Idebenone

GenSight Biologics Announces Initial Results from New Meta-Analyses on Visual Outcomes with LUMEVOQ® Gene Therapy at NANOS 2024

Retrieved on: 
Tuesday, March 12, 2024
Research, Neurology, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, Optical, Science, Mutation, Disease, Nadir, Patient, CRR, Eye, Idebenone, Natural history, Phase, Multimedia, Neuro-ophthalmology, ND4, Interval (mathematics), AAC, Medical imaging

The meta-analyses are the first to focus solely on patients with the m.11778G>A ND4 mutation, which is the most common mutation and one with a poor visual prognosis1.

Key Points: 
  • The meta-analyses are the first to focus solely on patients with the m.11778G>A ND4 mutation, which is the most common mutation and one with a poor visual prognosis1.
  • This gradient of recovery, based on the CRR measure of visual improvement, is observed at both eye level and patient level (response in one or both eyes).
  • There is no overlap in confidence intervals when LUMEVOQ® is compared to idebenone and to natural history, indicating a positive difference in visual outcomes.
  • Follow-up of patients in the Phase III REFLECT study of LUMEVOQ® is ongoing, with topline results at Year 4 of follow-up expected this month.

Santhera Announces Half-Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, September 7, 2023
Vamorolone, Marketing, European Medicines Agency, Lead, Prescription Drug User Fee Act, Committee, U.S. FDA, Patient, Committee for Medicinal Products for Human Use, News, Duchenne muscular dystrophy, Glucocorticoid, Becker muscular dystrophy, European Commission, EMA, CEST, PDUFA, Rare, FDA, Safety, Health care, LR, Duchenne, Publication, MAA, MHRA, CHMP, Catalyst Pharmaceuticals, Partnership, European Directive on Traditional Herbal Medicinal Products, NDA, BMD, EC, BST, Medical device, Pharmaceutical industry, Security (finance), Idebenone, EU, Chiesi Farmaceutici, LHON, Atlantic Ocean, Catalysis, DMD

Furthermore, we have secured a solid financial footing which allows us to press ahead toward our goal of European commercialization,” said Dario Eklund, CEO of Santhera.

Key Points: 
  • Furthermore, we have secured a solid financial footing which allows us to press ahead toward our goal of European commercialization,” said Dario Eklund, CEO of Santhera.
  • Total consideration to Santhera is up to USD 231 million (including equity investment) plus royalty payments from product sales.
  • After closing of the transaction in July 2023, Santhera received an upfront payment of USD 90 million (USD 75 million in cash and USD 15 million equity investment).
  • During the six months ending June 30, 2023, Santhera committed to the sale of Raxone/idebenone upon settlement of the French case and the transaction closed on July 28, 2023.

Santhera Completes Divestment of Raxone®/Idebenone Business to Chiesi Group

Retrieved on: 
Monday, July 31, 2023
Vamorolone, Marketing, LR, USD, EUR, Patient, FDA, Pharmaceutical industry, Chiesi Farmaceutici, Idebenone, LHON

53 LR

Key Points: 
  • 53 LR
    Santhera retains contingent value for LHON in U.S. and/or other indications worldwide
    Pratteln, Switzerland, July 31, 2023 – Santhera Pharmaceuticals (SIX: SANN) announces the full divestment of its Raxone®/idebenone business worldwide and for all indications to Chiesi Farmaceutici S.p.A., an international research focused healthcare group (Chiesi Group).
  • Under the terms of the agreement, Chiesi Group acquired all assets and certain liabilities related to idebenone in all indications worldwide, including Raxone in LHON, for which Chiesi already held exclusive license rights globally since 2019, except for North America and France.
  • The agreement simplifies the Raxone business significantly for both companies with Chiesi becoming the global brand owner while enabling Santhera to focus on the launch of vamorolone in Europe, subject to approval.
  • Chiesi Group also had the option to fully acquire said business outside North America.

Santhera Announces Preliminary Unaudited 2022 Annual Results Ahead of Full Report Publication by End of May and Provides Corporate Update

Retrieved on: 
Thursday, April 27, 2023
Vamorolone, Prescription Drug User Fee Act, Duchenne muscular dystrophy, Civil service commission, Education, Vitamin D deficiency, Health care, Glucocorticoid, European Medicines Agency, NDA, LR, European Directive on Traditional Herbal Medicinal Products, Biomarker, BMD, Committee, EMA, Vomiting, Depression, Prednisone, Publication, DMD, Vertebral compression fracture, PAMS, Patient, Annual report, Leber's hereditary optic neuropathy, European Commission, CEST, Syndrome, PDUFA, Committee for Medicinal Products for Human Use, MAA, FDA, LHON, CHF, EC, Safety, News, Bone fracture, MHRA, CHMP, BST, Cryptocurrency, Pharmaceutical industry, Medical device, Dietary supplement, Dentistry, EU, Idebenone

As permitted by SIX Exchange Regulation, the Company will publish the full 2022 Annual Report by the end of May.

