STOCK RECAP #PipelineProspector

Pipeline Prospector August 2025: Novo’s Wegovy approved for MASH, Tonix Pharma’s fibromyalgia drug okayed

In August, the global pharmaceutical industry witnessed several regulatory upheavals and policy shifts in the US. The month began with the US President Donald Trump announcing that planned tariffs on drug imports into the US may reach 250 percent within 12 to 18 months.In the last days of July, Vinay Prasad, the head of the US Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER) had stepped down (reportedly under pressure). On August 12, Prasad was back at CBER as its head. Then, around the month end, the White House fired Centers For Disease Control and Prevention (CDC) director Susan Monarez after a clash with Health and Human Services (HHS) Secretary Robert F. Kennedy Jr. Four senior CDC officials resigned soon after.Despite this chaos, the pharma indices showed resilience and climbed upwards. The Nasdaq Biotechnology Index (NBI) rose 5.22 percent from 4,445.39 to 4,677.63. The SPDR S&P Biotech ETF (XBI) gained 5.92 percent from 84.83 to 89.85. And the S&P Biotechnology Select Industry Index (SPSIBI) climbed 4.9 percent from 6,671.56 to 6,998.36. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel) Novo’s Wegovy gets approved for MASH; Precigen’s Papzimeos okayed for rare respiratory conditionThe FDA granted accelerated approval to Novo Nordisk’s Wegovy (semaglutide) for treating metabolic-associated steatohepatitis, or MASH — a progressive liver disease linked to obesity. MASH causes inflammation and scarring of the liver. This approval makes the blockbuster obesity drug the first glucagon-like peptide-1 (GLP-1) receptor agonist approved for this condition. The US regulator also granted an accelerated approval Hernexeos (zongertinib), a new drug from Boehringer Ingelheim, for adults with advanced non-small cell lung cancer (NSCLC) with specific HER2 mutations who have undergone prior treatment. In studies, the drug helped 75 percent of patients who had chemotherapy see their cancer shrink or disappear completely.Insmed (stock up 25 percent in August) secured FDA approval for its med Brinsupri (brensocatib). This is the first treatment for non-cystic fibrosis bronchiectasis, a chronic lung disease that leads to persistent cough, recurring infections, and damage to the airways.Maryland-based biotech Precigen bagged FDA approval for Papzimeos (zopapogene imadenovec-drba), making it the first treatment for recurrent respiratory papillomatosis. This is a rare condition that causes benign growths in the airway and voice box. Patients often require multiple surgeries each year to remove these growths, and until now there was no approved drug for this condition. Precigen saw its stock shoot up 182 percent during the month. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel) Ionis’ Dawnzera becomes first RNA-targeting therapy for angioedema; Tonix’s fibromyalgia med okayedIonis Pharmaceuticals gained FDA approval for Dawnzera (donidalorsen), the first RNA-targeting preventive therapy for hereditary angioedema. This rare genetic disorder causes sudden and unpredictable swelling that can be life-threatening if it affects the airway.New Jersey-based biotech Tonix Pharmaceuticals received approval for Tonmya (cyclobenzaprine), the first new fibromyalgia treatment in over 15 years. Fibromyalgia is a chronic disorder that causes widespread pain, fatigue, sleep problems, and memory issues.Teva Pharmaceuticals won an expanded indication for Ajovy (fremanezumab) to prevent migraines in children between six and 17 years old.Jazz Pharmaceuticals reported a mix of clinical and regulatory developments. Its drug Modeyso (dordaviprone) received accelerated approval for diffuse midline glioma, a rare and aggressive brain tumor that occurs in both children and adults. Jazz also strengthened its pipeline by licensing SAN2355, an epilepsy drug candidate, from Danish biotech Saniona. