FDA expansion for Symdeko in cystic fibrosis

pharmatimes | June 24, 2019

The US Food and Drug Administration (FDA) has extended the indication for Vertex Pharmaceuticals Symdeko (tezacaftor/ivacaftor) for the treatment of pediatric patients ages six years and older with cystic fibrosis who have certain genetic mutations. The approval comes after the FDA last year approved Symdeko to treat patients ages 12 and older who had the same specific genetic mutations. The initial FDA approval was based on three phase III, double blind, placebo-controlled trials, which demonstrated improvements in lung function and other key measures of the disease, including a reduction in exacerbations, and the extension data was extrapolated from patients age 12 years and older, with additional support from data in patients age six to 12 years. “Decades ago, patients with cystic fibrosis were generally expected to live until 10 years of age, with few surviving into their teenage years. Since then, wide-ranging research on the disease resulted in more treatments for this debilitating disease that have extended life expectancy and improved quality of life for patients, but there is still no cure,” said Banu Karimi-Shah, acting deputy director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research. “Based on their individual genetic makeup, individuals may respond differently to certain drugs, so it is important to provide a variety of options. Today’s approval of Symdeko for children as young as six years old provides an important treatment option for younger patients, and also provides more context on the safety and dosing specific to this population. The FDA remains committed to advancing novel treatment options for areas of unmet patient need, particularly in diseases impacting children.”

Spotlight

The Introduction to the Principles and Practice of Clinical Research (IPPCR) is a course to train participants on how to effectively conduct clinical research. The course focuses on the spectrum of clinical research and the research process by highlighting epidemiologic methods, study design, protocol preparation, patient monitoring, quality assurance, and Food and Drug Administration (FDA) issues…

Spotlight

The Introduction to the Principles and Practice of Clinical Research (IPPCR) is a course to train participants on how to effectively conduct clinical research. The course focuses on the spectrum of clinical research and the research process by highlighting epidemiologic methods, study design, protocol preparation, patient monitoring, quality assurance, and Food and Drug Administration (FDA) issues…

