ALR Technologies | June 30, 2022
ALR Technologies SG Pte. Ltd the diabetes management company, announces the completion of a definitive manufacturing agreement with Infinovo Medical Co. Ltd to manufacture and supply the Continuous Glucose Monitor hardware that will be utilized as part of the ALRT GluCurve Pet CGM. The Manufacturing Agreement gives ALRT the exclusive global rights to distribute the Infinovo CGM hardware for the animal health market, providing long-term production and supply. Subject to the satisfaction of all closing conditions it is expected that initial deliveries of product by Infinovo will be made during Q4 of 2022
“Executing the Manufacturing Agreement with Infinovo marks another major milestone in our path to commercialization and profitability. Our next targeted milestone is securing distribution for the ALRT GluCurve Pet CGM. We have been working with many of the largest global distributors and pharmaceutical companies in animal health, evaluating different distribution opportunities ranging from joint venture partnerships to sales and distribution agreements. We believe we have narrowed in on the right company and right deal structure to best bring value to our shareholders and to those who care for diabetic pets. We hope to announce the details of this pivotal next step in the near future.”
Sidney Chan, Chairman and CEO of ALRT
The Manufacturing Agreement is subject to certain closing conditions including the completion of a distribution agreement with a third party by July 31, 2022.
About Infinovo Medical Co. Ltd
Founded in 2016, Infinovo is an innovative medical technology company, focusing on developing an accurate and affordable CGM for patients which will be available for both Type 1 and Type 2 Diabetics.
ALR Technologies SG Pte. Ltd.
ALRT is a data management company that developed the ALRT Diabetes Solution, a comprehensive approach to diabetes care that includes an FDA-cleared and HIPAA compliant diabetes management system that collects data directly from blood glucose meters and continuous glucose monitoring devices, and a patent pending Predictive A1C algorithm to track treatment success between lab reports and an FDA-cleared Insulin Dosing Adjustment program. ALRT also offers an algorithm to provide prescribers support for timely non-insulin medication advancements. The overall goal is to optimize diabetes drug therapies to drive improved patient outcomes. The program tracks performance of all clinical activities to ensure best practices are followed. The ALRT Diabetes Solution gives healthcare providers a platform for remote diabetes care, helping to minimize patient exposure to potential infections in clinical settings. Currently, the Company is focused on diabetes and intends to expand its services to cover other chronic diseases anchored on verifiable data.
In addition, the animal health division of ALRT has identified an unmet need in diabetes care and has developed GluCurve; a solution to assist Veterinarian Doctors to determine the efficacy of insulin and to help to identify the appropriate dose and frequency of administration of insulin for companion animals, thereby delivering the same optimization of diabetic drug therapies to pets as to humans.
ALR Technologies SG Pte. Ltd. is controlled by ALR Technologies Inc., a Company with its shares traded on the OTC:QB under the symbol “ALRT”. On May 17, 2022, ALR Technologies Inc. announced an Agreement and Plan of Merger and Reorganization for the sole purpose of changing the Company’s jurisdiction of incorporation from Nevada to Singapore (the “Redomicile Merger Agreement”). The Redomicile Merger Agreement is subject to the required approval of the Company’s stockholders, requisite regulatory approvals, the effectiveness of the registration statement on Form F-4 filed by ALRT related to the Redomicile Merger, and other customary closing conditions. The Redomicile Merger is expected to be completed during the third quarter of 2022. See the Form 8-K filed May 20, 2022, by ALR Technologies Inc. for further information about the Redomicile Merger Agreement.
KemPharm | May 16, 2022
Arimoclomol is an NDA-stage, revenue-generating investigational drug candidate being developed for the treatment of Niemann-Pick disease type C a rare progressive neurodegenerative disease
KemPharm plans to refile the New Drug Application (NDA) for arimoclomol in NPC with the U.S. Food and Drug Administration as early as the First Quarter of 2023
CELEBRATION, Fla., May 15, 2022 KemPharm, Inc. a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system (CNS) diseases, announced a definitive agreement with Orphazyme A/S to acquire arimoclomol, an orally-delivered, first-in-class heat shock protein (HSP) amplifier being developed as a treatment for Niemann-Pick disease type C (NPC). NPC is a rare progressive neurodegenerative disease that impacts children, adolescents, and adults, and is characterized by an inability of the body to transport cholesterol and lipids inside of cells, which leads to the abnormal accumulation of these substances within various tissues of the body, including the brain. Arimoclomol is currently being made available to NPC patients in the U.S., France and Germany under Orphazyme’s Early Access Programs (EAP).
