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DATA COMPILATION #PharmaFlow

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Top first-in-class drug candidates of 2025: Ionis’ donidalorsen, Sanofi’s fitusiran, Cytokinetics’ aficamten await FDA approval
First‑in‑class drugs are therapies with entirely new approaches that improve patient outcomes and fundamentally change treatment paradigms.This week, PharmaCompass brings a compilation of 2025’s most promising first-in-class drug candidates. With their US Food and Drug Administration (FDA) action dates in 2025, many of these drugs, once approved, hold the potential of becoming blockbusters.Ionis’ donidalorsen, Sanofi’s fitusiran, Cytokinetics’ aficamten poised to transform patient careIonis’ donidalorsen is an exciting candidate designed to tackle hereditary angioedema (HAE), a rare condition marked by unpredictable and potentially life‑threatening swelling episodes.This novel antisense oligonucleotide works by “silencing” the messenger RNA that codes for prekallikrein — a protein that triggers inflammation leading to swelling. Instead of blocking a protein once it’s made, donidalorsen stops the cell from producing it in the first place. This precision approach could mean fewer infusions and reduced side effects compared to traditional treatments.Another promising candidate is Sanofi and Alnylam Pharmaceuticals’ fitusiran. It employs the technology of small interfering RNA (siRNA) to lower levels of antithrombin — a natural protein that, when in excess, can impair the blood’s ability to clot. By reducing antithrombin, fitusiran rebalances the blood’s clotting process in patients with hemophilia A and B.Similarly, Cytokinetics’ aficamten is designed to treat obstructive hypertrophic cardiomyopathy (oHCM), a condition in which the heart muscle becomes abnormally thick, obstructing blood flow. Aficamten works by inhibiting cardiac myosin, a protein that drives heart muscle contraction. This helps relieve symptoms like shortness of breath and chest pain. If approved, aficamten is expected to rake in annual sales of US$ 2.8 billion by 2030.UroGen, J&J’s candidates eye US$ 5 bn markets; AbbVie, Summit’s meds target lung cancerUroGen Pharma’s UGN‑102 is poised to redefine the treatment of non‑muscle‑invasive bladder cancer. This innovative formulation uses a reverse thermal gel technology that transforms from a liquid into a gel once inside the bladder. This change ensures that the chemotherapeutic agent, mitomycin, remains in contact with the bladder lining for a longer period, increasing its effectiveness at eradicating cancer cells and reducing the risk of recurrence.For patients, UGN‑102 could mean a non‑surgical, outpatient treatment option that is less invasive than traditional surgical methods. If approved, UGN-102 could address a US market of approximately 82,000 patients annually, translating into a market opportunity exceeding US$ 5 billion, according to company estimates.Johnson & Johnson’s nipocalimab is a new treatment aimed at helping people with autoimmune diseases — a group of conditions where the body’s defense system mistakenly attacks its own healthy cells. Nipocalimab works by blocking the neonatal Fc receptor (FcRn), thereby lowering the levels of pathogenic antibodies that drive many autoimmune disorders. J&J had acquired this FcRn blocker in 2020 as part of its US$ 6.5 billion takeover of Momenta Pharmaceuticals. J&J is trialing the drug in as many as 10 indications and expects peak annual sales in excess of US$ 5 billion.AbbVie’s antibody-drug conjugate (ADC), telisotuzumab vedotin, is designed as a “guided-missile” to deliver toxic drugs directly to cancer cells while sparing healthy tissues. Telisotuzumab vedotin targets c‑Met, a receptor that is over-expressed in certain cancers such as non‑small cell lung cancer (NSCLC). There are currently no approved anti-cancer therapies specifically for c-Met over-expressing NSCLC.Summit Therapeutics’ ivonescimab is a cutting‑edge bispecific antibody designed specifically for NSCLC. Unlike traditional therapies that target a single molecule, ivonescimab binds simultaneously to a tumor-specific antigen on cancer cells and to a receptor on T cells, effectively bringing these two together so the body’s own immune system can recognize and attack the tumor. This dual engagement strategy helps overcome common mechanism tumors.Insmed’s Brensocatib is an innovative, first‐in‐class oral inhibitor of dipeptidyl peptidase‑1 (DPP‑1) that targets a key step in the inflammatory cascade. DPP‑1 is responsible for activating neutrophil serine proteases — enzymes that, when overactive, can cause significant lung tissue damage in conditions such as non‑cystic fibrosis bronchiectasis. Brensocatib is expected to clock annual sales of US$ 2.8 billion by 2030.Gepotidacin, an investigational antibiotic developed by GSK, is poised to make a significant impact in the treatment of uncomplicated urinary tract infections (uUTIs) and urogenital gonorrhea. If approved, it would be the first oral antibiotic in a new class for uUTIs in over two decades.Arrowhead, Regenxbio, Ultragenyx address rare, genetic diseasesArrowhead Pharmaceuticals is developing an innovative gene-targeting therapy — plozasiran. This drug uses RNA interference (RNAi) to interrupt the gene responsible for producing apolipoprotein C‑III (APOC3), a protein that regulates blood triglyceride levels. For people with severe hypertriglyceridemia or familial chylomicronemia syndrome (FCS) — a severe and rare genetic disease for which there are currently no FDA‑approved treatments — high triglyceride levels can lead to pancreatitis and other serious cardiovascular issues. Regenxbio’s RGX‑121 is a one‑time gene therapy candidate for Hunter syndrome (MPS 2), a rare, inherited disorder that causes progressive neurological decline. The therapy uses a harmless virus to deliver a functional copy of the gene encoding iduronate‑2‑sulfatase (I2S) directly into the central nervous system. By restoring the missing enzyme, RGX‑121 aims to slow or even reverse the harmful buildup of substances in the brain that drive the disease’s progression.Another promising candidate is Ultragenyx Pharmaceuticals’ one-time intravenous gene therapy UX111 for Sanfilippo syndrome type A (MPS 3A). This devastating disorder, which leads to severe neurodegeneration in children, currently has no effective treatment. UX111 uses an adeno‑associated virus (AAV) to deliver a working copy of the SGSH gene, the deficiency of which is responsible for the disease. By enabling the body to produce the missing enzyme, UX111 holds the promise of slowing or even halting the progression of neurodegeneration.Our viewThis year has already seen several first-in-class approvals, such as Vertex’s suzetrigine (Journavx), a pioneering non‐opioid analgesic redefining pain management; SpringWorks’ mirdametinib (Gomekli), a breakthrough MEK inhibitor for a genetic disorder that often leads to the growth of benign tumors along nerves and; AstraZeneca and Daiichi’s ADC datopotamab deruxtecan (Datroway).Despite the volatile political environment in the US and its impact on federal agencies like the FDA, we are hopeful that many of the first-in-class drugs mentioned in this article will make a debut in 2025. 

