Breakthrough Drugs, Big Deals & Public Health Alerts – Biotech and Health News Roundup (Aug 22–23, 2025)

Regulatory Approvals & Clinical Milestones

• Ionis’ RNA Drug Approved for Rare Disease: The FDA approved Ionis Pharmaceuticals’ Dawnzera (donidalorsen) as the first RNA-targeted therapy to prevent hereditary angioedema (HAE) attacks pharmexec.com. Dawnzera, an antisense drug, is cleared for patients 12 and older and showed dramatic efficacy – cutting HAE attack rates by up to ~81% in trials biopharmadive.com. Ionis fully owns Dawnzera (unusual for the company) and priced it at ~$57,462 per dose, in line with rival HAE treatments biopharmadive.com biopharmadive.com. “Patients are looking for better efficacy, better convenience and better tolerability… at least one of those boxes is not checked with existing treatments,” noted Ionis CEO Brett Monia, predicting many will switch to the more potent Dawnzera biopharmadive.com biopharmadive.com. This is the third HAE drug to reach the U.S. market in 2025, entering a crowded field that includes CSL’s Andembry and KalVista’s Ekterly, but it’s the first-ever HAE medicine to work via RNA silencing biopharmadive.com biopharmadive.com.
• Major Trial Readouts – Alopecia & SMA: AbbVie announced Rinvoq (upadacitinib) hit key endpoints in a second Phase 3 trial for alopecia areata, significantly regrowing hair in patients with severe scalp hair loss news.abbvie.com. The positive results will be submitted to regulators, positioning Rinvoq to challenge Eli Lilly’s Olumiant in the alopecia market fiercepharma.com. Meanwhile, Scholar Rock published full Phase 3 data for its muscle-targeted therapy apitegromab in spinal muscular atrophy. In the 188-patient SAPPHIRE study, adding apitegromab led to statistically significant motor function gains (improvements in Hammersmith scores) over 52 weeks neurologylive.com neurologylive.com. “The robust apitegromab data reinforce that effective SMA treatment should address both motor neuron preservation and muscle function… The findings support the value of muscle-targeted therapies for children and adults living with SMA,” said Dr. Akshay Vaishnaw, Scholar Rock’s President of R&D neurologylive.com. Apitegromab’s U.S. Biologics License Application is under priority review, with an FDA decision expected by Sept. 22, 2025 neurologylive.com.

Pharma & Biotech Industry Moves

• Gilead’s Kite Buys In-Vivo CAR-T Platform: In a notable cell therapy acquisition, Gilead Sciences’ Kite Pharma agreed to acquire Interius BioTherapeutics for $350 million reuters.com. Interius is developing “in vivo” CAR-T gene delivery – a method to engineer T-cells directly inside the patient rather than in a lab. Kite’s EVP Cindy Perettie said “In vivo therapy is a promising frontier… By combining Interius’s novel platform with Kite’s deep expertise… we aim to advance best-in-class in vivo therapies to bring them to patients more efficiently.” interiusbio.com interiusbio.com The approach could eventually simplify CAR-T cancer treatment by eliminating costly cell-processing steps interiusbio.com interiusbio.com. In a related deal, California-based XOMA is acquiring oncology firm Mural for up to $36 million to beef up its royalties portfolio ts2.tech. “This transaction… achieves the goal of our strategic review to maximize shareholder value,” said Caroline Loew, Mural’s CEO, highlighting the premium for investors ts2.tech ts2.tech.
• Investments in Manufacturing & Markets: Pharma giants are pouring capital into production amid geopolitical pressures. Johnson & Johnson announced a $2 billion investment to build a state-of-the-art biomanufacturing facility in North Carolina, creating 120 jobs ts2.tech. “J&J has more manufacturing facilities in the US than any other country, and we continue to strengthen our presence here,” CEO Joaquin Duato said, citing a commitment to U.S. innovation under new trade policies ts2.tech. In India, Apollo Hospitals Enterprise saw a major shareholder sell a ₹14.9 billion stake (~$170 million) via block trade ts2.tech. ~1.25% of Apollo’s equity changed hands (reportedly from Managing Director Suneeta Reddy), signaling significant portfolio shifts in Asia’s hospital sector ts2.tech.
• AI Drug Discovery Partnership in Japan: Tokyo-based startup Elix, Inc. announced that Kyorin Pharmaceutical, a mid-sized Japanese drugmaker, has adopted the AI platform “Elix Discovery” to augment its R&D biopharmatrend.com biopharmatrend.com. Launched in 2022, Elix’s system uses machine learning models to generate novel molecular structures and predict drug properties, aiming to accelerate early drug discovery biopharmatrend.com biopharmatrend.com. Kyorin will deploy the platform in-house (with Elix’s support) to bolster its pipeline. The move underscores how pharma companies are embracing AI to boost productivity – Kyorin expects the system to speed up identification of new drug candidates and “improve the overall efficiency and quality of research” biopharmatrend.com biopharmatrend.com. The deal follows similar AI collaborations in Japan (e.g. Eisai adopted Elix’s platform earlier in 2025) and reflects growing confidence in machine learning to find next-generation medicines.

