Epileptic Syndromes

Danish pharma Lundbeck vastly increased its offer to buy Longboard Pharmaceuticals over six months as other parties came to the dealmaking table, a new SEC filing shows. Lundbeck initially proposed $29 per share $LBPH on April 19 to buy the Phase 3 neurology biotech. By Oct. 14, when the $2.6 billion cash deal was announced, it had raised its offer to $60, according to an SEC filing on Wednesday morning. At the time, Lundbeck CEO Charl van Zyl told Endpoints News the deal was within the “benchmark premium” for companies with assets in Phase 3. “We believe we have fundamentally not overpaid for this asset,” he said in the interview. Longboard said last month that its 5-HT2C superagonist known as bexicaserin had entered Phase 3 for Dravet syndrome and has broader plans to test the treatment in other epileptic encephalopathies like Lennox-Gastaut syndrome. Lundbeck had been intrigued by the Phase 1b/2a data that Longboard shared on Jan. 2 for patients who experience seizures associated with developmental and epileptic encephalopathies. That data skyrocketed Longboard’s stock price from $6.03 on Dec. 29 to $25.10 on Jan. 2. Shortly after the data release, Lundbeck representatives met with Longboard CEO Kevin Lind at the annual JP Morgan Healthcare Conference. Lundbeck would go on to increasingly raise its offer from $29.00 to $34.50, then $54.00 and $59.00. Lind wanted $62.00 a share, but they eventually settled on $60.00. During the process, Longboard had also talked with other undisclosed companies, codenamed Parties A, B, C and D, according to another SEC filing on Wednesday. Party A had submitted a few offers, including one as high as $60.00 on Sept. 27. But on Oct. 2, Party A told Longboard it was no longer interested because of “various perceived risks,” according to the SEC document.

– Pooled analysis from Phase 3 clinical trials highlights liver transplant rates in adult patients with HRS with rapid reduction in kidney function treated with TERLIVAZ1 – DUBLIN, Oct. 26, 2024 /PRNewswire/ -- Mallinckrodt plc, a global specialty pharmaceutical company, today announced a poster presentation on TERLIVAZ® (terlipressin) for injection in patients with hepatorenal syndrome (HRS) with rapid reduction in kidney function1 at Kidney Week 2024, the annual meeting of the American Society of Nephrology (ASN) taking place in San Diego from October 23-27, 2024. TERLIVAZ is the first and only FDA-approved product indicated to improve kidney function in adults with HRS with rapid reduction in kidney function,1 an acute and life-threatening condition requiring hospitalization.2 HRS involving rapid reduction in kidney function1 is estimated to affect more than 42,000 Americans annually, approximately 0.01% of the U.S. population,3 making it a very rare condition; and rates of hospitalizations are increasing.4 Please see Limitation of Use and Important Safety Information, including Boxed Warning, below. The poster presentation highlights a pooled analysis of data from the CONFIRM and REVERSE Phase 3 placebo-controlled trials of TERLIVAZ.5 The analysis examined the rates of hepatorenal syndrome-acute kidney injury (HRS-AKI) reversal, renal replacement therapy (RRT), and liver transplant (LT) among a subpopulation of patients listed for LT at baseline who met FDA label guidelines for treatment with TERLIVAZ (SCr <5 mg/dL, ACLF grade 0–2, and MELD score <35).5 In this patient subpopulation, treatment with TERLIVAZ increased the rate of HRS-AKI reversal and reduced the need for RRT at all time points assessed.5 The increase in HRS-AKI reversal with TERLIVAZ did not negatively impact the LT rate in patients listed for LT at baseline.5 The rate of HRS-AKI reversal was 43% in the TERLIVAZ group (n=53) and 20% in the placebo group (n=35).5 The rate of RRT in the TERLIVAZ and placebo groups was 28% and 46% by Day 30; 32% and 54% by Day 60; and 36% and 54% by Day 90, respectively.5 The LT rate was similar in the TERLIVAZ and placebo groups at all time points assessed: 53% and 51% by Day 30; 66% and 57% by Day 60; and 66% and 63% by Day 90, respectively.5 The limitations of this study include, but are not limited to, small sample sizes, variables in methodology, and possible errors and omissions within the data sets.5 "This analysis of data from our Phase 3 trials furthers our understanding of the