PHARMACY MARKET

M-Pharma and Kyowa Kirin Sign Exclusive License Agreement for Commercialization of Ilofotase Alfa in Japan

AM-Pharma | September 08, 2021

AM-Pharma B.V., an emerging leader focused on developing therapeutics for severe medical conditions, and Kyowa Kirin Co., Ltd, a global specialty pharmaceutical company that strives to create new value through the pursuit of advances in life sciences and technologies, today announced that they have entered into an exclusive license agreement under which Kyowa Kirin gains the rights to develop and commercialize ilofotase alfa, AM-Pharma’s proprietary recombinant human alkaline phosphatase. Ilofotase alfa is currently being evaluated in the global pivotal REVIVAL Phase III clinical study as the potential first disease-altering treatment for sepsis-associated acute kidney injury (SA-AKI). In July of this year, AM-Pharma announced the enrollment of the first patient in Japan as part of the ongoing REVIVAL trial.

“This is a significant milestone for AM-Pharma as this agreement will optimize the commercialization of ilofotase alfa in the Japanese market and expedite our ability to bring our therapeutic candidate to a substantial patient population in Japan post-approval.”

“Kyowa Kirin is a leading Japanese specialty pharmaceutical company deeply rooted in science that shares our commitment to addressing high unmet medical needs. Based on the number of successful international partnerships they have, they are the ideal partner to support the commercialization of ilofotase alfa in Japan,” stated Erik van den Berg, Chief Executive Officer at AM-Pharma. “This is a significant milestone for AM-Pharma as this agreement will optimize the commercialization of ilofotase alfa in the Japanese market and expedite our ability to bring our therapeutic candidate to a substantial patient population in Japan post-approval.”

Under the terms of the agreement, AM-Pharma will receive EUR 20 million upfront payment, and EUR 30 million related to milestones prior to regulatory submission, and up to EUR 195 million upon submission, NHI price listing and sales milestone payments bringing the overall deal value to EUR 245 million. In addition, AM-Pharma is entitled to tiered double-digit royalties on sales and a drug supply fee. Kyowa Kirin will gain the exclusive right to develop and commercialize ilofotase alfa in Japan. AM-Pharma is responsible for the completion of the REVIVAL pivotal Phase III study, as well as a Phase I pharmacokinetics, safety and tolerability study in Japan and drug supply, whereas Kyowa Kirin will be responsible for the regulatory approval process and commercialization of ilofotase alfa in Japan.

About REVIVAL
The REVIVAL trial is a Phase III pivotal study evaluating AM-Pharma’s proprietary recombinant alkaline phosphatase, ilofotase alfa, for the treatment of patients with SA-AKI. The primary endpoint of the study is all-cause mortality 28 days post-treatment start with ilofotase alfa at a dose of 1.6 mg/kg. In the Phase II study of ilofotase alfa, the patient group treated with this dose experienced a statistically significant 46% relative reduction in mortality compared to the group treated with placebo (p= 0.022). REVIVAL was initiated in November 2020 and is enrolling up to 1,600 patients in North America, Europe and Japan. As per the study protocol, four interim analyses for futility and/or efficacy will be conducted when enrollment hits certain levels and the first futility analysis will occur when 400 patients have been treated. Up to about 120 clinical sites worldwide, including as many as 11 sites in Japan, will enroll patients into the single global pivotal Phase III REVIVAL trial in SA-AKI patients. The U.S. Food and Drug Administration, European Medicines Agency and Japanese Pharmaceuticals and Medical Devices Agency have all approved the REVIVAL protocol. In addition to the REVIVAL study, the PMDA has approved enrollment into a Phase I pharmacokinetics (PK), safety and tolerability study in healthy Japanese subjects, which is being conducted in parallel to REVIVAL in Japan.

About ilofotase alfa
AM-Pharma’s therapeutic candidate is a proprietary recombinant human Alkaline Phosphatase (AP) constructed from two naturally occurring human isoforms of the AP enzyme. The Company’s compound is highly stable and active and has a dual mechanism of action. The recombinant enzyme displays exquisite activity towards dephosphorylating and detoxifying damage-associated molecular patterns (DAMPs) and pathogen-associated molecular patterns (PAMPs) such as, ATP, ADP, lipopolysaccharide (LPS) and other extracellular substrates that drive acute inflammation, coagulation and microvascular ischemia. Research has shown that ATP dephosphorylation has a double effect in protecting against kidney injury. When the pro-inflammatory ATP is dephosphorylated, the resulting adenosine further reduces inflammation through the activation of the immunosuppressive adenosine A2a receptor pathway (A2aR). AM-Pharma has shown that treatment of patients with ilofotase alfa not only reduces local and systemic inflammation but also protects the kidney, and possibly other organs, against further damage.