Startups & Innovation Highlights

• Amgen “Golden Ticket” Winner in Singapore: Amid a tough venture funding climate, bright spots emerged for biotech startups. Singzyme, a small Singaporean biotech, won Amgen’s 2025 Golden Ticket award for its breakthrough bioconjugation platform ts2.tech. The prize gives Singzyme a year of free lab space at Amgen’s incubator and mentorship to advance its novel peptide–drug ligation technology for next-gen antibody-drug conjugates ts2.tech ts2.tech. “Singzyme’s novel platform reflects the strong scientific thinking and innovation emerging from Singapore’s biotech sector,” said Dr. Alan Russell, Amgen’s VP of Research Biologics, praising the startup’s approach to improving targeted cancer therapies ts2.tech. Past Golden Ticket winners have leveraged the boost to raise funding (e.g. VerImmune and Albatroz Therapeutics both secured seed rounds after winning) ts2.tech.
• Allos Pharma Revives Autism Drug Program: Allos Pharma Inc. (Cambridge, MA) announced a string of milestones for its neurodevelopmental disorder programs. It acquired global rights to arbaclofen, a selective GABA-B agonist previously tested in autism spectrum disorder and fragile X syndrome allospharma.com allospharma.com. This transfer includes a wealth of clinical data and manufacturing assets on arbaclofen. “This marks a foundational moment for Allos,” said co-founder Dr. Randall Carpenter. “Owning full rights to arbaclofen enables us to move swiftly and with strategic clarity as we build a sustainable pipeline targeting core features of ASD and related syndromes.” allospharma.com Allos also reported encouraging trial results: recent placebo-controlled studies in Canada and Europe “independently replicate the clinically meaningful improvements in social function” first seen with arbaclofen in a prior U.S. trial allospharma.com. Along with a new grant from the Simons Foundation to support further research allospharma.com, Allos plans to advance arbaclofen into Phase 3 development for autism and fragile X, aiming to finally bring this long-discussed therapy to market.
• Major Fundraise for Gene Therapy Startup: In biotech financing news, Kriya Therapeutics closed a $313 million fundraising round to fuel its gene therapy pipeline ts2.tech. This mega-round – one of 2025’s largest – signals that investors will still back platform companies with strong science even as overall biotech venture funding remains subdued (global biotech VC spending fell ~57% year-on-year in Q2) ts2.tech. Analysts note that big pharma partnerships and incubator programs have become vital lifelines for startups until market conditions improve ts2.tech. Indeed, even smaller funding wins are making a difference: for example, after winning the Amgen Golden Ticket, Albatroz Therapeutics raised a seed round and another startup VerImmune closed a pre-Series A ts2.tech – showing that non-dilutive support programs can help young biotechs bridge the gap to venture funding.

Medical Research & Innovation

• Hearing Aids Could Curb Dementia Risk: A long-term population study offers compelling evidence that treating mid-life hearing loss might help prevent dementia in later years. Researchers tracking ~3,000 older adults in the Framingham Heart Study found that individuals under age 70 who used hearing aids had a 61% lower risk of developing dementia over ~20 years ts2.tech ts2.tech. The findings, published in JAMA Neurology, showed no significant benefit for those who only began using hearing aids after 70, suggesting earlier intervention is critical ts2.tech. With 20% of participants developing dementia, the protective effect was dramatic. Experts call the results a potential game-changer for public health: addressing hearing loss in middle age could delay or prevent thousands of dementia cases. It underscores “the importance of early intervention for hearing loss for possible prevention of dementia,” as the authors wrote ts2.tech. The study strengthens the case to proactively screen and treat hearing impairment as a modifiable dementia risk factor.
• Phantom Limbs Rewrite Neuroscience Textbook: A Nature Neuroscience study by NIH scientists has upended a classic theory about brain plasticity after limb loss. Using fMRI, researchers scanned patients’ brains before and after arm amputation and were stunned to find “little to no difference” in the brain’s hand-area activity maps even years after the limb was gone ts2.tech. This contradicts the long-held belief that the cortex rapidly remaps itself (with adjacent regions taking over the area for the missing limb). “For many decades, cortical remapping after amputation has been a literal textbook example of brain plasticity,” noted co-author Dr. Chris Baker – but instead, the brain seems to “remember” the missing arm or leg ts2.tech. The persistence of the hand’s representation in the brain explains why vivid phantom limb sensations are so common – the original neural circuits endure intact. These insights could lead to better neuroprosthetics and pain therapies for amputees ts2.tech. By leveraging the brain’s preserved body map, researchers might amplify phantom limb signals to control prosthetic devices, or dampen them to treat phantom pain, rather than trying to force the brain to reorganize.

