Promising early data for MSDs investigational HIV drug

Pharmatimes | July 24, 2019

Promising early data for MSDs investigational HIV drug
MSD has announced results from a Phase I study evaluating the pharmacokinetics and safety of a prototype subdermal drug-eluting implant for islatravir, an antiretroviral therapeutic for the prevention and treatment of HIV. The investigational drug is a nucleoside reverse transcriptase translocation inhibitor (NRTTI), and MSD is currently conducting a broad development program to evaluate the potential of the therapy for the treatment and prevention of HIV-1 infection. The Phase I double-blind, placebo-controlled study found that the implant containing 62mg of the drug was estimated to deliver levels well above the threshold at 12 months, providing early evidence for its potential as a once-yearly option for PrEP. The company is also exploring several potential options for PrEP, including a drug-eluting implant and a once-monthly oral formulation. The company is “encouraged by the results of this proof of concept study exploring the potential of delivering meaningful doses of islatravir over a 12-week period,” said Dr Roy Baynes, senior vice president and head of global clinical development, chief medical officer, MSD Research Laboratories. He continued, “At MSD, we recognise multiple options are needed to address the needs of individuals at risk of HIV-1, and we are committed to investigating those options. Through the application of our established expertise in drug delivery, we are seeking to capture the potential of islatravir for HIV-1 pre-exposure prophylaxis (PrEP).”

Spotlight

Global sales of oncology medicines were widely reported to have exceeded $100 billion for the first time in 2014, with the U.S. and European markets generating the majority of sales. This trajectory is expected to continue: Decision Resources Group (DRG) analysis suggests sales of oncology medicines will exceed sales of medicines in every other therapeutic area, growing at a nearly double-digit compound annual growth rate through 2021 to reach $170 billion (Figure 1). This surge will be driven by the approval and uptake of targeted small molecules and biologic therapies, reflecting the industry’s shift towards personalized treatments and a wide array of molecular and immunological targets.


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BUSINESS INSIGHTS

Pack4U acquires leading pharmacy technology company Catalyst Healthcare

Pack4U | January 07, 2022

Pack4U, a connected health services company, acquires long-time technology partner Catalyst Healthcare whose patented AdhereNet® platform links pharmacists, patients, and care teams to effectively manage drug complexity and improve medication adherence. "We are dedicated to improving patient outcomes at scale. Bringing this domain knowledge in-house is vital for Pack4U. Catalyst's CEO Shane Bishop is a pharmacy thought leader and we are excited about building the next phase of Pack4U with the Catalyst team on board. Catalyst's proprietary and patented platform strengthens our ability to meet the needs of patients, providers and health plans by offering a full, end-to-end, connected care solution seamlessly integrated with our vast clinical team across all states." Pack4U President, Rahul Chopra Pack4U provides in-home and virtual services that connect patients with pharmacists for oversight, medication delivery, remote monitoring, and care coordination. Our complete suite of connected health IP fortifies Pack4U's ability to personalize care, improve health outcomes, and lower total cost of care across North America. "I am extremely excited to see these organizations come together and to join the team that is transforming in-home healthcare," said Shane Bishop. "Pack4U's extensive and diverse team of pharmacists combined with Catalyst's adherence solutions, create opportunity for a new patient monitoring paradigm. With central fills for scale and community pharmacy adoption, we will reach more people to change more lives." "This collaboration follows our aggressive growth in 2021. It expands our vision of in-home care via a full suite of managed services featuring patient specific, in-home medicine dispensing, always-on patient engagement, electronic medicine administration record and supply chain software for centralized filling," said Rahul Chopra. About Pack4U Pack4U Inc is a tech-enabled health services company. We connect people with community pharmacists to manage drug complexity and maximize health benefits through personalized medication delivery, virtual monitoring, and proactive care. Our fully automated, high-volume central fill pharmacy hubs, proprietary health platform and medication adherence network power a robust, scalable solution to chronic condition management that leads to better patient outcomes and total cost of care reduction.

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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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PHARMA TECH

n-Lorem Foundation Partners with Alnylam to Expand Technology Access to RNAi Therapeutics for Patients with Nano-rare Diseases

