Catalyst Pharmaceuticals to License North American Rights to Vamorolone for Duchenne Muscular Dystrophy from Santhera Pharmaceuticals
Vamorolone is Currently Under Review with the FDA (PDUFA Date of
FDA has Granted Fast Track and Orphan Drug Designation for Vamorolone to Treat Duchenne Muscular Dystrophy
Catalyst Expects to Launch Vamorolone in the
Agreement to Include Commercial and Future Development Rights for Vamorolone for Additional Indications in
Transaction Also Includes a
Vamorolone was studied for the indication of DMD, a rare neuromuscular disorder. Santhera filed a New Drug Application (“NDA”) for vamorolone, supported by clinical data from the positive pivotal Phase 2b VISION-DMD study, which met the primary endpoint with statistical significance over placebo. The vamorolone NDA has been accepted and is currently under review by the
“We are extremely pleased to be entering into a partnership with Santhera on the vamorolone program and the prospect of delivering a potential therapy with a desirable profile for DMD patients," said
Under the terms of the agreement, Catalyst will make a
The agreement is structured as an all-cash transaction with no financing contingencies. The transaction is expected to be completed in the third quarter of 2023, subject to customary closing conditions and regulatory clearances in
Vamorolone is an investigational drug candidate with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity and as such, is considered a dissociative anti-inflammatory drug [2-5]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore vamorolone could emerge as an alternative to existing corticosteroids, the current standard of care in children and adolescent subjects with DMD. In the pivotal VISION-DMD study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile . The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity.
Vamorolone has been granted Orphan Drug status for DMD in the
 Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link.
 Mah JK et al (2022). JAMA Netw Open. 2022;5(1):e2144178. doi:10.1001/jamanetworkopen.2021.44178. Link.
 Guglieri M et al (2022) JAMA. doi:10.1001/jama.2022.4315
 Heier CR et al (2019). Life Science Alliance DOI: 10.26508
 Liu X et al (2020).
About Duchenne Muscular Dystrophy
DMD is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD.
For further information, please visit www.santhera.com.
About Catalyst Pharmaceuticals
With exceptional patient focus, Catalyst is committed to developing and commercializing innovative first-in-class medicines that address rare neurological and epileptic diseases. Catalyst's flagship
For Full Prescribing and Safety Information for FIRDAPSE®, please visit www.firdapse.com. For Full Prescribing Information, including Boxed WARNING for FYCOMPA®, please visit www.fycompa.com. For more information about Catalyst Pharmaceuticals, Inc., visit the Company's website at www.catalystpharma.com.
This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether the proposed agreement to license vamorolone and make the equity investment will be completed, (ii) whether the NDA for vamorolone will be approved by the PDUFA date, or at all, (iii) whether the licensing agreement, if it is completed and the NDA for vamorolone is approved by the FDA, can be successfully commercialized by Catalyst in the territory, (iv) whether if the vamorolone is commercialized by Catalyst, the results will prove to be accretive to Catalyst, (v) whether Catalyst and Santhera will successfully develop additional indications for vamorolone and obtain the ability to commercialize the product for those additional indications, (vi) whether Catalyst will, if vamorolone is commercialized by Catalyst, be successfully integrated into Catalyst's business activities, and (vii) those factors described in Catalyst's Annual Report on Form 10-K for the 2022 fiscal year, Catalyst's Quarterly Report on Form 10-Q for the first quarter of 2023, and Catalyst's other filings with the SEC, could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.
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