Key Points: 
  • As permitted by SIX Exchange Regulation, the Company will publish the full 2022 Annual Report by the end of May.
  • The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin D deficiency.
  • SIX Exchange Regulation has permitted Santhera to publish its 2022 Annual Report by May 31, 2023, at the latest.
  • The Company plans to publish its audited 2022 Annual Report during May 2023.

Santhera Concludes Agreement with French Authorities on Raxone® Reimbursement and Plans to Submit a Request for an Early Access Program for Vamorolone

Retrieved on: 
Wednesday, February 8, 2023
MAA, File, LHON, Leber's hereditary optic neuropathy, Duchenne muscular dystrophy, European Medicines Agency, NDA, AAP, Patient, FDA, Vamorolone, DMD, EMA, ATU, Health insurance, Pharmaceutical industry, EU, Idebenone

In addition, the Company plans to submit a request in France in the near-term for an early access program for vamorolone for the treatment of Duchenne muscular dystrophy (DMD).

Key Points: 
  • In addition, the Company plans to submit a request in France in the near-term for an early access program for vamorolone for the treatment of Duchenne muscular dystrophy (DMD).
  • Shortly, Raxone will be on the list of reimbursed products in France, and Santhera can resume generating sales.
  • “We are glad to have reached an agreement with the French authorities on the matter of Raxone reimbursement.
  • Earlier in January, Santhera announced FDA acceptance of the new drug application (NDA) for vamorolone for DMD for filing.

Santhera Reports 2021 Annual Results

Retrieved on: 
Friday, June 10, 2022
MAA, Company, Greater, Workforce, US FDA, Prednisone, Commercial, SEAL, Review, Annual, COVID-19, Bone fracture, LR, Association, CMO, Marketing, CEST, CEO, Treasury, BST, Mass meeting, Duchenne muscular dystrophy, NDA, EGM, Annual report, Contract, Proxy, Highbridge Capital Management, DMD, Vamorolone, Patient, Congenital muscular dystrophy, LEROS, AGM, Pratteln, USD, LHON, Exercise, Cystic fibrosis, CHF, US, Program, LLC, Comment, CF, Therapy, FDA, Growth, Clinical trial, Safety, Tutoring, Cryptocurrency, Medical device, Pharmaceutical industry, Airline, Animal, Security (finance), Duchenne, Idebenone, EU

Based on the agreement with the French authorities, Santhera has supplied Raxone free of charge from August 2021 following its removal from the list of reimbursed drugs.

Key Points: 
  • Based on the agreement with the French authorities, Santhera has supplied Raxone free of charge from August 2021 following its removal from the list of reimbursed drugs.
  • In March 2021, Santhera announced promising clinical trial data from a Phase 1b trial of lonodelestat in cystic fibrosis (CF).
  • Additional details on the pipeline products and operational progress can be found in the Annual Report 2021.
  • CEO Dario Eklund, CFO Andrew Smith and CMO Dr. Shabir Hasham will discuss the 2021 financial results and comment on ongoing corporate developments.

Santhera Announces Phase 4 LEROS Trial with Raxone® Met Primary Endpoint in Patients with Leber’s Hereditary Optic Neuropathy

Retrieved on: 
Wednesday, June 23, 2021
Branches of biology, Leber's hereditary optic neuropathy, Idebenone, Organ systems, Proteins, Nervous system, Optic neuropathy, Leber, Peripheral neuropathy, Mitochondrial optic neuropathies

Pratteln, Switzerland, June 23, 2021 Santhera Pharmaceuticals (SIX: SANN) announces positive topline results from its long-term Phase 4 LEROS study with Raxone (idebenone) in the treatment of Lebers hereditary optic neuropathy (LHON).

Key Points: 
  • Pratteln, Switzerland, June 23, 2021 Santhera Pharmaceuticals (SIX: SANN) announces positive topline results from its long-term Phase 4 LEROS study with Raxone (idebenone) in the treatment of Lebers hereditary optic neuropathy (LHON).
  • The primary endpoint, proportion of eyes with clinically relevant benefit after 12 months treatment with Raxone versus untreated patients from an external control group, was met with high statistical significance (p=0.002).
  • In September 2015, Raxone received the marketing authorization from the European Medicines Agency (EMA) for the treatment of patients with Leber's hereditary optic neuropathy (LHON).
  • Santhera out-licensed rights to its first approved product, Raxone (idebenone), outside North America and France for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group.