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel) Novartis’ Sjögren’s disease med posts trial win; IO Biotech’s melanoma vaccine faces phase 3 setbackNovartis reported success for its investigational monoclonal antibody ianalumab in a phase 3 study in patients with Sjögren’s disease. This is a chronic autoimmune condition in which the body’s immune system attacks its own moisture-producing glands, leading to dry eyes, dry mouth, and other complications. Similarly, Regeneron announced positive results for cemdisiran in generalized myasthenia gravis, a rare condition that causes muscle weakness.In negative news from trials, IO Biotech announced results from a late-stage trial of its experimental melanoma vaccine in combination with Merck’s Keytruda (pembrolizumab). Melanoma is a serious form of skin cancer. The vaccine’s benefits were not statistically significant. This setback dragged IO Biotech’s stock down by 28 percent over the month. The company still plans to file for FDA approval.Allogene reported a patient death in a phase 2 trial on its CAR-T cancer therapy — cemacabtagene ansegedleucel. And Agios Pharmaceuticals disclosed that four patients taking its anemia drug Pyrukynd (mitapivat) had died, according to FDA’s Adverse Event Reporting System (FAERS) database.Valneva faced a different kind of setback — FDA suspended the use of its chikungunya vaccine Ixchiq after 21 hospitalizations and three patient deaths, including one linked to encephalitis, a type of brain inflammation. With Ixchiq off the market, Bavarian Nordic’s Vimkunya is now the only chikungunya vaccine available in the US. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel)Merck KGaA-Skyhawk in US$ 2 bn neurology deal; AbbVie acquires Gilgamesh’s psychedelic drugMerck KGaA struck a strategic research deal worth more than US$ 2 billion with Massachusetts-based Skyhawk Therapeutics to develop RNA-targeting small molecules for neurological conditions. The deal, valued at over US$2 billion, includes upfront and milestone payments as well as tiered royalties on future product sales. Under the agreement, Skyhawk will lead discovery and preclinical work, while Merck KGaA will assume development and commercialization.The month saw several sub-US$ 2 billion deals. Bayer signed a US$ 1.3 billion agreement with California-based Kumquat Biosciences to develop a cancer drug targeting KRAS mutations. These mutations are among the most common in cancer and occur in roughly 25 percent of cases. Eli Lilly entered a US$ 1.3 billion partnership with Boston-based Superluminal Medicines, an artificial intelligence firm. The collaboration aims to identify small-molecule drugs for obesity and metabolic diseases.Jazz Pharmaceuticals also licensed an epilepsy therapy from Denmark-based Saniona in a deal that could reach US$ 1 billion in value. And AbbVie acquired rights to Gilgamesh’s experimental psychedelic drug for depression for up to US$ 1.2 billion.Moreover, there were reports of Novartis planning to acquire San Diego-based Avidity Biosciences in early August. Avidity is valued at about US$ 5.8 billion. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel) Our viewWhile it may seem like it’s business as usual in the global biopharma industry, the recent slowdown in big-ticket M&A deals suggests undercurrents of caution. Innovation and strong pipelines are keeping the momentum alive, but escalating geopolitical tensions, drastic cuts in federal funding in the US and, a rise in protectionism the world over are forcing companies to rethink their risk appetite. This conservative shift, though understandable, also signals that innovation and global collaboration may stand to suffer. Access the Pipeline Prospector Dashboard for August 2025 Newsmakers (Free Excel)

