New Medicines



New molecular entity

Development and Regulatory status

Jun 21Development discontinued [9].
Jun 21Biogen is waiting to evaluate the data from the STAR trial fully before deciding on its future, but it seems likely that with a comprehensive failure across the board it will be discontinued [8].
Jun 19Biogen acquires Nightstar Therapeutics [5].
Jun 18AAV-REP1 receives Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, based on the clinical data from a PI/II trial [3].
Jan 15NightstaRx receives orphan drug designations from the US FDA and the EMA for treatment of choroideremia [3].


Gene therapy product using an adeno-associated virus (AAV) vector encoding the Rab escort protein 1 (REP1). The procedure involves an injection of AAV under the retina with a very narrow needle under local anaesthetic by a retinal surgeon.
Prevalence of choroideremia is estimated to be 1 in 50,000 to 100,000 people. However, it is likely that this condition is underdiagnosed because of its similarities to other eye disorders. Choroideremia is thought to account for approximately 4% of all blindness [1]

Further information


Trial or other data

Jun 21Timrepigene fails to meet its primary or secondary treatment objectives in the PIII STAR study [8].
Feb 20PIII STAR trial is no longer recruiting and due to complete collection of primary outcome data in Nov 20 [7].
Dec 19UK trial sites: PIII NCT03496012 (STAR) - Manchester, Oxford [6]
Oct 18PI/II study published in Nature (NCT01461213). Despite complications in two patients, visual acuity improved in the 14 treated eyes over controls (median 4.5 letter gain, versus 1.5 letter loss, P = 0.04), with 6 treated eyes gaining more than one line of vision (>5 letters). The results suggest that retinal gene therapy can sustain and improve visual acuity in a cohort of predominantly late-stage choroideremia patients in whom rapid visual acuity loss would ordinarily be predicted [5].
Oct 18Newspapers report that single injection restores the sight of people with genetic blindness. Scientists have hailed the results of the world first gene therapy trial for blindness after 14 patients with one of the most common inherited forms of the disease (choroideremia) experienced significant gains or arrested deterioration of their vision. Of the 12 who received the treatment without suffering any complications, all have demonstrated improved or maintained vision since having the injection, some as long as five years ago [4].
Jan 18PII GEMINI trial to evaluate safety and efficacy of bilateral, sequential sub-retinal injection of AAV-REP1 in 15 male patients with choroideremia in the US & Germany starts (EudraCT2017-002395-75; NSR-REP-02) [3].
Sep 17PIII long-term follow-up study to evaluate the safety and efficacy of a sub-retinal injection of AAV2-REP1 in patients with choroideremia treated previously with adeno-associated viral vector encoding rab escort protein-1 (aav2-rep1) in an antecedent study starts (NSR-CHM-OS2; EudraCT2017-003104-42). The study intends to enrol approximately 111 patients in the US and Germany [3].
Aug 16PII REGENERATE trial to evaluate safety and efficacy of AAV-REP1 in 30 male patients with choroideremia in the Uk starts (REGEN2015; NCT02407678) [3].
Jul 16PIII STAR trial to investigate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in patients with choroideremia starts (NCT03496012). The primary endpoint of the study is change from baseline in best corrected visual acuity (BCVA). Approximately 140 male patients will be enrolled and randomised into one of three study arms: 56 patients receiving a high-dose of NSR-REP1 in one-eye; 28 patients receiving a low-dose of NSR-REP1 in one-eye; and 56 patients receiving no treatment (no-sham, parallel control arm). Enrolment will take placfe in the US, Canada, Finland, Germany, UK and Netherlands . Collection of primary outcome data is due to complete Mar 20 [2].
Jun 15Three PII studies start. PII THOR trial to evaluate safety and efficacy of AAV-REP1 in 6 male patients with choroideremia in Germany (NCT02671539); PII trial in six patients with choroideremia at the University of Miami Miller School of Medicine (NCT02553135); PII trial evaluating a single dose of AAV2 REP1 administered via a subretinal injection (NCT02077361) involving six patents in Canada [3].
Oct 11PI/II trial XIRIUS evaluating safety and tolerability of AAV2 REP1, in 14 patients with choroideremia in the UK completes (NCT01461213). The primary objectives of the trial were the safety and tolerability of the AAV2 REP1 vector administered at two differed doses to the retina [3]

Evidence based evaluations