BUSINESS INSIGHTS

OptimizeRx Acquires EvinceMed, a Specialty Drug Prescription Initiation Platform

OptimizeRx Corporation | March 16, 2022

OptimizeRx Corp. a leading provider of point-of-care technology solutions helping patients start and stay on therapy, announced a definitive agreement to acquire the EvinceMed platform and related assets. EvinceMed is a leader in delivering end-to-end automation for specialty pharmaceutical transactions. The acquisition includes the full Market Access Management Platform supporting pharma manufacturers, hub providers and pharmacies to improve patient access, speed to therapy and activation of affordability programs.

With the EvinceMed platform, OptimizeRx will be able to help patients get access to the drugs they need by simplifying the prescribing process for specialty products, automating manual steps to determine drug eligibility and affordability, and introducing electronic enrollment and medical documentation within workflow across the OptimizeRx network of over 300 electronic health record (EHR) systems, ePrescribing platforms, and account-based marketing technologies spanning more than 700 thousand providers. The integration of the EvinceMed and OptimizeRx platforms has already begun with an exclusive collaboration that began last year. Full integration of the technology assets will further the Company’s ability to help patients start and stay on therapy.

While specialty drugs account for only 2.2% of total prescription volume in the U.S, they accounted for over 45% of total pharmacy spend, or $218.6 billion in 2019 1,2. The cost and increasing use of specialty medications has exposed unique barriers to the prescription and patient access process. Manufacturer support programs provide much-needed services to prescription medication access, affordability, and adherence. However, a recent survey of 10,000 patients across multiple therapeutic areas found that only 20% of patients were aware of such programs3. Doctors and their staff can assist patients with enrollment into these programs, yet the enrollment process is often time-consuming, and doctors remain dissatisfied with current patient support options available. In addition, less than 10% of patient enrollment into these support programs is currently completed electronically4.

We are thrilled to have EvinceMed transition from an exclusive technology partner over the last year to being part of the team. Given the success we saw working together, this combination strengthens our position to serve our pharma customers across the full HCP network and throughout the patients’ journeys – from the moment the doctor logs into their EHR right through the point of dispense at the pharmacy. This acquisition offers significant high margin cross-selling opportunities across our growing customer base of leading life science companies, while further fortifying our business through vertical integration, and helping drive upward momentum to our key performance indicators. We expect this acquisition to benefit revenue growth in 2023 as we further develop our network around connectivity to additional partners.”

William Febbo, CEO of OptimizeRx

David Jackson, CEO of EvinceMed commented, “We are very excited to be joining the OptimizeRx team. The impact of our combined technologies and the reach and scale that OptimizeRx has with pharmaceutical manufacturers is a game-changer to patient access to specialty medications. By automating the processes and tasks required to be completed prior to the fill and dispense of specialty medications, we can get patients on treatment much faster and at a lower overall cost.”

OptimizeRx chief commercial officer, Steve Silvestro, added, “Our commitment to leveraging technology to assist providers and patients in accessing the tools they need to improve outcomes is a mission-driven effort. The EvinceMed assets give us additional resources to accomplish that while also expanding the network’s reach to point-of-dispense, a goal we have long pursued. Prescribing support through automation for providers will both accelerate workflows and remove barriers to the specialty prescribing process ensuring patients are able to readily access the life-changing innovative therapies that they need.”

OptimizeRx is committed to investing in innovation to bring more digital in-workflow solutions to execute against the commercialization goals of life science organizations. OptimizeRx currently provides solutions to 19 of the 20 largest pharma manufacturers and seeks to continue driving long-term, sustainable ROIs for its clients while supporting initiatives that improve health outcomes. Recent market research commissioned by OptimizeRx revealed specialty drug access support as being a $12B+ market. This acquisition further diversifies OptimizeRx revenue streams as the Company scales to address the client needs in this space.

Spotlight

Bioassays are required for a variety of purposes in the development and production of biopharmaceuticals including drug candidate selection, product releases, product stability assessment, and comparability to support proposed process changes. However, because of their complexity and susceptibility to many variables, bioassays often prove problematic and difficult to develop. Timely development of suitable assay systems represents a major investment on the part of the biopharmaceutical industry but late development often results in even more costly clinical holds. Recent and ongoing advances in biology and technology provide the possibility of new types of assay systems and readouts. Some offer significant logistical and scientific advantages so that, in addition to being investigated for new products, they are increasingly being considered as replacements for some existing bioassays.


