Premier Inc. Supports FDA, DEA Reforms and Regulatory Waivers to Counter Drug Shortages

Premier Inc. | July 24, 2020

Premier Inc. today provided the U.S. Food and Drug Administration (FDA) and the U.S. Drug Enforcement Administration (DEA) with a list of reforms and regulatory waivers that the company believes should be extended beyond the COVID-19 pandemic and made permanent thereafter in order to prevent drug shortages.

“During the height of the COVID-19 pandemic, we saw demand for many common pharmaceuticals surge by an average of more than 150 percent,” said Blair Childs, Premier’s Senior Vice President of Public Affairs. “Waivers from the FDA and DEA cleared away cumbersome barriers and helped providers manage surges in demand to avoid drug shortages. Lessons learned during the pandemic response demonstrate that these flexibilities could be implemented safely and were effective in mitigating drug shortages that otherwise could have been detrimental to patient care. The Administration should act on all these recommendations, easing regulatory burden and improving options for patients.”

Specifically, Premier recommends to the FDA that 503B compounding facilities be allowed to continue producing specific drugs that are not on the drug shortage list based on specific criteria, such as a short-term or regional shortages or demand surges for certain dosage strengths and/or packaging sizes. This waiver proved particularly helpful during the pandemic, as it allowed 503B compounders to quickly and seamlessly fill capacity gaps and alleviate spot shortages before they became severe enough to spread nationwide and make it onto the FDA drug shortage list.

Spotlight

Sales of pharmaceuticals in China are already the third highest in the world, and China is likely to become the world’s second-largest drug market within the next year. Multinational pharmaceutical corporations cannot afford to underperform in China if they want to achieve global prominence.

Spotlight

Sales of pharmaceuticals in China are already the third highest in the world, and China is likely to become the world’s second-largest drug market within the next year. Multinational pharmaceutical corporations cannot afford to underperform in China if they want to achieve global prominence.

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BUSINESS INSIGHTS

Astellas and MBC BioLabs Announce Astellas Future Innovator Prize to Help Biotech Start-ups Accelerate Early Drug Discovery and Research Efforts

Astellas Pharma Inc. | September 20, 2022

Astellas Venture Management LLC a wholly-owned venture capital subsidiary of Astellas Pharma Inc. and Mission Bay Capital BioLabs a life-science incubator, today announced their collaboration on the "Astellas Future Innovator Prize" formerly known as the Astellas Golden Ticket competition. Building on over three successful years, the competition offers entrepreneurial scientists or emerging biotechnology start-ups one year's priority usage of MBC BioLabs' state-of-the-art lab facility and access to Astellas' research and development (R&D) capabilities and business leaders. With a shared commitment to discovering and advancing innovative science for the potential future benefit of patients worldwide, AVM and MBC BioLabs are continuing their partnership to support scientists and early-stage companies to accelerate their novel therapeutic programs, modalities or platforms. The 2022 competition will prioritize innovative research that compliment Astellas' Focus Area Approach and pipeline. "We are truly thrilled to host the Astellas Future Innovator Prize again this year. Through our long-term collaboration with MBC BioLabs, we aim to create an environment that encourages innovation to thrive. The Astellas Future Innovator Prize enables us to provide biotech companies with expertise and capabilities to turn an innovative idea into a real solution, which could transform the lives of patients in need around the world." William Watt, President, AVM "We are pleased to continue our partnership with Astellas to uncover the innovators of tomorrow," said Douglas Crawford, MBC BioLabs General Manager. "The past winners of the Astellas Future Innovator Prize show what can be achieved by combining Astellas' support, advice and expertise with our laboratory incubator, and we are excited to see the result of our ongoing collaboration." Entrepreneurial scientists, emerging life-science and biotechnology start-ups have until November 4, 2022 to enter the Astellas Future Innovator Prize. About the Astellas Future Innovator Prize at MBC BioLabs Astellas is offering up to two prizes for pioneering scientists with innovative research that complements Astellas' areas of interest in alignment with its Focus Area Approach and pipeline, including Blindness & Regeneration, Mitochondria, Genetic Regulation, Immuno-Oncology, Cell Therapy, and other areas. The 2021 Astellas Future Innovator Prize winners were Vcreate and Weatherwax Biotechnologies, chosen for the potential of their innovations to deliver therapeutic advances for unmet medical needs and their potential synergy with Astellas' Focus Area Approach. About Astellas Venture Management LLC AVM is the wholly-owned venture capital organization within Astellas, dedicated to supporting pre-clinical, cutting-edge science that can bring VALUE to patients. For over 15 years, AVM has provided equity investments to private, early-stage companies developing therapeutic programs and platform technologies, helping them to advance their innovations faster. AVM is a strategic investor, making investments in science that will enhance the current Astellas R&D pipeline or that could catalyze new directions in discovery research. About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. About MBC BioLabs MBC BioLabs is dedicated to helping life-science startups succeed. By renting space as small as a single bench and providing these entrepreneurial scientists with access to millions of dollars of equipment, MBC BioLabs allows companies to be fast, focused, and frugal. It has three sites: one in the Dogpatch neighborhood in San Francisco and two campuses in San Carlos, California. Each site has a complete molecular biology core facility that allows companies to do experiments on day one. MBC BioLabs has partnerships with leading pharmaceutical and life-science companies as well as a built-in venture capital firm, Mission BioCapital. These partnerships provide entrepreneurs with valuable insights about where to focus their efforts and accelerates the innovation pipeline. Since opening in 2013, MBC BioLabs has helped launch and grow 290 companies. These companies have brought 58 programs to the clinic, produced 13 approved diagnostics, and raised over $14 billion.