appropriate patient subpopulations and the potential impact of treatment on liver transplantation rates," said Peter Richardson, MRCP (UK), Executive Vice President & Chief Scientific Officer. "For these critically ill patients with HRS-AKI, a liver transplant is the definitive treatment, and we know there are significant challenges associated with RRT; which makes continued research important." This study was sponsored by Mallinckrodt Pharmaceuticals. Presentation details can be found below: Poster #PO0102-2: Hepatorenal syndrome-acute kidney injury reversal and liver transplant rates in patients treated with terlipressin5 Presenter: Justin Belcher Session Type: Poster Presentation Session Title: AKI: Clinical, Outcomes, and Trials - Management Session Date and Time: Saturday, October 26, 2024; 10:00 a.m. – 12:00 p.m. PT INDICATION AND LIMITATION OF USE TERLIVAZ is indicated to improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function. Patients with a serum creatinine >5 mg/dL are unlikely to experience benefit. IMPORTANT SAFETY INFORMATION WARNING: SERIOUS OR FATAL RESPIRATORY FAILURE TERLIVAZ may cause serious or fatal respiratory failure. Patients with volume overload or with acute-on-chronic liver failure (ACLF) Grade 3 are at increased risk. Assess oxygenation saturation (e.g., SpO2) before initiating TERLIVAZ. Do not initiate TERLIVAZ in patients experiencing hypoxia (e.g., SpO2 <90%) until oxygenation levels improve. Monitor patients for hypoxia using continuous pulse oximetry during treatment and discontinue TERLIVAZ if SpO2 decreases below 90%. Contraindications TERLIVAZ is contraindicated: In patients experiencing hypoxia or worsening respiratory symptoms. In patients with ongoing coronary, peripheral, or mesenteric ischemia. Warnings and Precautions Serious or Fatal Respiratory Failure: Obtain baseline oxygen saturation and do not initiate TERLIVAZ in hypoxic patients. Monitor patients for changes in respiratory status using continuous pulse oximetry and regular clinical assessments. Discontinue TERLIVAZ in patients experiencing hypoxia or increased respiratory symptoms. Manage intravascular volume overload by reducing or discontinuing the administration of albumin and/or other fluids and through judicious use of diuretics. Temporarily interrupt, reduce, or discontinue TERLIVAZ treatment until patient volume status improves. Avoid use in patients with ACLF Grade 3 because they are at significant risk for respiratory failure. Ineligibility for Liver Transplant: TERLIVAZ-related adverse reactions (respiratory failure, ischemia) may make a patient ineligible for liver transplantation, if listed. For patients with high prioritization for liver transplantation (e.g., MELD ≥35), the benefits of TERLIVAZ may not outweigh its risks. Ischemic Events: TERLIVAZ may cause cardiac, cerebrovascular, peripheral, or mesenteric ischemia. Avoid use of TERLIVAZ in patients with a history of severe cardiovascular conditions or cerebrovascular or ischemic disease. Discontinue TERLIVAZ in patients who experience signs or symptoms suggestive of ischemic adverse reactions. Embryo-Fetal Toxicity: TERLIVAZ may cause fetal harm when administered to a pregnant woman. If TERLIVAZ is used during pregnancy, the patient should be informed of the potential risk to the fetus. Adverse Reactions The most common adverse reactions (≥10%) include abdominal pain, nausea, respiratory failure, diarrhea, and dyspnea. Please click here to see full Prescribing Information, including Boxed Warning. ABOUT HEPATORENAL SYNDROME (HRS) Hepatorenal syndrome (HRS) involving rapid reduction in kidney function1 is an acute and life-threatening condition that occurs in people with advanced liver disease.2 HRS is classified into two distinct types – a rapidly progressive type that leads to acute renal failure where patients are typically hospitalized for their care and a more chronic type that progresses over weeks to months.2 HRS involving rapid reduction in kidney function1 is estimated to affect more than 42,000 Americans annually, approximately 0.01% of the U.S. population,3 making it a very rare condition; and rates of HRS hospitalizations are increasing.4 If left untreated, HRS with rapid reduction in kidney function1 has a median survival time of less than two weeks and greater than 80 percent mortality within three months.6 