Under the terms of the agreement, KemPharm will purchase substantially all of the assets and operations of Orphazyme, including arimoclomol, for a cash payment of USD $12.8 million. The Company expects to finance the cash payment with a revolving line of credit secured by KemPharm’s balance sheet. KemPharm intends to retain the majority of Orphazyme’s current employees. In addition, KemPharm has agreed to assume an estimated reserve liability equal to approximately USD $5.2 million, which is an estimated future rebate due to the French regulatory authorities based on the revenue generated from the EAP in France. For the year ending December 31, 2022, the EAP is expected to generate at least USD $12 million in revenue based upon enrollment in France as of March 2022. The EAP is expected to remain in place until arimoclomol becomes commercially available in each of the current EAP markets. The transaction is expected to close on or before June 1, 2022, subject to customary closing conditions and approval by Orphazyme’s creditors and the Danish bankruptcy court. Canaccord Genuity LLC acted as a strategic advisor to KemPharm for the transaction.
“This strategic acquisition of arimoclomol is a transformative event that significantly expands our rare CNS disease development pipeline, bringing to KemPharm an NDA-stage, revenue-generating product upon which we intend to build commercial capabilities that allow KemPharm to create and retain value for the benefit of shareholders. Moreover, the financial structure of the acquisition combined with the revenue currently being generated by arimoclomol from the early access program in France affords us the opportunity to acquire the asset in a capital efficient manner that has the potential to create positive cash flow, while incurring no shareholder dilution.”
Richard Pascoe, Executive Chairman of KemPharm
Arimoclomol is administered orally and has been studied in ten Phase 1, four Phase 2, and three pivotal Phase 2/3 trials. Arimoclomol has received Orphan Drug Designation (ODD) for NPC in the United States and the European Union. Arimoclomol has received Fast-Track Designation (FTD), Breakthrough Therapy Designation (BTD), and Rare Pediatric Disease Designation (RPDD) from the FDA for NPC. If approved in the U.S., arimoclomol would also be eligible to receive a Pediatric Priority Review Voucher. On June 17, 2021, Orphazyme received a Complete Response Letter (CRL) from the FDA regarding its NDA for arimoclomol for the treatment of NPC. Orphazyme also withdrew its European Marketing Authorisation Application (MAA) for arimoclomol for the treatment of NPC ahead of a final vote and opinion by the Committee for Medicinal Products for Human Use.
“The acquisition of arimoclomol aligns perfectly with our strategy to build KemPharm’s value via the advancement and commercialization of novel treatments that address rare CNS conditions, including our lead clinical candidate, KP1077 in idiopathic hypersomnia,” stated Travis Mickle, Ph.D., President and Chief Executive Officer of KemPharm. “We have carefully evaluated the CRL issued by the FDA and the minutes from the subsequent Type A meeting, as well as the data that has been generated from the development work performed to date. We believe the efficacy signal for arimoclomol in NPC is convincing and that there is a viable regulatory path that could enable a successful NDA resubmission. KemPharm has significant experience with challenging regulatory situations, having successfully led or participated in three FDA product approvals, two of which followed an initial CRL. We welcome the opportunity to work with the FDA on the resubmission of the NDA for arimoclomol in NPC, which we expect to file as early as the first quarter of 2023.”
NPC is a rare progressive lysosomal storage disorder characterized by an inability of the body to transport cholesterol and lipids inside of cells. This leads to dysfunction in organs such as the brain, spleen and liver. NPC can range from a fatal disorder within the first few months after birth (neonatal period) to a late onset, chronic progressive disorder that remains undiagnosed well into adulthood. Disease progression is irreversible in all patients, and loss of neuro-cognitive function adversely impacts their daily life. The mean age of death is 13 years (Bianconi, 2019), and there are no approved treatments for NPC in the United States.
“NPC is an ultra-rare, inherited neurodegenerative disease that affects people of all ages from infancy to adulthood, and leads to progressive impairment of mobility, cognition, speech, and swallowing, culminating in premature death,” said Marc Patterson, MD, Professor of Neurology, Pediatrics and Medical Genetics at Mayo Clinic. “Therapies to treat NPC are desperately needed, and there is hope that a treatment such as arimoclomol could provide a solution to patients around the world who are living daily with the disease. It is encouraging that there is an opportunity to continue the regulatory process for arimoclomol with the FDA.”
