StarGen (SAR422459) / Oxford Biomedica - LARVOL DELTA

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration (clinicaltrials.gov) - P2 | N=27 | Active, not recruiting | Sponsor: Sanofi | Phase classification: P1/2 ➔ P2

Phase classification • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

The Promising Prospective Technology for Biofuel Production with Mixed Cultures Using Sorghum Grain (Sorghum bicolor (L.) Moench) Grown in a Temperate Climate: Characteristics of Fermentation Factors and Volatile Compounds Analysis. (PubMed, J Agric Food Chem) - "Analyses revealed that the granular starch fermentation method (with Stargen 002) combined with mixed cultures yielded higher bioethanol by about an average of 5% compared to single cultures...Distillates from fermentation with mixed cultures contained higher amounts of volatile byproducts (9.04-11.38 g/L) than single bacterial cultures (4.10-6.68 g/L) but less than single yeast cultures (9.27-13.14 g/L). These findings indicate the potential of mixed cultures in granular starch fermentation, at the same time suggesting that challenges such as selecting microbial consortia, accelerating the process, and ensuring consistent yields must be solved for widespread industrial application."

Journal

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration (clinicaltrials.gov) - P1/2 | N=27 | Active, not recruiting | Sponsor: Sanofi | Trial completion date: Aug 2034 ➔ Aug 2033 | Trial primary completion date: Aug 2034 ➔ Aug 2033

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Bioethanol production from sorghum grain with Zymomonas mobilis: Increasing the yield and quality of raw distillates. (PubMed, J Sci Food Agric) - "The study emphasizes the great potential of bioethanol production from sorghum with Z. mobilis using granular starch hydrolyzing enzyme Stargen 002, which leads to reduce water and energy consumption, especially when energy sources are strongly related to global climate change."

Journal

Three-year safety results of SAR422459 (EIAV-ABCA4) gene therapy in patients with ABCA4-associated Stargardt disease: An open-label dose-escalation phase I/IIa clinical trial, cohorts 1-5. (PubMed, Am J Ophthalmol) - "Subretinal treatment with EIAV-ABCA4 was well tolerated with only one case of ocular hypertension. No clinically significant changes in visual function tests were found to be attributable to the treatment. However, 27% of treated eyes showed exacerbation of RPE atrophy on FAF. There was significant reduction in macular flecks in one treated eye from the highest dose cohort. Additional follow-up and continued investigation in more patients will be required to fully characterize the safety and efficacy of EIAV-ABCA4."

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

Structural and spectral properties of generative models for synthetic multilayer air transportation networks. (PubMed, PLoS One) - "Besides a thorough study of the topological aspects of the ATNs created by the three models, our major contribution lays on an unprecedented investigation of their spectral characteristics based on Random Matrix Theory and on their resilience analysis based on both site and bond percolation approaches. Results have shown that ANGEL outperforms STARGEN and BINBALL to better capture the complexity of real-world ATNs by featuring the unique properties of building a multiplex ATN layer by layer and of replicating layers with point-to-point structures alongside hub-spoke formations."

Journal

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration (clinicaltrials.gov) - P1/2; N=27; Active, not recruiting; Sponsor: Sanofi; Trial completion date: Aug 2033 ➔ Aug 2034; Trial primary completion date: Aug 2033 ➔ Aug 2034

Clinical • Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Macular Degeneration • Ophthalmology • Retinal Disorders

Conversion of Starchy Waste Streams into Polyhydroxyalkanoates Using Cupriavidus necator DSM 545. (PubMed, Polymers (Basel)) - "Cupriavidus necator DSM 545, a well-known producer of PHAs, was adopted in a simultaneous saccharification and fermentation (SSF) process through an optimized dosage of the commercial amylases cocktail STARGEN™ 002. Broken rice was found to be the most promising carbon source with PHAs levels of up to 5.18 g/L. This research demonstrates that rice and sweet potato waste are low-cost feedstocks for PHAs production, paving the way for the processing of other starchy materials into bioplastics."

Journal

The comprehensive analysis of sorghum cultivated in Poland for energy purposes: Separate hydrolysis and fermentation and simultaneous saccharification and fermentation methods and their impact on bioethanol effectiveness and volatile by-products from the grain and the energy potential of sorghum straw. (PubMed, Bioresour Technol) - "The calorific value of sorghum straw was also measured, and amounted to 16,050-16,840 kJ/kg. The results have demonstrated the high value of sorghum as grain for bioethanol production and as straw as a valuable feedstock for forming pellets or briquettes."