Impressions: 6958

WEEKLY NEWS RECAP #Phispers

White House sacks CDC director; FDA okays Ionis’ Dawnzera, first RNA-based preventive for hereditary angioedema

The Trump administration has fired US Centers for Disease Control and Prevention (CDC) Director Susan Monarez, post a policy disagreement with Health and Human Services (HHS) Secretary Robert F. Kennedy Jr (RFK Jr). Four other senior CDC officials have also resigned over RFK Jr’s vaccine policy.The US Food and Drug Administration (FDA) has approved Ionis Pharmaceuticals’ Dawnzera (donidalorsen) for treating hereditary angioedema (HAE), a rare genetic disorder. This is the first RNA-targeting therapy cleared as a preventive treatment for HAE.FDA has suspended the use of Valneva’s chikungunya vaccine Ixchiq in the US, citing 21 hospitalizations and three deaths, including one from vaccine-linked encephalitis (inflammation of the brain).FDA has issued a Form 483 to Novo Nordisk’s Indiana plant, citing serious quality lapses and contamination risks.AbbVie has acquired Gilgamesh’s experimental psychedelic drug bretisilocin for US$ 1.2 billion. The drug is being tested for major depressive disorder. The drugmaker’s rheumatoid arthritis med Rinvoq (upadacitinib) has delivered strong results in two phase 3 trials for alopecia areata, a serious autoimmune hair-loss condition.Meanwhile, Eli Lilly’s oral GLP-1 pill — orforglipron — helped type 2 diabetes patients lose around 10.5 percent of body weight in late-stage studies.In news from other clinical trials, Akeso and Summit’s ivonescimab extended survival in EGFR-mutated non-small cell lung cancer (NSCLC). And Regeneron’s cemdisiran improved muscle function in patients with generalized myasthenia gravis, a rare disease that weakens muscles.Trump admin sacks CDC chief within weeks of hiring her; four officials resignThe White House has fired CDC Director Susan Monarez within a month of her appointment. Monarez refused to step down from her post after a policy disagreement with HHS Secretary RFK Jr. This prompted the Trump administration to fire Monarez as the head of America’s public health agency.According to the White House spokesman Kush Desai, Monarez was not “aligned with the President’s agenda of Making America Healthy Again”.The move has triggered resignations from at least four senior CDC officials, including the agency’s chief medical officer and leaders of immunization and emerging disease centers. The shakeup comes amid concerns over the agency’s approach to immunizations under RFK Jr, a known vaccine skeptic.FDA okays Ionis’ Dawnzera, first RNA-based preventive for hereditary angioedemaFDA has approved Ionis Pharmaceuticals’ Dawnzera (donidalorsen) for patients 12 and older with hereditary angioedema (HAE), a rare genetic disorder that causes swelling. This is the first RNA-targeting therapy cleared as a preventive treatment for HAE. The therapy, given as a self-injection every four or eight weeks, carries precautions for allergic reactions. Dawnzera will be launched in the coming days with a list price of US$ 57,462 per dose. Approves updated Covid-19 jabs for high-risk groups: FDA has approved updated Covid-19 vaccines from Moderna, Pfizer and Novavax , but for a limited group of adults (aged 65 or more) and children with underlying health conditions that put them at higher risk. Vaccines are no longer automatically authorized for healthy younger children, as the FDA has rescinded the broader emergency use approvals.FDA suspends use of Valneva’s chikungunya vaccine after deaths of three patientsFDA has suspended the use of Valneva’s chikungunya vaccine Ixchiq in the US, citing 21 hospitalizations and three deaths, including one from vaccine-linked encephalitis (inflammation of the brain). Ixchiq had generated €7.5 million (US$ 8.8 million) in first-half 2025 sales, about eight percent of Valneva’s revenue. With Ixchiq grounded, Bavarian Nordic’s FDA-approved Vimkunya is now the only chikungunya vaccine available in the US.AbbVie buys Gilgamesh’s depression drug for US$ 1.2 billionAbbVie will pay up to US$ 1.2 billion to acquire Gilgamesh’s experimental depression drug bretisilocin. The drug is being tested for major depressive disorder. Instead of buying the biotech outright, AbbVie is taking over the program while Gilgamesh spins out its other projects into a new company. Bretisilocin is a next-generation psychedelic that has shown to exert a shorter duration of psychoactive experience, while retaining an extended therapeutic benefit.Meanwhile, AbbVie’s rheumatoid arthritis drug Rinvoq (upadacitinib) has delivered strong results in two phase 3 trials for alopecia areata, a serious autoimmune hair-loss condition. AbbVie now plans to file for regulatory approval, potentially making Rinvoq a new rival to Eli Lilly’s Olumiant, Pfizer’s Litfulo (ritlecitinib), and Sun Pharma’s Leqselvi (deuruxolitinib). The drug is already a blockbuster, generating US$ 5.9 billion in 2024, up nearly 48 percent year-on-year. It is approved to treat several inflammatory diseases.J&J to invest US$ 2 bn in NC facility: Johnson & Johnson is investing US$ 2 billion over the next 10 years in order to increase its presence in North Carolina with an over 160,000 square foot dedicated manufacturing facility at the Fujifilm site in Holly Springs. This investment will create 120 new jobs, and also help in averting the import tariffs that may become applicable in future.FDA issues Form 483 to Novo’s Indiana plant; cites cat hair, contamination risksNovo Nordisk’s Bloomington, Indiana plant has received an FDA Form 483 after an agency inspection of the site in late June and July. The Form 483 has flagged serious quality lapses at the Indiana plant — a site it acquired through its US$ 16.5 billion acquisition of Catalent last year. Inspectors cited contamination risks ranging from cat hair to bacterial incursions, along with pest issues and repeated equipment failures.Regeneron’s experimental drug for myasthenia gravis scores phase 3 winRegeneron’s new drug cemdisiran has shown promising results in patients with generalized myasthenia gravis (gMG), a rare disease that weakens muscles. In a 24-week study, patients who received cemdisiran, a small RNA therapy injected under the skin once every three months, reported clear improvements in daily function. Regeneron plans to seek FDA approval in 2026.Lilly’s oral GLP-1 pill reduces 10.5% weight in phase 3 trial: Eli Lilly’s oral pill orforglipron helped people with type 2 diabetes and excess weight lose an average of 10.5 percent of body weight in a 72-week phase 3 trial. The pill also improved blood sugar levels. Analysts said the results matched expectations and should support an FDA submission this year.Akeso, Summit’s ivonescimab extends life in phase 3 lung cancer trialAkeso and Summit Therapeutics said their experimental cancer drug ivonescimab helped patients suffering from EGFR-mutated non-small cell lung cancer (NSCLC) live longer in a late-stage study. The phase 3 trial was held in China. This is the first time the medicine has shown that it can extend life, and not just slow disease progression.

Impressions: 1488