Lilly, BI's Jardiance still beats Novo’s Rybelsus when it comes to cost-effectiveness: ICER

FiercePharma | November 01, 2019

The Institute for Clinical and Economic Review (ICER) started giving Novo Nordisk a hard time about the cost-effectiveness of its oral version of semaglutide, Rybelsus, before the drug was even approved in September. Now the product has its price tag— $772 per month, on par with comparable injectables—yet ICER still isn’t happy. ICER released a report Friday concluding that if Rybelsus is prescribed as an add-on treatment to metformin in Type 2 diabetes, it will be less cost-effective than Eli Lilly and Boehringer Ingelheim’s Jardiance in the same setting. Rybelsus is the first GLP-1 receptor agonist that’s available in pill form, but Jardiance—a widely prescribed SGLT-2 inhibitor—is also an oral option. Hence “judging from the list price of oral semaglutide, its net price is likely to be much higher than that of competitor oral treatments…that appear to have similar benefits with fewer common side effects,” ICER said in a statement. ICER’s report came on the same day that Novo released its third-quarter earnings, and during a conference call with investors, chief financial officer Karsten Munk Knudsen griped that the cost-effectiveness review didn't take a broad enough approach. "Over decades, this is clearly cost-effective," he said of the new drug.

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More than 60 people from 16 states have been reported to have measles in the first four months of 2018, says the Centers for Disease Control and Prevention (CDC). New outbreaks continue to add to the number of cases. In 2017, the CDC says 118 people were confirmed with the highly contagious viral disease. "Measles is amongst the most infectious agents we know," says Dr. Pritish Tosh, an infectious diseases specialist at Mayo Clinic.

Spotlight

More than 60 people from 16 states have been reported to have measles in the first four months of 2018, says the Centers for Disease Control and Prevention (CDC). New outbreaks continue to add to the number of cases. In 2017, the CDC says 118 people were confirmed with the highly contagious viral disease. "Measles is amongst the most infectious agents we know," says Dr. Pritish Tosh, an infectious diseases specialist at Mayo Clinic.

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PHARMA TECH

Asahi Kasei Pharma to Sponsor Research for Drug Discovery Research and Technology: Open Innovation 2023

Asahi Kasei | October 17, 2022

Asahi Kasei Pharma, subsidiary of diversified Japanese multinational company Asahi Kasei, is publicly calling for new proposals for drug development research as part of its efforts for open innovation to promote pharmaceutical research and development through enhanced cooperation with universities, research institutes and enterprises around the world. The application period begins at 5:00 a.m. GMT on January 5, 2023 and ends at 8:00 a.m. GMT on February 28, 2023. Asahi Kasei Pharma established its Open Innovation Department in October 2016 with the mission to further strengthen and enhance global licensing and alliance-building activities, and has since funded around 5 proposals per year on average with a total of over 20 funded proposals for state-of-the-art pharmaceutical drug developments as well as collected a wealth of creative new ideas. The Open Innovation team is seeking to fund innovative proposals from researchers around the world for up to US$200,000 per year, per project for this upcoming round in 2023. Asahi Kasei Pharma will then pursue joint research opportunities with the drug discovery researchers together with their parent institutions in hopes to create new innovative drugs for diseases that currently have unmet needs or pioneer new platform technologies for drug discovery. Asahi Kasei Pharma has demonstrated a successful track record in developing new drugs in the realms of autoimmune, renal, neurological, and bone disease, so the organization can provide the greatest contribution from correlating research areas and subjects. "Asahi Kasei Pharma believes that nobody should have to give up what they would like to do because of illness. We believe great science comes from working together, and we are excited for the opportunity to accomplish this mission with researchers all over the world.” Osamu Matsuzaki, Primary Executive Officer and Head of R&D and Business Development at Asahi Kasei Pharma Open Innovation works to establish research collaborations related to innovative drug targets and candidate drug compounds up to the preclinical stage as well as advanced drug discovery technologies. Advancements in exploring a new mechanism of action for treating refractory autoimmune disease is one of the most promising outcomes of the research so far, with many more on the horizon. Asahi Kasei believes that these continued efforts to advance drug discovery and research technology will further contribute to life and living for people around the world. About Asahi Kasei The Asahi Kasei Group contributes to life and living for people around the world. Since its foundation in 1922 with ammonia and cellulose fiber businesses, Asahi Kasei has consistently grown through the proactive transformation of its business portfolio to meet the evolving needs of every age. With more than 46,000 employees around the world, the company contributes to a sustainable society by providing solutions to the world's challenges through its three business sectors of Material, Homes, and Health Care. Its health care operations include devices and systems for acute critical care, dialysis, therapeutic apheresis, transfusion, and manufacture of biotherapeutics, as well as pharmaceuticals and diagnostic reagents.

