Aptinyx PTSD Trial And Other News: The Good, Bad And Ugly Of Biopharma

Aptinyx PTSD Trial And Other News: The Good, Bad…

Aptinyx Reports Completion of Enrolment in NYX-783 Trial

Aptinyx (APTX) reported completion of enrollment in its NYX-783 trial. This phase 2 study aims to assess the safety and efficacy of NYX-783 in treating patients with post-traumatic stress disorder (PTSD). The company expects the data to be out later this year. The results will be used for informing further development for the drug candidate.

The Phase 2 exploratory study is a placebo controlled, multi-center, double blind, Sequential Parallel Comparison Design study. It aims to enroll 160 patients which will then be randomized into three different treatment cohorts. These cohorts will be given placebo, NYX-783 10 mg, or NYX-783 50 mg orally once-daily. Norbert Riedel, Ph.D., president and chief executive officer of Aptinyx said, “The completion of enrollment in our Phase 2 exploratory study of PTSD represents an important clinical development milestone for our company. We look forward to completing this study in the coming months and evaluating the data to determine the most appropriate next steps in development to address the unmet need in PTSD.”

The Phase 2 study is designed to last ten to thirteen weeks which will consists of a screening period, two treatment periods and a safety follow-up period. Patients randomized to receive the drug candidate will be administered over the period of four consecutive weeks. The primary endpoint is the change in CAPS-5 total score and sub-scores from baseline to the end of each of the four-week treatment periods. CAPS-5 or Clinician-Administered PTSD Scale for the DSM-5 is a standardized structured clinical interview designed to measure and diagnose symptom severity and frequency in PTSD patients.

The secondary endpoints for the study are related to the safety and tolerability of the drug candidate. The study also aims to identify the potential benefits of NYX-783 on various symptoms, such as sleep quality, cognitive function, anxiety, and depression in patients. The disorder is estimated to impact nearly seven percent in the general population but the incidence of this disorder is much higher in populations at risk for exposure to trauma, such as military service members and first responders.

Currently, PTSD patients are treated with a limited number of options available. There are only two approved conventional SSRI antidepressants which may be used for treating PTSD. However, these medications have limited efficacy and are prone to several side effects. Further, they also only provide symptomatic relief, without actually curing the underlying reasons.

NYX-783 is a novel, oral NMDA receptor modulator. The preclinical studies of the drug candidate showed that it had robust performance in psychiatry models, models of substance abuse and models of fear extinction. In its Phase 1 clinical study, the drug candidate exhibited strong central nervous system exposure. It also had favorable safety and tolerability profile and did not had any serious negative impact for a broad dose range. NYX-783 has been given Fast Track tag by the FDA for treating PTSD.

Aptinyx Inc. is a clinical-stage biopharmaceutical company. It is mainly engaged in discovering and developing proprietary synthetic small molecules for treating brain and nervous system disorders. The company also has a platform for discovering compounds which work by modulating NMDA receptors rather than blocking or over-activating them. These compounds also help in enhancing synaptic plasticity, which forms the base of neural cell communication.

Aptinyx has robust development pipeline with three product candidates in different phases. These drug candidates are mainly related to treating central nervous system indications such as PTSD, chronic pains and Parkinson’s disease.

Ultragenyx Receives FDA Nod for Crysvita

Ultragenyx Pharmaceutical Inc. (RARE) announced that the FDA has given its seal of approval to Crysvita for treating fibroblast growth factor 23 (FGF23)-related hypophosphatemia in tumor-induced osteomalacia. The drug candidate is a human antibody that works by blocking extra activity of FGF23, a hormone which is responsible for phosphate urinary excretion and suppressing active vitamin D production by the kidney.

Crysvita has already been approved by the FDA for treating X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older. The approval had come along in April, 2018 and the XLH indication was further expanded later in 2019 to include infants as young as six months of age. Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx said, “For approximately half of all individuals with TIO, surgical removal of the tumors is not possible, leaving these patients with no other treatment options. The FDA approval of Crysvita marks the first treatment option that addresses the cause of the severe hypophosphatemia and osteomalacia resulting from these rare tumors.”

Ultragenyx had used the data from its two single-arm Phase 2 studies for supporting its application to the FDA. The first study was a 144-week trial involving 14 adult patients conducted in the United States while the second was an 88-week trial with 13 adult patients in Japan and South Korea. Both the studies found Crysvita to enhance serum phosphorus and serum 1,25-dihydroxyvitamin D levels. The whole body scans showed declined tracer uptake with long term treatment.

Ultragenyx has been collaborating with Kyowa Kirin for developing and commercializing Crysvita globally.

Epizyme Receives Accelerated FDA Approval for Tazemetostat

Epizyme (EPZM) reported that the FDA has approved its tazemetostat drug candidate for treating adult patients with relapsed/refractory follicular lymphoma (NYSE:FL) whose tumors are positive for an EZH2 mutation. These patients also had been given minimum two prior systemic therapies and included adult patients with relapsed/refractory FL with no practical treatment options.

The drug candidate was approved under the FDA’s accelerated approval pathway with a priority review. Such approval is subjected to certain post-marketing requirements including a confirmatory study to validate the treatment benefits. TAZVERIK was initially given accelerated approval by FDA on January 23, 2020 for treating adult and pediatric patients aged 16 years and older suffering from metastatic or locally advanced epithelioid sarcoma which is not eligible for complete resection.

Epizyme is currently conducting a single global, randomized, adaptive confirmatory trial for assessing the combination of TAZVERIK with “R2” (Revlimid® plus rituximab) for treating FL patients in the second-line or later treatment setting. The company plans to enroll nearly 500 FL patients. Dr. Shefali Agarwal, chief medical officer of Epizyme said, “Based on this label, physicians will have the ability to use their clinical discretion to prescribe TAZVERIK for their relapsed or refractory patients regardless of EZH2 mutational status and without regard to a specific line of treatment where other options are not satisfactory.”

TAZVERIK is a methyltransferase inhibitor and is approved for treating various indications including the treatment of adult patients with relapsed or refractory follicular lymphoma who have no satisfactory alternative treatment options. The most common adverse reactions associated with the drug are pain, fatigues and nausea among others.

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