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BUSINESS INSIGHTS

KemPharm Announces Strategic Acquisition of Arimoclomol from Orphazyme, Expanding its Rare CNS Diseases Pipeline

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.” About KemPharm 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.

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PHARMACY MARKET

CASI PHARMACEUTICALS ANNOUNCES CNCT19 (CD19 CAR-T) GRANTED ORPHAN DRUG DESIGNATION BY THE U.S. FDA

CASI Pharmaceuticals, Inc. | January 19, 2022

CASI Pharmaceuticals, Inc. a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, announced today, that the U.S. Food and Drug Administration as granted Orphan Drug Designation for CNCT19, an investigational CD-19 directed CAR-T therapy, for the treatment of patients with Acute Lymphoblastic Leukemia. CNCT19 is currently being developed independently by Juventas to meet the urgent clinical needs of patients with hematologic malignancies globally. The National Medical Products Administration has granted CTA approval for CNCT19 in two indications in Nov. 2019. Currently, the Phase II clinical trials of CNCT19 are in progress. Positive clinical data for adult and pediatric patients with relapsed/refractory B-ALL has been presented at the December 2021 ASH annual meeting, which further demonstrated its safety and efficacy profile. CNCT 19 is expected to be the first domestic CD19 directed CAR-T product in China with independent intellectual property rights. "Our partner Juventas continues to make encouraging progress in developing their CD19 CAR-T therapy. The Orphan Drug Designation, from the FDA, and the Breakthrough Designation status, granted by the China Center of Drug Evaluation (CDE) in December 2020, represent significant milestones that demonstrate our belief that CNCT19's commercialization will not only be successful in China, but potentially on a global scale. CASI has worldwide co-commercial rights of CNCT19, and will start the global commercialization process according to CNCT19's regulatory progress outside China." Dr. Wei-Wu He, CASI's Chairman, and CEO About Juventas Juventas is a biopharmaceutical company headquartered in China dedicated to the development and commercialization of cell therapies globally. Utilizing innovative and integrated technology platforms, the company has developed a diverse pipeline of cellular immunotherapies for treatment of hematological malignancies, solid tumors, and other non-oncological conditions both in China and globally. At present, the company is conducting two pivotal clinical trials of CNCT19 for treating adult r/r-B-ALL and r/r-B-NHL in China. CNCT19 has the potential to become the first launched domestically developed CD19 CAR-T therapy in China and the first CAR-T product for the treatment of adult R/R B-ALL in China. About CASI Pharmaceuticals CASI Pharmaceuticals, Inc. is a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products in China, the United States, and throughout the world. The Company is focused on acquiring, developing and commercializing products that augment its hematology oncology therapeutic focus as well as other areas of unmet medical need. The Company intends to execute its plan to become a leader by launching medicines in the greater China market leveraging the Company's China-based regulatory and commercial competencies and its global drug development expertise. The Company's operations in China are conducted through its wholly-owned subsidiary, CASI Pharmaceuticals (China) Co., Ltd., which is located in Beijing, China. The Company has built a commercial team of more than 100 hematology oncology sales and marketing specialists based in China.

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PHARMACY MARKET

BioMatrix Specialty Pharmacy Granted Preferred Status with AscellaHealth for Home Infusion Therapies