GenSight Biologics Announces Publication of Indirect Comparison Showing Treatment Effect of LUMEVOQ® versus Natural History in Frontiers in Neurology

Retrieved on: 
Tuesday, June 1, 2021
Health, Medical devices, Genetics, Clinical trials, Pharmaceutical, Optical, Biotechnology, Health sciences, Health, Clinical research, Design of experiments, Antioxidants, Idebenone, Clinical trial, Leber's hereditary optic neuropathy, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), RESCUE and REVERSE, REALITY, GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC), RESCUE AND REVERSE, REALITY

Treated eyes refer to all eyes (LUMEVOQ and sham) from the RESCUE, REVERSE and CLIN06 trials (N=152 eyes from 76 patients).

Key Points: 
  • Treated eyes refer to all eyes (LUMEVOQ and sham) from the RESCUE, REVERSE and CLIN06 trials (N=152 eyes from 76 patients).
  • *A statistically significant difference between treated and natural history eyes is illustrated by the non-overlapping confidence intervals of the LOESS curves.
  • For a better characterization of LUMEVOQ efficacy, a natural history, or untreated, pool had to be assembled from patients carrying the same mutation as those in the clinical trials.
  • The natural history pool was formed from patients who were not treated with LUMEVOQ; they could, however, have been treated with idebenone.

Santhera Completes Capital Increase for Financing Arrangements

Retrieved on: 
Thursday, May 20, 2021
Antioxidants, Idebenone, Leber's hereditary optic neuropathy, XC2, Optic neuropathy, Leber, Share, Health, Branches of biology

b"Pratteln, Switzerland, May 20, 2021 \xe2\x80\x93 Santhera Pharmaceuticals (SIX: SANN) announces that it has issued 2,562,375 treasury shares.\nOn May\xc2\xa019, 2021, 2,562,375 shares were issued out of the existing authorized capital as treasury shares.

Key Points: 
  • b"Pratteln, Switzerland, May 20, 2021 \xe2\x80\x93 Santhera Pharmaceuticals (SIX: SANN) announces that it has issued 2,562,375 treasury shares.\nOn May\xc2\xa019, 2021, 2,562,375 shares were issued out of the existing authorized capital as treasury shares.
  • Santhera expects to use these shares for purposes of its financing arrangements, including the Senior Unsecured Convertible Bonds due 2024 (SAN21, CH0563348744) issued on May\xc2\xa04, 2021.
  • Santhera out-licensed ex-North American rights to its first approved product, Raxone\xc2\xae (idebenone), for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group.
  • For further information, please visit www.santhera.com .\nRaxone\xc2\xae is a trademark of Santhera Pharmaceuticals.\nFor further information please contact:\nThis communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG.

Santhera Announces First Trading Day of New Convertible Bonds on SIX Swiss Exchange

Retrieved on: 
Friday, May 7, 2021
Financial markets, Finance, Antioxidants, Idebenone, XC2, Money, Leber's hereditary optic neuropathy, SIX Swiss Exchange, Bond, Optic neuropathy, Convertible bond

b"Pratteln, Switzerland, May 7, 2021 \xe2\x80\x93 Santhera Pharmaceuticals (SIX:\xc2\xa0SANN) announces that trading of its CHF\xc2\xa030,270,375 Senior Unsecured Convertible Bonds due 2024 is starting today on the SIX Swiss Exchange.\nOn May\xc2\xa04, 2021, Santhera Pharmaceuticals Holding AG issued Senior Unsecured Convertible Bonds due 2024 (the 2021/24 Bonds) in the aggregate principal amount of CHF\xc2\xa030,270,375.

Key Points: 
  • b"Pratteln, Switzerland, May 7, 2021 \xe2\x80\x93 Santhera Pharmaceuticals (SIX:\xc2\xa0SANN) announces that trading of its CHF\xc2\xa030,270,375 Senior Unsecured Convertible Bonds due 2024 is starting today on the SIX Swiss Exchange.\nOn May\xc2\xa04, 2021, Santhera Pharmaceuticals Holding AG issued Senior Unsecured Convertible Bonds due 2024 (the 2021/24 Bonds) in the aggregate principal amount of CHF\xc2\xa030,270,375.
  • The 2021/24 Bonds have been admitted to trading on the SIX Swiss Exchange.
  • The first trading day is today, May\xc2\xa07, 2021.
  • Santhera out-licensed ex-North American rights to its first approved product, Raxone\xc2\xae (idebenone), for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group.