Neurocrine Biosciences and Voyager Therapeutics Announce Phase I Results for VY-AADC in Patients with Parkinson's Disease at the American Academy of Neurology Annual Meeting
Treatment with VY-AADC improved good ON time (ON time without troublesome dyskinesia) by 1.7 hours from baseline and reduced OFF time by 2.2 hours at 12 months from baseline in patients with Parkinson's disease. Exploratory analyses in four of the eight patients with low or no dyskinesia or absence of impulse control disorder (ICD) at baseline demonstrated a greater improvement in motor function including a 3.2-hour improvement in good ON time at 12 months. Infusions of VY-AADC were well tolerated with no serious adverse events (SAEs) reported. These Phase I results show that the posterior trajectory is an additional surgical delivery route in patients with Parkinson's disease.
"The results from this Phase I trial in patients with Parkinson's disease provide further evidence that VY-AADC administration can allow neurons in the brain to convert levodopa to dopamine and improve motor function," said Eiry W. Roberts, M.D., Chief Medical Officer at
VY-AADC Motor Function Results from the Phase I (PD-1102) Trial
The PD-1102 trial includes eight patients with advanced Parkinson's disease. On average, patients' baseline characteristics in PD-1102 were consistent with patients' baseline from a separate, ongoing Phase Ib trial (PD-1101) employing a frontal (i.e., from the top of the head) surgical delivery approach. Two patients in PD-1102 were identified as having impulse control disorder while no patients were identified as having impulse control disorder in PD-1101. At baseline, patients' mean good ON time was 9.1 hours and mean OFF time was 6.8 hours.
Treatment with VY-AADC improved patients' motor function from baseline to twelve months across multiple assessments. These assessments include patient self-reported diary entries of ON and OFF times (including good ON time), Unified Parkinson's Disease Rating Scales, and activities of daily living measures. In addition, patients' reported an ability to maintain motor function with less Parkinson's disease medication, as patients' reported a mean 28% reduction in the dosage of Parkinson's disease medication (measured as levodopa equivalents) at six months and at twelve months from a baseline mean of 1,500 mg/day.
Treatment with VY-AADC improved patients' good ON time by 1.7 hours from baseline and reduced OFF time by 2.2 hours from baseline to 12 months. Exploratory analyses in PD-1101 suggested that patients with high dyskinesia or an ICD at baseline may show different outcomes, especially in patient-reported diary measures. A clinical assessment of the subgroup of patients (n=4) with no or low baseline dyskinesia as measured by the Unified Dyskinesia Rating Scale score (≤ 30) and absence of ICD at baseline as determined by the investigator, indicated that VY-AADC improved good ON time from baseline by 3.2 hours and reduced OFF time by 3.2 hours in patients at 12 months.
In addition to motor function, VY-AADC improved patients' quality of life as measured by the patient-reported 39-item Parkinson's Disease Questionnaire (PDQ-39). For PDQ-39, VY-AADC improved (reduced) patients' score by a mean change from baseline to 12 months of -7.6. Infusions of VY-AADC have been well tolerated in the eight patients treated in PD-1102 with no serious adverse events (SAEs) reported.
About the Phase II RESTORE-1 Clinical Trial
Based on the results from PD-1101 and PD-1102, Voyager initiated RESTORE-1, a Phase II, randomized, placebo-surgery controlled, double-blinded, multi-center, clinical trial to evaluate the safety and efficacy of VY-AADC in patients who have been diagnosed with Parkinson's disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day as measured by a validated self-reported patient diary.
For more information about the RESTORE-1 clinical trial, including eligibility criteria, please visit restore1study.com.
In the first quarter of 2019,
About Parkinson's Disease and VY-AADC
Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disease that affects approximately one million people in the U.S. and six million people worldwide1. Parkinson's disease is characterized by a loss of dopamine and its function. Dopamine is a chemical "messenger" that is produced in the brain and is involved in the control of movement. Dopamine is made in the brain when the enzyme AADC (Aromatic l-amino acid decarboxylase) converts the chemical levodopa to dopamine. Levodopa, AADC, and dopamine are each present at normal levels in healthy people. As Parkinson's disease worsens, there is less AADC enzyme in parts of the brain where it is needed to convert levodopa to dopamine. When this happens, patients' motor function may worsen with a less predictable response to medications.
VY-AADC, an investigational gene therapy, is designed to put the AADC enzyme into brain cells where it can convert levodopa to dopamine. To do this, the AADC gene is delivered inside a transporter called "adeno-associated viral vector" (AAV). Interim results from an open-label Phase Ib trial demonstrated that administration of VY-AADC to the putamen using intraoperative monitoring with MRI facilitated targeted delivery of the investigational gene therapy with dose-dependent increases in AADC enzyme expression and improvements in clinical measures and has been well-tolerated to date.
Neurocrine Biosciences Forward-Looking Statements
In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are risks that the product candidates licensed from Voyager may not obtain regulatory approval from the
Voyager Forward-Looking Statements
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "might," "will," "would," "should," "expect," "plan," "anticipate," "believe," "estimate," "undoubtedly," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the initiation, timing, progress, activities, goals and reporting of results of its preclinical programs and clinical trials and its research and development programs, the potential benefits and future operation of the collaboration agreements with Sanofi Genzyme,
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Neurocrine Biosciences Media & Investor Relations: Navjot Rai, Director, Corporate Communications, 858-617-7623, IR@neurocrine.com; or Voyager Therapeutics Media & Investor Relations: Matt Osborne, Vice President of Corporate Affairs, Communications and Investor Relations, 857-259-5353, firstname.lastname@example.org