About AKI and Sepsis
Acute Kidney Injury (AKI) involves inflammatory processes in the kidney which can lead to complete loss of renal function. Hospital‐acquired AKI affects annually around 3 million patients in the US, Europe, and Japan, and is associated with mortality in roughly 700,000 patients. It occurs in 40-60% of critical care admissions. Depending on the severity and cause of renal injury, mortality occurs in up to 60% of the cases. In the US alone, hospitals spend around $10 billion each year on managing this major medical problem. Sepsis is a condition that is responsible for 1 out of 3 deaths in hospitals and is defined as life-threatening organ dysfunction caused by a dysregulated host response to infection. The kidney is the most commonly affected organ, resulting in SA-AKI and significantly increasing the risk for mortality and morbidity in sepsis. No singular effective therapy to alter the progression of these devastating conditions has been approved.4

About AM-Pharma
AM-Pharma's purpose is to save and improve the lives of patients confronted with severe medical conditions. Our initial focus is sepsis-associated acute kidney injury, the cause of death for hundreds of thousands of people hospitalized each year. Our proprietary compound, ilofotase alfa, has the potential to become the first treatment for sepsis-associated acute kidney injury and is now in a global pivotal Phase III clinical trial. We are a dedicated team driven to bring treatment options to severely ill patients, their families and acute care professionals. Find out more about us online at: www.am-pharma.com.

About Kyowa Kirin
Kyowa Kirin strives to create and deliver novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company with a more than 70-year heritage, the company applies cutting-edge science including an expertise in antibody research and engineering, to address the needs of patients and society across multiple therapeutic areas including Nephrology, Oncology, Immunology/Allergy and Neurology. Across our four regions – Japan, Asia Pacific, North America and EMEA/International – we focus on our purpose, to make people smile, and are united by our shared values of commitment to life, teamwork/Wa, innovation, and integrity.

Spotlight

Suicide is the act of intentionally taking your own life. Suicidal feelings can range from being preoccupied by abstract thoughts about ending your life, or feeling that people would be better off without you, to thinking about methods of suicide, or making clear plans to take your own life.

Spotlight

Suicide is the act of intentionally taking your own life. Suicidal feelings can range from being preoccupied by abstract thoughts about ending your life, or feeling that people would be better off without you, to thinking about methods of suicide, or making clear plans to take your own life.

Related News

Jazz Pharmaceuticals Announces New Research Collaboration with Redx Pharma to Discover and Develop Two Targeted Cancer Therapies

Jazz Pharmaceuticals | September 09, 2020

Jazz Pharmaceuticals plc today announced that the company has signed a new research collaboration agreement with Redx Pharma plc (AIM: REDX) to discover and develop drug candidates for two cancer targets in the Ras/Raf/MAP kinase (MAPK) pathway. This research collaboration follows the previously announced sale of Redx's pre-clinical pan-RAF inhibitor program to Jazz for the potential treatment of Raf and Ras mutant tumors in July 2019. Under the terms of the research collaboration agreement, Jazz will make an upfront payment to Redx of $10 million followed by another $10 million in year two, provided research work is continuing. Following delivery of an IND-ready molecule, Redx will be eligible to receive up to a further $200 million from Jazz in development, regulatory and commercial milestone payments for each program

Read More

PHARMACY MARKET

Virpax Announces MMS019 Manufacturing and Supply Agreement

Virpax Pharmaceuticals | August 27, 2021

Virpax® Pharmaceuticals, Inc., a company specializing in developing product candidates for pain management, CNS and anti-viral indications, today announced that it has entered into a commercial manufacturing and supply agreement with Seqens, an integrated global leader in pharmaceutical solutions with 24 manufacturing sites worldwide and seven research and development facilities throughout the U.S. and Europe. The agreement with Seqens provides for both the supply material for Virpax’s clinical studies as well as the long-term commercial supply of MMS019. Seqens will conduct process development and validation of additional large scale commercial quantities of MMS019 at its facilities in Devens and Newburyport, Massachusetts. “Establishing a collaboration with a strong partner capable of supplying clinical and commercial scale quantities of MMS019 is another important advancement in our MMS019 product development strategy. Seqens has a demonstrated expertise in developing and manufacturing highly-complex molecules for large scale production,” said Anthony Mack, Chairman and CEO of Virpax. “Importantly, we expect this collaboration to support future development and supply additional Molecular Envelope Technology programs under development, including Envelta™ and PES200, our post-traumatic stress disorder product candidate,” concluded Mr. Mack. About MMS019 MMS019 is a drug product candidate based on a type of nanotechnology that enables the exclusive delivery of a metabolically labile peptide drug into the brain via intranasal delivery. MMS019 is manufactured using industrially relevant equipment and processes (high pressure homogenization and spray drying). There is pharmacological evidence of activity of molecular envelope technology (MET) enabled enkephalin in morphine-tolerant animals. The MET nanoparticles are well tolerated via the nasal route at the dose administered. MMS019 demonstrated comparable preclinical activity to morphine in all animal pain models tested without the drug seeking and tolerance associated with opioids. About Seqens SEQENS is an integrated global leader in pharmaceutical solutions and specialty ingredients. With 24 production sites and 7 R&D centers in Europe, North America and Asia, SEQENS develops and manufactures tailor-made solutions and ingredients for the most demanding industries such as healthcare, electronics, cosmetics, food and home care. Driven by a culture of excellence and a strong entrepreneurial spirit, our 3,200 employees are committed to providing our customers with the highest level of service and quality while acting ethically in accordance with our corporate social responsibility program.” About Virpax Pharmaceuticals Virpax is developing branded, non-addictive pain management product candidates using its proprietary technologies to optimize and target drug delivery. Virpax is initially seeking FDA approval using its three patented drug delivery platforms. Epoladerm™ is a topical diclofenac metered-dose spray film formulation being developed to manage acute musculoskeletal pain and osteoarthritis. Probudur™ is a single injection liposomal bupivacaine formulation being developed to manage post-operative pain. Envelta™ is an intranasal molecular-envelope enkephalin formulation being developed to manage acute and chronic pain, including pain associated with cancer. Virpax is also using its intranasal Molecular Envelope Technology (MET) to develop its PES200 product candidate to manage post-traumatic stress disorder (PTSD) and its MMS019 product candidate to inhibit viral replication caused by influenza or SARS-CoV-2.

