Genetown Hotbed: Churning Along During the COVID-19 Pandemic
BioSpace’s Genetown Hotbed is at the very heart of the biopharma industry in the United States, largely in the Boston area. As the biopharma industry responds to the COVID-19 pandemic, some of the area's companies are at the very frontlines of drug development against the disease, while others play supporting roles for the entire industry. Others are continuing to work on developing cutting-edge therapies for a wide range of diseases while ensuring current products continue to be manufactured to meet patients’ needs. Here's a look.
Sarepta Therapeutics. Based in Cambridge, Massachusetts, has two products on the market, Exondys 51 and Vyondys 53, both for different forms of Duchenne muscular dystrophy (DMD). At this time, the company has several ongoing clinical trials for DMD and other rare diseases, such as Limb-Girdle Muscular Dystrophies (NGMD) and Charcote-Marie-Tooth (CMT) disease. The company is closely monitoring the COVID-19 pandemic and its potential effects on clinical trials and the company, with a focus on critical infrastructure and the integrity of its supply chain for its commercial products.
AbbVie. Although its corporate headquarters are in Chicago, AbbVie has a major presence in Cambridge, Massachusetts. On March 9, AbbVie announced it was supporting the COVID-19 global activities, including the experimental use of its Kaletra/Aluvia (lopinavir/ritonavir) for COVID-19. As such, it is working with the U.S. Food and Drug Administration (FDA), Centers for Disease Control and Prevention (CDC), National Institutes of Health, Biomedical Advanced Research and Development Authority (BARDA), as well as its industry partners and European health authorities on these efforts.
Amgen. With headquarters in Thousand Oaks, California, Amgen also has significant activities in Cambridge, Massachusetts. On March 23, Amgen and the Amgen Foundation announced an initial commitment of up to $12.5 million in support of U.S. and global relief efforts for the pandemic. The monies will be used to support emergency response efforts, patient-focused organizations, and international relief efforts by Direct Relief and International Medical Corps.
Bristol Myers Squibb. Headquartered in New York, with offices in Cambridge, Bristol Myers Squibb and the Bristol Myers Squibb Foundation have donated more than $5 million in financial support and protected equipment to relief efforts in the U.S., Wuhan city and Hubei province (China) and the Lombardy region of Italy. On March 23, the company indicated it would delay beginning new clinical trials for at least three weeks, until April 13, and that all studies involving healthy volunteers would be paused if they were at a natural stopping point.
Black Diamond Therapeutics. Based in Cambridge and in New York, Black Diamond Therapeutics focuses on precision oncology. On March 24, the company announced it had initiated a Phase I/II clinical trial of BDTX-189 in solid tumors. And in February 2020, the company completed an initial public offering (IPO) raising $231.3 million.
Cygnal Therapeutics. Founded in 2017 by Flagship Pioneering, Cygnal Therapeutics came out of stealth mode in October 2019 with a $65 million financing round. The company focuses on the field of exoneural biology. Using its Exoneural Medicine Platform, it is working on developing drugs starting with cancer and inflammatory diseases.
Dicerna Pharmaceuticals. Located in Lexington, Massachusetts, Dicerna Pharmaceuticals focuses on RNA interference (RNAi) therapeutics. On March 18, the FDA granted its DCR-A1AT orphan drug designation for the treatment of alpha-1 antitrypsin (A1AT) deficiency. This is a genetic disorder that can result in lung and liver disease.
Intellia Therapeutics. Headquartered in Cambridge, Intellia focuses on using CRISPR-cas9 gene editing to develop therapies for various diseases, including transthyretin amyloidosis, hereditary angioedema (HAE), sickle cell disease, acute myeloid leukemia and solid tumors. At its full-year and fourth quarter report on February 27, the company indicated it had selected NTLA-5001 for its AML program and planned to pick a third development candidate in the first half of the year for HAE. It also expected in the second half of the year to begin dosing ATTR patients with NTLA-2001.
Ipsen Biopharmaceuticals. An affiliate of France’s Ipsen with offices in Cambridge, the company presented results of the PRESTO study on March 11 by the European Neuroendocrine Tumor Society (ENETS) and published the results in Advances in Therapy. PRESTO was a multi-national, multi-center study that enrolled 90 nurses with more than two years of experience injecting with the Somatuline Depot (lanreotide) syringe and/or the syringe for long-acting release octreotide. The objective was to assess nurse preference.
The company has also announced its efforts to ensure business continuity at its manufacturing sites, which are continuing to operate.
Lantheus Medical Imaging. Lantheus is based in North Billercia, Massachusetts. The company develops, manufactures and commercializes essential diagnostic imaging agents and products. On February 25, the company released its annual and fourth-quarter financial results. The company had a decent year with global revenue of $89.3 million for the quarter and $347.3 million for the full year, representing an increase of 3.6% and 1.2% over the previous year, respectively.
New England BioLabs. Located in Ipswich, Massachusetts, the company is a leader in the discovery and production of enzymes for molecular biology applications. As such, the company’s technology and reagents are critical to laboratory testing and research into the COVID-19 coronavirus. The company noted, “Despite a ‘Stay At Home’ advisory being put in place in Massachusetts, USA, we are deemed an essential business, and our manufacturing and distribution teams continue to be fully operational. We are continuing to work with our suppliers and distribution partners to ensure uninterrupted access to our products and technical support.”
Nitto Avecia. Located in Milford and Marlborough, Massachusetts, Nitto Avecia offers a variety of services including process and analytical development, preclinical and CGMP oligo manufacture, and quality control testing, including on-site ICH stability storage and testing. In response to the pandemic, the company announced, “We want to assure you that all of our sites in the USA are open, and we’re committed to continuing to support your important work. We have business continuity plans in place to allow us to operate with minimal disruption.”
Ovid Therapeutics. Based in New York and Cambridge, Ovid focuses on rare neurological disorders. Its lead program, OV101 (gaboxadol) is in clinical development for Angelman syndrome and Fragile X syndrome. It is also developing OV935 (soticlestat) in collaboration with Takeda Pharmaceutical Company for rare developmental and epileptic encephalopathies (DEE). The company expects initial data form the ongoing open-label Phase II ARCADE study in CDKL5 deficiency disorder and Dup15q syndrome by the end of the first quarter of 2020. It also expects data from its signal-finding Phase II ROCKET trial in the second quarter for Fragile X syndrome.
Rentschler Biopharma. Germany-based Rentschler Biopharma has a facility in Milford, Massachusetts. The company is a contract development and manufacturing organization (CDMO). The site in Milford is relatively new and supports both clinical and commercial manufacturing programs. It is being qualifying as a multi-product manufacturing facility.
In September 2019, Ralf Otto, chief operating officer of Rentschler Biopharma and president and chief executive officer of Rentschler Biopharma, said that, “The Milford site isn’t an extended workbench of the German site. Instead, it’s both a CDMO and an innovation center.”