Completed Phase 2 study of NYX-2925 in painful DPN -- did not meet primary endpoint, but NYX-2925 demonstrated robust analgesic activity in a large and mechanistically relevant patient sub-group
Reported positive data from interim analysis of Phase 2 exploratory neuroimaging study of NYX-2925 in patients with fibromyalgia -- study completion and data expected in 1H 2019
Confirmed novel NMDAr modulatory mechanism in humans through EEG studies in healthy volunteers
Initiated first-in-patient Phase 2a study of NYX-783 in patients with PTSD -- data expected in 1H 2020
Nearing completion of Phase 1 study of NYX-458in healthy volunteers -- data expected in 1H 2019
Conference call today at
“This past year was marked by a number of important clinical and preclinical study results showing activity and safety of the product candidates in our pipeline, informing our ongoing development of innovative therapies for CNS disorders that are vastly underserved by the treatments available today,” said
Fourth Quarter 2018 and Recent Highlights
- Initiated Phase 2a study of NYX-783: In
February 2019, Aptinyxinitiated a Phase 2a clinical study of NYX-783 to characterize its safety, pharmacokinetics, and effects on symptoms in patients with post-traumatic stress disorder (PTSD). The study will enroll approximately 144 patients randomly assigned to receive either placebo or 50 mg of NYX-783 over the course of the eight-week study. Multiple efficacy endpoints will be evaluated in the study to assess the impact of NYX-783 across the spectrum of PTSD symptoms. The company expects to report data from this study in the first half of 2020.
- Reported top-line results from Phase 2 study of NYX-2925 in painful DPN:
Aptinyxrecently completed a Phase 2 clinical study of NYX-2925 in patients with painful diabetic peripheral neuropathy (DPN). While the primary endpoint in the study was not met, the data generated from the study address key considerations for future development, including the safety of NYX-2925 in a DPN patient population, the most active dose level across a 20-fold dose range, activity across multiple endpoints relevant to chronic pain, and key patient characteristics to inform inclusion and exclusion criteria for future studies.
January 2019, the company reported top-line results from the study. NYX-2925 was safe and well-tolerated with no serious adverse events reported; all adverse events were mild or moderate in nature and similar to those observed in the placebo group. In the total study population of 300 patients, NYX-2925 did not demonstrate statistically significant separation from placebo on the primary endpoint, change in patients’ average daily pain scores on the Numeric Rating Scale (NRS). However, NYX-2925 exhibited signals of analgesic activity, particularly in the 50 mg dose group. In the 50 mg dose group, an average pain reduction from baseline to week four of 1.61 points on the NRS was observed. This reflected an incremental pain reduction of 0.38 points compared to placebo. When evaluating only those patients that were not taking a concomitant analgesic medication, this separation from placebo improved to 0.58 points. The effect in the 50 mg dose group across several endpoints improved in a linear manner over the four weeks of treatment and did not appear to have reached a plateau by week four.
March 2019, Aptinyxannounced additional analyses from the study, which focused on a sub-group of 127 patients with advanced DPN—a DPN diagnosis for four years or longer—representing over 42% of the patients in the study. These patients are particularly relevant to the mechanism of NYX-2925, which addresses the increasingly centralized pain these patients perceive when they experience chronic pain over a prolonged period of time. Consistent with the dose response observed in the total study population, the 50 mg dose group showed the greatest treatment benefits in this advanced DPN patient population. In the 50 mg dose group, an average pain reduction from baseline to week four of 1.93 points on the NRS was observed in advanced DPN patients. This reflected an incremental pain reduction of 1.21 points compared to placebo (p=0.004). Importantly, these effects were consistent across numerous endpoints in the study, including worst pain, pain on walking, sleep interference, and other pain and quality of life measures. Also consistent with the observations in the total study population, in advanced DPN patients the effects of NYX-2925 were even more pronounced in those patients not taking a concomitant analgesic medication—in the 50 mg group, the separation from placebo improved to 1.85 points in these patients.
Based on the detailed analysis of the study and the clear signals of analgesic activity that were observed in this mechanistically relevant patient sub-group, the company plans to initiate an additional clinical study of NYX-2925 for the treatment of painful DPN in the second half of 2019.
Aptinyxplans to present further details from the analysis of the data generated in this study at the American Pain Society Scientific Meeting in April 2019.
- Reported positive data from interim analysis of Phase 2 exploratory neuroimaging study of NYX-2925 in fibromyalgia: In
December 2018, Aptinyxreported positive results from an interim analysis of an exploratory neuroimaging study of NYX-2925 in patients with fibromyalgia. The design of the study was based on that of a post-approval study conducted with Lyrica® (pregabalin), an approved fibromyalgia treatment, which showed that changes in glutamate in certain brain regions are correlated with a reduction in clinical pain. In 11 patients, NYX-2925 was shown to induce statistically significant changes in these imaging biomarkers associated with pain processing and alleviation, including reductions in glutamatergic activity and connectivity in and between pre-specified brain regions. Reductions in patient-reported pain and other fibromyalgia symptoms were also observed. The study is ongoing, and the company expects to complete it and report data in the first half of 2019. Based on the results of the interim analysis, the company expects to initiate a larger Phase 2 study in the second half of 2019.
- Reported positive data from exploratory clinical studies evaluating biomarkers of NMDAr-mediated pharmacodynamic activity of NYX-2925: In
November 2018, Aptinyxreported results from two exploratory clinical studies, conducted in healthy volunteers, in which NYX-2925 was shown to have rapid and persistent effects on NMDAr-mediated pathways in the brain, evaluated using EEG measures. In the studies, NYX-2925 was shown to have clear effects on synaptic plasticity and sleep architecture, validating the results observed in preclinical studies and providing the first clinical evidence of pharmacodynamic activity of a small-molecule product candidate from the Aptinyxdiscovery platform.
- Presenting detailed results of NYX-2925 Phase 2 study in painful DPN at APS Scientific Meeting in
- Completion of, and reporting data from, NYX-2925 exploratory Phase 2 study in fibromyalgia in 1H 2019.
- Completion of, and reporting data from, Phase 1 study of NYX-458 in healthy volunteers in 1H 2019.
- Completion of, and reporting top-line data from, Phase 2a first-in-patient study of NYX-783 in PTSD in 1H 2020.
Fourth Quarter and Full Year 2018 Financial Results
Cash Position: Cash and cash equivalents were
Collaboration and Grant Revenue: Revenue was
Research and Development (R&D) Expenses: R&D expenses were
General and Administrative (G&A) Expenses: G&A expenses were
Net Loss: For the fourth quarter of 2018, net loss was
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the company’s business plans and objectives, including future plans or expectations for NYX-2925, therapeutic effects of the company’s product candidates, expectations regarding the design, implementation, timing, and success of its current and planned clinical studies, the timing for the company’s receipt of data from its clinical studies, expectations regarding its preclinical development activities, and expectations regarding its uses and sufficiency of capital. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of the company’s product candidate development activities and planned clinical studies; the company’s ability to execute on its strategy; positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies; regulatory developments in
CONDENSED BALANCE SHEETS
|Assets||December 31, 2018||December 31, 2017|
|Cash and cash equivalents||$||150,637||$||92,136|
|Prepaid expenses and other current assets||1,784||1,960|
|Total current assets||153,251||95,033|
|Property and equipment and other long-term assets||2,363||2,289|
|Liabilities and stockholders’ equity (deficit)|
|Accrued expenses and other current liabilities||3,996||2,835|
|Total current liabilities||5,885||4,372|
|Other long-term liabilities||418||282|
|Convertible preferred stock||—||132,386|
|Stockholders’ equity (deficit)||149,311||(39,718||)|
|Total liabilities and stockholders’ equity (deficit)||$||155,614||$||97,322|
CONDENSED STATEMENTS OF OPERATIONS
(in thousands, except per share data)
|Three Months Ended
|Collaboration and grant revenue||$||1,039||$||1,185||$||6,574||$||4,962|
|Research and development||10,928||6,714||48,788||31,644|
|General and administrative||4,822||1,733||12,674||5,551|
|Total operating expenses||15,750||8,447||61,462||37,195|
|Loss from operations||(14,711||)||(7,262||)||(54,888||)||(32,233||)|
|Net loss and comprehensive loss||$||(14,093||)||$||(7,227||)||$||(53,281||)||$||(32,068||)|
|Net loss per share - basic and diluted||$||(0.42||)||$||(1.36||)||$||(2.64||)||$||(6.17||)|
|Weighted average shares outstanding - basic and diluted||33,286||5,305||20,199||5,196|
Stern Investor Relations, Inc.
Source: Aptinyx Inc.