Read More

PHARMA TECH

Invitae to Acquire Ciitizen to Strengthen its Patient-Consented Health Data Platform to Improve Personal Outcomes and Global Research

Invitae | September 07, 2021

Invitae , a leading medical genetics company, today announced it has entered into a definitive agreement to acquire Ciitizen, a patient-centric consumer health tech company. Backed by a16z, Section 32 and Verily, Ciitizen is working to build a global platform to help patients collect, organize, store and share their medical records digitally. The acquisition would enhance Invitae's platform by providing patients an easy-to-use, centralized hub for their genomic and clinical information, which together comprise a powerful dataset with the potential to drive research and improve healthcare decision making. "Invitae is relentlessly pursuing a day when genetics is used routinely to improve healthcare for billions of people around the globe. We believe combining Ciitizen's state-of-the-art, transparent and patient-consented platform with our technologies and services will accelerate our evolution into a genome information company that informs healthcare throughout one's life. This would give us the ability to engage patients, create an innovative patient-centered data ecosystem and deliver better outcomes for everyone. Invitae views the acquisition of Ciitizen as an important part of its strategy to be the industry leader across the genetic testing, software and health information technology spaces," said Sean George, Ph.D., co-founder and CEO of Invitae. "Ciitizen has already helped patients take ownership of their medical records to optimize therapy for themselves and contribute their data towards a variety of goals, including research on rare diseases," said Anil Sethi, CEO and founder of Ciitizen. "Patients leave a breadcrumb trail of records behind them, and we believe access to this real-world data is key to personalized therapeutics in the future. The combination of lifelong health history together with Invitae's world-class genetic and data services would enable a digital ecosystem of personalized services for each patient. Democratization of patient-consented data is expected to improve outcomes by providing access to third-parties for whom the patient has granted consent. Unlike many health data companies, we operate on the premise that it's the patient's data, not ours." Invitae is working to aggregate results from the world's genetic tests into a single, easy-to-use service that makes genetic information accessible to all who can benefit from it - always with patient consent. Over the past five years, Invitae has completed 13 acquisitions that have expanded its testing menu, lowered the cost of its tests, improved its customer experience and enhanced its ability to attract partners. Invitae raised more than $1.4 billion since the beginning of 2021, in part to identify and acquire key assets that bring the company closer to delivering on its vision. The addition of Ciitizen is expected to empower Invitae to more quickly extend its platform, adding a complementary patient service that streamlines the process of collecting and organizing health information from any source and uses machine learning to transform the unstructured (and largely untapped) health information to enable better care decisions, find clinical trials, and power research using real-world evidence. In addition, as the use of Invitae testing and the Ciitizen service expands, third-party access to consented genomic and other clinical data can be democratized to power a variety of applications that can enhance Invitae's business and growth strategy. About Invitae Invitae Corporation is a leading medical genetics company, whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. About Ciitizen Ciitizen is a patient-centric consumer health technology platform that enables patients to collect, store and share their medical records in a single location. Ciitizen developed proprietary machine learning and AI technologies to abstract critical information from unstructured health records to advance the understanding of diseases, match patients with potentially life-saving clinical trials, and enable better care decisions. Ciitizen also provides valuable big data analytics and data quality technologies to the network of Health Information Exchanges (HIEs) to produce research-ready, regulatory-grade data within minutes, instead of hours or days, and ensure HIEs can comply with the Information Blocking regulations.

Read More