BioMatrix Specialty Pharmacy | February 09, 2022

BioMatrix Specialty Pharmacy announced inclusion as a preferred provider in AscellaHealth's home infusion pharmacy network. AscellaHealth, a global specialty pharmacy and healthcare solutions company, has launched a site of care optimization strategy designed to cut costs for third-party payers without compromising quality of care. BioMatrix was included in the network to help the organization achieve its goals of improving drug affordability; increasing price transparency; reducing disparities in quality of care and safety; enabling care delivery in the highest-quality, lowest-cost care setting; and increasing patient convenience. Determining the optimal site of care for patients to receive therapy is an important component of treatment success. BioMatrix Specialty Pharmacy provides robust specialty infusion services, including site of care coordination. Whether administering in-home with assistance from one of our home infusion nurses, in-office, or at an ambulatory infusion center, our clinical team works with patients, payers, and prescribers to make therapy administration as safe and convenient as possible. Using a nationwide network of highly trained specialty infusion nurses, BioMatrix coordinates over 1,200 home nursing visits every month. "We are pleased to be included as a preferred provider in AscellaHealth's home infusion network. Our shared focus on positive outcomes for all patients, reduced cost, and price transparency will help AscellaHealth's members effectively manage their specialty infusion spend without compromising quality care." Ken Trader, BioMatrix VP of Managed Markets "By selecting top pharmacies for preferred status, we are taking an important step toward reducing the cost of specialty biologic medications and encouraging patients to move from higher-cost settings, like big hospitals and institutions, to lower-cost settings, such as physician's in-office infusion suites or stand-alone infusion centers," says Dea Belazi, president and CEO, AscellaHealth. "As part of our mission to help our clients better manage the high costs of specialty medication, this initiative exemplifies our efforts to increase care accessibility by encouraging the use of clinically appropriate, lower-cost care settings." About BioMatrix Specialty Pharmacy BioMatrix Specialty Pharmacy, an Inc. 5000 company, offers comprehensive, nationwide specialty pharmacy services and digital health technology solutions for patients with chronic, difficult to treat conditions. Our commitment to every patient is to provide individualized pharmacy services, timely access to care, and focused education and support. We offer a tailored approach for a wide range of therapeutic categories, improving health and empowering patients to experience a higher quality of life. About AscellaHealth LLC AscellaHealth is a global Specialty Pharmacy and Healthcare services organization serving patients, payers, life sciences and providers, offering a comprehensive portfolio of customized, tech-enabled specialty pharmaceutical and medical management services. An Inc. 5000 2021 winner, AscellaHealth's unique, patient-centric approach is built upon proprietary technology processes for innovative programs and services optimizing health outcomes and quality of life for patients with complex chronic conditions or rare diseases that require specialty medications and/or gene and cell therapies.