n-Lorem a nonprofit Foundation | January 20, 2022

n-Lorem, a nonprofit Foundation, discovering and providing experimental antisense oligonucleotide medicines to nano-rare disease patients for free, for life, today announced a new partnership with Alnylam Pharmaceuticals Inc. “Discovering, developing and providing individualized medicines for nano-rare patients is a monumental task. At n-Lorem, using ASO technology, we take advantage of three decades of experience, unparalleled quality control and integrated safety databases that are used to select frequency and dosage for each individual ASO therapy. As we expand our highly selective and versatile model to other amendable technologies, it is important for us to maintain a high level of quality,” said Stanley T. Crooke, M.D., Ph.D., Founder, Chief Executive Officer and Chairman, n-Lorem Foundation. “Alnylam’s RNAi therapeutic technology is the perfect complement to Ionis’ ASO technology, both are well understood, proven drug discovery technologies. Adding Alnylam as a partner is another step in the creation of a broad network of partner stakeholders committed to treating nano-rare patients.” “We are pleased to partner with the n-Lorem Foundation in identifying opportunities to bring our novel RNAi technology platform to potentially help patients with nano-rare diseases. At Alnylam, we are committed to identifying pathways to expand access to our medicines and we applaud the work of the foundation in bringing together industry partners who are driven to provide options for patients that have little or no options for treatment,” Akshay Vaishnaw, M.D., Ph.D., President at Alnylam “Although we have the tools and expertise to understand nano-rare patients at a genetic level, the extreme rarity of these patients present insurmountable challenges to the current healthcare systems,” said Dr. Sarah Glass, Chief Development Officer, n-Lorem Foundation. “n-Lorem is addressing these challenges one nano-rare patient at a time and is supported through a consortium of well-renowned and diverse organizations worldwide. These include our biotechnology and pharmaceutical partners, including Ionis Pharmaceuticals, Biogen, Ultragenyx and today Alnylam; our clinical research partners, including Charles River Labs, Korea Institute of Toxicology and Parexel; our contract manufacturing partners, including ChemGenes, Cytiva and Nitto Avecia; and many other collaborations that make n-Lorem’s mission possible.” Alnylam is leading 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 genetic, cardio-metabolic, hepatic infectious, and central nervous system ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam’s commercial RNAi therapeutic products are ONPATTRO®, GIVLAARI® (givosiran), and OXLUMO®, as well as Leqvio®, which is 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. About n-Lorem n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 100 applications for treatment with more than 40 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics.

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BUSINESS INSIGHTS

Treatment to Expand Global Library of Medicine with Dermatologic Diseases

Treatment.com International Inc. | January 05, 2022

Treatment.com International Inc. a healthcare AI technology company, has entered into a research project agreement with the University of Minnesota to add more than 220 dermatologic diseases to their proprietary, responsive AI engine known as the Global Library of Medicine (GLM). The addition of these diagnoses will bring another level of accuracy and specificity to the GLM, enhancing soon-to-be-released products such as the Treatment Mobile app, which is set for release this month. Kevin A. Peterson, MD, MPH, FRCS(Ed), FAAFP and Chief Medical Officer of Treatment, says, “Engaging in research with the University of Minnesota Dermatology department brings extensive academic medical expertise that enhances our ability to provide richly detailed descriptions of dermatologic concerns. The rich experience of the University of Minnesota Medical School will help the GLM provide more accurate descriptions of complex presentations of skin diseases. Combining our advanced AI infrastructure with the rich clinical and research expertise of the Medical School will substantially enhance our AI platform’s ability to provide better access to high quality information and directly supports the mission of the GLM to enhance health outcomes for individuals around the world.” “We are proud that the University of Minnesota is participating with us on this latest research project, which is in addition to other mutually beneficial agreements we’ve entered into with the University. The Global Library of Medicine is one way we are realizing Treatment’s plan to provide the most accurate and reliable data about health concerns, made available to clinicians, organizations, and patients alike.” John Fraser, Treatment’s CEO The Global Library of Medicine is the foundation of Treatment’s Clinical solutions, which offer unique ways to bring healthcare into the digital age by providing better access to up-to-the-minute healthcare information and promoting better health outcomes around the world. Treatment Mobile and the intelligent digital assistant will be released in select North American clinics in 2022. About Treatment.com Treatment.com is a disruptive healthcare technology company that is harnessing the power of AI to help global citizens improve their health through personalized recommendations and insights. Based in Vancouver with a US subsidiary, the company spent the last five years working with a team of world-class doctors, engineers, mathematicians, and AI specialists to develop a complex AI engine that leverages the most robust, personalized data to generate highly predictive and accurate insights. Treatment.com is the parent company of Treatment Mobile, and an intelligent digital health assistant which will empower people to take control of their health through an innovative mobile app, powered by Treatment’s exclusive AI engine.

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Spotlight

Global sales of oncology medicines were widely reported to have exceeded $100 billion for the first time in 2014, with the U.S. and European markets generating the majority of sales. This trajectory is expected to continue: Decision Resources Group (DRG) analysis suggests sales of oncology medicines will exceed sales of medicines in every other therapeutic area, growing at a nearly double-digit compound annual growth rate through 2021 to reach $170 billion (Figure 1). This surge will be driven by the approval and uptake of targeted small molecules and biologic therapies, reflecting the industry’s shift towards personalized treatments and a wide array of molecular and immunological targets.

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