Catalyst Pharmaceuticals Inc (NASDAQ:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today reported financial results for the second quarter and six months ended June 30, 2016 and provided a product development update.
“We were pleased to have reached agreement with the FDA during the quarter on the clinical trial protocol for our second pivotal Phase 3 trial for Firdapse® in the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS)”, said Patrick J. McEnany, Chief Executive Officer of Catalyst. “We expect to initiate a small, efficient short term study with Firdapse during the second half of 2016, as we continue to expend additional research dollars studying other potential neuromuscular indications for Firdapse. As stated previously, we believe that our existing capital resources are adequate to get us to an accepted NDA submission without the need for additional financing.”
Q2 and Recent Highlights
- Agreement with FDA on a second Phase 3 Study Protocol for Firdapse in LEMS
- Publication of detailed results from the LMS-002 Phase 3 study of Firdapse in patients with LEMS in the May 2016 issue of Muscle & Nerveauthored by Dr. Shin Oh, et al.
- Operating expense management plan to align resources is now complete
- Ended June 30, 2016 with approximately $48 million in cash and investments and no debt
PRODUCT DEVELOPMENT UPDATE
Firdapse for LEMS NDA resubmission plan
- Finalizing logistics to launch a small, efficient short-term second Phase 3 trial in the fourth quarter 2016
- Have met with most of our key opinion leaders and they are fully supportive of our plan for the second Phase 3 trial
- Anticipate having two clinical trial sites, one each on the east and west coasts of the U.S.
- Anticipate enrolling up to 24 subjects, utilizing a cross-over design that we believe will be adequately powered
- The trial will have the same co-primary endpoints as the first Phase 3 trial (LMS-002)
- FDA is permitting us to enroll subjects currently participating in our Expanded Access Program
- Expect top-line data and an NDA resubmission (assuming favorable data) in the second half of 2017
Firdapse for Congenital Myasthenic Syndromes (CMS)
- Phase 3 trial currently underway and continues to identify and recruit patients at four sites
- Based on discussions with the FDA, the study is being expanded to include adult CMS patients in addition to the pediatric population and to expand the total subjects in this trial to approximately 20 patients
- FDA is currently reviewing amended protocol and statistical analysis plan
- Expect top-line data in the second half of 2017
- Assuming favorable data, we plan to include CMS in our NDA resubmission or as a supplement to the NDA
- Patients will be eligible to enroll in our Expanded Access Program at the completion of the study
Firdapse for MuSK antibody positive Myasthenia Gravis
- Approximately 5-8% of the Myasthenia Gravis patient population is estimated to be MuSK antibody positive
- Small phase 3 investigator-sponsored study currently underway at the Carlo Besta Neurological Institute in Milan, Italy
- Anticipate top line data in early 2017
- Assuming positive data from current study, we plan to initiate a registration-quality trial in the U.S.
- We have submitted a Special Protocol Assessment request to the FDA for the proposed U.S. based trial
CPP-115 (next generation GABA-AT inhibitor)
- Additional dose optimization studies are required
- Other studies required to make CPP-115 “Phase 2 ready” include: long-term toxicology studies in two species; development and reproductive toxicology; and additional ADME studies
- Current cash resources are devoted to advancing Firdapse, as a result we are in discussions with several potential partners for the continued development of CPP-115
Generic Sabril® (generic ingredient vigabatrin)
- Sabril marketed in U.S. by Lundbeck, and all Hatch-Waxman exclusivities expire by April 2017
- Indicated for the treatment of refractory complex partial seizures and infantile spasms
- As of July 21, 2016 FDA has relaxed the requirements under the REMS program for physicians to prescribe Sabril
- U.S. Sabril sales in 2015 as reported were approximately $144 million up 15% from 2014
- Based on currently available information, Catalyst is hopeful that any ANDA submission that it makes for this product will be one of the first ANDA’s submitted for a generic version of Sabril
For the quarter ended June 30, 2016, Catalyst reported a GAAP net loss of $4,568,914 or 6 cents per basic and diluted share, as compared to a GAAP net loss of $4,558,503 or 6 cents per basic and diluted share, for the same period in 2015. Excluding non-cash gain of $152,783 attributable to the change in fair value of liability-classified warrants, Non-GAAP1 net loss was $4,721,697 or 6 cents per basic and diluted share for the second quarter of 2016. In comparison, Non-GAAP1 net loss for the second quarter of 2015 was $4,892,459 or 6 cents per basic and diluted share, which excludes non-cash gain of $333,956 attributable to the change in fair value of liability-classified warrants.
