monitored his heart function and brain health. In addition to receiving many medications, he also underwent surgery for a perforated bowel, which has up to a 40% mortality rate. "They were on top of it every step of the way. They really gave him a fighting chance," said Randall Keen. "They were really honest with us during the whole journey about what his chances looked like. They made sure we were well-informed and kept us involved in all the decision-making." After more than six months in the hospital, Nash was finally able to go home in January 2025. He will continue to be monitored for ongoing health issues, including a minor heart defect, and is currently being weaned from oxygen. Nash is still on a feeding tube and wears hearing aids. Although he has had some developmental delays, Nash is getting stronger and more interactive with the help of ongoing therapy sessions, according to his mother. Stanford shared her ultimate goal for Nash — "that by the time he's 5 years old when he goes to kindergarten, no one will know that he was born so early." "Nash's remarkable outcome reflects the progress we've made by building on the experiences of those patients who came before him," she added. For more Health articles, visit Mollie Keen shared that she wants Nash to know how loved he is — and "how many people have cheered him on from the very beginning." "I want him to grow up and be healthy, happy and confident in who he is. I want him to see his story as a source of strength."
