SPK-7001 / Roche - LARVOL DELTA

Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations (clinicaltrials.gov) - P1/2 | N=15 | Completed | Sponsor: Spark Therapeutics | Active, not recruiting ➔ Completed

Trial completion • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

AAV2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial. (PubMed, Ophthalmology) - "VA was within 15 letters of baseline after the subfoveal AAV2-hCHM injections in 13/15 (87%) of the patients. Acute foveal thinning with unchanged perifoveal function in one patient and macular hole in a second suggests foveal vulnerability to the subretinal injections. Longer observation intervals will help establish the significance of the minor differences in sensitivities and rate of disease progression observed between intervention and control eyes."

Journal • P1/2 data • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Short-term Assessment of Subfoveal Injection of Adeno-Associated Virus-Mediated hCHM Gene Augmentation in Choroideremia Using Adaptive Optics Ophthalmoscopy. (PubMed, JAMA Ophthalmol) - "To use adaptive optics scanning light ophthalmoscopy (AOSLO) to assess the short-term integrity of the cone mosaic following subretinal injections of adeno-associated virus vector designed to deliver a functional version of the CHM gene (AAV2-hCHM) in patients with choroideremia...Minor foveal thinning observed following surgery corresponds with short-term cone outer segment shortening rather than cone cell loss. Foveal cone loss in 1 participant raises the possibility of individual vulnerability to the subretinal injection."

Journal • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

[VIRTUAL] Cone mosaic integrity in choroideremia following gene augmentation via subretinal injection of AAV2-hCHM (ARVO 2021) - "Cone mosaic integrity is maintained following subretinal delivery of AAV2-hCHM, providing strong evidence in support of the safety of the surgical injections and short-term exposure to this viral vector. Individual vulnerability to the subfoveal injection may account for the foveal cone loss observed in one patient. Future studies will evaluate the integrity of the cone mosaic following long-term AAV2-hCHM gene augmentation."

Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations (clinicaltrials.gov) - P1/2; N=15; Active, not recruiting; Sponsor: Spark Therapeutics; Trial completion date: Oct 2032 ➔ Oct 2022; Trial primary completion date: Oct 2032 ➔ Oct 2022

Clinical • Trial completion date • Trial primary completion date • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Spark Therapeutics: Annual Report 2015 (Spark Therapeutics) - Anticipated expiry of patent in US related to plasmid used in manufacture of SPK-CHM in 2032

Anticipated patent expiry • Inherited Retinal Dystrophy • Ophthalmology

"Nice list of upcoming catalysts$events next few months please add to it $LXRX $TSRO $GBT $ABUS $KPTI $NVAX $BLUE" - @tgtxdough 

Anticipated clinical data • Gene Therapies • Ophthalmology

SPK-7001: Additional analyses of earlier and later stage disease cohorts in P1/2 trial (NCT02341807) for choroideremia in Q4 2018 (Spark Therapeutics) - Corporate Overview

P1/2 data • Inherited Retinal Dystrophy • Ophthalmology

SPK-CHM: Anticipated patent expiry in US related to a certain plasmid used in the manufacture of SPK-CHM in 2032 (Spark Therapeutics) - Annual Report 2016

Anticipated patent expiry • Inherited Retinal Dystrophy • Ophthalmology

SPK-7001: “At interim analysis, 4/10 later-stage participants showing nonstatistically significant indications of efficacy on 1 or more endpoints” (Spark Therapeutics) - Corporate Presentation

P1/2 data • Inherited Retinal Dystrophy • Ophthalmology

Spark Therapeutics: Corporate Presentation (Spark Therapeutics) - Anticipated initial efficacy analysis from P1/2 trial (NCT02341807) for choroideremia in early 2017

Anticipated P1/2 data • Inherited Retinal Dystrophy • Ophthalmology

Short-term assessment of subfoveal injection of AAV2-hCHM gene augmentation in Choroideremia using adaptive optics ophthalmoscopy (ARVO 2020) - "Foveal cone loss in one patient raises the possibility of individual vulnerability to the subfoveal injection. Further observation is needed to evaluate long-term safety and efficacy."

Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations (clinicaltrials.gov) - P1/2; N=15; Active, not recruiting; Sponsor: Spark Therapeutics; Trial completion date: Oct 2019 ➔ Oct 2032; Trial primary completion date: Oct 2019 ➔ Oct 2032

Clinical • Trial completion date • Trial primary completion date

Subfoveal gene augmentation therapy for choroideremia: One-year results from a Phase I/II trial of AAV2-hCHM (ARVO 2019) - "Conclusions AAV2-hCHM was well-tolerated over a one year interval. Longer-term evaluation of the advancing edge of degeneration is needed to determine whether treatment changes the progression rate in these patients with preserved foveal function."

P1/2 data

AAV2-hCHM Subretinal Delivery to the Macula in Choroideremia: 2 year Results of an Ongoing Phase I/II Gene Therapy Trial (ARVO 2019) - "Residual islands of relatively preserved retina continued to shrink in both injected and uninjected eyes. Longer observation intervals are required to better evaluate the significance of these observations."

P1/2 data

Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations (clinicaltrials.gov) - P1/2; N=15; Active, not recruiting; Sponsor: Spark Therapeutics; Trial completion date: Jan 2019 ➔ Oct 2019; Trial primary completion date: Jan 2019 ➔ Oct 2019

Clinical • Trial completion date • Trial primary completion date

Spark Therapeutics reports 2018 financial results and recent business progress (Spark Therapeutics Press Release) - "In the year ended December 31, 2018, we recognized $64.7 million in total revenue, of which $27.0 million was net product sales of LUXTURNA and $37.8 million was contract revenue associated with our agreements with Pfizer and Novartis....These costs were partially offset by $7.9 million less in expenses related to LUXTURNA and $3.8 million less in expenses related to the SPK-FIX and SPK-CHM clinical programs."

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