Rhythm Pharmaceuticals Announces Successful Phase 3 Trial Results for Obesity In November 2020, when the U.S. FDA granted approval to the Rhythm Pharmaceuticals (RYTM) product IMCIVREE™ (setmelanotide) for obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/Kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency, the firm has since announced positive results from a pivotal . . . This content is for paid subscribers. Please click here …

Inovio Phase 1 Data of INO-4800 for COVID-19 Peer-reviewed Phase 1 data shows Inovio COVID-19 DNA Vaccine Candidate, INO-4800, to be immunogenic in 100% of subjects, inducing neutralizing antibody and/or T cell responses. INO-4800 demonstrates favorable safety and tolerability. No serious adverse events reported. Offers best-in-class thermostability, including a five-year projected shelf life at normal refrigeration temperature and no frozen transport or storage requirements. INOVIO …

Regenxbio Regenxbio (RGNX) announced that it is selling a portion of its royalty rights from the net sales of Zolgensma® by Novartis to entities managed by Healthcare Royalty Management, LLC (HCR) for $200 million. This transaction provides immediate, non-dilutive capital to Regenxbio in order continue the development of its gene therapy pipeline products and complete its internal manufacturing capabilities. Morgan Stanley & Co. LLC served …

Compugen Exclusive License Agreement with AstraZeneca In April 2018 Compugen (CGEN) and AstraZeneca (AZN) entered into . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Gilead Sciences Company, Kite Pharma, News Kite Pharma, a Gilead Company (GILD), announced that the European Commission has granted conditional marketing authorization for Tecartus™ (autologous, anti-CD19-transduced CD3+ cells; formerly KTE-X19). Tecartus is a chimeric antigen receptor (CAR) T cell therapy for adult patients with relapsed or refractory mantle cell lymphoma after two or more lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor. Conditional …

Intellia Therapeutics in the News Intellia Therapeutics (NTLA) presented the first demonstration of physiological protein levels of human alpha-1 antitrypsin (AAT) in non-human primates (NHPs) following a single administration.  Intellia Therapeutics Liver Gene Insertion Technology Intellia’s targeted liver gene insertion technology has the ability to achieve therapeutic levels of protein . . . This content is for paid subscribers. Please click here to subscribe or here to …

Acquisitions  AstraZeneca Acquires Alexion Pharmaceuticals AstraZeneca (AZN) and Alexion Pharmaceuticals (ALXN) entered into a definitive agreement for . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

CRISPR, Intellia and Editas, Our Gene-Editing Sisters, are Rallying The three CRISPR gene-editing firms that we have chosen are enjoying rallies. CRISPR Therapeutics (CRSP) and Intellia Therapeutics (

Prohost Letter #443 The Biotechnology Industry The Biotechnology Industry revolutionized the arts of life sciences with accomplishments promising a tremendous improvement in the treatment of chronic, debilitating, progressive and life-threatening diseases. The improved approaches have been game changers in the treatment of cancers and genetic-derived hereditary and non-hereditary chronic diseases. The out-performance was made possible through the biotech industry’s creativity, innovation and remarkable productivity. The …

Reata Pharmaceuticals: Why its Stock Price is Down Reata Pharmaceuticals (RETA) announced that it has priced a public offering of 2,000,000 shares of its Class A common stock at a price to the public of $140.85 per share for gross proceeds of $281.7 million. Reata has granted the underwriters a 30-day option to purchase 300,000 additional . . . This content is for paid subscribers. …

Intellia Therapeutics Intellia Therapeutics (NTLA) stock price headed south probably because some robots learned that the firm commenced an underwritten public offering of $150 million of shares of its common stock. In addition to the commenced public offering Intellia announced its intention to grant the underwriters a 30-day option to purchase up to an additional fifteen percent (15%) of the shares . . . This content is …

Regenxbio Dosed First Patient with RGX-111 for MPS I Regenxbio (RGNX) has dosed the first patient with RGX-111 in the Phase I/II trial for the treatment of Mucopolysaccharidosis Type I (MPS I). About Regenxbio Product RGX-111 RGX-111 is an investigational one-time gene therapy designed to deliver the α-l-iduronidase (IDUA) gene directly to the central nervous system (CNS) using the NAV AAV9 vector. RGX-111 is a …

Rhythm Pharmaceuticals FDA Approval of IMCIVREE™ for Genetic Obesity At the end of the trading week last Friday, November 27, 2020, the United States FDA approved Rhythm Pharmaceuticals’ (RYTM) product IMCIVREE™ (setmelanotide). The product is designed to treat chronic obese adult patients and pediatric patients 6 years and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/Kexin type . . . This content is …