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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.

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PHARMACY MARKET

Alnylam Announces FDA Approval of Supplemental New Drug Application for OXLUMO® (lumasiran) in Advanced Primary Hyperoxaluria Type 1

Alnylam Pharmaceuticals, Inc. | October 07, 2022

Alnylam Pharmaceuticals, Inc. the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration approved a label expansion for OXLUMO® an RNAi therapeutic administered via subcutaneous injection, now indicated for the treatment of primary hyperoxaluria type 1 to lower urinary oxalate and plasma oxalate levels in pediatric and adult patients. The approval is based on positive efficacy and safety results of the ILLUMINATE-C Phase 3 study of OXLUMO in patients with severe renal impairment, including those on hemodialysis. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction in the liver. The excess production of oxalate results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones and nephrocalcinosis, which can progress to kidney failure. PH1 can also lead to oxalate deposition in multiple organs beyond the kidney, a condition known as systemic oxalosis. The label expansion for OXLUMO is based on positive six-month results from ILLUMINATE-C, where treatment with lumasiran resulted in a substantial reduction in POx from baseline to Month Six in both dialysis-independent and -dependent patients. Patients in both cohorts had a reduction in POx as early as Month One. In Cohort A treatment with OXLUMO led to a 33% least squares (LS) mean reduction in POx from baseline to Month Sixand in Cohort B, treatment with OXLUMO led to a 42% LS mean reduction in POx from baseline to Month Six. OXLUMO demonstrated an encouraging safety and tolerability profile with no deaths or drug-related serious adverse events among enrolled patients. There were two treatment discontinuations due to adverse events in the extension period of the study, neither of which was drug related. The most common AE related to lumasiran was injection-site reaction reported in five of 21 patients which were all mild and transient. “The significance of the label expansion of OXLUMO cannot be overstated, as this milestone provides crucial reassurance among a patient population with the highest unmet need, as well as their caregivers and loved ones, that OXLUMO is available in the US for patients living with PH1, including those with advanced disease. We are grateful to Alnylam for their continued commitment to the PH1 community and for ensuring that all patients — no matter how severe their disease — have the same opportunity to potentially benefit from OXLUMO treatment.” Kim Hollander, Executive Director of the Oxalosis and Hyperoxaluria Foundation In November of 2020, OXLUMO was approved by the FDA for the treatment of PH1 to lower UOx levels in pediatric and adult patients. It was also approved by the European Medicines Agency (EMA) for the treatment of PH1 in all age groups. The Committee for Medicinal Products for Human Use (CHMP) of the EMA delivered a positive opinion recommending variation to the marketing authorization of OXLUMO based on ILLUMINATE-C data from patients with advanced PH1 in September 2022. Lumasiran is also being evaluated in a global Phase 2 study as an investigational treatment in patients with recurrent kidney stone disease and elevated UOx levels. About OXLUMO OXLUMO® is an RNAi therapeutic targeting hydroxyacid oxidase 1. HAO1 encodes glycolate oxidase. Thus, by silencing HAO1 and depleting the GO enzyme, OXLUMO inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. OXLUMO has received regulatory approvals from the U.S. Food and Drug Administration for the treatment of primary hyperoxaluria type 1 to lower urinary and plasma oxalate levels in pediatric and adult patients and from the European Medicines Agency for the treatment of PH1 in all age groups. In the pivotal ILLUMINATE-A study, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. In the ILLUMINATE-C study, OXLUMO resulted in substantial reductions in plasma oxalate in patients with advanced PH1. Across all three studies, injection site reactions were the most common drug-related adverse reaction. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly beginning one month after the last loading dose at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. About Alnylam Pharmaceuticals Alnylam has led the translation of RNA interference into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding 20 years ago, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® GIVLAARI® OXLUMO® AMVUTTRA® and Leqvio® being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.

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