- Pivotal readout for vamorolone in Duchenne muscular dystrophy (DMD) expected in the second quarter of 2021
- Organizational restructuring to reduce workforce by 50%; annualized cost reductions of CHF 10 million
- Cost reductions combined with Highbridge facility amendment position Santhera for the readout of the VISION-DMD study, Santhera’s next key value inflection point
- Partnering opportunities under evaluation for vamorolone and lonodelestat in non-core indications and geographies to enable proactive portfolio management
Kristina Sjöblom Nygren , Chief Medical Officer and Head of Development, to leave Company by end 2020
Pratteln,
Vamorolone, the only dissociative steroid in development, is currently in a pivotal Phase 2b study in DMD patients with a 6-month readout expected in the second quarter of 2021. If successful, the results could lead to an NDA submission with the FDA in the fourth quarter of 2021 which is subject to a fast track review. Santhera currently anticipates to be first to market with a dissociative steroid in the US in 2022.
The refocusing on vamorolone, following discontinuation of Puldysa, necessitates a realignment of the organization to bring it in line with its new priorities and an extension of cash reach to advance the pipeline, above all vamorolone. Concluded amendments of the existing financing agreements with certain funds managed by
At the Executive Management level, Santhera will see a change with the departure of
“Moving forward as a Company with a focus on vamorolone, upon acquisition of global rights in all indications through agreements with Idorsia and ReveraGen and the termination of the Puldysa program, has forced us to take difficult decisions but at the same time has opened up new opportunities,” said
Reorganization to realize cost reduction and advance vamorolone towards approval and market entry
The adjustment of Santhera’s business activities is driven by the focus on vamorolone and the US, which is expected to be the first market for this drug candidate, and the need to extend cash reach to Santhera’s next major value enhancing inflection point in the second quarter of 2021. Santhera is reducing its workforce by more than 50 positions to 47 full-time equivalent (FTE) employees. This restructuring will result in one-time costs of approximately CHF 3 million against recurring annual cost reductions of CHF 10 million. In addition, external development, marketing and other operating costs will decrease significantly as activities related to the terminated Puldysa program are closed out.
Furthermore, the Company will incur a one-time non-cash expense of CHF 9 million resulting from an impairment of inventory and other assets related to Puldysa as well as the reduction in infrastructure use arising from the organizational restructuring. Following the impairment, annualized amortization costs will be reduced by approximately CHF 1 million.
Cash runway sufficient to reach next inflection point and continue operations as planned
The existing financing with Highbridge has been amended to provide up to CHF 15 million in senior secured notes exchangeable by Highbridge, with CHF 5 million available immediately and the balance subject to certain conditions. These amendments replace Highbridge's existing unused financing commitments and are required to allow further drawdown following the termination of the Puldysa program. The Company’s cash balances of CHF 10 million (as of
In addition to balance sheet related cash inflows, the Company is entitled to staggered milestone payments of up to EUR 49 million, if and when license holder
Santhera’s strategic near- and mid-term priorities
In summary, the near-term focus of Santhera will be on advancing its late-stage clinical drug candidates vamorolone and lonodelestat and securing sufficient funding to support operations beyond the second quarter of 2021. The Company expects a positive 6-month readout of the vamorolone VISION-DMD trial and a restructuring of the convertible bond to provide the basis to source additional capital aimed at meeting future needs. For the mid-term, the Company is pursuing proactive portfolio management strategy through outlicensing agreements for both vamorolone (in non-DMD indications and geographies outside the US and
About vamorolone and the path forward
Santhera recently announced the completion of a license assignment from Idorsia (SIX: IDIA) and the early exercise of a licensing option to vamorolone with
The 6-month topline data from the fully enrolled pivotal VISION-DMD (VBP15-004 [9]) study are expected in the second quarter of 2021, based on which a regulatory submission to the FDA is planned for the fourth quarter of 2021. Subject to FDA approval, Santhera anticipates to be the first to market with a dissociative steroid in the US in 2022. The European regulatory authorities require 12-month treatment data and a filing with the EMA could occur in the first half of 2022 followed by launch approximately one year later. The Company estimates the peak sales potential for vamorolone for the DMD indication alone to be in excess of USD 500 million in the US and the largest five EU countries combined.
In parallel to the DMD program, Santhera is pursuing partnering opportunities for vamorolone in additional indications outside DMD and in geographies outside the US and
Vamorolone has been granted Orphan Drug status in the US and in
References:
[1] Heier CR at al. (2013). VBP15, a novel anti‐inflammatory and membrane‐stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med 5: 1569–1585.
[2] Reeves EKM, et al (2013) VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem 21(8):2241-2249
[3] Heier CR et al. (2019). Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Science Alliance DOI 10.26508/lsa.201800186.
[4] Liu X et al. (2020). Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment.
[5] ClinicalTrials.gov Identifier: NCT02760277, Link
[6] Hoffman EP et al. (2019). Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology 93: e1312-e1323.
[7] ClinicalTrials.gov Identifier: NCT03038399, Link
[8] Smith E, et al. (2020). Efficacy and safety of vamorolone in Duchenne muscular dystrophy: an 18-month interim analysis of a non-randomized open-label extension study. PLOS Medicine, Link
[9] ClinicalTrials.gov Identifier: NCT03439670, Link
[10] ReveraGen website, Link
About Santhera
Raxone® is a trademark of
For further information please contact:
public-relations@santhera.com or
Phone: +41 79 875 27 80
eva.kalias@santhera.com
Disclaimer / Forward-looking statements
This communication does not constitute an offer or invitation to subscribe for or purchase any securities of
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Attachment
- 2020-11-02_SANN_GoingFoward_e_finalx
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