ABOUT MALLINCKRODT Mallinckrodt is a global business consisting of multiple wholly owned subsidiaries that develop, manufacture, market and distribute specialty pharmaceutical products and therapies. The company's Specialty Brands reportable segment's areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, hepatology, nephrology, pulmonology, ophthalmology, and oncology; immunotherapy and neonatal respiratory critical care therapies; analgesics; and gastrointestinal products. Its Specialty Generics reportable segment includes specialty generic drugs and active pharmaceutical ingredients. To learn more about Mallinckrodt, visit . CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING STATEMENTS This release contains forward-looking statements, including with regard to TERLIVAZ®, its potential to improve health and treatment outcomes, and its potential impact on patients. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: the effects of Mallinckrodt's recent emergence from bankruptcy; satisfaction of, and compliance with, regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; issues with product quality, manufacturing or supply, or patient safety issues or adverse side effects or adverse reactions associated with TERLIVAZ; and other risks identified and described in more detail in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Mallinckrodt's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and other filings with the SEC, all of which are available on its website. The forward-looking statements made herein speak only as of the date hereof and Mallinckrodt does not assume any obligation to update or revise any forward-looking statement, whether as a result of new information, future events and developments or otherwise, except as required by law. CONTACT Media Inquiries Green Room Communications 908-577-4531 [email protected] Investor Relations Derek Belz Vice President, Investor Relations 314-654-3950 [email protected] Mallinckrodt, the "M" brand mark, TERLIVAZ, and the Mallinckrodt Pharmaceuticals logo are trademarks of a Mallinckrodt company. Other brands are trademarks of a Mallinckrodt company or their respective owners. ©2024 Mallinckrodt. US-2400773 10/24 References 1 TERLIVAZ® (terlipressin) for Injection. Prescribing Information. Mallinckrodt Hospital Products Inc. 2023. 2 National Organization for Rare Disorders. Hepatorenal Syndrome. Available at: . Accessed October 2024. 3 United States Census Bureau: Quick Facts. Available at: . Accessed October 2024. 4 Singh J., Dahiya D.S., Kichloo A., et al. Hepatorenal Syndrome: A Nationwide Trend Analysis from 2008 to 2018. Annals of Med. 2021;53:1. 2018-2024 doi.org/10/1080/07853890. 5 Belcher J.M., Regner K.R., Mujtaba M.A., et al. Hepatorenal syndrome-acute kidney injury reversal and liver transplant rates in patients treated with terlipressin. Abstract to be presented at the American Society of Nephrology (ASN) 2024 Annual meeting. October 2024. 6 Flamm, S.L., Brown, K., Wadei, H.M., et al. The Current Management of Hepatorenal Syndrome–Acute Kidney Injury in the United States and the Potential of Terlipressin. Liver Transpl. 2021;27:1191-1202. . SOURCE Mallinckrodt plc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Fundraising campaign will fuel research to change the course of tuberous sclerosis complex SILVER SPRING, Md., Oct. 26, 2024 /PRNewswire/ -- Last night during the organization's 50th Anniversary Gala in New York City, the TSC Alliance® announced the public phase of a major fundraising campaign called the 50 Forward Fund. The effort aims to raise $40 million for research into tuberous sclerosis complex (TSC), a rare disease affecting 50,000 in the United States and 1 million worldwide. To date, the campaign has raised more than $18.5 million. "The 50 Forward Fund bolsters the momentum we've built since our founding while ambitiously looking forward to a future where everyone affected by TSC can live their fullest lives," said Kari Luther Rosbeck, TSC Alliance President & CEO. "The TSC Alliance is committed to further catalyzing research to create meaningful outcomes for people living with TSC and their families, and major investments in TSC research through this campaign will help us achieve this vision." Since 1984, the organization has cumulatively funded more than $37 million in TSC research and leveraged $505 million in additional Federal funding. Research accomplishments to date include: Backing the first-ever preventative clinical trial for epilepsy in the United States. Creating a Biosample Repository with more than 2,650 samples linked to clinical data in the Natural History Database. Building a Preclinical Consortium to help move new treatment ideas toward clinical trials. Funding basic research that ultimately contributed to FDA approval of three drugs specifically for TSC. The 50 Forward Fund is the TSC Alliance's largest research initiative in its 50-year history. It was designed as part of the organization's Research Business Plan to guide research efforts for TSC individuals with the following goals: Focusing research to better understand TSC and identify new treatments; Altering the course of the disease and improving quality of life through prenatal diagnoses and preventative treatments; and Educating medical professionals to achieve more rapid diagnosis and better treatment aligned with evidence-based standards of care. "Funding from the 50 Forward campaign will help the TSC Alliance expand our research investment in four key areas," explained Steven L. Roberds, PhD, Chief Scientific Officer. "These include our Natural History Database and Biosample Repository, Clinical Research efforts, Preclinical Consortium and Innovative Research grants program." Natural History Database and Biosample Repository: TSC affects everyone differently. To understand which drugs will be most effective for each individual, researchers need biosamples, clinical data and patient-reported outcomes. These require efficient collection processes, standardized analysis protocols and robust data management. Clinical Research: Clinical studies require extensive planning and collaboration. The TSC Alliance aims to alleviate the obstacles that stand in the way of efficient, effective and safe clinical trials. Preclinical Consortium: This project fills a critical research gap for standardized, rigorous testing of potential new TSC therapies prior to clinical trials. The innovative platform provides industry and academic members access to reproducible preclinical models and outcome measures, providing an unbiased platform for this critical stage in drug development. The Preclinical Consortium removes risks associated with developing new drugs and will encourage more companies to focus on treatments for the TSC community. Innovative Research: This effort's goal is to award more research grants annually, forge stronger connections between the top researchers around the world and bring stakeholders together at Innovation Workshops. "To achieve these ambitious research goals, we're counting on continued investment, collaboration and unwavering commitment of our community," Rosbeck continued. "The 50 Forward Fund will lead to research outcomes that will truly make a meaningful, positive impact on the lives of all those touched by TSC." To learn more about the 50 Forward Fund, view the campaign video and donate online, visit tscalliance.org/50-forward-fund today. About tuberous sclerosis complex Tuberous sclerosis complex (TSC) is a rare genetic disease that TSC causes tumors to grow in different organs and can impair their function, primarily the brain, heart, kidneys, skin and lungs. The disease is the leading genetic cause of epilepsy, including infantile spasms, and can also cause cognitive, behavioral and psychiatric disabilities; aggressive behaviors; and more. A strong correlation also exists between TSC and autism—an estimated 40-50% of individuals with TSC have autism spectrum disorder. TSC is a linchpin disease, meaning its genetic pathway also plays a role in other diseases and disorders. Advancements in TSC research may lead to a better understanding of autism, epilepsy, traumatic brain injury, diabetes and cancer—diseases that affect more than 65 million people in the United States alone. About the TSC Alliance The TSC Alliance improves quality of life for everyone affected by tuberous sclerosis complex by catalyzing new treatments, driving research toward a cure and expanding access to lifelong support. For more information, visit tscalliance.org. Contact Jaye Isham Chief Outreach Officer TSC Alliance (301) 562-9890 [email protected] SOURCE TSC Alliance WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED