Akcea LICA data look 'solid,' as expected, says Stifel » 10:3901/2201/22/20
Stifel analyst Paul…
Stifel analyst Paul Matteis said the "solid" APOCIII-LRx data announced by Akcea Therapeutics (AKCA), an affiliate of Ionis (IONS), align with his expectations that a lower dose, LICA-conjugated medicine can offer "a much improved therapeutic index" compared to Waylivra. The safety looked clean and the drug showed no signs of reduced platelet counts or impact on liver or kidney function, added Matteis. Akcea conveyed plans to pursue FCS and although Matteis sees a high probability-of-success in such an indication, he is skeptical about the size of the market potential. Matteis has a Hold rating on Akcea shares.
Akcea Therapeutics and Ionis report Phase 2 results of Akcea-ApocIII-LRx » 07:1001/2201/22/20
Akcea Therapeutics (AKCA), a majority-owned affiliate of Ionis Pharmaceuticals (IONS), announced positive topline results from the Phase 2 study of AKCEA-APOCIII-LRx in the treatment of patients with hypertriglyceridemia who are at risk for or have established cardiovascular disease. The study met the primary endpoint of significant triglyceride lowering and multiple secondary endpoints with a favorable safety and tolerability profile. Statistically significant dose-dependent reductions in fasting triglycerides compared to placebo at all dose levels; at the highest once monthly dose of 50 mg, more than 90% of patients achieved serum triglycerides of less than or equal to 150 mg/dL, compared to less than 5% of patients in the placebo group; mean triglyceride levels of patients at baseline was 285 mg/dL. Significant reductions in multiple additional risk factors, including apoC-III, very low-density lipoprotein (VLDL-C) and remnant cholesterol, compared to placebo. Treatment-emergent adverse events were comparable between active and placebo groups. The most common adverse event was injection site reactions. AKCEA-APOCIII-LRx was discovered by Ionis and has been co-developed by Akcea and Ionis.
|Over a week ago|
Akcea, Ionis announce initiation of CARDIO-TTRansform Phase 3 clinical trial » 07:1101/1001/10/20
Akcea Therapeutics, Inc.…
Akcea Therapeutics, Inc. (AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (IONS), announced the initiation of the CARDIO-TTRansform Phase 3 cardiovascular outcomes study for AKCEA-TTR-LRx in patients with transthyretin-mediated amyloid cardiomyopathy. AKCEA-TTR-LRx is an antisense drug developed using Ionis' proprietary LIgand Conjugated Antisense technology platform and is designed to inhibit production of TTR. It was discovered by Ionis and is being co-developed by Ionis and Akcea. In a Phase 1 clinical trial, patients treated with AKCEA-TTR-LRx experienced reductions in TTR of up to 94 percent at the highest dose.
Ionis Pharma appoints Onaiza Cadoret-Manier as CCO, Corporate Development » 07:0901/0901/09/20
Ionis Pharmaceuticals has…
Ionis Pharmaceuticals has appointed Onaiza Cadoret-Manier as Chief Corporate Development and Commercial Officer reporting to CEO Brett P. Monia. In this newly created role, Cadoret-Manier will serve as a member of Ionis' executive leadership team and be responsible for corporate strategy, commercialization, and operational leadership. Before joining Ionis, Cadoret-Manier was the chief commercial officer for Grail Biosciences, an early detection genomics company, where she developed the commercial strategy and business model. Prior to Grail, she was vice president of the Respiratory Franchise at Genentech.
Akcea announces publication of results from Phase 2 study of AKCEA-APO(a)-LRx » 08:0501/0201/02/20
Akcea Therapeutics (AKCA), a majority-owned affiliate of Ionis Pharmaceuticals (IONS), announced that results from a Phase 2 study evaluating AKCEA-APO(a)-LRx, also known as TQJ230, in patients with established cardiovascular disease and elevated levels of lipoprotein(a), or Lp(a), were published today in The New England Journal of Medicine. The article is titled "Lipoprotein(a) Reduction in Persons with Cardiovascular Disease." Elevated Lp(a) is an independent, genetic risk factor for CVD that cannot be well controlled with lifestyle modifications such as diet or exercise or with treatment using existing lipid-lowering therapies. It is estimated that there are more than eight million patients living with CVD and elevated levels of Lp(a) worldwide. AKCEA-APO(a)-LRx was discovered by Ionis and co-developed through Phase 2 by Akcea and Ionis. It is an antisense medicine developed using Ionis' advanced LICA technology designed to inhibit the production of apolipoprotein(a) and thus reduce Lp(a) levels. Following treatment, patients experienced significant dose-dependent reductions in Lp(a) at all dose levels studied with up to an 80% reduction in Lp(a) at the highest cumulative dose regimen. Approximately 98% of patients at the 80 mg monthly dose regimen achieved Lp(a) levels below 125 nmol/L. Significant dose-dependent reductions in LDL-C, apoB, OxPL-apoB and OxPL-apo(a) from baseline were also observed. The majority of adverse events were mild or moderate, with the most frequent adverse events being injection site reactions. ISRs occurred in 27% and 6% of patients on treatment and those on placebo, respectively, and were mostly mild. One patient discontinued treatment due to an ISR. There were no differences in platelet counts, liver and renal parameters, or flu-like symptoms in patients administered AKCEA-APO(a)-LRx. The randomized, double-blind, placebo-controlled, dose-ranging study of 286 patients across five countries with established CVD and elevated Lp(a) is the largest ever conducted specifically for people with elevated Lp(a). It is also the largest and longest study to date evaluating the Ionis LICA technology platform, with patients treated for up to 1 year and all patients treated for a minimum of six months. The goal was to assess the safety and tolerability of AKCEA-APO(a)-LRx and inform the dose and dose frequency for the planned Phase 3 cardiovascular outcomes study, being led by Novartis. Patients were administered AKCEA-APO(a)-LRx or placebo subcutaneously for six to 12 months.
