Ensifentrine

OhtuvayreTM (ensifentrine) recorded net product sales of $36.6 million in Q4 and $42.3 million in 2024 More prescriptions filled through February 2025 than in Q4 2024 Over 4,600 unique prescribers with ~55% of Tier 1 HCPs prescribing Ohtuvayre through February 2025 Phase 2 programs in bronchiectasis and fixed-dose combination in COPD advance Conference call today at 9:00 a.m. EST / 2:00 p.m. GMT LONDON and RALEIGH, N.C., Feb. 27, 2025 (GLOBE NEWSWIRE) — Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), a biopharmaceutical company focused on respiratory diseases, announces its financial results for the fourth quarter and full year ended December 31, 2024, and provides a corporate update. “2024 was another transformational year for Verona Pharma with the US approval and launch of Ohtuvayre (ensifentrine), the first novel inhaled therapy available for the maintenance treatment of Chronic Obstructive Pulmonary Disease (“COPD”) in over 20 years,” said David Zaccardelli, Pharm. D., President and Chief Executive Officer. “We are very pleased to report the extremely strong start to the launch continues to build momentum with more prescriptions dispensed through February 2025 than in the entire fourth quarter of 2024. “More than 4,600 unique healthcare professionals (“HCPs”) including approximately 55% of our Tier 1 HCPs have prescribed Ohtuvayre. Additionally, to date, more than 275 HCPs have prescribed Ohtuvayre to over 20 patients in their practice. Ohtuvayre is being prescribed for maintenance therapy across a broad COPD population including those receiving background single, dual and approximately 50% on triple therapy. These data strengthen our belief that ensifentrine’s novel bronchodilator and non-steroidal anti-inflammatory activity will redefine the treatment paradigm for COPD. “Alongside our commercialization efforts, in the third quarter we initiated two Phase 2 clinical trials: a dose-ranging trial with glycopyrrolate, a long-acting muscarinic antagonist (“LAMA”), to support a nebulized fixed-dose combination of ensifentrine and glycopyrrolate for the maintenance treatment of COPD, and a study assessing the safety and efficacy of nebulized ensifentrine in patients with non-cystic fibrosis bronchiectasis (“bronchiectasis”). We are pleased to report the glycopyrrolate dose-ranging trial was successfully completed and we plan to initiate a dose ranging Phase 2b trial with a fixed-dose combination of ensifentrine and glycopyrrolate in the second half of 2025. “In addition to our successful Ohtuvayre launch, our development partner in Greater China, Nuance Pharma, announced in February 2025 that Ohtuvayre was approved in Macau, the first approval outside of the US, for the maintenance treatment of COPD. In addition, Nuance Pharma has completed enrollment in its own pivotal Phase 3 trial in China to evaluate ensifentrine for the maintenance treatment of COPD and expects to report results in mid-2025. “In 2025, we will continue to build on the successful commercialization of Ohtuvayre in the US while progressing our Phase 2 programs. We are also initiating activities with regulatory authorities in the European Union and the UK for potential marketing authorization application submissions for Ohtuvayre.” Program Updates and Key Milestones The Company’s near-term milestones include: In the second half of 2025, the Company plans to start a dose-ranging Phase 2b trial to assess the safety and efficacy of a fixed-dose nebulized combination of ensifentrine with glycopyrrolate. The Company has successfully completed a Phase 2 dose-ranging trial with glycopyrrolate to support this program. The Company continues to enroll subjects in a Phase 2 trial to assess the efficacy and safety of nebulized ensifentrine in patients with bronchiectasis. In 2025, the Company plans to progress the regulatory activities for potential marketing authorization application submissions for Ohtuvayre for the maintenance treatment of COPD in the European Union and in the UK. In mid-2025, the Company’s development partner in Greater China, Nuance Pharma, is expected to report results from its pivotal Phase 3 trial evaluating ensifentrine for the maintenance treatment of COPD in China. Fourth Quarter and Recent Highlights In October 2024, the Company gave four oral presentations and presented two posters on analyses from the ENHANCE studies at CHEST Annual Meeting 2024. These included subgroup data supporting improvements in lung function, symptoms and quality of life, as well as reductions in the rate of exacerbations, regardless of COPD severity (moderate or severe), smoking status (current or former) and chronic bronchitis (with or without). An analysis of Ohtuvayre’s impact on reducing exacerbation rates and COPD-related healthcare resource utilization was also presented. In November 2024, the 2025 GOLD