On March 5, 2020 Newron Pharmaceuticals S.p.A. ("Newron") (SIX: NWRN, XETRA: NP5), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system, reported its financial results and operational highlights for the year ended December 31, 2019, and provided an outlook for 2020 (Press release, Newron Pharmaceuticals SpA, MAR 5, 2020, View Source [SID1234555228]).
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Highlights:
Sarizotan (Rett syndrome)
Newron successfully completed enrolment of 129 patients in the STARS Phase III study and advanced the study towards the end of the 24-week, double-blind treatment period, with the clinical database locked and blinded
Newron participated in a meeting with the U.S. Food and Drug Administration (FDA) to discuss the STARS study statistical analysis plan, ahead of STARS clinical trial data unblinding, and now expects top-line results in H1 2020
The FDA granted Rare Pediatric Disease Designation for sarizotan, following an earlier decision to grant sarizotan Orphan Drug Designation
Evenamide (Schizophrenia)
Newron and the FDA have agreed on the design and conduct of explanatory studies with Evenamide required to address previously announced potential safety issues raised by the FDA, including a four-week explanatory study in patients with schizophrenia; initial results in rats and humans are expected in Q3 2020
Subject to the successful completion of these additional studies, Newron intends to commence its proposed Phase III clinical trial program with Evenamide comprising of two efficacy studies
Xadago/safinamide (Parkinson’s disease)
Zambon and its regional partners have launched safinamide in Australia, Canada and Colombia and received marketing authorization in Brazil and the UAE; Meiji Seika and partner Eisai have launched safinamide in Japan
Successful negotiations with the Italian authorities have resulted in the reimbursement cap being removed, allowing for further potential revenue growth
Progress made in plans to perform the levodopa-induced dyskinesia (PD LID) study with Xadago
Zambon previously held discussions with the FDA on the design of a potentially pivotal efficacy study to evaluate the effects of Xadago/safinamide in patients with PD LID
Intention is to perform the study in the US, Europe and Asia/Australia
Zambon acknowledges Newron’s experience in the development of Xadago in patients with Parkinson’s disease; discussions to have Newron as the party responsible for conducting the study; Zambon will remain associated with the study
Financial terms to stay unchanged
Corporate
Newron received two tranches of funding from the European Investment Bank of EUR 10 million and EUR 7.5 million respectively, out of a total of up to EUR 40 million; this funding is used to boost the Company’s R&D activities and support its pivotal and post-approval CNS development programs
In addition to its primary listing on the Swiss Stock Exchange, Newron began trading in Germany on the Düsseldorf Stock Exchange and XETRA to facilitate access to Newron’s shares for investors based in the EU via EU brokers
Cash (incl. Other current financial assets) as of December 31, 2019 is EUR 39.2 million
Stefan Weber, Newron’s Chief Executive Officer, commented:
"2019 has been a successful year of strategy execution for Newron. We are pleased to have continued to develop and mature our pipeline of innovative therapies for central and peripheral nervous system diseases. We have made significant progress with our STARS clinical study, advancing the study to the end of the 24-week, double-blind treatment period, with the clinical database locked and blinded. We now expect to announce top-line results from the study later in H1 2020."
Stefan Weber continued: "We are pleased that our partners worldwide were successful in launching safinamide in Australia, Canada, Colombia and Japan and we hope that more patients worldwide will gain access to safinamide through further launches in the coming months. Prior to the start of our Phase III development program with Evenamide in schizophrenia, we have initiated additional short-term explanatory studies which we expect to report initial results in Q3 2020. We would like to thank all our shareholders for their continued support and confidence in Newron, and we look forward to updating markets on our progress throughout 2020."
Sarizotan
In 2019, Newron made significant progress with its STARS clinical study, completing patient recruitment, with 129 Rett syndrome patients qualified and enrolled, and more than 85% of enrolled patients completing the 24-week, double-blind period, have continued into the long-term open-label extension. This is an indicator of the critical need within the Rett syndrome community and demonstrates the potential of a new treatment option such as sarizotan.