1 Statements made in this press release include a non-GAAP financial measure. Such information is provided as additional information and not as an alternative to Catalyst’s financial statements presented in accordance with generally accepted accounting principles (GAAP). This non-GAAP financial measure is intended to enhance an overall understanding of Catalyst’s current financial performance. Catalyst believes that the non-GAAP financial measure presented in this press release provides investors and prospective investors with an alternative method for assessing Catalyst’s operating results in a manner that Catalyst believes is focused on the performance of ongoing operations and provides a more consistent basis for comparison between periods. The non-GAAP financial measure in this press release excludes from the calculation of net loss the expense (or the income) associated with the change in fair value of the liability-classified warrants. Non-GAAP net loss per share is calculated by dividing non-GAAP net loss by the weighted average common shares outstanding.
For the six months ended June 30, 2016, Catalyst reported a GAAP net loss of $9,955,151, or 12 cents per basic and diluted share, as compared to a GAAP net loss of $9,968,762, or 13 cents per basic and diluted share, for the same period in 2015. Excluding non-cash gain of $886,139 attributable to the change in fair value of liability-classified warrants, Non-GAAP1 net loss was $10,841,290 or 13 cents per basic and diluted share for the first six months of 2016. In comparison, Non-GAAP1 net loss for the first six months of 2015 was $9,122,440, or 12 cents per basic and diluted share, which excludes non-cash expense of $846,322 attributable to the change in fair value of liability-classified warrants.
Research and development expenses for the second quarter of 2016 were $2,508,897 as compared to $2,577,508 in the second quarter of 2015. For the six months ended June 30, 2016, research and development expenses were $6,055,288 as compared to $4,927,060 in the same period in 2015. Research and development expenses for the first six months of 2016 increased when compared to the same period in 2015, due primarily to consulting fees relating to regulatory matters, activities related to the Firdapse expanded access program, including manufacturing of related drug, and increased activities in ongoing studies and trials. We expect that costs related to research and development activities will continue to be substantial throughout the balance of 2016 and into 2017.
General and administrative expenses for the second quarter of 2016 totaled $2,305,555 as compared to $2,319,822 in the second quarter of 2015. For the six months ended June 30, 2016, general and administrative expenses were $4,996,700 as compared to $4,262,185 in the same period in 2015. The increase when compared to the same period in 2015 is primarily due to increases in pre-commercialization expenses, payroll and benefits expenses during the first half of 2016, including approximately $600,000 for severance costs related to the reduction in force that occurred in May 2016, partly offset by our initiatives to conserve resources. We expect general and administrative expenses to decrease during the remainder of 2016 as we continue taking steps to conserve our available resources.
As a development-stage biopharmaceutical company, Catalyst had no revenues in the second quarter of 2016 and 2015 or the first six months of 2016 and 2015.
At June 30, 2016, Catalyst had cash and cash equivalents, certificates of deposit and short-term investments of $48.0 million and no debt. Catalyst believes that these resources give it sufficient runway for at least the next year. However, until Catalyst finalizes the details and logistics of its required confirmatory study evaluating Firdapse for the treatment of LEMS, it will be difficult for Catalyst to provide more details regarding how far its existing resources will take it. Notwithstanding, and while there can be no assurance, Catalyst continues to believe that its currently available resources will be sufficient to complete the development of Firdapse and get to an accepted NDA submission for Firdapse without the need for additional financing. (Original Source)
Shares of Catalyst Pharmaceutical closed today at $0.788, up $0.018 or 2.29%. CPRX has a 1-year high of $4.52 and a 1-year low of $0.51. The stock’s 50-day moving average is $0.77 and its 200-day moving average is $1.00.
On the ratings front, CPRX has been the subject of a number of recent research reports. In a report issued on June 14, H.C. Wainwright analyst Andrew Fein reiterated a Buy rating on CPRX. Separately, on April 26, Piper Jaffray’s Charles Duncan downgraded the stock to Hold and has a price target of $1.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Andrew Fein and Charles Duncan have a total average return of 14.5% and -6.4% respectively. Fein has a success rate of 49% and is ranked #186 out of 4105 analysts, while Duncan has a success rate of 42.5% and is ranked #3827.
Catalyst Pharmaceuticals, Inc. is a biopharmaceutical company. It focuses on development and commercialization of prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette’s disorder.