Related News

PHARMA TECH

Silverback Therapeutics and ARS Pharmaceuticals Announce Merger

Silverback Therapeutics and ARS Pharmaceuticals | July 22, 2022

Silverback Therapeutics, Inc. and ARS Pharmaceuticals, Inc. announced that the companies have entered into a definitive agreement under which ARS will merge with Silverback in an all-stock transaction. The combined company will focus on the potential regulatory approval and commercialization of neffy, ARS’s investigational epinephrine nasal spray for the treatment of Type I allergic reactions including anaphylaxis. The combined company is expected to have approximately $265M in cash, cash equivalents and marketable securities at closing. Upon stockholder approval, the combined company is expected to operate under the name ARS Pharmaceuticals and trade on the Nasdaq Capital Market under the ticker symbol “SPRY.” The merger is currently expected to close in the fourth quarter of 2022. Type I severe allergic reactions are serious and potentially life-threatening events that can occur within minutes of exposure to an allergen and require immediate treatment with epinephrine, the only FDA-approved medication for these reactions. While epinephrine autoinjectors have been shown to be highly effective, there are well published limitations that result in many patients and caregivers delaying or not administering treatment in an emergency situation. These limitations include fear of the needle, lack of portability, needle-related safety concerns, lack of reliability, and complexity of the devices. There are approximately 25 million people in the United States who experience Type I severe allergic reactions. Of those, only 3.3 million currently have an active epinephrine autoinjector prescription, and of those, only half consistently carry their prescribed autoinjector. Even if patients or caregivers carry an autoinjector, more than half either delay or do not administer the device when needed in an emergency. ARS designed neffy to provide injection-like absorption of epinephrine, in a small, easy-to-carry, easy-to-use, rapidly administered, and reliable nasal spray device. With its needle-free administration, neffy may help eliminate the anxiety and hesitation associated with using an autoinjector. “We are extremely pleased to announce this proposed merger with Silverback, which we believe enables ARS to maximize the paradigm-changing opportunity of neffy. neffy is on the cusp of achieving what has not been possible before – the ability to deliver epinephrine with comparable pharmacokinetics to an intramuscular injection, but with a simple to administer nasal spray. We have completed a comprehensive registration program with neffy and based on a favorable pre-NDA meeting with the U.S. Food and Drug Administration (“FDA”), we are preparing to submit our New Drug Application (“NDA”) in the third quarter of 2022. This merger positions ARS and our experienced team to execute on the potential launch of neffy in 2023 by providing the requisite capital needed for launch. ARS was founded with a mission of solving many of the issues that patients and caregivers express about their epinephrine autoinjectors. Today is an important step toward bringing this novel treatment to patients and caregivers to improve their treatment options for these serious and potentially life-threatening allergic reactions.” Richard Lowenthal, M.Sc., MSEL, co-founder and chief executive officer of ARS Data across three registration studies supports that neffy should meet all clinical endpoints recommended by regulators and that its pharmacokinetics are within the range of approved efficacious epinephrine injection products. In addition, neffy has been well-tolerated to date with more than 500 individuals having received at least one dose, and many with repeat administration. The majority of adverse events in clinical trials were mild in nature and comparable to injection products. Based on the totality of data, ARS is preparing to submit its NDA for neffy for use in adults and pediatric patients who are 30 kg or greater in the third quarter of 2022. If approved, ARS is planning to launch neffy in the United States in 2023. “This transaction represents the result of a thorough and thoughtful strategic review process by Silverback,” said Laura Shawver, Ph.D., chief executive officer of Silverback. “ARS is an exciting late-stage company with compelling clinical data demonstrated with neffy, a path to near-term commercialization in a large and dissatisfied market, and an expert team with proven experience in launching and commercializing market-leading nasal spray products, such as NARCAN. I believe we have found the optimal partner to provide value for our stockholders, and even more so, the potential to transform treatment for millions of people with or at-risk for Type I severe allergic reactions.” About the Proposed Merger Under the terms of the merger agreement, assuming that Silverback’s net cash at closing is $240 million, Silverback equity holders are expected to own approximately 37% of the combined company and pre-merger ARS equity holders are expected to own approximately 63% of the combined company on a fully-diluted basis on a treasury stock method. The percentage of the combined company that Silverback’s equity holders will own as of the close of the transaction is subject to certain adjustments as described in the merger agreement, including the amount of Silverback’s net cash at closing. Upon closing of the transaction, Silverback will be renamed ARS Pharmaceuticals, Inc. and will be headquartered in San Diego, California. Richard Lowenthal, M.S., MBA, will serve as chief executive officer and president of the combined company. The merger agreement provides that the Board of Directors of the combined company will be comprised of ten members, including seven from ARS and three from Silverback. The merger agreement has been approved by the Board of Directors of each company, and the transaction is expected to close in the fourth quarter of 2022, subject to approvals by the stockholders of each company and other customary closing conditions. About Silverback Therapeutics, Inc. Silverback Therapeutics, Inc. is a biopharmaceutical company focused on leveraging its proprietary ImmunoTAC technology platform to develop systemically delivered and tissue targeted therapeutics for the treatment of chronic viral infections, cancer, and other serious diseases. Silverback’s platform enables the strategic pairing of proprietary payloads that modulate key disease modifying pathways with monoclonal antibodies directed at specific disease sites. Silverback Therapeutics is located in Seattle, Washington.

Read More

BUSINESS INSIGHTS

Novo Nordisk to acquire Forma Therapeutics and expand presence in sickle cell disease and rare blood disorders

