Enterprise Therapeutics granted ‘rare pediatric disease designation’ in the US for novel cystic fibrosis investigational therapy ETD001

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  • ETD001 is a novel, first in class blocker of the epithelial sodium channel (ENaC) aimed at treating people with cystic fibrosis without current effective therapies
  • ETD001 commenced Phase 2 clinical trials in July 2024, expected to complete in 2025

Brighton, UK, 26 September 2024: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of those suffering from respiratory disease, today announced its novel cystic fibrosis (CF) investigational therapy, ETD001, has been granted ‘rare pediatric disease designation’ (RPD) in the US by the Food and Drug Administration (FDA).1

ETD001 is a low molecular weight compound with first-in-class potential, which targets the sodium channel (ENaC) in the airway epithelium to increase the hydration and clearance of mucus. Enterprise announced dosing of the first person with cystic fibrosis (pwCF) in its Phase 2a trial of ETD001 on 23rd July 20242. The trial aims to deliver clinical proof-of-concept and to assess the safety and efficacy of ETD001 in the ~10% of pwCF with the highest unmet medical need. The study will assess lung function (FEV1) in pwCF who are either ineligible for or are not receiving CFTR modulator therapy.

The RPD designation was based on the assessment of CF as a serious or life-threatening disease, primarily affecting individuals aged from birth to 18 years.

CF is estimated to affect over 100,000 people worldwide, with an average life expectancy of only 50 years. Failed mucociliary clearance and mucus congestion in the lungs leads to cycles of infection and inflammation and an ongoing decline in lung function. Increasing fluid volume in the lung by inhibiting ENaC with ETD001 will hydrate mucus, improve clearance, reduce mucus congestion, and is expected to drive substantial improvements in lung function. ETD001 has previously demonstrated a well-tolerated profile in healthy subjects in a Phase 1 trial and has been shown to be long-acting in pre-clinical studies3.

Dr John Ford, CEO, Enterprise Therapeutics, said: “We’re delighted with the FDA’s decision and would like to thank the Office of Pediatric Therapeutics and the Office of Orphan Products Development for their consideration. As we progress though our Phase 2a trial of ETD001, this RPD designation will further support our mission to advance this novel approach for treating pwCF with the highest unmet medical need, as rapidly as possible.”

Annabella Amatulli, Head of Regulatory Affairs, commented: “We are thrilled to be granted the RPD designation by the FDA, a regulatory framework intended to encourage and accelerate innovative therapies, in recognition of the significance of our programme in addressing an unmet medical need. The RPD designation will give Enterprise access to valuable incentives and support from the FDA during the development of ETD001, including the eligibility to request a Priority Review Voucher (PRV) at the time of marketing approval.”

  1. https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions
  2. https://enterprisetherapeutics.com/enterprise-therapeutics-doses-first-person-with-cystic-fibrosis-in-phase-2-trial-for-novel-therapy-etd001/
  3. https://enterprisetherapeutics.com/enterprise-therapeutics-publishes-preclinical-profile-of-etd001-a-novel-inhaled-enac-blocker/

Enterprise Therapeutics appoints Annabella Amatulli as Head of Regulatory Affairs

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  • Appointment brings extensive regulatory experience as lead asset ETD001 progresses through Phase 2 clinical trial for treatment of cystic fibrosis

Brighton, UK, 11 September 2024: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of those suffering from respiratory disease, today announced the appointment of Annabella Amatulli as Head of Regulatory Affairs.

In this newly created role, Annabella will be responsible for driving the Company’s regulatory strategy and working in close collaboration with clinical, medical, patient engagement, and market access teams. The primary focus of her role will be to support the ongoing Phase 2 clinical trial of lead asset ETD001, a novel long acting ENaC blocker which offers the potential to treat people with cystic fibrosis (CF) not currently benefitting from CFTR modulator therapies1. Annabella will also define the regulatory pathways for Enterprise’s pipeline of other novel low molecular weight compounds which also have first-in-class and best-in-class potential.

Annabella has more than 14 years’ experience working in pharmaceutical companies, with a focus on regulatory affairs. Prior to joining Enterprise, she was Head of Global Regulatory Sciences at Alfasigma, where she contributed to the growth of the company and the regulatory strategy for all products, across varying phases of development. Annabella has an extensive range of regulatory experience throughout Europe, the US and in other regions of the world. She has led interactions with health authorities including EMA, FDA, MHRA and NMPA to obtain Orphan Drug Designation, Fast Track and Breakthrough approvals for numerous candidates, negotiated Paediatric Investigation Plans, set up a global early access programme, and obtained global approval for a rare disease therapeutic.

Prior to Alfasigma, Annabella held various senior regulatory positions in both SMEs and large pharmas, including Chief Regulatory Officer at Napo Therapeutics and Global Regulatory Affairs Biotech & Development Director at Dompè Farmaceutici, as well as roles within Janssen-Cilag (J&J), Schering-Plough, and Mylan. As a member of the Pharma Trade Association, she led the regulatory workstream dedicated to advanced therapies (ATMPs). Annabella is also one of the founders of the Milan chapter of the Healthcare Businesswomen’s Association and is currently Regional Chair of the Southern Europe Region.

Dr John Ford, CEO, Enterprise Therapeutics, said: “We welcome Annabella as Head of Regulatory Affairs. Her broad experience in managing regulatory teams and liaising with health authorities will be hugely beneficial as we continue to progress our respiratory therapeutics programmes. Annabella’s expertise will prove especially valuable as we transition through Phase 2 clinical trials for our lead asset ETD001, moving closer to providing a novel treatment for all people with CF, including those with the highest unmet medical need.”

Annabella Amatulli, Head of Regulatory Affairs, Enterprise Therapeutics, added: “I am delighted to join Enterprise at such a pivotal time. The work that the Company is doing has the potential to vastly improve the lives of people with respiratory diseases. I look forward to working with the leadership team to help drive the continued development of the pipeline.”