Public Health & Policy Developments

• Global Health Funding Cuts Hit Developing Nations: A new analysis warns of a steep decline in global health aid in 2025, with developing countries – especially in Africa – bearing the brunt. The IHME (Institute for Health Metrics and Evaluation) reported that development assistance for health (DAH) fell 21% from 2024 to 2025, largely due to U.S. budget cuts thinkglobalhealth.org. The United States, historically the top global health donor, slashed its contributions by an unprecedented 67% (over $9 billion) following a change in administration thinkglobalhealth.org thinkglobalhealth.org. “The climate has fundamentally changed,” said IHME’s Joseph Dieleman, noting the drastic drop is reshaping programs worldwide thinkglobalhealth.org. Low-income countries in sub-Saharan Africa are most affected – many rely heavily on aid and cannot fill the gap thinkglobalhealth.org. For example, Gambia, Lesotho, Malawi, and Mozambique have lost up to 16% of their health budgets, and Nigeria saw the largest absolute cut (> $400 million) thinkglobalhealth.org. The funding pullback is already hindering efforts against HIV, malaria and other diseases thinkglobalhealth.org. Major NGOs and UN agencies are downsizing: the World Food Programme is cutting ~6,000 staff after a 40% funding reduction, even as hunger crises deepen in places like Gaza and Sudan thinkglobalhealth.org. These trends raise alarms that progress on global health issues could regress without alternate funding or policy reversals.
• Famine Declared in Gaza’s Humanitarian Crisis: United Nations agencies officially declared a famine in Gaza for the first time on Aug 22, as the territory’s humanitarian situation reached a breaking point who.int. In a joint statement, WHO, UNICEF, WFP and FAO confirmed that extreme food scarcity is causing widespread malnutrition and starvation in Gaza amid ongoing conflict who.int. They reiterated urgent calls for an immediate ceasefire and unhindered humanitarian access to prevent mass casualties from hunger who.int. This grim milestone – famine – underscores the severity of the crisis: weeks of blocked aid, displacement, and infrastructure destruction have left huge numbers of people without sufficient food, clean water or medical care. Health officials warn that already, child wasting and stunting rates are surging, and communicable disease risks are rising as the population’s immunity plummets. The UN agencies stressed that only a durable political solution and sustained relief corridor can avert an even higher death toll from starvation and disease in the coming weeks.
• Heat Stress Threat Prompts New Global Guidelines: With climate change driving temperatures to record highs, the WHO and World Meteorological Organization (WMO) issued a new report and guidance to protect workers from extreme heat who.int. The technical report, drawing on five decades of data, warns that rising heat is already severely hurting worker health and productivity worldwide who.int. (Notably, 2024 was the hottest year ever recorded, per WMO.) The guidance urges employers and governments to implement stronger heat-safety measures as heatwaves become more common – from mandated rest breaks and hydration, to adjusted work hours and cooling interventions for outdoor and non-air-conditioned workplaces. According to the report, excessive workplace heat exposure is a growing public health emergency that heightens risks of heat stroke, dehydration, heart and kidney stress, and on-the-job accidents. “The health and productivity of workers are severely impacted by rising temperatures,” the WHO/WMO report states, underscoring that without adaptation, hundreds of millions of workers (especially in agriculture, construction, and manufacturing) face increasing heat-related health hazards who.int. The agencies call for urgent adoption of their heat-protection guidelines, noting that protecting worker health is also critical for economic development in a warming world.
• Chikungunya Outbreak Spans 100+ Countries: Health authorities are racing to contain the largest recorded global spread of chikungunya virus – a mosquito-borne disease typically confined to the tropics, which in 2025 has surged across over 119 countries, even reaching temperate regions drugtargetreview.com. Outbreaks have swept the Indian Ocean islands (Réunion, Mauritius, Mayotte) and autochthonous (local) transmission has been confirmed in parts of Europe (including France’s Grand Est and southeast) drugtargetreview.com drugtargetreview.com. The WHO estimates a staggering 5.6 billion people may be at risk of exposure this year as Aedes mosquitoes (which transmit chikungunya, dengue and Zika) expand their range drugtargetreview.com drugtargetreview.com. Experts attribute the spike to a perfect storm of factors: climate change enabling mosquitoes to thrive in new latitudes, rapid urbanization creating breeding grounds, increased international travel seeding the virus in new locales, and lack of immunity in populations that haven’t seen the virus before drugtargetreview.com. Chikungunya causes high fevers and severe joint pain; it’s rarely fatal but can lead to chronic arthritis-like symptoms. With no specific antiviral or widely available vaccine, the response has focused on vector control (e.g. mosquito mitigation) and accelerating R&D. Global biotech firms and academic labs are urgently working on chikungunya vaccines and treatments, some now in clinical trials, but widespread deployment is likely a few years off. Public health agencies are on alert, and in the interim the WHO has called for improved surveillance and community measures (like removing standing water and using repellents) to curb the spread drugtargetreview.com.
• Upheaval at U.S. Health Agencies: Turmoil hit U.S. federal health institutions as the new administration continued its shake-up of agency leadership and staff. The CDC quietly laid off 500–600 employees this week – a dramatic downsizing of the nation’s public health workforce, The Washington Post first reported kffhealthnews.org kffhealthnews.org. A federal official confirmed the mass terminations (part of a broader restructuring), which have raised alarm given the CDC’s frontline role in outbreak response and health security. Morale is low: in a separate development, 750 HHS employees signed a letter to Health Secretary Robert F. Kennedy Jr., accusing him of “dangerous and deceitful statements and actions” that undermine science and public trust kffhealthnews.org. The letter – an extraordinary rebuke to a sitting HHS chief – comes after a series of controversial moves by HHS, including rolling back certain vaccine promotion efforts and ousting longtime agency scientists. (Secretary Kennedy, an environmental lawyer and vaccine skeptic, has drawn criticism for pushing unproven treatments and conspiracy-tinged claims since taking office.) The administration defends the changes as needed “reforms,” but public health experts worry the loss of experienced staff and erosion of scientific norms at agencies like CDC, FDA, and NIH could cripple the government’s ability to manage health crises. Congress has begun to inquire into the CDC layoffs, even as agency veterans call the purge “devastating in scope” and warn it will take years to rebuild expertise. The situation underscores a broader politicization of U.S. health policy that observers say may hinder responses to everything from pandemics to chronic disease prevention.