report added Ohtuvayre to the COPD treatment algorithm. Also in November 2024, the Company completed enrollment in a Phase 2 dose-ranging trial with glycopyrrolate, a LAMA, supporting a fixed-dose combination program for the maintenance treatment of COPD via a nebulizer. On January 1, 2025, Ohtuvayre’s product specific J-code became effective. In February 2025, the Company’s development partner in Greater China, Nuance Pharma, announced Ohtuvayre has been approved in Macau for the maintenance treatment of COPD in adult patients. This is the first regulatory approval outside of the US. Fourth Quarter 2024 Financial Results Cash position: Cash and cash equivalents at December 31, 2024, were $399.8 million (December 31, 2023: $271.8 million). Product sales: Net sales of Ohtuvayre were $36.6 million for the fourth quarter ended December 31, 2024 (Q4 2023: $0 million). The Company received FDA approval on June 26, 2024 and the product was commercially available beginning in August 2024. Cost of sales: Cost of sales was $2.0 million for the fourth quarter ended December 31, 2024 (Q4 2023: $0 million), which included Ohtuvayre manufacturing costs incurred after US approval, inventory overhead costs and sales-based royalties. R&D Expenses: Research and development (“R&D”) expenses were $7.9 million for the fourth quarter ended December 31, 2024 (Q4 2023: $4.1 million). This increase of $3.8 million was primarily due to an increase of $3.2 million in clinical trial and other development costs as we initiated two Phase 2 studies in the third quarter of 2024 related to the combination of nebulized ensifentrine and glycopyrrolate and for Ohtuvayre in patients with bronchiectasis, and an increase of $1.1 million in share-based compensation. SG&A Expenses: Selling general and administrative expenses (“SG&A”) were $45.1 million for the fourth quarter ended December 31, 2024 (Q4 2023: $15.0 million). The increase of $30.1 million was driven primarily by an increase of $9.8 million in marketing and other commercial related activities, including travel, primarily related to the launch of Ohtuvayre, and an increase of $2.6 million in professional and consulting fees, information technology costs and other support costs due to the continued buildout of our commercial organization. Additionally, we had an increase of $8.8 million in people-related costs as we built out our commercial organization including much of the field sales team as well as an increase of $7.8 million related to share-based compensation. Net loss: Net loss was $33.8 million for the fourth quarter ended December 31, 2024 (Q4 2023: Net loss $14.1 million). Full Year 2024 Financial Results Product sales: Net sales were $42.3 million for the year ended December 31, 2024 (2023: $0 million) related to product sales of Ohtuvayre. Cost of sales: Cost of sales was $2.6 million for the year ended December 31, 2024 (2023: $0 million), which included Ohtuvayre manufacturing costs incurred after US approval, inventory overhead costs and sales-based royalties. R&D Expenses: R&D expenses were $44.6 million for the year ended December 31, 2024 (full year 2023: $17.2 million), an increase of $27.4 million. This increase was primarily due to an increase of $17.5 million in clinical trial and other development costs we incurred related to the two Phase 2 studies which were initiated in the third quarter of 2024 related to the combination of nebulized ensifentrine and glycopyrrolate and for Ohtuvayre in patients with bronchiectasis, the $6.3 million approval milestone, $3.1 million increase in share-based compensation, $2.0 million increase for people-related costs and $1.1 million related to pre-launch manufacturing costs for commercial supply. This was partially offset by a decrease of $1.3 million in consultant and professional fee costs which were higher in the prior year due to service costs associated with our New Drug Application and the related approval process. SG&A Expenses: SG&A expenses were $149.8 million for the year ended December 31, 2024 (full year 2023: $50.4 million), an increase of $99.4 million. This increase was driven primarily by an increase of $29.7 million in marketing and other commercial related activities, including travel, primarily related to the launch of Ohtuvayre, a charge of $15.0 million for the first sale milestone and an increase of $7.3 million in professional and consulting fees, information technology costs and other support costs due to the continued buildout of our commercial organization. Additionally, we had an increase of $26.8 million in people-related costs as we built out our commercial organization including much of the field sales team as well as an increase of $18.8 million related to share-based compensation. Net loss: Net loss was $173.4 million for the year ended December 31, 2024 (full year 2023: $54.4 