In addition, the U.S. Food and Drug Administration (FDA) decided to grant a Rare Pediatric Disease Designation to sarizotan, which followed earlier decisions by the U.S. and EU authorities to grant Orphan Drug Designations to this compound for the U.S. and the EU. Newron believes that this highlights the large unmet medical need in Rett syndrome, as well as represents progress towards potential marketing authorization with U.S., Canadian and EU regulatory agencies in the future. This designation also represents progress towards qualifying sarizotan for a rare pediatric disease priority review voucher upon potential future U.S. marketing approval.
A communication from the FDA in December suggested that Newron discuss the Company’s statistical analysis plan in a meeting prior to unblinding the STARS clinical trial results. The clinical database has been locked and remains blinded. We currently expect unblinding of the STARS clinical trial data and disclosure of the top-line results in H1 2020. Subject to a positive study outcome, our goal is to initiate discussions with the regulatory agencies towards filing of the dossiers for marketing authorization. Upon regulatory approval, Newron intends to commercialize sarizotan for Rett syndrome in the U.S. and – if viable – in key EU territories.
As part of its commitment to the rare disease patient community, Newron is conducting a landmark International Burden of Illness study, partnered with the global Rett syndrome community. The survey outreach launched in the beginning of November 2019 in the U.S. and aims to deliver data and analytics to quantify the physical, emotional and financial challenges of Rett syndrome for patients, their families and caregivers. In February 2020, this survey outreach was expanded to reach families and caregivers in the U.K., Germany, Italy and Australia.
Evenamide
In May, the FDA requested that Newron complete additional short-term explanatory studies in rats and human subjects to address questions on findings from a recently completed pre-clinical study of Evenamide. The Company engaged with the FDA in order to address the agency’s concerns prior to the initiation of the Phase III development program. In early January 2020, Newron announced that it had reached agreement with the FDA on the design and conduct of these explanatory studies with Evenamide, as well as the protocol for a first, four-week explanatory study in patients with schizophrenia. Newron expects to see initial results from these additional studies in rats and humans in Q3 2020.
Subject to the successful completion of these studies, Newron intends to commence its proposed Phase III clinical trial program with Evenamide in two pivotal efficacy studies in patients with schizophrenia. One for patients experiencing worsening of psychosis on atypical antipsychotics, and the other study in ultra-treatment-resistant schizophrenia patients not responding to clozapine, with the latter representing an orphan-like indication affecting approximately 20,000 to 25,000 patients in the U.S. (with similar numbers in the EU). Positive results in both studies could lead to Evenamide being the first add-on therapy for the treatment of patients with positive symptoms of schizophrenia who show an inadequate response to their current atypical medication. In key territories, Newron expects to commercialize Evenamide itself in the treatment-resistant schizophrenia indication.
Xadago/safinamide
In 2019, Meiji Seika together with Eisai announced the approval and launch of safinamide in Japan, under the brand name Equfina. Seqirus launched Xadago (safinamide) in Australia, Zambon in Colombia, and Valeo Pharma in Canada under the brand name Onstryv. Xadago has received marketing approval in Brazil and the United Arab Emirates and dossiers for marketing authorization are currently under review in Mexico and Israel. Newron is pleased with this progress and remains optimistic for additional launches in 2020.
Newron’s total income from the marketed territories increased by 75% over the prior year, to EUR 7.0 million, of which EUR 2.3 million is due to one-time non-refundable milestone payments. The Company expects further growth in Europe, aided by the cap on reimbursement being removed in Italy effective March 1, 2019.
Newron progresses in the plans to perform the LID study with Xadago: Zambon had previously held discussions with the FDA on the design of a potentially pivotal efficacy study to evaluate the effects of Xadago/safinamide in patients with PD LID. The intention is to perform the study in the U.S., Europe and Asia/Australia.