Novo Nordisk | September 02, 2022

Novo Nordisk and Forma Therapeutics, Holdings Inc. announced that they have entered into a definitive agreement under which Novo Nordisk will acquire Forma Therapeutics for $20 per share in cash, which represents a total equity value of $1.1 billion. Forma Therapeutics is a clinical-stage biopharmaceutical company focused on transforming the lives of patients with sickle cell disease (SCD) and rare blood disorders. The acquisition of Forma Therapeutics, including its lead development candidate, etavopivat, is aligned with Novo Nordisk’s strategy to complement and accelerate its scientific presence and pipeline in hemoglobinopathies, a group of disorders in which there is abnormal production or structure of the hemoglobin protein in the red blood cells. “Novo Nordisk has worked for more than 40 years to develop and deliver transformative medicines to patients around the world with rare and devastating diseases. By adding Forma’s differentiated approach to address unmet needs for patients, we are taking a step forward in enhancing our sickle cell disease pipeline,” said Ludovic Helfgott, executive vice president and head of Rare Disease at Novo Nordisk. “We have an ambition to build a leading portfolio with standalone and combination treatments to tackle the complications and underlying causes of sickle cell disease.” Etavopivat, an investigational oral, once-daily, selective pyruvate kinase-R (PKR) activator, is being developed to improve anemia and red blood cell health in people with SCD, a seriously debilitating, life-threatening and life shortening disease. Etavopivat is currently being evaluated in a global phase 2/3 clinical trial (Hibiscus) in patients with SCD, and in a phase 2 trial (Gladiolus) in patients with transfusion-dependent SCD and another inherited hemoglobinopathy called thalassemia. “Today’s announcement is an exciting milestone that accelerates Forma’s purpose to transform the lives of patients with sickle cell disease and other serious hematological diseases,” said Frank D. Lee, president and chief executive officer of Forma. “Novo Nordisk will partner closely with the sickle cell community to amplify our impact for patients around the world who urgently need new treatment options. We look forward to working together with Novo Nordisk to serve as a trusted partner to our communities and to advance innovation, access and health equity for patients.” The transaction will not impact Novo Nordisk’s previously communicated operating profit outlook for 2022 or the ongoing share buy-back program. Novo Nordisk will fund the acquisition from financial reserves. About the transaction Under the terms of the agreement, Novo Nordisk will initiate a tender offer to acquire all outstanding shares of Forma Therapeutics’ common stock at a price of $20 per share in cash (or aggregated value of $1.1 billion) and a premium of 92% to Forma Therapeutics’ volume-weighted average price per share over the past 30 days ended August 31, 2022. The transaction has been unanimously approved by the Forma Therapeutics Board of Directors. The closing of the tender offer will be subject to certain conditions, including the tender of shares representing a majority of Forma Therapeutics’ outstanding shares, receipt of applicable regulatory approvals and other customary conditions. Upon the successful completion of the tender offer, Novo Nordisk’s acquisition subsidiary will merge into Forma Therapeutics, and any shares of common stock of Forma Therapeutics not tendered into the offer will receive the same USD per share price payable in the tender offer. The transaction is expected to close in the fourth quarter of 2022. In addition, certain affiliates of RA Capital Management, L.P., which collectively own approximately 19% of Forma Therapeutics’ outstanding shares, have entered into a support agreement pursuant to which they committed to tender their shares in the tender offer. Novo Nordisk is represented by Moelis & Company UK LLP as financial advisor and Davis Polk & Wardwell LLP as legal advisor. Forma Therapeutics is represented by Centerview Partners LLC as financial advisor and Goodwin Procter LLP as legal advisor. About sickle cell disease Sickle cell disease (SCD) is a chronic and progressive inherited disorder associated with a decrease in the health and lifespan of red blood cells. People living with SCD have red blood cells that are crescent shaped, rendering them inflexible, fragile, and unable to effectively deliver oxygen. The health of these sickle red blood cells is impaired and characterized by reduced cellular energy, poor deformability, decreased membrane repair, and increased adhesion. Around 17 million people worldwide live with SCD, including approximately 100,000 people in the United States, as well as approximately 30,000 in France, Germany, Italy, Spain, and the United Kingdom. SCD can cause serious health problems, including anemia, fatigue, episodes of pain known as vaso-occlusive crises (VOCs), and chronic, progressive end-organ damage. Despite recent advances in treatment, most patients with SCD still suffer from pain crises, lifelong disability, reduced quality of life, and shortened life expectancy. About etavopivat Etavopivat is an investigational, once-daily, selective pyruvate kinase-R (PKR) activator designed to be a disease-modifying therapy with the potential to improve red blood cell health and transform the lives of people living with SCD. Employing a multimodal approach, etavopivat works by activating the red blood cell’s natural PKR activity to decrease levels of the metabolite 2,3-DPG, allowing sickle hemoglobin to hold on to oxygen longer, resulting in decreased polymerization, hemolysis, and sickling. Etavopivat-mediated PKR activation also increases adenosine triphosphate (ATP) levels, to improve red blood cell function, which can lead to improved deformability, capacity for membrane repair, red blood cell health, and lifespan. Together, these effects are anticipated to improve the health of sickle red blood cell and lead to a reduction in anemia, hemolysis, vaso-occlusive crises, and end organ damage. In a phase 1 trial, etavopivat improved anemia and red blood cell health and appeared to have a safe and well-tolerated profile, demonstrating a potential to improve the lives of patients with SCD, including increases in hemoglobin, improvements in red blood cell health, and decreases in vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) has granted etavopivat Fast Track, Rare Pediatric Disease and Orphan Drug designations. Additionally, etavopivat was granted Orphan Drug designation from the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency for the treatment of patients with SCD. About Forma Therapeutics Forma Therapeutics is a clinical-stage biopharmaceutical company focused on the research, development and commercialization of novel therapeutics to transform the lives of patients with rare hematologic diseases and cancers. Forma Therapeutics R&D engine combines deep biology insight, chemistry expertise and clinical development capabilities to create drug candidates with differentiated mechanisms of action focused on indications with high unmet need. Our work has generated a broad proprietary portfolio of programs with the potential to provide profound patient benefit. About Novo Nordisk Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity and rare blood and endocrine disorders. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 50,800 people in 80 countries and markets its products in around 170 countries.

