Key Facts
- Weight-Loss Pill Success: Eli Lilly’s oral GLP-1 drug orforglipron delivered up to a 10.5% body weight reduction (approx. 23 lbs) in a Phase 3 trial for obese patients with diabetes Fiercebiotech Fiercebiotech. All tested doses met their primary endpoints, significantly improving weight, blood sugar, and heart risk factors over 72 weeks Fiercebiotech Fiercebiotech. A high-dose group saw 28% of patients lose at least 15% of body weight, approaching results of leading injectable therapies Fiercebiotech Fiercebiotech. Lilly plans regulatory filings based on these results. Company scientists are optimistic – calling the pill’s efficacy “as good as it gets for GLP-1 monotherapy…in the once-a-day small molecule space” Fiercebiotech.
- Historic FDA Approval for Rare Disease: The FDA granted full approval (no confirmatory trial required) to Precigen’s gene therapy Papzimeos (zopapogene imadenovec) – the first-ever treatment for adult recurrent respiratory papillomatosis (RRP), a rare airway tumor disease Cgtlive Cgtlive. Administered as four injections over 12 weeks, Papzimeos triggers an immune attack on HPV-6/11-infected cells, reducing the need for repeated throat surgeries Cgtlive Cgtlive. “Today marks a historic turning point…all adult RRP patients are now eligible for access to the first and only therapy that targets the root cause of the disease,” said Precigen CEO Helen Sabzevari Cgtlive. FDA’s Dr. Vinay Prasad noted the broader significance: “Randomized trials are not always needed to approve medical products and this approval is proof of that philosophy” Cgtlive.
- Big Pharma’s Psychedelic $1.2B Bet: AbbVie struck a deal worth up to $1.2 billion to acquire bretisilocin, an experimental fast-acting psychedelic-based depression drug from partner Gilgamesh Pharmaceuticals Reuters Reuters. The move thrusts AbbVie into the nascent psychedelic therapeutics field, which analysts peg as a potential $50B market Reuters Reuters. “Large Pharma has been less active exploring psychedelic compounds due to potential regulatory concerns…making today’s deal more significant,” observed BMO analyst Evan Seigerman Reuters. Bretisilocin, a short-acting 5-HT2A agonist, aims to provide the therapeutic benefit of psilocybin-like drugs with a shorter hallucinogenic experience Reuters. AbbVie will advance it into late-stage trials.
- Cancer & Rare Disease Trial Milestones: A bispecific antibody immunotherapy that targets PD-1 and VEGF (Akeso & Summit’s ivonescimab) became the first of its class to show improved overall survival in a Phase 3 lung cancer trial Fiercepharma Fiercepharma. Used with chemotherapy in EGFR-mutated lung cancer patients after TKI therapy, ivonescimab significantly extended patients’ lives, validating hopes that its tumor control benefits would translate into a survival gain Fiercepharma Fiercepharma. “Meeting the gold-standard overall survival endpoint is an important accomplishment,” wrote Evercore analysts, noting skepticism had lingered until this final analysis Fiercepharma. In another win, Regeneron announced its RNAi drug cemdisiran hit Phase 3 goals in generalized myasthenia gravis, reducing symptom scores by 2.3 points vs placebo – a result the company had earlier said “anything in the 2s…would be a great result” Fiercebiotech. Regeneron is now gearing up for FDA filing to challenge rivals in the competitive neuromuscular disease market Fiercebiotech Fiercebiotech.
- Global Regulatory Roundup: Beyond the Papzimeos approval, regulators saw other notable actions. In Europe, Abbott’s dissolving stent (Esprit BTK) for below-the-knee peripheral artery disease earned CE Mark clearance, after U.S. approval last year
ttps://www.fiercebiotech.com/medtech/abbotts-absorbable-below-knee-stent-nets-european-approval-peripheral-artery-disease?utm_source=ts2.tech#:~:text=Abbott%20has%20collected%20a%20green,the%20FDA%20early%20last%20year" target="_blank" rel="noreferrer noopener">Fiercebiotech
Fiercebiotech. The bioresorbable scaffold, which delivers everolimus then vanishes in 3 years, can help avert limb amputations in critical limb ischemia patients
Fiercebiotech Fiercebiotech. Meanwhile, U.S.-based Inovio announced the FDA agreed to a
rolling BLA submission for its DNA immunotherapy
INO-3107 – aiming to become the second RRP therapy on the market
Inovio Inovio.
“We believe INO-3107 has the potential to become the preferred product for RRP,” said Inovio CEO Jacqueline Shea, noting FDA’s openness to accelerating novel rare-disease treatments
Inovio. (Not all decisions were positive: investors braced for an Aug. 27 FDA verdict on Outlook Therapeutics’ Wet-AMD drug, a high-stakes
“inflection point” for the company
Medpath.)
M&A, Financing and Industry Moves: The deal flow stayed strong. Alongside AbbVie’s headline-grabbing buy, China’s Fosun Pharma licensed a Phase 2 inflammatory disease drug to UK startup Sitala Bio for $670 million in potential payments Fiercebiotech Fiercebiotech – Fosun’s second big out-licensing deal this month as Chinese innovators tap Western partners. On the startup front, the biotech IPO drought finally broke: LB Pharmaceuticals filed for a NASDAQ IPO (seeking $100M) to fund Phase 3 trials of its schizophrenia drug after a 6-month dry spell in biotech listings Biospace Biospace. And in manufacturing, Genentech (Roche) broke ground on a $700M biologics facility in North Carolina – its first East Coast plant Pharmaceuticalcommerce Pharmaceuticalcommerce. The 700,000 sq. ft. site will produce next-generation metabolic and obesity drugs and create 400 jobs by 2029 Pharmaceuticalcommerce Pharmaceuticalcommerce. “We are proud to break ground in North Carolina…taking action to deliver more life-changing medicines to patients faster, contribute to the local economy, and bolster innovation in the US,” said Genentech CEO Ashley Magargee Pharmaceuticalcommerce.