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PHARMACY MARKET

TC BioPharm Begins Dosing Phase 2B Clinical Study Evaluating its Lead Compound, OmnImmune®, in Patients with Acute Myeloid Leukemia

TC BioPharm | November 23, 2022

TC Biopharm a clinical stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer today announced the dosing of its first three patients within its Phase 2b clinical trial of OmnImmune®, an allogeneic unmodified cell therapy focused on treating Acute Myeloid Leukemia. The initial 5 patients in the trial are deemed a "safety cohort", spaced two weeks apart with safety review by an oversight board to confirm no drug related toxicity issues, subsequent to 5 patients being dosed the study will advance to open enrollment. This safety cohort is in line with TCBP's step-wise clinical trial advancement, moving from donor matching in the Phase 1b to a universal donor model with no HLA matching of donor to patient. "The launch of our Phase 2B trial is a key milestone in the development of our lead therapeutic, OmnImmune®, for patients with AML and for TC BioPharm's emerging pipeline of 'off-the-shelf' gamma-delta T cell therapies. This study design includes a 5 patient safety cohort prior to open enrollment, we expect to complete the safety cohort before the end of 2022. The next step in the study is a 19 patient interim review, which will allow TCBP to review dosing and increase dosing to a higher level should our team deem it necessary for efficacy, or we can elect to maintain our current dosing level of 7x10^7 or 700 million cells per dose. We look forward to moving ahead with our Phase 2b trial with a target for open enrollment in January 2023, as well as our efforts to expand our clinical efforts in the US in the first half of 2023." Bryan Kobel, CEO of TC BioPharm TC BioPharm's Phase 2B trial, dubbed ACHIEVE, will enroll adults diagnosed with AML who have either relapsed or are refractory to prior treatments as well as a cohort for patients with myelodysplastic syndromes conditions that can occur when the blood-forming cells in the bone marrow become abnormal. The trial is expected to enroll approximately 37 patients. About TC BioPharm PLC TC BioPharm is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of gamma-delta T cell therapies for the treatment of cancer with human efficacy data in acute myeloid leukemia. Gamma-delta T cells are naturally occurring immune cells that embody properties of both the innate and adaptive immune systems and can intrinsically differentiate between healthy and diseased tissue. TC BioPharm uses an allogeneic approach in both unmodified and CAR modified gamma-delta T cells to effectively identify, target and eradicate both liquid and solid tumors in cancer. TC BioPharm is the leader in developing gamma-delta T cell therapies, and the first company to conduct phase II/pivotal clinical studies in oncology. The Company is conducting two investigator-initiated clinical trials for its unmodified gamma-delta T cell product line - Phase 2b/3 pivotal trial for OmnImmune® in treatment of acute myeloid leukemia using the Company's proprietary allogenic CryoTC technology to provide frozen product to clinics worldwide. TC BioPharm also maintains a robust pipeline for future indications in solid tumors as well as a significant IP/patent portfolio in the use of CARs with gamma-delta T cells and owns our manufacturing facility to maintain cost and product quality controls.

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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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