RESEARCH, PHARMA TECH
Asklepios BioPharmaceutical, Inc. (AskBio) | March 01, 2023
Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG, recently announced that the European Commission (EC) has granted orphan drug designation to AB-1003 (also known as LION-101) for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is an investigational recombinant adeno-associated virus (AAV) based gene therapy that is being developed as a one-time intravenous infusion for patients with LGMD type 2I/R9.
The disease affects 4.5 people per million worldwide, with more than 5,000 people affected in the EU and the US. The EC decision was received through AskBio's EU-based subsidiary BrainVectis, following a positive opinion from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP)
AB-1003 is currently being investigated in a Phase 1/2 multicenter study in the US to evaluate its tolerability, safety, and efficacy in adult subjects with genotypically confirmed LGMD2I/R9.
The EC for medicinal products grants orphan drug designation developed to treat a life-threatening disease that affects no more than five people in 10,000 in the EU, provided there is no other adequate treatment option or the medicine can bring significant value to those affected by a specific condition. This designation will provide special incentives in the EU, including eligibility for protocol assistance and possible reductions or exemptions in certain regulatory fees. Additionally, if the medicine is approved for marketing, this designation will provide ten years of marketing exclusivity.
Limb-girdle muscular dystrophy (LGMD) is a group of diseases that cause progressive weakening and wasting of the muscles in the arms and legs. The severity, features of LGMD and age of onset vary among the many subtypes of the condition and are often inconsistent, even within the same family. Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) is a form of LGMD caused by mutations in the FKRP gene. Signs and symptoms of LGMD2I/R9 often appear in late childhood and may include difficulty walking and running. The symptoms intensify with time, eventually leading to considerable disability, and afflicted persons typically require a wheelchair for movement roughly 23-26 years after beginning. Unfortunately, there is presently no treatment that slows the advancement of the disease, and management is based on the indications and symptoms that each individual exhibits.
About Asklepios BioPharmaceutical, Inc. (AskBio)
AskBio is a leading, fully integrated gene therapy company dedicated to developing innovative gene therapeutics and life-saving medicines. With operations worldwide, its pipeline includes gene therapy candidates for various diseases, including the central nervous system, neuromuscular, metabolic, and cardiovascular diseases. AskBio's global headquarters are located in RTP, NC, with European headquarters in Edinburgh. In addition, the company operates AAV research centers in Philadelphia, PA; Columbus, OH; and Paris, and modern clinical and commercial facilities in San Sebastian, Spain.
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BUSINESS INSIGHTS, PHARMACY MARKET
Businesswire | March 27, 2023
Massive Bio, Inc., a private AI-enabled oncology startup that provides virtual and in-person concierge services for cancer patients, and NeoGenomics, Inc. a leading provider of cancer-focused genetic testing services and global oncology contract research services, today announced a collaboration with the goal of accelerating the development of new cancer therapies and ultimately improving the lives of millions of cancer patients around the world. NeoGenomics will identify patients in real time who may be eligible for clinical trials based on biomarker status. Following initial contact and outreach provided directly from NeoGenomics to the treating physician, Massive Bio will help obtain patient consent and expediate additional screening and potential enrollment. This partnership will help to quickly identify patients eligible for clinical trials and help patients and providers make an informed decision regarding their potential treatment avenue.
By combining their respective strengths in biomarker testing, data analysis, machine learning, and biomarker and genomic profiling, the collaboration between NeoGenomics and Massive Bio have achieved a significant milestone in the oncology industry.
"Our mission at Massive Bio is to provide cancer patients with the best possible care and treatment options,” said Selin Kurnaz, PhD, CEO and co-founder of Massive Bio. “By partnering with NeoGenomics, a leading player in the cancer diagnostics industry that shares Massive Bio’s commitment to advancing cancer research and improving patient outcomes, we can leverage their expertise in oncology diagnostics to accelerate the identification of patients who may be eligible for clinical trials."
“NeoGenomics’ advanced diagnostic tools and U.S. footprint, combined with Massive Bio's AI capabilities and concierge services in oncology, will enable us to match patients to clinical trials faster and more efficiently, resulting in improved outcomes and reduced costs,” said Vishal Sikri, President of the Advanced Diagnostics Division of NeoGenomics. “We are thrilled to partner with Massive Bio to advance precision medicine and improve the delivery of healthcare services to patients, pharmaceutical partners, and healthcare providers."
Dr. Arturo Loaiza-Bonilla, MD, co-founder and Chief Medical Officer of Massive Bio, added, "This collaboration between NeoGenomics and Massive Bio will enable us to provide patients with personalized care and support throughout their cancer journey, and move the needle forward in precision oncology and research."
AboutMassive Bio
Massive Bio empowers cancer patients to find their best treatment options, using artificial intelligence to improve equitable access and precision targeting for clinical trial matching, drug matching, and drug development. Massive Bio combines its best-in-class AI platform with technology-enabled services to remove barriers in clinical trial enrollment, value-based oncology decisions, and data-driven cancer treatment. The company serves more than two dozen pharmaceutical companies, contract research organizations, and hospital networks, and has been awarded an SBIR contract by the National Cancer Institute. Massive Bio was founded in 2015 by clinical, technology, and M&A executives, and has a global presence with nearly 100 people in 12 countries.
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BUSINESS INSIGHTS, PHARMA TECH
Innate Pharma | January 17, 2023
On January 16, 2023, Innate Pharma SA announced the publication of preclinical findings demonstrating the control of acute myeloid leukemia (AML) cells by a trifunctional NKp46-CD16a-NK cell engager (NKCE) targeting CD123 in the scientific journal Nature Biotechnology. Innate and Sanofi conducted the experiments, which were published in Nature Biotechnology on January 12, 2023.
This study demonstrates that CD64 expression on AML blasts imparts resistance to anti-CD123 antibody-dependent cell cytotoxicity (ADCC) and that redirecting NK cells against cancer targets by binding to CD16a and NKp46 circumvents this resistance. Moreover, through their binding to NKp46, CD123-NKCE primarily target NK cells and have substantial antitumor activity against primary AML blasts; they stimulate NK cell activation and cytokine secretion exclusively in AML cells' presence.
In vivo, its antitumor activity observed in a mouse tumor model surpasses that of the anti-CD123 antibody used as the comparator. Furthermore, in vitro in human peripheral blood mononuclear cells and in vivo in nonhuman primates, the efficacy of CD123-NKCE was associated with the induction of low pro-inflammatory cytokine release and the absence of toxicity.
These results support CD123-NKCE clinical development. IPH6101/SAR'579 (SAR443579), the first NKp46/CD16-based CD123-targeted ANKETTM NK cell engager, is currently being evaluated in a Phase 1/2 clinical trial by Sanofi in patients with relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia (B-ALL), or high-risk myelodysplastic syndrome (HR-MDS).
About Innate Pharma
Headquartered in Marseille (France), Innate Pharma SA is a global, clinical-stage oncology-focused biotech firm dedicated to enhancing patient treatment and clinical outcomes using therapeutic antibodies that employ the immune system to combat cancer. Its extensive antibody pipeline has numerous potentially first-in-class clinical and preclinical candidates for cancers with a high unmet medical need. In addition, Innate has been a pioneer in the study of natural killer cell (NK cell) biology and developed its expertise in tumor antigens, tumor microenvironment, and antibody engineering. This innovative strategy has resulted in a varied proprietary portfolio and substantial alliances with biopharmaceutical industry leaders, including Sanofi, Bristol-Myers Squibb, Novo Nordisk A/S, and a multi-product collaboration with AstraZeneca.
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