Sources: Ionis HAE drug approval pharmexec.com biopharmadive.com; BioPharmaDive on Dawnzera pricing and positioning biopharmadive.com biopharmadive.com; AbbVie Rinvoq AA trial via FiercePharma fiercepharma.com; Lancet Neurology SAPPHIRE trial results neurologylive.com neurologylive.com; Reuters – Kite/Gilead’s Interius acquisition reuters.com and Interius press release interiusbio.com; PharmExec on XOMA-Mural deal ts2.tech; Reuters – J&J $2B NC facility and quote ts2.tech ts2.tech; Reuters – Apollo Hospitals stake sale ts2.tech; BiopharmaTrend on Kyorin adopting Elix AI platform biopharmatrend.com biopharmatrend.com; Laotian Times – Amgen Golden Ticket (Singzyme) and quote ts2.tech ts2.tech; Allos Pharma press release on arbaclofen rights & Dr. Carpenter quote allospharma.com allospharma.com; Allos press – ASD trial results allospharma.com; BiopharmaDive on VC funding and Kriya Therapeutics raise ts2.tech; Neurology Live – Hearing aids & dementia study (JAMA Neuro) ts2.tech ts2.tech; NIH news – Phantom limb cortical maps (Nature Neuro) ts2.tech ts2.tech; WHO/UN joint release – Gaza famine declaration who.int; WHO/WMO report on heat stress who.int; Drug Target Review – Chikungunya outbreak global spread drugtargetreview.com drugtargetreview.com; KFF Health News/Washington Post – CDC layoffs & HHS staff letter kffhealthnews.org kffhealthnews.org.