Synovial Sarcoma

iStock, hurca Adaptimmune is rolling out its T cell therapy Tecelra for synovial sarcoma, recording $1.2 million in sales since its approval in August 2024. Nevertheless, it is pausing development of two oncology assets to save money. Despite the “increasing” momentum of its engineered T cell therapy Tecelra, Adaptimmune on Thursday expressed concern about its ability to continue as a business. “There is substantial doubt about our ability to continue,” the biotech wrote in a news release, adding that it will continue to “implement cost reduction measures.” In particular, Adaptimmune revealed on a Thursday investor call that it would pause work on two early-stage assets : the PRAME-targeting ADP-600 being developed for solid tumors and the anti-CD70 molecule ADP-520 for hematological malignancies. Both programs were undergoing IND-enabling studies. The company’s cash crunch comes as it works on launching Tecelra, the first FDA-approved engineered T cell therapy for solid tumors. The therapy won the FDA’s blessing in August 2024, allowing its use in patients with unresectable or metastatic synovial sarcoma. Adaptimmune has so far prepared 10 patients for treatment across 20 activated authorized treatment centers, according to its Thursday release. Adaptimmune is also currently exploring “all strategic options” for its business and all of its programs, for which it has enlisted TD Cowen’s help, CEO Adrian Rawcliffe said on the investor call. “This includes potential partnerships or collaborations, strategic combinations, various financial transactions and multiple pipeline monetization opportunities,” he added. Adaptimmune had just under $147 million in cash, cash equivalents and marketable securities at the end of 2024, according to its Q4 and full-year business update . The biotech expects to hit profitability in 2027, Rawcliffe said on the call. With two patients invoiced and no reimbursement denials to date, Tecelra has so far made $1.2 million for Adaptimmune. The company expects to have its full network of 30 treatment centers online by the end of 2025. “We feel very confident that the consensus analyst forecast of approximately $25 million of sales in 2025 is very achievable,” Rawcliffe said on the call. Meanwhile, Adaptimmune is also building toward its next potential launch, the TCR T cell therapy lete-cel, which in November 2024 cleared the mid-stage IGNYTE-ESO trial. In patients with advanced or metastatic synovial sarcoma or myxoid/round cell liposarcoma, lete-cel elicited a 42% response rate that lasted for a median of 12.2 months in the overall study population. Adaptimmune is working on filing a Biologics License Application for lete-cel later this year and expects the therapy to hit the market in 2027, Rawcliffe said on Thursday, forecasting peak sales of around $400 million. “We estimate that lete-cel will eventually make up over 60% of our combined sarcoma franchise revenue,” he added.

FY 2024 revenue of $228.4 million, in-line with company guidance Commercial Cell & Gene Therapy revenue rose to $26 million in FY 2024, up 20% year-over-year Supporting a record total of 701 global clinical trials as of December 31, 2024 NASHVILLE, Tenn., March 4, 2025 /PRNewswire/ -- Cryoport, Inc. (NASDAQ: CYRX) (Cryoport), a global leader in supply chain solutions for the life sciences industry, today announced financial results for the fourth quarter (Q4) and year ended (FY) December 31, 2024. Jerrell Shelton, CEO of Cryoport, commented, "Cryoport ended 2024 with solid results across the company including total full year revenue of $228.4 million, which was in-line with our expectations. We continued to see considerable revenue growth from our support of commercial Cell & Gene therapies where revenue rose 37% for the fourth quarter and 20% for the full year compared to the prior year periods. "Our Life Sciences Services business continued its expansion, partially attributed to the double-digit year-over-year growth in BioStorage/BioServices revenue for both the fourth quarter and full year periods. In the fourth quarter our Life Sciences Products business began to show signs of market demand stability and continued to provide positive free cash flow. "As previously reported, during 2024, we implemented cost reduction and capital realignment strategies, making significant progress in improving our cost structure. Notably, our gross margin improved to 45.8% in Q4 2024, up from 40.6% in the same period last year. We remain confident that our actions will lead us to a return to positive adjusted EBITDA during 2025 as we further implement our pathway to profitability. "We believe that as we enter 2025, we are prepared to capitalize on the anticipated growth in the Cell & Gene Therapy market. We intend to grow our leading market position and open additional revenue streams that have been under development through new services and product introductions. We will supplement this through potential strategic collaborations and partnerships. We are excited about our prospects for this year, and we believe we have all the necessary tools in place to execute on our growth plans and to reach our long-term objective of sustainable profitability," concluded Mr. Shelton. In tabular form, Q4 2024 and FY 2024 revenue compared to Q4 2023 and FY 2023, respectively, was as follows: BioStorage/BioServices revenue continues to grow double digits year-over-year, increasing 11% in FY 2024 as we continue to introduce our expanded capabilities to existing customers, as well as add new customers into our global network, and as more allogeneic clinical and commercial therapies progress in the number of patients treated. Revenue from the support of commercially approved Cell & Gene therapies grew to $25.9 million, up 20% year-over-year, for FY 2024 and increased to $7.9 million, up 37% year-over-year, for Q4 2024. During FY 2024, five (5) new therapies were approved including Mesoblast's Ryoncil® for the treatment of graft versus host disease, Adaptimmune's Tecelra® for the treatment of adults with unresectable or metastatic synovial sarcoma, ImmunityBio's Anktiva® for BCG-unresponsive non-muscle invasive bladder cancer, Iovance Biotherapeutics' Amtagvi™ therapy for advanced melanoma, and Immuneel's Qartemi® for the treatment of non-Hodgkin Lymphoma. Qartemi® is the first cell therapy developed and approved in India and is supported by CRYOPDP's logistics network of 14 facilities inside the country. Our total commercial therapy count was nineteen (19) as of December 31, 2024. As of December 31, 2024, Cryoport supported a total of 701 global clinical trials, a net increase of 26 clinical trials over December 31, 2023, with 81 trials in Phase 3. The number of trials by phase and region are as follows: A total of eleven (11) Cryoport supported Biologic License Applications (BLA)/Marketing Authorization Applications (MAA) were filed in 2024, of which three (3) were filed during the fourth quarter. Following the end of the year, three (3) filings occurred in January 2025. For 2025, we anticipate up to an additional twenty-three (23) application filings, five (5) new therapy approvals and an additional five (5) approvals for label/geographic expansions or moves to earlier lines of treatment. Financial Highlights Revenue Total revenue for Q4 2024 was $59.5 million compared to $57.3 million for Q4 2023, a year-over-year increase of 4.0% or $2.3 million. Life Sciences Services revenue for Q4 2024 was $39.6 million compared to $37.0 million for Q4 2023, up 6.8% year-over-year, including BioStorage/BioServices revenue of $4.0 million, up 10.4% year-over-year. Life Sciences Products revenue for Q4 2024 was $20.0 million compared to $20.2 million for Q4 2023, down 1.3% year-over-year. Total revenue for FY 2024 was $228.4 million, compared to $233.3 million for FY 2023, a year-over-year decrease of 2.1%. Life Sciences Services revenue for FY 2024 was $153.7 million compared to $144.1 million for FY 2023, up 6.6% year-over-year, including BioStorage/BioServices revenue of $15.0 million, up 10.6% year-over-year. Life Sciences Products revenue for FY 2024 was $74.7 million compared to $89.2 million for FY 2023, down 16.2%. Gross Margin Total gross margin was 45.8% for Q4 2024 compared to 40.6% for Q4 2023. Gross margin for Life Sciences Services was 46.2% for Q4 2024 compared to 40.8% for Q4 2023. Gross margin for Life Sciences Products was 45.1% for Q4 2024 compared to 40.4% for Q4 2023. Total gross margin was 43.6% for FY 2024 compared to 42.6% for FY 2023. Gross margin for Life Sciences Services was 44.5% for FY 2024 compared to 43.2% for FY 2023. Gross margin for Life Sciences Products was 41.7% for FY 2024 compared to 41.6% for FY 2023. Operating Costs and Expenses Operating costs and expenses decreased to $41.2 million for Q4 2024 compared to operating costs and expenses of $93.1 million for Q4 2023, which includes a non-cash impairment charge to goodwill of $49.6 million related to the MVE Biological Solutions business unit. Operating costs and expenses increased to $230.5 million for FY 2024 (which includes a non-cash impairment charge of $63.8 million), compared to $214.5 million for FY 2023 (which includes a non-cash impairment charge of $49.6 million). Net Loss Net loss for Q4 2024 and FY 2024 was $18.7 million and $114.8 million, respectively, compared to a net loss of $62.4 million and $99.6 million for the same periods in 2023, respectively. Net loss attributable to common stockholders was $20.7 million, or $0.42 per share, and $122.8 million, or $2.49 per share, for Q4 2024 and FY 2024, respectively. This compares to a net loss attributable to common stockholders of $64.4 million, or $1.31 per share, and $107.6 million, or $2.21 per share, for Q4 2023 and FY 2023, respectively. Adjusted EBITDA Adjusted EBITDA was a negative $1.3 million for Q4 2024, compared to a negative $6.6 million for Q4 2023. Adjusted EBITDA for FY 2024 was a negative $15.1 million, compared to a negative $8.3 million for FY 2023. Cash, Cash equivalents, and Short-Term Investments Cryoport held $261.7 million in cash, cash equivalents, and short-term investments as of December 31, 2024. Convertible Debt repurchases During FY 2024, the Company repurchased $185.0 million in aggregate principal amount of its Convertible Senior Notes due in 2026 for an aggregate repurchase price of $163.2 million. The Company has approximately $73.9 million in total of repurchase authorization available under its repurchase programs as of December 31, 2024. Note: All reconciliations of GAAP to adjusted (non-GAAP) figures above are detailed in the reconciliation tables included later in the press release. Outlook The Company is