million). Conference Call and Webcast Information Verona Pharma will host an investment community webcast and conference call at 9:00 a.m. EST / 2:00 p.m. GMT on Thursday, February 27, 2025, to discuss the fourth quarter and full year 2024 financial results and the corporate update. To participate, please dial one of the following numbers and ask to join the Verona Pharma call: +1-800-836-8184 for callers in the United States +1-646-357-8785 for international callers A live webcast will be available on the Events and Presentations link on the Investors page of the Company’s website, www.veronapharma.com, and the audio replay will be available for 90 days. An electronic copy of the fourth quarter and full year 2024 results press release will also be made available today on the Company’s website. Important Safety Information Indication Ohtuvayre is a prescription medicine used to treat COPD in adults. COPD is a chronic (long-term) lung disease that includes chronic bronchitis, emphysema, or both. What is the most important information I should know about Ohtuvayre? Ohtuvayre can cause serious side effects, including: Sudden breathing problems immediately after inhaling your medicine. If you have sudden breathing problems immediately after inhaling your medicine, stop using Ohtuvayre and call your healthcare provider right away or go to the nearest hospital emergency room right away. Mental health problems including suicidal thoughts and behavior. You may experience mood or behavior changes when taking Ohtuvayre. Call your healthcare provider right away if you have any of these symptoms, especially if they are new, worse, or worry you: thoughts of suicide or dying, attempt to commit suicide, trouble sleeping (insomnia), new or worse anxiety, new or worse depression, acting on dangerous impulses, and/or other unusual changes in your behavior or mood. Do not use Ohtuvayre to treat sudden breathing problems. Always have a rescue inhaler with you. Who Should Not use Ohtuvayre? Do not use Ohtuvayre if you have had an allergic reaction to ensifentrine or any of the ingredients in Ohtuvayre. What should I tell my healthcare provider before using Ohtuvayre? Before you use Ohtuvayre, tell your healthcare professional if you have or have had a history of mental health problems including depression and suicidal behavior; have liver problems; are pregnant or plan to become pregnant; are breastfeeding. It is not known if Ohtuvayre may harm your unborn baby. It is not known if the medicine in Ohtuvayre passes into your breast milk and if it can harm your baby. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. What are the most common side effects of Ohtuvayre? The most common side effects of Ohtuvayre include back pain, high blood pressure, bladder infection and diarrhea. These are not all the possible side effects of Ohtuvayre. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. This summary does not include all the information about Ohtuvayre and is not meant to take the place of a discussion with your healthcare provider about your treatment. For further information, please see the full Prescribing Information, including the Patient Information Leaflet. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. For further information please contact: About Verona Pharma Verona Pharma is a biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of chronic respiratory diseases with significant unmet medical needs. OhtuvayreTM (ensifentrine) is the Company’s first commercial product and the first inhaled therapy for the maintenance treatment of COPD that combines bronchodilator and non-steroidal anti-inflammatory activities in one molecule. Ensifentrine has potential applications in non-cystic fibrosis bronchiectasis, cystic fibrosis, asthma and other respiratory diseases. For more information, please visit www.veronapharma.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. All statements contained in this press release other than statements of historical fact should be considered forward-looking statements. Words such as “anticipate,” “believe,” “plan,” “expect,” “intend,” “may,” “potential,” “prepare,” “possible” and similar words and expressions are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements regarding the potential benefits and efficacy of our drug Ohtuvayre to treat adult patients in the US with COPD, statements regarding our two recently initiated Phase 2 clinical trials, the Company’s plans to initiate a Phase 2b clinical trial, potential regulatory approvals in the EU and UK, and Nuance Pharma’s results from its pivotal Phase 3 trial, and the timing of any of the foregoing. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from our expectations expressed or implied by the forward-looking statements, including, but not limited to, the following: risks related to our limited operating history; our need for additional funding to complete development and commercialization of Ohtuvayre which may not be available and which may force us to delay, reduce or eliminate our development or commercialization efforts; our reliance on the success of Ohtuvayre, our only commercial product; our reliance on third-party manufacturers and suppliers; the efficacy of Ohtuvayre compared to competing drugs; our ability to successfully commercialize Ohtuvayre; serious adverse, undesirable or unacceptable side effects associated with Ohtuvayre which could adversely affect our ability to commercialize Ohtuvayre; failure to develop Ohtuvayre for additional indications, alternate delivery methods, or as a combination therapy; failure to obtain approval for and commercialize Ohtuvayre in multiple major pharmaceutical markets; our commercial capabilities and infrastructure, including sales, marketing, operations, distribution, and reimbursement infrastructure, may not be adequate to successfully commercialize Ohtuvayre; lawsuits related to patents covering Ohtuvayre and the potential for our patents to be found invalid or unenforceable; lawsuits related to our licensing of patents and know-how from third parties for the commercialization of Ohtuvayre; changes in our tax rates, unavailability of certain tax credits or reliefs or exposure to additional tax liabilities or assessments that could affect our profitability, and audits by tax authorities that could result in additional tax payments for prior periods; the terms of our credit agreement and the revenue interest purchase and sale agreement (“RIPSA”) place restrictions on our operating and financial flexibility, and if we fail to comply with certain covenants in the RIPSA, our results of operations and financial condition may be harmed; our vulnerability to natural disasters, global economic factors, geo-political actions and unexpected events, including health epidemics or pandemics; and the other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the period ended December 31, 2024, filed with the Securities and Exchange Commission (“SEC”) on February 27, 2025, as such factors may be updated from time to time in our other filings with the SEC. We disclaim any obligation to update or revise any forward-looking statement contained in this press release, even if subsequent events cause our views to change, except as required under applicable law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release. Verona Pharma plc Consolidated Financial Summary (unaudited) (in thousands, except share and per share amounts)

In 2024, the range of diseases treated by globally approved new drugs was diverse, with cancer and rare diseases leading in the total number of new drug approvals. Several new drugs were also approved in the fields of anti-infectives and cardiovascular diseases. Among the approved small molecule drugs, 8 were designated as "first-in-class" therapies with breakthrough therapy status. These drugs not only possess unique mechanisms of action but also provide significant therapeutic improvements over previous treatments. As the development technologies for peptide and nucleic acid-based drugs have matured, their proportion among FDA-approved new drugs has stabilized. In 2024, the approved nucleic acid-based drugs included the first telomerase inhibitor, Rytelo (imetelstat). This drug targets and binds to the RNA template of telomerase, inhibiting its activity. It received FDA approval in June 2024 for the treatment of adult patients with myelodysplastic syndromes (MDS). A major challenge for peptide-based drugs is their susceptibility to degradation in the human body, making it difficult to maintain their efficacy. However, advancements in peptide modification technologies have led to longer half-lives for these drugs, offering patients long-acting therapies with reduced treatment burdens. Yorvipath, a long-acting parathyroid hormone prodrug approved in 2024, links the parathyroid hormone prodrug to a polyethylene glycol carrier to control the hormone's release in the body. It received FDA approval in August 2024 as the first treatment for adult hypoparathyroidism. In 2024, CBER approved 8 cell and gene therapies. These therapies not only represent technological innovations but also provide the first approved treatments for patients with various types of diseases. Amtagvi (lifileucel): On February 16, 2024, the U.S. FDA granted accelerated approval to Iovance Biotherapeutics' tumor-infiltrating lymphocyte (TIL) therapy, Amtagvi (lifileucel), for the treatment of advanced melanoma. Amtagvi is the first approved TIL therapy and the first T-cell therapy approved for solid tumors, marking another milestone in cell therapy. Lenmeldy (atidarsagene autotemcel): On March 18, 2024, the FDA approved Lenmeldy (atidarsagene autotemcel, arsa-cel), a