Impressions: 5760

https://www.pharmacompass.com/radio-compass-blog/top-first-in-class-drug-candidates-of-2025-ionis-donidalorsen-sanofi-s-fitusiran-cytokinetics-aficamten-await-fda-approval

#Phispers by PHARMACOMPASS
27 Feb 2025

STOCK RECAP #PipelineProspector

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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: 286

https://www.pharmacompass.com/pipeline-prospector-blog/pipeline-prospector-august-2025-novo-s-wegovy-approved-for-mash-tonix-pharma-s-fibromyalgia-drug-okayed

#PharmaFlow by PHARMACOMPASS
04 Sep 2025

NEWS #PharmaBuzz

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https://investor.insmed.com/2025-08-12-FDA-Approves-BRINSUPRI-TM-brensocatib-as-the-First-and-Only-Treatment-for-Non-Cystic-Fibrosis-Bronchiectasis,-a-Serious,-Chronic-Lung-Disease

PRESS RELEASE
13 Aug 2025

https://www.businesswire.com/news/home/20250813362324/en/Amber-Specialty-Pharmacy-Begins-Dispensing-Newly-Approved-BRINSUPRI-brensocatib-for-Non-Cystic-Fibrosisbronchiectasis-NCFB

BUSINESSWIRE
13 Aug 2025

https://www.prnewswire.com/news-releases/vytlone-announces-new-partnership-with-insmed-302529214.html

PR NEWSWIRE
13 Aug 2025

https://www.prnewswire.com/news-releases/pantherx-rare-selected-by-insmed-to-dispense-brinsupri-brensocatib-for-the-treatment-of-non-cystic-fibrosis-bronchiectasis-302528964.html

PR NEWSWIRE
13 Aug 2025

https://www.prnewswire.com/news-releases/brensocatib-shows-consistent-efficacy-and-safety-across-three-prespecified-subgroups-in-new-data-from-landmark-aspen-study-302462008.html

PR NEWSWIRE
21 May 2025

https://www.prnewswire.com/news-releases/seven-oral-presentations-innovent-to-present-breakthrough-clinical-data-of-ibi363pd-1il-2-biasand-other-novel-drug-candidates-at-the-2025-asco-annual-meeting-302436193.html

PR NEWSWIRE
23 Apr 2025