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.
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.
BioMarin Pharmaceutical Inc. | March 08, 2023
On March 7, 2023, BioMarin Pharmaceutical Inc., a global biotech company focused on improving lives via genetic discovery, announced that the US FDA has accepted its supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection to expand the treatment of achondroplasia in children under the age of 5.
Achondroplasia is a common form of disproportionate short stature. The FDA has set an action date of October 21, 2023, for the sNDA. The sNDA is supported by data from a Phase 2 double-blind, randomized, placebo-controlled clinical trial, which showed similar safety and efficacy profiles in children under five years of age compared to those aged five years and older.
The European Medicines Agency (EMA) also validated BioMarin's application for VOXZOGO in January to treat children under the age of 2. If approved, VOXZOGO could be prescribed for over 1,000 additional children from birth for achondroplasia.
VOXZOGO is the first EMA and FDA-approved treatment for achondroplasia with open bone growth plates. The treatment uses a new class of therapy, C-type natriuretic peptide (CNP) analog, which promotes bone growth by acting as a positive regulator of the signaling pathway downstream of the fibroblast growth factor receptor 3 gene (FGFR3).
The drug is approved in the EU, Brazil, and Australia for children aged two years and above with epiphyses (bone growth plates) and in Japan for children from birth who have achondroplasia with open growth plates. BioMarin has enrolled 250 children with achondroplasia in seven clinical studies in eight countries to evaluate the safety and efficacy of VOXZOGO.
About BioMarin Pharmaceutical Inc.
BioMarin is a leading biotechnology firm established in 1997 with a mission to transform people's lives by using genetic discovery. The company creates targeted therapies that address the underlying cause of genetic disorders to improve the quality of life for individuals with rare genetic diseases. It has developed eight first or best-in-class treatments and has a range of product candidates to address various genetic disorders using the same science-based approach. As BioMarin continues to create new and innovative solutions, it has the potential to impact the lives of even more people. The company is committed to prioritizing patients and their needs in its approach to drug development.