providing full year 2025 revenue guidance in the range of $240 - $250 million. The Company's 2025 guidance is dependent on its current business and expectations, which may be further impacted by, among other things, factors that are outside of our control, such as national economic factors, the global macroeconomic and geopolitical environment, supply chain constraints, inflationary pressures, and/or the effects of foreign currency fluctuations, as well as the other factors described in the Company's filings with the Securities and Exchange Commission ("SEC"), including in the "Risk Factors" section of its most recently filed periodic reports on Form 10-K and Form 10-Q, as well as in its subsequent filings with the SEC. Additional Information Further information on Cryoport's financial results is included in the attached condensed consolidated balance sheets and statements of operations, and additional explanations of Cryoport's financial performance are provided in the Company's Annual Report on Form 10-K for the year ended December 31, 2024, which is expected to be filed with the SEC on March 7, 2025. Additionally, the full report will be available in the SEC Filings section of the Investor Relations section of Cryoport's website at . Earnings Conference Call Information IMPORTANT INFORMATION: In addition to the earnings release, a document titled "Cryoport Fourth Quarter and Full Year 2024 in Review", providing a review of Cryoport's financial and operational performance and a general business update, will be issued at 4:05 p.m. ET on Tuesday, March 4, 2025. The document is designed to be read in advance of the questions and answers conference call and will be accessible at . Cryoport management will host a conference call at 5:00 p.m. ET on March 4, 2025. The conference call will be in the format of a questions and answers session and will address any queries investors have regarding the Company's reported results. A slide deck will accompany the call. Conference Call Information Please allow 10 minutes prior to the call to visit this site to download and install any necessary audio software. The questions and answers call will be recorded and available approximately three hours after completion of the live event in the Investor Relations section of the Company's website at for a limited time. To access the replay of the questions and answers click here. A dial-in replay of the call will also be available to those interested, until March 11, 2025. To access the replay, dial 1-844-512-2921 (United States) or 1-412-317-6671 (International) and enter replay entry code: 1116296#. About Cryoport, Inc. Cryoport, Inc. (Nasdaq: CYRX), is a global leader in supply chain solutions for the Life Sciences with an emphasis on cell & gene therapies. Cryoport enables manufacturers, contract manufacturers (CDMOs), contract research organizations (CROs), developers, and researchers to carry out their respective business with products and services that are designed to derisk services and provide certainty. We provide a broad array of supply chain solutions for the life sciences industry. Through our platform of critical products and solutions including advanced temperature-controlled packaging, informatics, specialized bio-logistics services, bio-storage, bio-services, and cryogenic systems, we are "Enabling the Future of Medicine™" worldwide, through our innovative systems, compliant procedures, and agile approach to superior supply chain management. Our corporate headquarters, located in Nashville, Tennessee, is complemented by over 50 global locations in 17 countries, with key sites in the United States, United Kingdom, France, the Netherlands, Belgium, Portugal, Germany, Japan, Australia, India, and China. For more information, visit or follow via LinkedIn at or @cryoport on X, formerly known as Twitter at for live updates. Forward-Looking Statements Statements in this press release which are not purely historical, including statements regarding Cryoport's intentions, hopes, beliefs, expectations, representations, projections, plans, or predictions of the future, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, those related to Cryoport's industry, business, long-term growth prospects, plans, strategies, acquisitions, future financial results and financial condition, such as Cryoport's outlook and guidance for full year 2025 revenue and the related assumptions and factors expected to drive revenue, projected growth trends in the markets in which Cryoport operates, and Cryoport's plans and expectations regarding the launch of new products and services, such as the expected timing and benefits of such products and services launches. It is important to note that Cryoport's actual results could differ materially from those in any such forward-looking statements. Factors that could cause actual results to differ materially include, but are not limited to, risks and uncertainties associated with the effect of changing economic and geopolitical conditions, supply chain constraints, inflationary pressures, the effects of foreign currency fluctuations, trends in the products markets, variations in Cryoport's cash flow, market acceptance risks, and technical development risks. Cryoport's business could be affected by other factors discussed in Cryoport's SEC reports, including in the "Risk Factors" section