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PHARMACY MARKET

Virpax Pharmaceuticals Recaps Milestones and Highlights Product Portfolio

Virpax® Pharmaceuticals, Inc. | December 29, 2021

Virpax® Pharmaceuticals, Inc. "Company" achieved significant milestones relating to its pipeline of product candidates as of the end of 2021, which are summarized below. “I believe that Virpax has made significant progress this past year toward our stated goalsThese accomplishments include accelerated development of our existing product candidates, broadening our pipeline of product candidates, and utilizing grants where appropriate. We recently announced that we anticipate commencing our initial human trial for Epoladerm™ in the second quarter of 2022. Anthony P. Mack, Chairman and CEO of Virpax “Virpax initially focused strictly on pain product candidates. However, our unique Molecular Envelope Technology delivery platform licensed from Nanomerics, Ltd. has enabled the development of product candidates for central nervous system and anti-viral indications. In the second half of 2021, we advanced the development of AnQlar™, a prophylactic, over-the-counter anti-viral product candidate formulated to prevent the spread of respiratory infections like influenza, SARS-CoV-2 and rhinovirus. We added VRP324 which is an intranasally delivered cannabidiol formulation for the management of epilepsy in adults and children. Our Envelta™ IND-enabling studies, completed by the National Center for Advancing Translational Sciences as a part of our Cooperative Research and Development Agreement, determined that the MET intranasal delivery formulation bypasses the liver which we believe reduces drug-to-drug interaction concerns for treatments using this technology. “On the corporate front, we strengthened our Board by appointing two new members adding expertise in commercialization and financial strategy. Additionally, in 2021 we raised approximately $58 million in aggregate gross proceeds from our initial public offering and an underwritten follow-on public offering. These funds are being used for research and development activities of our product candidates. We are on track to add accomplishments in 2022 and I remain confident that we have the significant financial strength to continue advancing our pipeline,” concluded Mr. Mack. Epoladerm™ Epoladerm is a diclofenac topical spray film product candidate that is being developed for pain associated with osteoarthritis of the knee. Virpax recently reported successful results of a Charles River Laboratories single dose toxicology and pharmacokinetic study of dermal administration of Epoladerm in minipigs as part of the required Investigational New Drug Application enabling trials. Single-dose transdermal delivery of Epoladerm was well-tolerated in all minipigs. There were no treatment-related clinical observations, changes in body weight, or dermal irritation observed. The maximum plasma concentration was reached at 4 hours post-dose, and concentrations of plasma Epoladerm remained at 24-hour post-dose for all animals. Upon completion of all the required IND enabling studies and subsequent review by the FDA, Virpax intends to conduct a Phase 1 study to evaluate the relative bioavailability and pharmacokinetics of Epoladerm™. Virpax recently announced the execution of a clinical trial agreement with Altasciences Company, Inc., to conduct this study in Canada. Virpax anticipates enrollment of the first patient by early second quarter of 2022. Probudur™ Probudur is an injectable bupivacaine liposomal hydrogel for postoperative pain management which Virpax believes has improved onset and extended duration of action compared to existing treatment options. Additional pre-clinical trials are being conducted with Lipocure, the product developer, to improve the formulation to potentially enhance manufacturing efficiencies, prolong duration and extend patent protection. Once completed, we plan to perform seven preclinical animal studies as part of required FDA IND enabling trials. Envelta™ Envelta is an endogenous enkephalin intranasal spray for acute and chronic pain, including pain associated with cancer. This product leverages Nanomeric’s MET platform technology which Virpax licensed to deliver the endogenous enkephalin formulation through an intranasal delivery enabling the enkephalin to permeate the blood-brain barrier while bypassing the liver. This product candidate is being funded through an in-kind CRADA with the NCATS. Virpax recently announced that under this CRADA, the National Institutes of Health has awarded multiple contracts to support the research, development and manufacturing of Envelta. These contracts are to support Good Manufacturing Practices production of drug substance and drug product, as well as to support Good Laboratory Practices toxicology, safety studies and preclinical efficacy studies. The NIH has contracted with a clinical research organization to conduct additional pre-clinical efficacy studies and has procured a device to be used with the manufactured GMP drug product for preclinical and clinical studies. The NIH has also engaged a firm to manufacture Leu-enkephalin, the active ingredient in Envelta, and a company to manufacture the MET carrier that delivers L-ENK to the brain to promptly suppress pain. AnQlar™ AnQlar is a high-density intranasal molecular masking spray in development as an anti-viral OTC product for protection against respiratory infections, such as SARS-CoV-2 and influenza, that Virpax anticipates will be used as an adjuvant to barrier-based personal protective equipment. Virpax recently announced a manufacturing and supply agreement with Seqens to provide AnQlar for both clinical studies and the long-term commercial supply of AnQlar. Additionally, Virpax engaged Sinclair Research to initiate IND-enabling studies for AnQlar. The Company anticipates that these preclinical animal studies will begin in early 2022. VRP324 Virpax has acquired the exclusive worldwide rights from Nanomerics to use its MET platform for the nasal delivery of CBD for the management of epilepsy in children and adults. Under this agreement, Virpax has the global rights to develop, manufacture, market and sell VRP324, the first investigational formulation delivered via the nasal route to enhance CBD transport to the brain. This product candidate will be formulated to potentially treat seizures associated with tuberous sclerosis complex, Lennox-Gastaut syndrome and Dravet syndrome in patients one year of age and older. Lennox-Gastaut syndrome and Dravet syndrome are rare CNS diseases considered serious epileptic encephalopathies that cause epileptic seizures, as well as cognitive and behavioral changes, and are generally resistant to treatment. Preclinical studies of VRP324 have been initiated by Nanomerics which it anticipates will be completed in the first quarter of 2022. Upon completion, Virpax will collaborate with RRD International, a clinical drug development company which Virpax has engaged, to prepare the pre-IND briefing documents for the FDA. About Virpax Pharmaceuticals Virpax is developing branded product candidates for non-addictive pain management and neurological disorders using its proprietary technologies that optimize and target drug delivery. Virpax is initially seeking FDA approval of its three different patented drug delivery platforms. Epoladerm™ is a topical diclofenac spray film formulation being developed to manage pain associated with osteoarthritis of the knee. Probudur™ is a single injection long-acting liposomal bupivacaine formulation being developed to manage post-operative pain. Envelta™ is an intranasal Molecular Envelope Technology enkephalin formulation being developed for the management of acute and chronic pain, including pain associated with cancer, as well as post-traumatic stress disorder under the name PES200. MET technology is also used in AnQlar™, a candidate to inhibit viral replication caused by influenza or SARS-CoV-2. Virpax acquired global rights to VRP324, a product candidate for the nasal delivery of a pharmaceutical-grade cannabidiol for the management of epilepsy in children and adults.

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Spotlight

Bioassays are required for a variety of purposes in the development and production of biopharmaceuticals including drug candidate selection, product releases, product stability assessment, and comparability to support proposed process changes. However, because of their complexity and susceptibility to many variables, bioassays often prove problematic and difficult to develop. Timely development of suitable assay systems represents a major investment on the part of the biopharmaceutical industry but late development often results in even more costly clinical holds. Recent and ongoing advances in biology and technology provide the possibility of new types of assay systems and readouts. Some offer significant logistical and scientific advantages so that, in addition to being investigated for new products, they are increasingly being considered as replacements for some existing bioassays.

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