LONDON, UK / ACCESSWIRE / October 16, 2017 / Pro-Trader Daily takes a look at the latest corporate events and news making the headlines for Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), following which we have published a free report that can be viewed by signing up at http://protraderdaily.com/optin/?symbol=IONS. The Company, which is a leading pharmaceutical firm in RNA-targeted drug discovery and development, announced on October 13, 2017, that it has commenced the Phase-1/2a clinical study of IONIS-MAPTRx in patients with mild Alzheimer's disease (AD). Ionis also proclaimed that it has received a $10 million milestone payment from Biogen for initiating this study. For immediate access to our complimentary reports, including today's coverage, register for free now at: http://protraderdaily.com/register/.

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How IONIS-MAPTRx Works?

IONIS-MAPTRx is an antisense drug, which has been designed to selectively lessen the production of microtubule-associated protein tau (MAPT), or tau protein, in the brain.

Neurofibrillary tangles, which are created by the misfolding of Tau inside brain cells, are a trademark feature of neurodegenerative diseases known as tauopathies. Neurodegenerative diseases include Alzheimer's disease (AD) as well as some other forms of frontotemporal dementia (FTD). Of these, AD is the most common form of dementia; and it accounts for about 70% of cases. On the other hand, FTD is a rare form of early-onset dementia, which is a result of neuronal damage in the frontal and temporal lobes of the brain.

C. Frank Bennett, Ph.D., Senior Vice President of research at Ionis Pharma, highlighted that tau deposits are spatially and temporally related to the brain regions where atrophy occurs and neurocognitive deficits begin. This makes it different from amyloid plaques that may start to deposit in the brain for up to 20 years before the commencement of AD. And IONIS-MAPTRx is devised to reduce the production of all forms of tau in all regions of the brain.

Phase-1/2a Clinical Study Design

  • Phase-1/2a Clinical Study is a three-month randomized, placebo-controlled, dose escalation study. It has been designed to assess the safety and efficacy of IONIS-MAPTRx in nearly 44 patients, who have mild AD.

  • For the purpose of the study, IONIS-MAPTRx would be administered as a once-monthly intrathecal injection directly into the cerebral spinal fluid, similar to the way SPINRAZA® (nusinersen) is administered.
  • The estimated primary completion date for the study is January 31, 2020.

Ionis Pharma and Biogen's Strategic Collaboration

Ionis Pharma and Biogen's collaboration is a strategic move as it combines Biogen's expertise in neurology with Ionis Pharma's leadership in antisense technology. Both Companies are working together to develop innovative therapies for the treatment of neurological disorders. SPINRAZA, which is an antisense drug developed with Ionis Pharma's proprietary antisense technology, is the first commercial drug from this collaboration. SPINRAZA has already been approved in the global markets for the treatment of patients with spinal muscular atrophy (SMA).

Under the collaboration agreement, Biogen is accountable for the commercialization of SPINRAZA. Also under this collaboration, Ionis Pharma and Biogen are developing IONIS-MAPTRx for amyotrophic lateral sclerosis (ALS), and IONIS-BIIB5Rx, IONIS-BIIB6Rx, and IONIS-BIIB7Rx for the treatment of undisclosed neurodegenerative diseases.

Addressing an Unmet Medical Need

Ionis Pharma and Biogen are venturing into a challenging area that has witnessed a series of clinical failure in AD by drug developers who have been striving to create successful new drugs for this disease. In fact, just a few drug successes have ever won marketing approvals up till now, and those that have succeeded have only slowed progression of symptoms by 6 to 12 months. Over the past year, pharmaceutical giants such as Merck & Co., Eli Lilly, and Fujifilm have acknowledged clinical failures for AD candidates. A Cleveland Clinic study conducted in 2014 established a failure rate of 99.6% for clinical trials for AD drug candidates between 2002 and 2012. The study discovered high attrition rates for AD treatments, with 72% of agents failing in Phase-I, 92% failing in Phase-II, and 98% failing in Phase-III.

B. Lynne Parshall, Chief Operating Officer of Ionis Pharma, shared his views about the development. He mentioned that IONIS-MAPTRx is another exciting program within Ionis Pharma's strategic collaboration with Biogen. MAPT treatment represents an extensive opportunity for addressing the unmet need in rare as well as more prevalent tauopathies. Biogen has retained the option to develop and commercialize IONIS-MAPTRx. He shared that although the first clinical study is to evaluate the efficacy of IONIS-MAPTRx in patients with AD, simultaneously, the Company plans to develop IONIS-MAPTRx for patients suffering with FTD, which represents a rare disease population. Parshall believes that this strategy has the potential to accelerate the drug's path to market.

Last Close Stock Review

Ionis Pharma's share price finished last Friday's trading session at $59.10, advancing 2.37%. A total volume of 1.96 million shares have exchanged hands, which was higher than the 3-month average volume of 1.22 million shares. The Company's stock price skyrocketed 11.49% in the last three months, 40.18% in the past six months, and 96.35% in the previous twelve months. Additionally, the stock surged 23.56% since the start of the year. Shares of the Company have a PE ratio of 281.43 and currently have a market cap of $7.33 billion.

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