After Krystal Biotech announced the official opening of its new cGMP facility located in Pittsburgh, H.C. Wainwright analyst Joseph Pantginis said the plant opening represents an “important box checked” and demonstrates that the company is favorably positioned to address the current and future manufacturing needs of its assets. The analyst, who said KB103 is “moving quickly towards pivotal,” keeps a Buy rating and $32 price target on Krystal Biotech shares
Wednesday, March 6, 2019
Pfizer licenses AnTolRx’s immune tolerance nanomed for Type 1 diabetes
Back in 2016, Pfizer led a $4 million round for AnTolRx to fund the research and development of its antigen-specific immune tolerance treatment for diabetes. Now, the Big Pharma is pulling the trigger on an option to take AnTolRx’s lead candidate forward, in exchange for the usual upfront, milestone and royalty payments, all undisclosed.
“We are delighted to confirm that the discovery phase of the program at AnTolRx has transitioned successfully to Pfizer for further development of our antigen-specific immunotherapy for Type 1 diabetes,” said AnTolRx CEO Mark Carthy, in a statement.
AnTolRx’s work is based on the immunoregulatory research of Francisco Quintana, a professor of neurology at Brigham and Women’s Hospital in Boston and the company’s scientific cofounder. Its pipeline comprises nanoparticle therapeutics focused on immune tolerance rather than immune suppression. AnTolRx is targeting a range of autoimmune ailments, including rheumatoid arthritis, inflammatory bowel disease, psoriasis and multiple sclerosis, as well as Type 1 diabetes.
The nanotechnology is designed to bring about immune tolerance to specific self-antigens—in the case of diabetes, a tolerance-inducing treatment could eliminate immune attacks on beta cells in the pancreas. And since it is antigen-specific, the technology is expected to avoid the side effects of systemic immunosuppression.
“There is an urgent need for disease-modifying Type 1 diabetes immunotherapies to slow down the autoimmune process and delay or block progression to symptomatic insulin-dependent diabetes,” said JDRF President and CEO Derek Rapp.
Pfizer isn’t the only Big Pharma that has gotten into immune tolerance. In 2017, both Eli Lilly and Novartis struck deals to at least prep an entry into the space. Lilly bagged an option from Evotec’s spinout Topas Therapeutics on immune tolerance drugs that have been applied in conditions including multiple sclerosis, Type 1 diabetes and celiac disease. And Novartis secured the worldwide rights to Parvus Therapeutics’ Navacims nanomedicine tech, specifically for Type 1 diabetes patients.
Alnylam’s givosiran looks approvable on Phase 3 data, says Piper Jaffray
After Alnylam announced the Phase 3 ENVISION trial of givosiran in acute hepatic porphyria patients hit the primary endpoint of reduction in the annualized rate of composite porphyria attacks, Piper Jaffray analyst Edward Tenthoff said he believes the “robust significance across all components” of the primary endpoint should enable approval. Tenthoff, who expects approvals in early 2020, reiterated his Overweight rating and $142 price target on Alnylam shares, which is down $2.58, or 2.93%, to $85.72 in morning trading.
https://thefly.com/landingPageNews.php?id=2875213
https://thefly.com/landingPageNews.php?id=2875213
Bio-Path gains on positive prexigebersen data
Nano cap Bio-Path Holdings (NASDAQ:BPTH) is up 54% premarket on increased volume in response to updated data from an open-label Phase 2 clinical trial evaluating prexigebersen (BP1001) plus low-dose cytarabine (LDAC) compared LDAC alone in newly diagnosed patients with acute myeloid leukemia (AML).
The response rate improved to 65% (n=11/17) from 47% reported almost a year ago, including five complete responders. Six patients experienced stable cancer implying a disease control rate of 100% (n=17/17). The company says these data are impressive since the treatment group included a majority of secondary AML patients who typically do not respond well.
A second cohort has been added evaluating the combination of prexigebersen and decitabine in the same population of patients.
Prexigebersen [Liposomal Grb-2 (Growth factor Receptor Bound protein-2)] suppresses the expression of Grb-2, an adaptor protein that links tyrosine kinases with their downstream signaling molecules. Tyrosine kinases function as “on” “off” switches in many cellular functions so interrupting their signaling may have an inhibitory effect on rapidly-growing cancer cells.
Tandem Diabetes business ‘is just getting going,’ says Lake Street
While the shares of Tandem Diabetes have had a nice move, the business is “just getting going,” Lake Street analyst Brooks O’Neil tells investors in a research note partially titled “Why $100 And How Fast?” The analyst believes Tandem has the best pump on the market today coupled with a pipeline of future products “that will bring market leading innovation to diabetics world-wide over the next few years.” He sees the shares going to $100 and affirms a Buy rating on Tandem Diabetes.
https://thefly.com/landingPageNews.php?id=2875171
https://thefly.com/landingPageNews.php?id=2875171
Departure of Gottlieb brings some biopharma uncertainty, says Piper Jaffray
Piper Jaffray remains “largely bullish” on the biopharma sector but acknowledges a “new and unexpected” source of uncertainty now with the announced departure of FDA commissioner Scott Gottlieb, analysts led by Christopher Raymond tell investors in a research note. With a track record of favoring innovation and pushing for increased regulatory efficiency while also advocating for more competition, Dr. Gottlieb was about as “market friendly, patient focused and industry friendly as one could have hoped,” Piper writes. The firm adds that while it has no reason to believe the next commissioner “will be quite as radical and potentially damaging to the industry,” it does think investors would do well to watch the selection process very closely.
Alnylam says givosiran meets primary, majority of secondary endpoints in study
Alnylam Pharmaceuticals announced that the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 in development for the treatment of acute hepatic porphyria, met its primary efficacy endpoint and the majority of secondary endpoints. Specifically, givosiran met the primary endpoint of reduction in the annualized rate of composite porphyria attacks relative to placebo and achieved statistically significant results for five of nine secondary endpoints, with a safety and tolerability profile that the company believes is encouraging, especially in this high unmet disease. Based on these results, the company plans to complete its rolling submission of a New Drug Application and file a Marketing Authorization Application in mid-2019. Givosiran has received Breakthrough Therapy and Prime designation by the FDA and EMA, respectively, and has Orphan Drug status in the U.S. and EU for the treatment of AHP. The Phase 1 results of givosiran were recently published in The New England Journal of Medicine. Full ENVISION study results will be presented in an oral plenary session on Saturday, April 13 at EASL in Vienna, Austria. The ENVISION results have not yet been reviewed by regulatory authorities.
https://thefly.com/landingPageNews.php?id=2874973
https://thefly.com/landingPageNews.php?id=2874973
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