Read More

BUSINESS INSIGHTS

invoX Pharma Extends Tender Offer to Acquire F-star Therapeutics, Inc.

invoX Pharma Limited | August 05, 2022

invoX Pharma Limited a wholly owned subsidiary of Sino Biopharmaceutical Limited focused on research and development and business development activities outside of China, and F-star Therapeutics, Inc. a clinical-stage biopharmaceutical company pioneering bispecifics in immunotherapy so more people with cancer can live longer and improved lives, today announced that invoX has extended the expiration of its previously announced tender offer for all of the issued and outstanding shares of F-star common stock for a price of $7.12 per share. The tender offer is now scheduled to expire at 05:00 p.m., Eastern Time, on September 19, 2022, unless it is further extended. The tender offer was previously scheduled to expire at one minute after 11:59 P.M., Eastern time, on August 3, 2022. The tender offer is being extended in order to allow additional time for the satisfaction of the regulatory conditions to the offer. The depositary for the tender offer has advised invoX that as of the previous expiration time there were validly tendered and not withdrawn a total of approximately 13,026,582 shares of F-star common stock, and approximately 2,704,867 shares of F-star common stock tendered pursuant to a notice of guaranteed delivery. All terms and conditions of the tender offer remain unchanged during the extension period. F-star shareholders who have already tendered their shares do not have to re-tender their shares or take any other action as a result of the extension. Complete terms and conditions of the tender offer are set forth in the Offer to Purchase, Letter of Transmittal and other related materials, which have been filed by invoX with the Securities and Exchange Commission on July 7, 2022, as amended. In addition, F-star filed a Solicitation/Recommendation Statement on Schedule 14D-9 with the SEC on July 7, 2022, as amended, which includes, among other things, the recommendation of F-star’s board of directors that F-star stockholders tender all of their shares in the tender offer. The Information Agent for the tender offer is Innisfree M&A Incorporated. The Depositary and Paying Agent for the tender offer is Computershare Trust Company, N.A. For all questions relating to the tender offer, please call the Information Agent, Innisfree M&A Incorporated toll-free at (888) 750-5830; banks and brokers may call collect at (212) 750-5833. About invoX invoX was incorporated in March 2021 and is a wholly owned subsidiary of Sino Biopharm, a global top 40 pharmaceutical company with more than 24,000 employees. United Kingdom-based invoX is Sino Biopharm’s international expansion platform, focusing on R&D and business development activities outside of China, with a core focus on oncology and respiratory therapeutics. At its core, invoX aspires to improve patients’ lives by creating access to innovative medicine. About Sino Biopharm Sino Biopharm, together with its subsidiaries, is a leading, innovative research and development driven pharmaceutical conglomerate in China, with a business scope that is vertically integrated including research and development, manufacturing and sales and marketing infrastructure. Sino Biopharm’s product offerings include a variety of biologics and small molecule drugs, and in therapy areas that include hepatology, oncology, cardiovascular and cerebrovascular diseases, orthopaedics, digestive and immune and respiratory diseases. About F-star F-star is a clinical-stage biopharmaceutical company pioneering bispecifics in immunotherapy so more people with cancer can live longer and have improved lives. F-star is committed to working towards a future free from cancer and other serious diseases, through the use of tetravalent (2+2) bispecific antibodies to create a paradigm shift in treatments. F-star has four second-generation immuno-oncology therapeutics in the clinic, each directed against some of the most promising immuno-oncology targets in drug development, including LAG-3 and CD137. F-star’s proprietary antibody discovery platform is protected by an extensive intellectual property estate. F-star has over 500 granted patents and pending patent applications relating to its platform technology and product pipeline. F-star has attracted multiple partnerships with biopharma targeting significant unmet needs across several disease areas, including oncology, immunology, and CNS.

Read More