gene therapy developed by Orchard Therapeutics, for the treatment of pediatric patients with metachromatic leukodystrophy (MLD) who meet specific criteria. This is the first FDA-approved gene therapy for this rare genetic disorder. Tecelra (afamitresgene autoleucel): On August 1, 2024, the FDA approved Adaptimmune Therapeutics' engineered T-cell therapy, Tecelra (afami-cel), for the treatment of adult patients with advanced MAGE-A4+ synovial sarcoma of certain HLA types who have previously received chemotherapy. Tecelra is the first engineered T-cell therapy approved for solid tumors and the first effective treatment for synovial sarcoma in over a decade. Kebilidi (eladocagene exuparvovec): On November 13, 2024, the FDA granted accelerated approval to Kebilidi (eladocagene exuparvovec), a gene therapy developed by PTC Therapeutics, for the treatment of pediatric and adult patients with aromatic L-amino acid decarboxylase deficiency (AADCD), regardless of disease severity. This is the first FDA-approved gene therapy administered via direct injection into the brain. Ryoncil (remestemcel): On December 18, 2024, the FDA approved Ryoncil (remestemcel), developed by Mesoblast, for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients aged 2 months and older. The FDA's press release highlighted that this is the first FDA-approved mesenchymal stromal cell (MSC) therapy. In addition, the FDA approved Moderna's respiratory syncytial virus (RSV) vaccine, Mresvia, marking the first approval of an mRNA vaccine for a disease other than COVID-19, demonstrating the potential of mRNA technology. Top 10 Potential Blockbuster Therapies of 20241. Rezdiffra (resmetirom): The First FDA-Approved Therapy for Metabolic Dysfunction-Associated Steatohepatitis (MASH) Rezdiffra (resmetirom), developed by Madrigal Pharmaceuticals, is a "first-in-class," once-daily, oral thyroid hormone receptor (THR)-β selective agonist designed to target the underlying causes of MASH. Thyroid hormone plays a central role in liver function by activating β receptors in hepatocytes, influencing a range of health parameters, from serum cholesterol and triglyceride levels to the pathological accumulation of fat in the liver. Rezdiffra received FDA approval in March 2024, becoming the first approved therapy for MASH and marking a significant milestone in this field. 2. Winrevair (sotatercept): A "First-in-Class" Therapy for Pulmonary Arterial Hypertension Winrevair is a "first-in-class" activin receptor type IIA (ActRIIA) fusion protein. It combines a modified extracellular domain of ActRIIA with the Fc region of an antibody. This therapy blocks the binding of activin to its cell membrane receptors, thereby reducing activin-mediated signaling. Merck acquired this innovative therapy through its approximately $11.5 billion acquisition of Acceleron Pharma in 2021. Winrevair received FDA approval in March 2024 for the treatment of pulmonary arterial hypertension. Merck believes it has the potential to become a cornerstone therapy for this condition. 3. Anktiva: A "First-in-Class" IL-15 Receptor Agonist for Non-Muscle Invasive Bladder Cancer (NMIBC) Anktiva, developed by ImmunityBio's Altor BioScience, consists of an IL-15 mutant (IL-15N72D) bound to an IL-15 receptor α/IgG1 Fc fusion protein. Compared to natural IL-15, Anktiva exhibits superior pharmacokinetic properties in patients, persists longer in lymphoid tissues, and demonstrates enhanced anti-tumor activity. In April 2024, Anktiva received FDA approval for use in combination with Bacillus Calmette-Guérin (BCG) to treat adult patients with BCG-unresponsive NMIBC accompanied by carcinoma in situ (CIS). In clinical trials, patients treated with Anktiva achieved a complete response rate of 62%. 4. mResvia: The First mRNA Respiratory Syncytial Virus (RSV) Vaccine Moderna's mRNA-based RSV vaccine, mResvia (mRNA-1345), received FDA approval in May 2024 for protecting adults aged 60 and older from lower respiratory tract disease (RSV-LRTD) caused by RSV infection. This is Moderna's second FDA-approved mRNA vaccine. It consists of an mRNA sequence encoding a stabilized prefusion F glycoprotein, delivered using the same lipid nanoparticle (LNP) technology as Moderna's COVID-19 vaccine. 5. Rytelo (imetelstat): The First Telomerase Inhibitor In June 2024, the U.S. FDA approved Geron's "first-in-class" telomerase inhibitor, Rytelo (imetelstat), for the treatment of adult patients with low- to intermediate-1-risk myelodysplastic syndromes (MDS). This approval marked the arrival of the first telomerase-targeted therapy, nearly 40 years after the discovery of telomerase in 1984. Telomeres are protective caps at the ends of chromosomes that naturally shorten with each cell division. In MDS, abnormal bone marrow cells often express telomerase, which rebuilds telomeres, leading to uncontrolled cell division. Preclinical and clinical data show that Rytelo inhibits telomerase activity, curbing the uncontrolled proliferation of cancerous stem and progenitor cells and inducing apoptosis in malignant cells, demonstrating disease-modifying activity. 