Zambon acknowledges Newron’s experience in the development of Xadago in patients with Parkinson’s disease and there have been discussions to have Newron as the party responsible for conducting the study. Zambon will remain associated to the study. Financial terms will stay unchanged: Newron will make a fixed financial contribution to the study, in return for a one-time milestone payment and a greater share of royalties should the study lead to a label extension.
Financial Highlights:
In 2019, Xadago-related payments received increased by 75% (EUR 7.0 million versus EUR 4.0 million in 2018), including a one-time milestone payment for approval in Japan of net EUR 2.0 million.
At the same time, Newron’s net R&D expenses increased to EUR 17.4 million from EUR 9.8 million in 2018, largely due to the ongoing STARS study in Rett syndrome and work relating to the preparation for the two pivotal efficacy studies in patients with schizophrenia.
Newron received Italian R&D tax credits of EUR 5.0 million that can be offset with future tax and social contribution payments by the Company, versus EUR 5.9 million in 2018.
In 2019, G&A expenses reached EUR 9.9 million compared to EUR 8.8 million in 2018 (increase refers to evaluation of and preparation for additional listings of Newron’s shares).
In 2019, Newron reported a net loss of EUR 20.2 million, compared to EUR 15.0 million in 2018.
Cash used in operating activities has increased to EUR 22.2 million from EUR 16.0 million in 2018.
Cash and Other current financial assets at December 31, 2019 were at EUR 39.2 million, compared to EUR 43.9 million at the beginning of the year.
Financial Summary (IFRS):
(1): The Group adopted in 2019, for the first time, the new standard IFRS 16 Leases applying the full retrospective method that requires the restatement of previous financial statements.
Newron’s full 2019 Annual Report is available on www.newron.com/financial-report-2019
Outlook for 2020:
"2020 will be an important year for Newron, with sarizotan in late-stage clinical development and Evenamide poised to enter a Phase III pivotal program. We look forward to reporting on our meeting with the FDA on the statistical plan for our STARS study and remain confident that we can address the FDA’s questions around Evenamide. We are encouraged by the continued success of our global partners in the approvals and launches of safinamide and expect these to continue into 2020. We started 2020 with total available funds of up to EUR 61.7 million, including the EUR 22.5 million of EIB funds not yet drawn, which will cover the pursuit of Newron’s development programs and operations as currently contemplated beyond 2021," outlined Stefan Weber, CEO of Newron.
2020 Shareholders’ Meeting Agenda:
Newron’s Board of Directors has approved the below agenda for the March 31, 2020, Shareholders’ meeting, which will take place at the Company’s registered office (Via Antonio Meucci 3) in Bresso (Mi), Italy, starting at 10:00 am CET. The formal invitation to shareholders will be issued and disclosed in the statutory papers on or around March 5. The full invitation and supporting material will be made available on the Company’s website on the same date. The agenda is as follows:
Approval of the balance sheet as of 31 December 2019
Appointment of the members of the Board of Directors, after determination of the relevant number, for the financial years 2020, 2021 and 2022 and, therefore, until the approval of the financial statements as of December 31st, 2022, as follows:
– Ulrich Köstlin, in quality of Chairman of the Board and non-executive director;
– Stefan Weber, in quality of executive director;
– Patrick Langlois, in quality of non-executive director;
– Robert Leslie Holland, in quality of non-executive director;
– Luca Benatti, in quality of non-executive director; and,
– Donald deBethizy, in quality of non-executive director
Determination of the remuneration of the Board of Directors
Dial-in details to the media/analyst/investor conference on March 5, 2020, 04:00 pm CET:
The Newron management team will present the 2019 full-year results and provide an update and guidance for 2020. The conference call can be accessed via the following dial-in numbers:
Switzerland/Europe: +41 (0)58 310 50 00
United Kingdom: +44 (0)207 107 0613
United States: +1 (1)631 570 5613
The slide deck for the call is available at www.newron.com/downloads/reports-presentations–webcasts/2020
Upcoming events:
AGM 2020: March 31, 2020
Half-year report 2020: September 15, 2020