KemPharm is a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system diseases through its proprietary LAT® platform technology. KemPharm utilizes its proprietary LAT® platform technology to generate improved prodrug versions of FDA-approved drugs as well as to generate prodrug versions of existing compounds that may have applications for new disease indications. KemPharm’s prodrug product candidate pipeline is focused on the high need areas of idiopathic hypersomnia (IH) and other CNS/rare diseases. In addition, the U.S. Food and Drug Administration (FDA) has approved AZSTARYS®, a new once-daily treatment for ADHD in patients age six years and older containing KemPharm’s prodrug, serdexmethylphenidate (SDX), which is being commercialized by Corium, Inc. in the U.S., and APADAZ®, an immediate-release combination product containing benzhydrocodone, KemPharm’s prodrug of hydrocodone, and acetaminophen, which is being commercialized by KVK-Tech, Inc. in the U.S.
Innocoll Pharmaceuticals | July 07, 2022
Innocoll Pharmaceuticals Limited, a commercial-stage biotechnology company and portfolio business of Gurnet Point Capital, announced the publication of the results of Study BU-002-IM evaluating the safety and efficacy of POSIMIR® for infiltration use in 107 patients undergoing arthroscopic subacromial decompression. The peer-reviewed paper, entitled “SABER-Bupivacaine Reduces Postoperative Pain and Opioid Consumption After Arthroscopic Subacromial Decompression: A Randomized, Placebo-Controlled Trial,” was published online on May 17, 2022 in the Journal of the American Academy of Orthopedic Surgeons Global Research & Reviews.
According to the report, POSIMIR 5 mL administered into the subacromial space at the end of arthroscopic subacromial decompression surgery in this double-blind, randomized, controlled, multicenter study, reduced mean postoperative pain on movement over 72 hours by 1.3 points on a 0-10 scale compared with vehicle control, a relative reduction of 20%. Simultaneous improvements in postoperative opioid use during the same 72-hour period provided evidence that the observed pain reduction was clinically meaningful. The median time to first request for opioid rescue medication was 12.4 hours among patients treated with POSIMIR compared with 1.2 hours in patients who received vehicle control. The median total opioid consumption in intravenous (IV) morphine milligram equivalents over 72 hours was 4.0 mg in the POSIMIR group vs 12.0 mg in the vehicle control group. The proportion of patients who were opioid free at 72 hours was 40% in the POSIMIR group vs 16% in the vehicle control group.
As reported in the paper, the most common treatment-emergent adverse events were headache, nausea, musculoskeletal pain, cardiac disorders, skin and subcutaneous tissue disorders, injury and procedural complications, general disorders and administration site conditions and respiratory, thoracic, and mediastinal disorders. Of 2 serious adverse events reported among patients in these treatment groups, only 1 occurred during the immediate 2-week postsurgical follow-up period, and none was considered related to treatment. There were no TEAEs leading to study discontinuation.
“These very compelling results, demonstrating up to three days of local analgesia after a single intra-operative injection, make POSIMIR an important new tool in treating postoperative pain from arthroscopic subacromial decompression,” said Sten Rasmussen, MD PhD, Professor and Head of Department of Clinical Medicine, Aalborg University, Denmark, the senior author and a principal investigator in the POSIMIR study.
“We are excited to publish the positive results of this pivotal clinical trial of POSIMIR, the second approved product in our portfolio of nonopioid, extended-duration, locally-acting analgesics for postsurgical pain control. As the only company with two non-opioid extended release bupivacaine products on the market, we look forward to bringing POSIMIR to the orthopedic community,”
Louis Pascarella, Innocoll President and Chief Executive Officer
POSIMIR for infiltration use contains more bupivacaine in a single dose than any other approved, sustained-release bupivacaine product. It employs a proprietary extended-release technology that enables the continuous release of bupivacaine, a non-opioid local anesthetic, directly to the surgical site for 3 days after administration. POSIMIR was approved in February, 2021 by the US Food and Drug Administration. POSIMIR is indicated in adults for administration into the subacromial space under direct arthroscopic visualization to produce post-surgical analgesia for up to 72 hours following arthroscopic subacromial decompression. Use in any other surgical procedure is investigational. POSIMIR is a registered trademark of Innocoll Pharmaceuticals Limited.