6. Ohtuvayre (ensifentrine): The First Inhaled Therapy with a Novel Mechanism of Action for COPD Maintenance Treatment in Over 20 Years Ohtuvayre, developed by Verona Pharma, is a "first-in-class" dual phosphodiesterase 3/4 (PDE3/4) inhibitor. It received FDA approval in June 2024 as a maintenance treatment for adult patients with chronic obstructive pulmonary disease (COPD). Ohtuvayre is the first inhaled COPD therapy to combine bronchodilator and non-steroidal anti-inflammatory effects. Additionally, it represents the first inhaled therapy with a novel mechanism of action for COPD maintenance treatment in over two decades. Ohtuvayre works by inhibiting PDE3, leading to relaxation of airway smooth muscles and bronchodilation, while its inhibition of PDE4 reduces inflammation by decreasing the release of pro-inflammatory cytokines. 7. Kisunla (donanemab): The First Alzheimer’s Disease (AD) Therapy Allowing Treatment Discontinuation Upon Meeting Criteria Kisunla, developed by Eli Lilly, is an anti-amyloid monoclonal antibody that specifically binds to the N3pG subtype of amyloid. By targeting this subtype, Kisunla selectively binds to amyloid plaques in the brain, facilitating their clearance. In July 2024, it received FDA approval for the treatment of adult patients with early symptomatic AD, including those with mild cognitive impairment (MCI) or mild dementia stage AD, confirmed by the presence of amyloid pathology. According to the press release, Kisunla, administered monthly, is the first amyloid plaque-targeting therapy with evidence supporting treatment discontinuation after amyloid plaque clearance, potentially reducing treatment costs and the frequency of infusions. 8. Ebglyss (lebrikizumab): A New First-Line Biologic Therapy for Atopic Dermatitis Ebglyss, co-developed by Almirall and Eli Lilly, is an anti-IL-13 antibody. It received FDA approval in September 2024 for the treatment of moderate-to-severe atopic dermatitis (AD) in adults and adolescents (aged 12 years and older, weighing at least 40 kg) whose disease remains uncontrolled despite topical treatments. IL-13 is a key cytokine in AD, driving type 2 inflammation in the skin, which leads to impaired skin barrier function, itching, skin thickening, and infections. Ebglyss's targeted mechanism of action, along with its demonstrated efficacy and safety in both short- and long-term use, makes it a compelling option for patients with moderate-to-severe AD inadequately controlled by topical therapies. Its once-monthly maintenance dosing offers convenience for patients. 9. Cobenfy (xanomeline, trospium chloride): The First Novel Mechanism Drug for Schizophrenia in Decades Cobenfy received FDA approval in September 2024 for the treatment of adult patients with schizophrenia. The FDA press release highlighted that Cobenfy is the first antipsychotic drug targeting cholinergic receptors, diverging from the long-standing standard therapies that target dopamine receptors. This approval marks the first novel mechanism drug for schizophrenia in decades. Cobenfy combines two active ingredients, xanomeline and trospium chloride, designed to activate muscarinic acetylcholine receptors in the brain while minimizing peripheral effects. By stimulating M1 and M4 muscarinic receptors, xanomeline alleviates negative symptoms such as apathy and reduced social drive, improves cognitive function, and helps manage other psychiatric symptoms like hallucinations and delusions. Originally developed by Karuna Therapeutics, Cobenfy was acquired by Bristol Myers Squibb in December 2023 through a merger agreement. 10. Attruby (acoramidis): The First Approved Product with Labeling Indicating Near-Complete Stabilization of Transthyretin (TTR) In November 2024, the FDA approved Attruby (acoramidis), developed by BridgeBio Pharma, for the treatment of adults with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) to reduce cardiovascular mortality and cardiovascular-related hospitalizations. The press release noted that Attruby is the first approved product with labeling indicating near-complete stabilization of TTR. The drug is designed to mimic the protective function of the TTR T119M mutation, maintaining the normal tetrameric conformation of TTR and preventing the formation of toxic amyloid deposits. In a Phase 3 clinical trial, Attruby reduced all-cause mortality and recurrent cardiovascular-related hospitalizations by 42% compared to placebo at 30 months of treatment. How to obtain the latest research advancements in the field of biopharmaceuticals? In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!