Novartis AG is expanding its position in the optogenetics space with the acquisition of Vedere Bio Inc., bringing with it a program aimed at vision loss prevention and treatment. Vedere shareholders received $150 million up front and are eligible for up to $130 million in milestone payments, bringing the total to $280 million. The acquired cell and gene therapy technologies include light-sensing proteins for use in the retina and the delivery vectors to get them there. The optogenetics technology is based on work done by Cambridge, Mass.-based Vedere’s scientific founders at the University of California, Berkeley and technology for studying ocular gene therapy delivery developed at Berkeley and the University of Pennsylvania’s school of veterinary medicine.
Aurora phase II results mixed with avacopan in HS; Chemocentryx moving ahead
Chemocentryx Inc. disclosed long-awaited, COVID-19-delayed top-line data from the company’s phase II Aurora study with avacopan for hidradenitis suppurativa (HS). The first-in-class oral complement C5a receptor inhibitor turned up statistically significant, dose-dependent improvement on the HS Clinical Response scale vs. placebo in Hurley stage III (severe HS) patients at 12 weeks, yielding numerical improvement in the overall study population, but missed the primary endpoint: clinical response in all patients, moderate Hurley stage II as well as severe. Chemocentryx said the findings provide a path forward for avacopan in HS, also called acne inversa, a disabling autoimmune skin disease characterized by recurrent, painful nodules, boils and abscesses. As Wall Street sorted the data, company shares (NASDAQ:CCXI) were trading midday at $49, down $3.24.
Tricida shares docked as FDA seeks evidence of veverimer CKD impact
Shares of Tricida Inc. (NASDAQ:TCDA) fell 47% by midday after it said the FDA would require data on how veverimer, its investigational therapy for metabolic acidosis, impacts renal disease progression to support an accelerated approval. The news followed a complete response letter for the NDA in August. Company president and CEO Gerrit Klaerner called the data requirement "a major setback" to the timeline for veverimer’s development and announced a significant reduction in Tricida's headcount to extend its financial runway.
Bio-Europe: Taking stock of the COVID-19 pipeline
DUBLIN – The global product development pipeline for COVID-19 is now twice as big as for any particular cancer indication, a major disease like Alzheimer’s or any other infectious disease indication. Since the virus sequence became available in January, 808 development programs have been initiated, David Thomas, BIO’s vice president of industry research, said during a virtual Bio-Europe session on COVID-19 therapeutics. Small companies have been to the fore in populating that pipeline. “Seventy-three percent of these drugs originate in small companies. Even though the larger companies are, a lot of the time, driving the phase III trials, if you dig deep enough, they are actually originating from small companies. Fifty-two percent of the programs originate in the U.S.; China comes next with six percent, while Germany, the U.K. and Switzerland are also active.
Many genes with weak effects are key to drug response, too
CYBERSPACE – “We are not very good at predicting drug response in the clinic,” Ayesha Muhammad told the audience at the 2020 annual meeting of the American Society of Human Genetics (ASHG), “though it is not for lack of trying.” Nevertheless, adverse drug reactions are among the top 10 causes of in-hospital mortality. At a Thursday platform session, Muhammad, who is a graduate student at Vanderbilt University, described attempts to improve genome-based predictions to drug responses by assuming that the response to drugs is much like other complex genetic traits. That is, it is best predicted not by focusing on large effects of a few genes, but teasing out weaker effects of a larger group of candidates.
CDC: Complexity, sales factor in generic development
The simpler a new chemical entity (NCE), and the more revenue it generates, the more likely it is to face generic competition as soon as its U.S. patents and exclusivity expire. While those findings from an FDA Center for Drug Evaluation and Research (CDER) investigation into the factors influencing generic competition aren’t surprising, they do put some numbers to the brands that probably will be targeted by generics. For instance, abbreviated new drug applications were nearly four times more likely to be submitted referencing NCEs with annual revenues greater than $250 million for up to a four-year period after approval than for a product generating sales of less than $10 million, according to the CDER researchers.
Also in the news
Abivax, AB Science, Aerie, Affyimmune, Allakos, Amryt, Annovis, Apstem, Ariz, Asieris, Astrazeneca, Avrobio, Biolinerx, Centrexion, Chemocentryx, CNS, Connect, Cstone, Daewoong, Dr. Reddy's, Elpis, Field Trip Health, Forte, Frequency, Galecto, Genprex, Hookipa, Horizon, Humanigen, IM, Incyte, Insignis, Jaguar, Kintor, Legochem, Lianbio, Lilly, Linear Clinical Research, Mesoblast, Novocure, Oncosec, Pepromene, Plus, Polyphor, Protalix, Q32, Rafael, Regeneron, Sanofi, Scholar Rock, Spark, Syneos, Takeda, Tricida, Ultragenyx