These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include:
Pipeline and key clinical candidates for these companies:
Xoma is a biotech royalty aggregator that says it is "playing a distinctive role in helping biotech companies achieve their goal of advancing novel therapeutic candidates aimed at improving human health"
VBL Therapeutics is developing targeted medicines for immune-inflammatory diseases. VBL’s lead immunology product candidate VB-601 is a targeted antibody for immune-inflammatory applications that has shown disease-modifying activity across multiple preclinical models including multiple sclerosis, rheumatoid arthritis and inflammatory bowel disease. At present, VBL is evaluating options for development of its assets, which may include partnering and other strategic options.
BioMarin is focused on therapies for people with serious and life-threatening genetic diseases and medical conditions. The company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases.
uniQure is focused on gene therapy with a goal of "single treatments with potentially curative results." The company is advancing a pipeline of gene therapies to treat patients with hemophilia B, Huntington's disease, refractory temporal lobe epilepsy, Fabry disease, and other diseases.
Mirati Therapeutics is focused on bringing forward therapies that address areas of high unmet medical need, including lung cancer, and advancing a pipeline of novel therapeutics targeting the genetic and immunological drivers of cancer.
Recent news on these stocks:
November 23
BioMarin Pharmaceutical announced advancements in the U.S. FDA review of the Biologics License Application of ROCTAVIAN for adults with severe hemophilia A. The company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. Previously, the FDA communicated to the company that it did intend to hold an advisory committee meeting but did not specify a date. The company also remains on track to host the scheduled FDA Pre-Licensure Inspection of BioMarin's gene therapy manufacturing facility located in Novato, CA.
Piper Sandler analyst Christopher Raymond cautions that "reading FDA tea leaves can be a dangerous game," but with that said he thinks it is "reasonable to connect a couple of recent data points from FDA" that he thinks bode well for Roctavian's U.S. approval process, including yesterday's approval of uniQure's (QURE) hemophilia B gene therapy Hemgenix and today's announcement that the FDA no longer intends to conduct an AdCom to review BioMarin's (BMRN) Roctavian hemophilia A application. The analyst, who reminds investors that Roctavian's EU launch is currently underway and that his recent hematologist survey makes it appear there is "some degree of pent-up demand for the therapy," has an Overweight rating and $128 price target on BioMarin shares.
Truist analyst Robyn Karnauskas raised the firm's price target on uniQure to $65 from $51 and keeps a Buy rating on the shares. The analyst cites the company's announcement yesterday regarding the FDA approval of Hemgenix with a "clean label". Karnauskas adds that she is updating her model to reflect the $3.5M price in U.S., modeling a peak $800M in royalties vs. $600M previously.
November 22
uniQure announced that its partner CSL (CSLLY) has received approval from the U.S. Food and Drug Administration for Hemgenix, a one-time gene therapy for the treatment of adults 18 years of age and older living with hemophilia B. The product is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. CSL licensed the exclusive global rights to Hemgenix from uniQure in May 2021 and is now solely responsible for the further development, registration, and commercialization of the therapy. "Today's landmark approval represents a major milestone in the field of genomic medicine and ushers in a new treatment paradigm for patients living with hemophilia B," said Matt Kapusta, chief executive officer of uniQure. "This is uniQure's second internally-developed gene therapy to achieve approval and the world's first gene therapy for hemophilia B, an historic achievement based on more than a decade of research and clinical development, as well as the tireless dedication of our employees, clinicians, patients and their families. For nearly 25 years, uniQure has been driven by a singular mission: to transform peoples' lives by harnessing the power of gene therapy. This groundbreaking approval delivers on this promise by providing people with hemophilia B the possibility of being liberated from burdensome infusions and an ability to unlock the potential in their daily lives. We offer our sincere gratitude to the hemophilia community, without whose years of dedication and invaluable support this medical breakthrough could not have been achieved."
Mirati Therapeutics is garnering renewed takeover interest from pharmaceutical companies ahead of updates on its drug pipeline, Michelle Davis and Dinesh Nair of Bloomberg reported, citing people familiar with the matter. The stock rose as much as 16% on the news. Mirati has reportedly been mulling strategic options including a sale for a while.
After Bloomberg reported that Mirati Therapeutics is drawing fresh takeout interest ahead of updated PD1 + adagrasib combination data at next month's ESMO I/O meeting, BMO Capital analyst Evan Seigerman said he expects Mirati to build upon data shared in conjunction with the June ASCO meeting that showed "encouraging efficacy" when adagrasib was combined with PD1. A takeout is likely to reflect full valuation for usage in the first line NSCLC setting, said Seigerman, who could see Mirati being valued at up to $200 per share in an "upside scenario." He has an Outperform rating and $110 price target on Mirati shares, which are up $11.90, or 16%, to $86.44 following Bloomberg's report.
Xoma acquired all rights and title to ebopiprant held by ObsEva (OBSV) for $15 million plus certain earn-out payments. XOMA has assumed the ebopiprant intellectual property estate and all license agreements, including the 2021 exclusive license agreement from ObsEva to Organon related to the development and commercialization of ebopiprant. Under the terms of the agreement, XOMA has acquired all rights to ebopiprant held by ObsEva, including the Organon/ObsEva license agreement and the IP associated with the asset. XOMA will now be entitled to receive up to $475 million in development, regulatory, and sales-based milestone payments under the ObsEva/Organon license agreement. XOMA will pay to ObsEva a portion of the development and regulatory milestones, as well as certain sales milestones, up to $98 million. Upon commercialization, XOMA will receive royalties that range from low- to mid-teens from Organon and will make a mid-single-digit royalty payment to Merck KGaA, Darmstadt, Germany.
About “Biotech Alert”
The Fly will report on a selection of biotech stocks seeing a surge in interest from retail and financial professional investors, based on data from InvestingChannel.
This Fly exclusive recap reveals the biotech stocks that are seeing a spike in searches among the 15-plus million retail and financial professional investors through InvestingChannel's online financial news media ecosystem.
This increased attention from the investors may be in response to, or advance of, outsized moves for stocks in the biotech sector, which tend to be volatile and prone to sharp swings in share price around binary events such as clinical study results and FDA approvals.
Xoma
+0.12 (+0.48%)
VBL Therapeutics
+
BioMarin
-0.76 (-0.78%)
uniQure
+1.125 (+4.28%)
Mirati Therapeutics
+2.24 (+2.34%)
CSL
+0.15 (+0.15%)
ObsEva
+