About Subacromial Decompression Shoulder Surgery
Subacromial decompression is a type of shoulder surgery used to treat impingement syndrome, a common repetitive-use injury that causes pain when the arm is raised over the head. The procedure is performed arthroscopically, meaning that several small incisions are made in the skin and muscle of the shoulder through which a camera (arthroscope) and surgical instruments are inserted during surgery. Arthroscopic subacromial decompression is generally performed as outpatient surgery, and most patients go home within a few hours of surgery. The most intense pain typically occurs during the first 3 days and is often managed with a combination of nerve block and oral opioids. There are over 600,000 surgeries involving arthroscopic subacromial decompression performed each year in the U.S.
About Innocoll Pharmaceuticals Limited
Innocoll Pharmaceuticals Limited is a global biotech pharmaceutical company headquartered in Athlone, Ireland and is a subsidiary of Innocoll Biotherapeutics Holding Limited. The Innocoll group of companies is focused on the development and commercialization of pharmaceutical technologies to meet some of today’s most important healthcare challenges.
About Gurnet Point Capital
Gurnet Point Capital is a unique healthcare investment platform within the B-Flexion Group and led by a team with deep expertise in an industry for which they share a passion, both as investors and senior executives. GPC invests long-term capital and supports entrepreneurs in building a new generation of companies that deliver outsized returns through active ownership. Based in Cambridge, MA, its remit encompasses life sciences and health care focused businesses, with a particular emphasis on businesses that have high growth potential in the product development and commercialization stages of their evolution. With its strategy of driving best in class operational transformation for these businesses, to create social impact while generating significant economic value, Gurnet is able to deliver differentiated results for its investors and partners.
Integral Molecular | June 09, 2022
Integral Molecular and Optimeos Life Sciences announce a partnership to develop next-generation mRNA and DNA therapeutics that will use antibody-based molecular targeting to direct vaccines and gene therapies to relevant tissues in a patient's body. This partnership combines Integral Molecular's experience in antibody discovery and mRNA immunization with Optimeos' technology for nanoparticle-based drug delivery systems.
DNA and mRNA therapeutics harness a patient's own molecular machinery to encode proteins. Integral Molecular and Optimeos plan to encapsulate these therapeutic molecules within nanoparticle spheres that are decorated with antibodies on the outer surface. The antibodies can then target the particles to specific tissues. Currently, gene therapies and mRNA vaccines are delivered into the body via infusion or injection, but only a fraction of the molecules arrive at their intended site. The proposed molecular targeting strategy has the potential to transform the delivery of vaccines and therapeutics by dramatically reducing dosages, decreasing toxicity, and enabling scalable administration.
Integral Molecular will apply its industry-leading antibody discovery expertise against complex cell-surface proteins to provide targeting moieties for the therapies. Its specialized MPS Antibody Discovery platform is tailored to work with challenging membrane protein targets and routinely uses mRNA-based immunization strategies. "Precision targeting of an isolated cell type with gene therapy holds great promise for genetic diseases and may provide brand-new directions in cancer immunotherapy including in vivo CAR-T cell therapeutics," said Joseph Rucker, Co-founder and VP of R&D at Integral Molecular.
Optimeos' technology allows the robust and scalable incorporation of mRNA and DNA therapeutics and other biologics into customizable nanoparticles. "We are excited to work with Integral Molecular towards the next generation of gene-therapy that no longer relies on viral-based delivery systems," said Robert Prud'homme, Co-founder and Chief Technical Officer of Optimeos.
Integral Molecular and Optimeos will be attending the BIO International Convention in San Diego later this month.
About Integral Molecular
Integral Molecular is the industry leader in developing and applying innovative technologies that advance the discovery of therapeutics against difficult protein targets. With 20 years of experience focused on membrane proteins and antibodies, Integral Molecular's technologies have been integrated into the drug discovery pipelines of over 400 biotech and pharmaceutical companies to help discover new therapies for cancer, diabetes, autoimmune disorders, and viral threats such as SARS-CoV-2, Ebola, Zika, and dengue viruses.
About Optimeos Life Sciences
Optimeos Life Sciences has developed a novel, highly differentiated, non-viral, nanoparticle technology, for delivery of new therapeutic drugs to address important unmet medical needs. The technology is a patented and scalable polymeric drug delivery system, which was developed and refined in Professor Prud'homme's lab at Princeton University over 20 years. Optimeos' technology enables encapsulation of mRNA, DNA, peptides, proteins or small molecules, as well as their targeted delivery to cells and tissues beyond the liver.