BUSINESS INSIGHTS, PHARMA TECH
Sumitovant Biopharma, Inc. | January 20, 2023
On January 19, 2023, Neochromosome, Inc., announced a partnership with Sumitovant Biopharma, Inc., a technology-driven biopharmaceutical firm expediting the discovery and commercialization of novel therapeutics.
This collaboration blends Neochromosome's neoYeast™ platform with Sumitovant's DrugOME™ computational ecosystem to identify new proteins that would benefit from non-canonical amino acid chemistries that are unavailable in nature.
As part of the collaboration, Sumitovant used a combination of natural language processing (NLP), artificial intelligence (AI), machine learning (ML), and computational algorithms to identify opportunities for Neochromosome to enhance the discovery and production of proteins limited by evolution and traditional fabrication processes. For example, non-canonical amino acid inclusion allows new-to-nature proteins with improved structures and functions but often requires chemical synthesis, which is expensive, size- and complexity-restricted, and environmentally damaging.
neoYeast™, based on Neochromosome's world-leading genome-scale cell engineering technology stack, enables the highly efficient and environmentally friendly integration of non-canonical amino acids into proteins, with both discovery and manufacturing possible in a single genome-engineered yeast strain. Sumitovant used DrugOME to explore libraries of traditional and non-traditional sources of data to recommend targeted protein development candidates for neoYeast.
Co-Founder and CEO of Neochromosome, Leslie Mitchell, commented, "The combination of Sumitovant's technology platform and life-sciences expertise with Neochromosome's ability to both discover and biomanufacture novel proteins in a single system enables Neochromosome to accelerate and streamline the delivery of better therapies for patients." She added, "We are excited to partner with the world-class team at Sumitovant to identify opportunities to create better versions of existing drugs, along with new-to-nature compounds, and to manufacture them efficiently at scale."
(Source – Business Wire)
Sumitovant Biopharma, Inc.
The firm has supported the development of multiple FDA-approved products and a strong pipeline of early-through late-stage investigational assets addressing unmet needs in pediatrics, oncology, urology, women's health, specialty respiratory, and infectious diseases through its proprietary computing and data platforms, scientific expertise, and diverse company portfolio. It is a wholly-owned subsidiary of Sumitomo Pharma.
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.
BUSINESS INSIGHTS, PHARMACY MARKET
Accord Healthcare | March 14, 2023
On March 13, 2023, Accord Healthcare informed that it has expanded its portfolio with Teriflunomide, a film-coated tablet equivalent to Sanofi Genzyme's Aubagio. The drug treats relapsing forms of MS in adults, including clinically-isolated syndrome and active secondary progressive disease. The company offers Teriflunomide in 14 mg and 7 mg tablets, now available for shipping.
Teriflunomide has been granted FDA approval as an immunomodulatory agent for patients aged 18 and above. Its purpose is to reduce the frequency and severity of relapses, which can lead to new or worsening neurological symptoms associated with the disease.
Accord Healthcare US Vice President of Sales Jim Brown stated, "We're proud to add Teriflunomide to our portfolio. This is an important oral therapy for the millions of patients living with relapsing MS." He added, "Generic medicine allows broad access to effective therapy, to help improve disease course and potentially slow progression of MS."
(Source – Cision PR newswire)
Teriflunomide is an immunomodulatory medication that inhibits an enzyme critical to a metabolic pathway that supports lymphocyte proliferation. This results in fewer activated lymphocytes, which minimizes inflammation and immunological reactivity, resulting in lower recurrence rates and protection of brain volume against inflammatory nerve cell attacks.
Around 2.8 million people globally have multiple sclerosis, with the majority diagnosed with relapsing-remitting MS that typically begins in their twenties or thirties. Diagnosis involves experiencing two or more attacks or relapses while maintaining stability. New or exacerbated symptoms characterize relapses, and the immune system attacks the myelin sheath surrounding nerve fibers, ultimately causing long-term damage. Symptoms include numbness, fatigue, double vision, and balance problems. Besides, relapsing-remitting MS can also progress to active secondary progressive disease.
About Accord Healthcare
Accord Healthcare, a subsidiary of Intas Pharmaceuticals, is recognized as a prominent player in the generic pharmaceutical industry. The company's vision is to enhance patient access to medicines and revolutionize the pharmaceutical experience for healthcare providers and patients. With an emphasis on advanced manufacturing technology and in-house research, Accord Healthcare delivers a wide range of affordable, complex, and essential medicines. The company's dedication to forward-thinking and innovative solutions remains at the core of its mission. Headquartered in Durham (North Carolina), its products are marketed in 80 countries through the Intas network.