Interim report January-September 2021
November 4, 2021
Emcitate® receives FDA Fast Track Designation
Financial overview July – September
· Quarterly net revenue MSEK 6.2 (2.6)
· Quarterly loss MSEK -22.5 (-24.7)
· Cash and cash equivalents MSEK 173.2 (159.4)
· Cash flow for the period MSEK-34.5 (-24.6)
· Loss per share before/after dilution SEK -0.1 (-0.5)
Financial overview January – September
· Net revenue for the period MSEK 35.0 (35.8)
· Loss for the period MSEK -76.2 (-103.7)
· Cash and cash equivalents MSEK 173.2 (159.4)
· Cash flow for the period MSEK -115.4 (-95.8)
· Loss per share before/after dilution SEK -0.5 (-1.9)
Significant events during the period July – September
· Egetis receives approval in Turkey for compassionate use of Emcitate® for MCT8 deficiency.
· Egetis Therapeutics launches disease awareness initiatives to support identification of subjects with suspected MCT8 deficiency.
· Preparation for the pivotal Phase IIb/III study for Aladote continues targeting study start early 2022, pending the COVID-19 pandemic situation.
Significant events after the reporting period
· Egetis Therapeutics receives FDA Fast Track Designation for Emcitate® for MCT8 deficiency.
· New data published confirms long-term efficacy and safety of Emcitate® (tiratricol) in MCT8 deficiency patients.
Comments from the CEO
In the third quarter, Egetis Therapeutics continued its good progress with lead candidate Emcitate®, while the next clinical study with Aladote® is targeting study start early 2022, pending the COVID-19 pandemic situation.
New data published confirms long-term efficacy and safety of Emcitate® in MCT8 deficiency patients
In October we announced that strong data from long-term treatment, up to 6 years, of 67 patients with the company’s leading drug candidate Emcitate has been published in the Journal of Clinical Endocrinology & Metabolism. The data comes from an investigator-initiated real-life cohort study at 33 sites conducted by the Erasmus Medical Center, Rotterdam, Netherlands, where the efficacy and safety of Emcitate (tiratricol) was investigated in 67 patients with MCT8 deficiency. These long-term data confirm the positive results from our previous trial with Emcitate, TRIAC Trial I, and indicate that the beneficial effects are maintained over time, up to six years. The consistent efficacy seen across several key clinical and biochemical parameters regardless of age, further supports the use of Emcitate in the treatment of MCT8 deficiency. We are currently evaluating how this data can be used as additional evidence in forthcoming regulatory submissions of a Marketing Authorisation Application (MAA) in Europe and a New Drug Application (NDA) in the US.
Fast Track Designation granted for Emcitate
In early October, the U.S. Food and Drug Administration (FDA) granted Emcitate Fast Track Designation for the treatment of MCT8 deficiency. This designation is an acknowledgement from the FDA of the importance of Emcitate to address the significant unmet medical need in MCT8 deficiency. With a Fast Track Designation come opportunities to expedite both the NDA submission and FDA’s review which can enable an earlier regulatory approval of Emcitate. Emcitate, has already been granted Orphan Drug Designation (ODD) in both EU and the US, as well as a US Rare Pediatric Disease designation (RPD).
Patient recruitment to the TRIAC II study with Emcitate progressed well in the third quarter, and the recruitment is targeted to be completed in Q4 2021
We continue to recruit patients to the clinical phase IIb/III study TRIAC II despite the challenging Covid-19 situation. TRIAC II is an international, open label, multi-center study in children younger than 30 months with MCT8 deficiency, conducted in both Europe and North America.
Emcitate supplied on compassionate use and named patient basis
As we continue our clinical program with Emcitate, we see a continued interest from physicians across the globe to treat patients that suffer from MCT8 deficiency with our lead candidate drug. Emcitate is supplied on a named patient or compassionate use basis, following individual regulatory approval from the national regulatory agency. Compassionate use and named patient programs are mechanisms to allow early access to a medicine prior to regulatory marketing approval, granted to pharmaceuticals under development for conditions with high unmet medical needs and where no available treatment alternatives exist or are suitable.
More than 130 patients in more than 25 countries have been granted such named patient approval and are being treated with Emcitate, underlining the significant unmet medical need in this patient population and verifies the interest to treat patients that suffer from MCT8 deficiency.
In September, we received approval from the Turkish Medicines Agency for a compassionate use program of Emcitate.
Launch of campaign to raise awareness of MCT8 deficiency
We are committed to help transforming and extending the lives of patients with rare diseases such as MCT8 deficiency. One important pillar in this commitment is raising disease awareness, and with this purpose we launched an awareness campaign, including the website www.mct8deficiency.com in September. In addition to other disease educational activities, e.g. at scientific and medical conferences targeting health care professionals, the website will be used for educational purposes through the expanding network of key opinion leaders, physicians and patient advocacy groups focused on MCT8 deficiency.
With these activities, we aim to shorten the time toe diagnosis of MCT8 deficiency and relieve some of the heavy burden that MCT8 deficiency places on the affected individuals and the caregivers they are heavily dependent on.
Preparations for the Aladote pivotal Phase IIb/III study are ongoing
Preparations for the planned Phase IIb/III study with Aladote are ongoing in the US, UK and EU together with the selected CRO. The Covid-19 pandemic is still making it challenging to start a clinical study in an emergency/intensive care setting. Therefore, pending how the situation evolves, we expect study start will likely take place early next year.
We remain committed to the continued development of Aladote, which has the potential to be the first approved drug to benefit patients with an increased risk of liver injury, who are not adequately treated with NAC after a paracetamol overdose. Aladote has been granted ODD in the US. An application for an ODD in the EU was submitted in March, and we have an ongoing dialogue with EMA on the appropriate indication for an ODD in the EU.
We reported a cash position of approximately 173 million SEK on September 30, 2021, which is planned to finance the continued clinical development of Emcitate and Aladote.
I am grateful to employees and directors for their support and strong belief in the company. This was illustrated among other things by executives in Egetis Therapeutics acquiring additional shares in late August.
Our focus on our clinical candidates with their opportunity to provide treatment for patients suffering from rare and serious diseases remains firm as we shape the future of Egetis, our exciting company focusing on the orphan drug and rare disease segment. I believe we are well positioned to deliver on our projects Emcitate and Aladote and their respective development programs. I look forward to relaying news to you around the projects and the progress of Egetis Therapeutics.
Nicklas Westerholm, CEO