On January 11, 2016 Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) reported that the Company expects to significantly improve upon its financial guidance for 2015 (Press release, Isis Pharmaceuticals, JAN 11, 2016, View Source;p=RssLanding&cat=news&id=2128373 [SID:1234508772]). The Company expects to end 2015 with a pro forma net operating loss (NOL) in the low $20 million range and more than $775 million in cash.
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"Over the past several years, we have created a strong and consistent financial foundation. Our corporate partnerships have produced increasing levels of revenue as our partnered programs have matured and expanded. We have been able to support the advancement of our very large pipeline with modest expense growth due to the efficiency of our technology platform as well as the significant in-kind contributions of our partners. With three potentially transformational medicines close to commercialization, we look forward to adding product revenues and royalties to our revenue base over the next few years," said Elizabeth L. Hougen, chief financial officer of Ionis Pharmaceuticals. "We expect to end 2015 with a pro forma NOL in the low $20 million range, which represents a 60% improvement over our original guidance. In addition, we expect to end 2015 with more than $775 million in cash, substantially exceeding our 2014 year-end cash balance and our 2015 cash guidance. Importantly, our significant increase in revenue over the last several years reflects the successes in all of our collaborations as the drugs in those collaborations advanced."
The Company intends to provide financial guidance for 2016 in connection with its year-end 2015 financial results in February.
Pipeline Update
Ionis management will present an update today on its drug development pipeline at the J.P. Morgan conference, including an update on its ongoing open-label Phase 2 clinical study of nusinersen in infants with Type I spinal muscular atrophy (SMA). Previously the company reported data from this study on event-free survival, measures of muscle function and assessments of developmental milestones. The data reported today show continued increases in median event-free survival and muscle function scores. The safety and tolerability profile of nusinersen to date continues to support further development.
In 2015, Ionis added nine new drugs to its pipeline. Already in 2016, the company has added two new drugs to its pipeline: IONIS-BIIB5Rx and IONIS-BIIB6Rx. Both new drugs are being developed together with Biogen and are designed to address undisclosed targets for the treatment of patients with neurodegenerative diseases. Ionis currently has a total of 38 drugs in its pipeline.
"We believe we are just beginning to realize the value we have created. We have three potentially groundbreaking medicines for which we have completed target enrollment in the respective pivotal Phase 3 trials and preparations are underway for each of these drugs for the necessary regulatory filings for marketing authorization. In addition, we and our partners are well along in preparing to commercialize these medicines. As our pipeline continues to mature, we have many other medicines coming along behind these three late-stage drugs," said B. Lynne Parshall, chief operating officer at Ionis. "We have also demonstrated in 2015 that the advances we are making in our technology continue to make better and better medicines, which allows us to expand the application of our drugs to new tissues, new targets and new diseases. All of this sets us up for an exciting and event-filled 2016."
In January 2016, Ionis terminated its license agreement with Sanofi Genzyme for KYNAMRO (mipomersen sodium).
"We are disappointed that we have had to terminate our contract with Genzyme to commercialize KYNAMRO," said Ms. Parshall. "We know that KYNAMRO is an important drug for patients with homozygous FH. Genzyme is continuing to support patients and physicians as we explore other potential commercialization options."
In addition, Ionis announced that results from the Phase 1/2 study with IONIS-AR-2.5Rx in heavily pretreated, late-stage prostate cancer patients demonstrated encouraging data, including several durable reductions in PSA levels. The drug also exhibited a good safety and tolerability profile supportive of continued development. Data from this study will be presented later in the year. Ionis plans to continue the development of IONIS-AR-2.5Rx independent of AstraZeneca.