William Blair says Wave 'disappointment' leaves Ionis, Roche in Huntington lead » 15:2512/3012/30/19
IONS, WVE, RHHBY
After Wave Life Sciences…
After Wave Life Sciences (WVE) announced data from an interim analysis of the ongoing Phase Ib/IIa PRECISION-HD2 trial of WVE-120102 for the treatment of Huntington's disease, William Blair analyst Myles Minter said that Wave's data is not in the same range as Roche (RHHBY) and Ionis' (IONS) Phase I/IIa 9-month dosing data for IONIS-HTT-Rx. Given that Wave's interim analysis "disappoints," Minter sees Roche and Ionis in the lead for disease-modifying HD therapeutic development and believes the duo are "potentially years ahead of meaningful oligonucleotide competition," he tells investors. Minter has a Market Perform rating on Ionis shares.
|Over a month ago|
Ionis Pharmaceuticals licenses investigational Alzheimer's therapy » 07:0912/1912/19/19
Ionis Pharmaceuticals (IONS) announced today that Biogen (BIIB), a collaboration partner for neurological diseases, has licensed IONIS-MAPTRx, an antisense therapy designed to selectively reduce production of microtubule-associated protein tau, or tau, in the central nervous system. MAPT is believed to contribute to or cause several neurodegenerative diseases, including Alzheimer's disease and some forms of frontotemporal degeneration. Ionis earned a $45M license fee from Biogen and is eligible to earn up to $155M in additional milestone payments. Ionis is also eligible to receive royalties on sales of the medicine in the low- to mid-teens. Under terms of the collaboration, Ionis will be responsible for the Phase 1 clinical study of IONIS-MAPTRx in patients with mild AD that was initiated in 2017 and a one-year long-term extension study that began this year. Biogen will have responsibility for all subsequent studies and any further development, including regulatory filings, and commercialization.
Ionis Pharmaceuticals appoints Frank Bennett to Chief Scientific Officer » 07:1212/1812/18/19
Ionis Pharmaceuticals appointed C. Frank Bennett as Chief Scientific Officer, and Eric Swayze as Senior Vice President of Research, effective January 1, 2020. In his new role, Bennett will lead a new program to identify technologies to enhance Ionis' drug discovery programs as well as, identify potentially new therapeutic platforms. Additionally, Bennett will continue in his current role as Franchise Leader for Neurology Drug Discovery and serve as a senior strategic advisor to Ionis' research and development programs. Bennett is one of the founding members of Ionis. Swayze will succeed Bennett as Senior VP of Research. He will be responsible for leading preclinical antisense drug discovery and antisense technology research. Swayze joined Ionis in 1994. Since 2012, Swayze has led the neuroscience drug discovery group.
Akcea Therapeutics appoints Lynne Parshall as chair, increases board to ten » 07:4212/1612/16/19
AKCA, IONS, MRK
Akcea Therapeutics (AKCA), a majority-owned affiliate of Ionis Pharmaceuticals (IONS), announced the appointments of Lynne Parshall, J.D., as chair of the Akcea Board of Directors and Barbara Yanni as a new member of the board. Both positions are effective immediately. Parshall has served as a member of Akcea's Board of Directors since January 2015. She replaces Christopher Gabrieli, who will continue to serve as a board member. Parshall is also currently a director and senior strategic advisor to Ionis Pharmaceuticals. Yanni was vice president and chief licensing officer at Merck & Co. (MRK) from November 2001 until her retirement in March 2014. With the addition of Yanni, Akcea has now increased the authorized number of members of the Board to 10 and increased the number of independent board members from six to seven.
Ionis Pharmaceuticals initiated with an Outperform at Oppenheimer » 16:2712/1212/12/19
Oppenheimer analyst Esther Rajavelu initiated coverage of Ionis Pharmaceuticals with an Outperform rating and $77 price target. The analyst is positive on the company's "solid base business of revenue-generating assets", along with its "broadly applicable discovery platform" and strategic partnerships, saying the stock offers a diversified opportunity in the RNAi sub-segment of biotechnology, and stands "well positioned for growth with its current portfolio of commercialized assets". Rajavelu also cites Ionis Pharma's "incremental value inflection" opportunities through its developing pipeline.