of its most recently filed periodic reports on Form 10-K and Form 10-Q, as well as in its subsequent filings with the SEC. The forward-looking statements contained in this press release speak only as of the date hereof and Cryoport cautions investors not to place undue reliance on these forward-looking statements. Except as required by law, Cryoport disclaims any obligation, and does not undertake to update or revise any forward-looking statements in this press release. Note Regarding Use of Non-GAAP Financial Measures To supplement our financial statements, which are presented on the basis of U.S. generally accepted accounting principles (GAAP), the following non-GAAP measures of financial performance as defined in Regulation G of the Securities Exchange Act of 1934 are included in this release: revenue at constant currency, revenue growth rate at constant currency, and adjusted EBITDA. Non-GAAP financial measures are not calculated in accordance with GAAP, are not based on any comprehensive set of accounting rules or principles and may be different from non-GAAP financial measures presented by other companies. Non-GAAP financial measures, including revenue at constant currency, revenue growth rate at constant currency and adjusted EBITDA, should not be considered as a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. We believe that revenue growth is a key indicator of how Cryoport is progressing from period to period, and we believe that the non-GAAP financial measures, revenue at constant currency and revenue growth rate at constant currency, are useful to investors in analyzing the underlying trends in revenue. Under GAAP, revenue received in local (non-U.S. dollar) currency is translated into U.S. dollars at the average exchange rate for the period presented. As a result, fluctuations in foreign currency exchange rates affect the results of our operations and the value of our foreign assets and liabilities, which in turn may adversely affect results of operations and cash flows and the comparability of period-to-period results of operations. When we use the term "constant currency," it means that we have translated local currency revenue for the current reporting period into U.S. dollars using the same average foreign currency exchange rates for the conversion of revenue into U.S. dollars that we used to translate local currency revenue for the comparable reporting period of the prior year. Revenue growth rate at constant currency refers to the measure of comparing the current reporting period revenue at constant currency with the reported GAAP revenue for the comparable reporting period of the prior year. However, we also believe that data on constant currency period-over-period changes have limitations, particularly as the currency effects that are eliminated could constitute a significant element of our revenue and could significantly impact our performance. We therefore limit our use of constant currency period-over-period changes to a measure for the impact of currency fluctuations on the translation of local currency revenue into U.S. dollars. We do not evaluate our results and performance without considering both period-over-period changes in non-GAAP constant currency revenue on the one hand and changes in revenue prepared in accordance with GAAP on the other. We caution the readers of this press release to follow a similar approach by considering revenue on constant currency period-over-period changes only in addition to, and not as a substitute for, or superior to, changes in revenue prepared in accordance with GAAP. Adjusted EBITDA is defined as net loss adjusted for net interest expense, income taxes, depreciation and amortization expense, stock-based compensation expense, acquisition and integration costs, cost reduction initiatives, investment income, unrealized (gain)/loss on investments, foreign currency (gain)/loss, gain on insurance claim, net gain on extinguishment of debt, impairment loss, changes in fair value of contingent consideration and charges or gains resulting from non-recurring events, as applicable. Management believes that adjusted EBITDA provides a useful measure of Cryoport's operating results, a meaningful comparison with historical results and with the results of other companies, and insight into Cryoport's ongoing operating performance. Further, management and the Company's board of directors utilize adjusted EBITDA to gain a better understanding of Cryoport's comparative operating performance from period to period and as a basis for planning and forecasting future periods. Adjusted EBITDA is also a significant performance measure used by Cryoport in connection with its incentive compensation programs. Management believes adjusted EBITDA, when read in conjunction with Cryoport's GAAP financials, is useful to investors because it provides a basis for meaningful period-to-period comparisons of Cryoport's ongoing operating results, including results of operations, against investor and analyst financial models, helps identify trends in Cryoport's underlying business and in performing related trend analyses, and it provides a better understanding of how management plans and measures Cryoport's underlying business. SOURCE Cryoport, Inc. 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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!