Health Policy Showdowns:Drug pricing tensions escalated in the UK, where Health Secretary Wes Streeting took an unusually hard line after talks with pharma companies broke down Theguardian Theguardian. He blasted the “shortsighted” industry for rejecting a “serious and generous” NHS offer and making “unaffordable” demands Theguardian. The standoff comes as US pricing pressure rises – President Trump’s push to cut U.S. drug prices to European levels has global firms wary of granting the UK (just ~2.5% of the market) any special low-price deals Theguardian Theguardian. Observers note pharma is determined not to let UK prices undercut higher U.S. revenues. In the U.S., the Biden-to-Trump administration transition (2025) continues to reshape health policy: HHS announced a new outside Healthcare Advisory Committee to recommend reforms to Medicare and Medicaid Wordandbrown Wordandbrown. HHS Secretary Robert F. Kennedy Jr. and CMS chief Dr. Mehmet Oz are seeking expert advice to “improve how care is financed and delivered” in federal programs Wordandbrown. “This committee will help us cut waste, reduce paperwork, expand preventive care…and modernize CMS programs with real-time data,” said Dr. Oz Wordandbrown. Secretary RFK Jr. added that the goal is high-quality, affordable care “without red tape, corporate greed, or excessive costs”, vowing to “unite the best minds in healthcare to deliver real results” Wordandbrown. On the trade front, the U.S. averted a transatlantic pharma trade war – Trump backed down from a threat to impose 100–250% tariffs on EU pharmaceutical imports, after a new deal excluded drugs from the spat Wordandbrown. However, hefty 50% U.S. tariffs on some Indian exports took effect Aug. 27 (part of broader trade measures), with pharmaceuticals pointedly exempted – underlining the sector’s critical supply-chain importance Livemint Reuters.
Public Health Alerts: Health agencies sounded alarms over resurgent infectious diseases. The Pan American Health Organization warned of a pertussis (whooping cough) resurgence across the Americas, now compounded by antibiotic-resistant strains Paho Paho. Cases of pertussis exploded from ~4,100 in 2023 to 43,000+ in 2024, with over 18,000 cases and 128 deaths already in the first half of 2025 Paho Paho. PAHO’s Dr. Pilar Ramón-Pardo urged immediate action: “Vaccination, surveillance, and the responsible use of antibiotics are critical to preventing pertussis from becoming a serious public health threat again…We still have time to contain this issue, but we must act now” Paho Paho. In the U.S., late-summer COVID-19 infections have been creeping upward due to a new Omicron subvariant nicknamed “Stratus” (XFG). Wastewater data shows Stratus now accounts for ~60% of circulating variants Latimes Latimes. COVID hospitalizations in hot spots (e.g. the U.S. Southwest) are rising, though still about half of last summer’s peak Latimes. Health officials note this year’s wave appears “less intense than last year”, but caution that updated fall vaccine rollouts have been delayed amid policy shifts Latimes Latimes. Meanwhile, New York City grappled with its worst Legionnaires’ disease outbreak in a decade: as of Aug. 26, 113 cases and 6 deaths have been confirmed in a Harlem cluster linked to cooling towers Kcra Nyc. City health teams are disinfecting sites and urging vigilance for flu-like symptoms in the area. And in an unusual case, U.S. officials confirmed a rare New World screwworm infection in a traveler returning from Central America – the first human case seen in years Hhs Hhs. The parasitic fly larvae was contained and poses “very low” risk to the public, but USDA has launched precautionary surveillance to ensure the livestock pest doesn’t gain a foothold Hhs Hhs.
Science & Tech Breakthroughs: Researchers unveiled innovations with game-changing potential. A new malaria-fighting technology developed at UCSF could provide year-long protection against mosquito-borne illnesses from just a simple dispenser. The team found that a novel insecticide formula, applied on a sheet of material the size of paper, can continuously repel and kill mosquitoes for up to 12 months Ucsf. This approach proved effective not only against malaria vectors but also mosquitoes carrying dengue, Zika, yellow fever and West Nile virus Ucsf. If deployed widely in endemic regions, such long-lasting “insecticide sheets” could radically reduce infections by creating protective perimeters in homes and public spaces. And in neuroscience, scientists reported progress in regenerative medicine for hearing loss: Lineage Cell Therapeutics inked a $12M partnership to advance an inner-ear cell transplant for restoring hearing in patients with cochlear damage Fiercebiotech Fiercebiotech. Early research suggests implanted otic progenitor cells might improve hearing function, offering hope to millions with sensorineural deafness. In oncology, Genmab’s antibody-drug conjugate Rina-S (rinatabart sesutecan) earned an FDA Breakthrough Therapy designation for advanced endometrial cancer Genmab, after Phase 2 data showed promising tumor responses. This status will expedite Rina-S’s development and review, potentially bringing a much-needed option to women with recurrent endometrial tumors Genmab. Overall, the past 48 hours showcased a whirlwind of progress – from cutting-edge drug trials and approvals to bold corporate moves and urgent public health interventions – underscoring the rapid pace of change in biotech, pharma, medicine and global health.
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