MHRA gives Bavencio, Inlyta combo Early Access to Medicines status

Pharmatimes | July 22, 2019

Merck and Pfizer have announced that the UK Medicines and Healthcare Products Regulatory Agency MHRA has issued an Early Access to Medicines Scheme EAMS positive scientific opinion for Bavencio avelumab used in combination with Inlyta axitinib. The opinion is for the combination as first-line treatment of adult patients with advanced renal cell carcinoma (RCC), and is based on information relating to the benefit and risks of the medicines and enables clinicians to prescribe the unlicensed treatment under their own responsibility. The combination was approved by the FDA in May this year based on positive results from the Phase III JAVELIN Renal 101 study (NCT02684006), in which the combination significantly improved median progression-free survival (PFS) compared with sunitinib by more than five months. The study included patients regardless of PD-L1 expression and across IMDC (International Metastatic Renal Cell Carcinoma Database) prognostic risk groups. There is a significant unmet need for RCC first-line treatments that delay progression and have an acceptable safety profile, as approximately 20% to 30% of patients are first diagnosed with RCC at the advanced stage, and 30% of patients treated for an earlier stage go on to develop metastases.

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Public and private health plans, managed Medicaid fee-for-service (FFS) states, and Medicare FFS voluntarily participated in a national effort to measure the use of Alternative Payment Models (APMs) as well as progress toward the Health Care Payment Learning & Action Network’s (LAN) goal of tying 30 percent of U.S. healthcare payments to APMs by 2016 and 50 percent by 2018, according to a new infographic by the Health Care Payment LAN.


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BUSINESS INSIGHTS

Alnylam Announces 3-Month Extension of Review Period for New Drug Application for Vutrisiran

Alnylam Pharmaceuticals, Inc. | April 05, 2022

Alnylam Pharmaceuticals, Inc. the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration has extended the review timeline of the New Drug Application for vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated amyloidosis, to allow for the review of newly added information related to the new secondary packaging and labeling facility. Alnylam recently learned that the original third-party secondary packaging and labeling facility the Company planned to use for the vutrisiran launch was recently inspected and the inspection requires classification for the FDA to take action on the vutrisiran NDA. The inspection observations were not directly related to vutrisiran. In order to minimize delays to approval, Alnylam has identified a new facility to pack and label vutrisiran and submitted an amendment to the NDA for review by the FDA. The updated Prescription Drug User Fee Act goal date to allow for this review is July 14, 2022. No additional clinical data have been requested by the FDA. “We are committed to working with the FDA and the new facility to bring this important treatment option as quickly as possible to patients living with the polyneuropathy of hATTR amyloidosis, an inherited, progressively debilitating, and fatal disease. We are confident in our regulatory application and the body of data supporting the efficacy and safety of vutrisiran.” Pushkal Garg, M.D., Chief Medical Officer & EVP, Development and Medical Affairs Vutrisiran is also under review by the European Medicines Agency the Brazilian Health Regulatory Agency and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). We aim to minimize the impact of this issue on these reviews through timely resolution of the matter or utilization of an alternative facility for one or more of these regions. Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis, and in Japan for transthyretin type familial amyloidosis with polyneuropathy. About Vutrisiran Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hATTR and wild-type ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of wild-type and variant transthyretin protein before it is made. Quarterly, and potentially biannual, administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s Enhanced Stabilization ChemistryGalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections. 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® (givosiran), OXLUMO® 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 benefitingpatients 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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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.

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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

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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Public and private health plans, managed Medicaid fee-for-service (FFS) states, and Medicare FFS voluntarily participated in a national effort to measure the use of Alternative Payment Models (APMs) as well as progress toward the Health Care Payment Learning & Action Network’s (LAN) goal of tying 30 percent of U.S. healthcare payments to APMs by 2016 and 50 percent by 2018, according to a new infographic by the Health Care Payment LAN.

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