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Ionis Pharmaceuticals Inc (NASDAQ:IONS) should be smiling after the company, along with Biogen (NASDAQ:BIIB), announced that SPINRAZATM (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset (consistent with Type 2) SMA.

Ionis Pharmaceuticals shares reacted to the news, rising nearly 11% to $30 in pre-market trading Monday.

The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment. SPINRAZA demonstrated a favorable safety profile in the study.

“These results, along with our successful trial in infantile-onset SMA, reinforce the potential of SPINRAZA to benefit a broad range of SMA patients,” said Michael Ehlers, M.D., Ph.D., executive vice president, head of Research and Development at Biogen. “We will make regulators around the globe aware of this data and will continue working closely with them to bring SPINRAZA to families affected by SMA as quickly as possible.”

Biogen is preparing for the potential launch of SPINRAZA in the U.S. possibly as early as the end of 2016 or the first quarter of 2017.

Results From the CHERISH Interim Analysis

CHERISH is a fifteen-month study investigating SPINRAZA in 126 non-ambulatory patients with later-onset SMA (consistent with Type 2), including patients with the onset of signs and symptoms at greater than 6 months and an age of 2 to 12 years at screening.

Results from the primary endpoint of the pre-specified interim analysis demonstrated a difference of 5.9 points (p= 0.0000002) at 15 months between the treatment (n=84) and sham-controlled (n=42) study arms, as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE). From baseline to 15 months of treatment, patients who received SPINRAZA achieved a mean improvement of 4.0 points in the HFMSE, while patients who were not on treatment declined by a mean of 1.9 points. The HFMSE is a reliable and validated tool specifically designed to assess motor function in children with SMA, and a change of three points or greater in the HFMSE has previously been identified as clinically meaningful. Data from the other endpoints analyzed were consistently in favor of children who received treatment. SPINRAZA demonstrated a favorable safety profile. The majority of the adverse events were considered to be either related to SMA disease, common events in the general population, or events related to the lumbar puncture procedure. No patients discontinued the study.

With the positive interim analysis, the CHERISH study will be stopped and participants will be able to transition into the SHINE open-label extension study to receive SPINRAZA. Full study results will be presented at future medical congresses.

“These data further validate the potential of SPINRAZA as a treatment for patients with SMA,” said B. Lynne Parshall, chief operating officer of Ionis Pharmaceuticals. “We are grateful to all the families and clinicians who have participated in all of the SPINRAZA studies. Without their commitment and support, this program would not have been able to progress so quickly.”

The U.S. Food and Drug Administration (FDA) recently accepted the company’s New Drug Application (NDA) for SPINRAZA as a treatment for SMA and communicated they plan to act early on the NDA under an expedited review. Additionally, the European Medicines Agency (EMA) recently validated Biogen’s Marketing Authorization Application (MAA) in the EU. The EMA’s Committee for Medicinal Products for Human Use (CHMP) granted Accelerated Assessment status and the FDA granted Priority Review to SPINRAZA. Biogen is initiating regulatory filings in other countries in the coming months.

Biogen initiated a global expanded access program (EAP) in infantile-onset SMA earlier this year. The company will continue to explore where and when the EAP may be broadened to include patients with later-onset SMA (consistent with Type 2).

The SPINRAZA Clinical Trial Program

SPINRAZA has been studied in both presymptomatic and symptomatic patients with SMA including patients likely to develop or diagnosed with SMA Types 1, 2, and 3.

The SPINRAZA Phase 3 program is comprised of two registrational studies, ENDEAR and CHERISH. ENDEAR is a thirteen-month study investigating SPINRAZA in 122 patients with infantile-onset SMA, including patients with the onset of signs and symptoms of SMA at up to six months of age. The endpoint pre-specified for the interim analysis of the study evaluated the proportion of motor milestone responders from the motor component of the Hammersmith Infant Neurological Examination (HINE). Given the results of the positive interim analysis, the ENDEAR study is being stopped and participants are able to transition into the SHINE open-label study, in which all patients will receive SPINRAZA.

Additionally, the SHINE open-label extension study for patients who previously participated in ENDEAR or CHERISH is open and is intended to evaluate the long-term safety and tolerability of SPINRAZA.

Two additional Phase 2 studies, EMBRACE and NURTURE, were designed to collect additional data on SPINRAZA. EMBRACE is studying a small subset of patients with infantile or later-onset SMA who do not meet the age and other criteria of ENDEAR or CHERISH. NURTURE is an open-label, ongoing study in pre-symptomatic infants who are up to six weeks of age at time of first dose to determine if treatment before symptoms begin would prevent or delay the onset of SMA symptoms. An interim analysis of NURTURE showed that infants treated for up to one year with SPINRAZA achieved motor milestones in timelines more consistent with normal development than what is observed in the natural history of patients with Type 1 SMA. Three infants experienced adverse events considered possibly related to SPINRAZA, all of which resolved. In addition, no infants have discontinued or withdrawn from the study and no new safety concerns have been identified. NURTURE is currently active and enrolling. All studies are being conducted on a global scale. (Original Source)

On the ratings front, Ionis Pharmaceuticals has been the subject of a number of recent research reports. In a report issued on November 1, Piper Jaffray analyst Joshua Schimmer reiterated a Buy rating on IONS. Separately, on October 10, Wells Fargo’s Jim Birchenough maintained a Buy rating on the stock.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Joshua Schimmer and Jim Birchenough have a yearly average loss of 13.2% and a return of 17.7% respectively. Schimmer has a success rate of 28% and is ranked #4011 out of 4162 analysts, while Birchenough has a success rate of 45% and is ranked #88.

Overall, 3 research analysts have assigned a Hold rating and 4 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $42.00 which is 55% above where the stock closed last Friday.

Ionis Pharmaceuticals, Inc. engages in the development and commercialization of antisense drug discovery. It operates its business through the Ionis Core and Akea Therapeutics segments. The Ionis Core segment involves a a novel drug discovery platform generate a broad pipeline of drugs. The Akea Therapeutics segment develops and commercializes drugs for cardiometabolic disorders.