Journal • Biosimilar

SAR422459: Data from P1/2 trial (NCT01736592) in Stargardt’s macular degeneration in 2034 (Sanofi) - Q3 2018 Results

P1/2 data • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

SAR422459: Regulatory submission in Stargardt disease in 2022 or later (Sanofi) - Q3 2018 Results

Regulatory • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Oxford Biomedica: Biotech and Money Investival Showcase (Biotech and Money Investival Showcase, Oxford Biomedica) - Anticipated initiation of pivotal P2b/P3 trial in Stargardt disease by the end of 2018

Anticipated new P2/3 trial • Inherited Retinal Dystrophy • Ophthalmology

Oxford Biomedica: Biotech and Money Investival Showcase (Biotech and Money Investival Showcase, Oxford Biomedica) - Anticipated initiation of pivotal P2b/P3 trial in Stargardt disease by the end of 2018

Anticipated new P2/3 trial • Inherited Retinal Dystrophy • Ophthalmology

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration (clinicaltrials.gov) - P1/2; N=27; Active, not recruiting; Sponsor: Sanofi; N=46 ➔ 27

Clinical • Enrollment change • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Gene Therapies • Genetic Disorders • Macular Degeneration • Ophthalmology • Rare Diseases • Retinal Disorders

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration (clinicaltrials.gov) - P1/2; N=46; Active, not recruiting; Sponsor: Sanofi; Trial completion date: Jun 2034 ➔ Aug 2033; Trial primary completion date: Jun 2034 ➔ Aug 2033

Trial completion date • Trial primary completion date

The potential of fiber-depleted starch concentrate produced through air currents assisted particle separation of barley flour in bio-ethanol production. (PubMed, Bioresour Technol) - "Interestingly, simultaneous saccharification and fermentation of BSC using a 0.25X dose of STARGEN™ 002 alone enabled efficient ethanol production, though a requirement for phosphorus supplementation was identified. This study proposes a biorefining strategy that supports the generation of a value-added co-product, starch concentrate, while significantly reducing the enzyme requirements for bioethanol production."

Journal

SAR422459: Data from P1/2 trial (NCT01736592) in Stargardt’s macular degeneration in 2034 (Sanofi) - Q3 2019 Results

P1/2 data

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration (clinicaltrials.gov) - P1/2; N=46; Active, not recruiting; Sponsor: Sanofi; Enrolling by invitation ➔ Active, not recruiting

Clinical • Enrollment closed

Phase I/II Study of SAR422459 in Patients With Stargardt's Macular Degeneration (clinicaltrials.gov) - P1/2; N=27; Terminated; Sponsor: Sanofi; N=41 ➔ 27; Recruiting ➔ Terminated; Study stopped not for safety reasons. Due to review of clinical development plans and priorities, Sponsor decided to stop development of the product.

Clinical • Enrollment change • Trial termination

SAR422459: Data from P1/2a trial (NCT01367444) for Stargardt's macular degeneration in 2019 (Sanofi) - Q2 2019 Results

P1/2 data

Consolidated bioprocessing of raw starch with Saccharomyces cerevisiae strains expressing fungal alpha-amylase and glucoamylase combinations. (PubMed, FEMS Yeast Res) - "When ateA was co-expressed with the previously reported Aspergillus tubingensis glucoamylase gene (glaA), the amylolytic S. cerevisiae Y294[AteA-GlaA] strain produced 45.77 g l-1 ethanol after 6 days. Ethanol production by this strain was improved with the addition of either 2.83 μl STARGENTM 002 (54.54 g l-1 ethanol and 70.44% carbon conversion) or 20 μl commercial glucoamylase from Sigma-Aldrich (73.80 g l-1 ethanol and 90.19% carbon conversion) This is the first report of an engineered yeast strain that can replace up to 90% of the enzymes required for raw starch hydrolysis, and thus contributes to the realisation of a CBP yeast for starch-based biofuel production."

Journal

SAR422459: Data from P1/2a trial (NCT01367444) for Stargardt's macular degeneration in 2020 (Sanofi) - Q1 2019 Results: Data from P1/2 trial (NCT01736592) in Stargardt’s macular degeneration in 2034

P1/2 data

Efficacy and toxicity of mustargen, vincristine, procarbazine and prednisone (MOPP) for the treatment of relapsed or resistant lymphoma in cats. (PubMed, J Feline Med Surg) - "MOPP is a safe protocol for the treatment of relapsed or refractory feline lymphoma, with a promising overall response rate and median remission time."

Clinical • Journal

Phase I/II Study of SAR422459 in Patients With Stargardt's Macular Degeneration (clinicaltrials.gov) - P1/2; N=41; Recruiting; Sponsor: Sanofi; Trial completion date: Feb 2020 ➔ Jun 2019; Trial primary completion date: Feb 2020 ➔ Jun 2019

Clinical • Trial completion date • Trial primary completion date

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration (clinicaltrials.gov) - P1/2; N=46; Enrolling by invitation; Sponsor: Sanofi; Recruiting ➔ Enrolling by invitation; Trial completion date: Feb 2035 ➔ Jun 2034; Trial primary completion date: Feb 2035 ➔ Jun 2034

Clinical • Enrollment status • Trial completion date • Trial primary completion date