Enterprise Therapeutics Ltd.

The US Food and Drug Administration (FDA) has granted rare paediatric disease (RPD) designation to Enterprise Therapeutics’ investigational cystic fibrosis therapy. Estimated to affect more than 100,000 people globally, cystic fibrosis is a genetic disease that causes sticky mucus to build up in the lungs, digestive system and other organs in the body. Symptoms usually begin in early childhood and vary between patients, but the condition gets progressively worse over time and causes increasing damage to the affected organs. Enterprise’s ETD001, which targets the sodium channel in the airway epithelium to increase the hydration and clearance of mucus, has already demonstrated a well-tolerated profile in a phase 1 trial of healthy participants and has been shown to be long-acting in pre-clinical studies. A phase 2a trial is now aiming to assess the safety and efficacy of the candidate in patients who are ineligible for, or are not receiving, cystic fibrosis transmembrane conductance regulator modulator therapy. FDA RPD designation aims to incentivise drug development for serious or life-threatening diseases that affect fewer than 200,000 people in the US and primarily impact patients aged under 18 years. Under the programme, a sponsor that receives an approval for a RPD product may qualify for a priority review voucher (PRV) that can be redeemed to expedite the review of a different product, or be sold or transferred to another sponsor. John Ford, Enterprise’s chief executive officer, said: “As we progress through our phase 2a trial of ETD001, this RPD designation will further support our mission to advance this novel approach for treating people with cystic fibrosis with the highest unmet medical need, as rapidly as possible.” Annabella Amatulli, head of regulatory affairs at the company, added: “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 PRV at the time of marketing approval.” The FDA’s decision comes just two months after Enterprise announced that the first cystic fibrosis patient had been dosed in its phase 2a trial, which Ford said represented “an incredible milestone”.

BRIGHTON, England--( BUSINESS WIRE )--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 23 rd July 2024 2 . 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 studies 3 . 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.” https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions https://enterprisetherapeutics.com/enterprise-therapeutics-doses-first-person-with-cystic-fibrosis-in-phase-2-trial-for-novel-therapy-etd001/ https://enterprisetherapeutics.com/enterprise-therapeutics-publishes-preclinical-profile-of-etd001-a-novel-inhaled-enac-blocker/

Industry veteran Doug Williams is back with a new approach to CAR-T cell therapies that he expects to take into the clinic in the US by year’s end. After a brief stint at Sana Biotechnology , leaving his R&D chief post in the spring, Williams is returning to the role of CEO at a little-known biotech. In recent weeks, he took the top spot at China-born and now California-headquartered biotech TriArm Therapeutics . It’s a position he held at Codiak BioSciences , a startup that tried turning exosomes into medicines, but the relatively nascent idea couldn’t survive on the public markets in 2023. Now, he’s driving TriArm’s mission to “democratize” CAR-T cell therapies using what it calls a “fast-in-time” (FIT) and non-viral manufacturing process, Williams told Endpoints News . He said TriArm’s technology platform has led to a two-day manufacturing process. “There’s also some potential advantages to cell fitness, the actual fitness of the CAR, the composition of cells that we’re reinfusing back into the patient,” Williams said. “We’re preserving a population of cells using this FIT process that may end up being more fit from a biologic perspective because they’re enriched in STEM and naïve T cells,” he added. TriArm has been relatively quiet since launching in 2019. Its financial backers include Panacea Venture , MSD Capital and Lombard Odier , and it employs about 100 people across Shanghai, Taipei and San Mateo, CA. The company aims to speed up the testing of CAR-T therapies and make faster go/no-go R&D decisions by employing an investigator-initiated model in China and then moving into clinical studies in the US, Williams said. “As somebody that is a drug developer by choice and by training, to be able to get a glimpse of what is arguably real data in a population of patients as sort of a preamble to filing an IND and really moving ahead in larger, much more expensive trials, it’s a fantastic setup to have available,” Williams said. Its first US-based trial, testing a CD19-directed autologous therapy, is slated to start later this year, Williams said. A gene therapy could soon enter a similar patient setting with the emergence this week of a new biotech called Vironexis . — Kyle LaHucik → Biogen ’s board continues to evolve under chair Caroline Dorsa , who replaced Stelios Papadopoulos last year. On Jan. 1, ex- AstraZeneca BioPharmaceuticals R&D chief Mene Pangalos will take a seat on the board, and Lloyd Minor — the dean of Stanford University’s School of Medicine — will become a board member much earlier on Oct. 1. After the hubbub surrounding the departure of board member Alex Denner and the appointment of his romantic partner Susan Langer in June 2023, Biogen then elected ex- 3M president and CFO Monish Patolawala to the board in November. Richard Mulligan and William Jones also left the board last year. Pangalos retired from AstraZeneca in April and was replaced by Sharon Barr . → Vir Biotechnology ’s revamp took the spotlight in early August, as CEO Marianne De Backer made the decision to prop up its hepatitis B and D development and wind down its Covid-19, flu and T cell-based viral vector programs. On Wednesday, Vir said that Jason O’Byrne will succeed Sung Lee as CFO on Oct. 2. O’Byrne had the same title at Caribou Biosciences since February 2021 and is the former SVP of finance at Audentes Therapeutics . He spent 13 years with Genentech in a number of roles, including head of commercial finance for the Asia-Pacific region. To accommodate the makeover, Vir laid off about a quarter of its staff. → BridgeBio Oncology Therapeutics has selected Yong Ben as chief medical and development officer. Readers may know Ben from BeiGene , where he was CMO, solid tumors from 2019-22, and he’s been a venture partner at Eight Roads for the past two years. Ben was the global clinical leader of immuno-oncology clinical development at AstraZeneca when Imfinzi received its first approval for advanced bladder cancer in May 2017, and a week later, he became CMO of BioAtla . BridgeBio’s oncology spinout debuted in May with a $200 million infusion from Cormorant Asset Management , Omega Funds , GV and others. → As Endpoints News reported on Monday, Eli Lilly ended a three-month search by promoting Lucas Montarce to CFO. He replaces current Alphabet finance chief Anat Ashkenazi , with an interim stint by controller Gordon Brooks sandwiched in between. Montarce’s career at Lilly began in 2001 and has held enough positions to scroll through ever since. He was elevated to president and general manager for the Spain, Portugal and Greece hub in February after nearly three years as group VP, corporate controller and chief financial officer for Lilly Research Laboratories . He takes over as CFO at a time when the Indianapolis pharma’s market cap has exceeded $800 billion. → Pfizer CSO Mikael Dolsten has nabbed a spot on the board of directors at Rocket Pharmaceuticals . Dolsten announced in July that he would leave Pfizer after 15 years, but he’s sticking around until the company finds a successor. In late June, questions about manufacturing resulted in a CRL for marnetegragene autotemcel ( marne-cel ), Rocket’s gene therapy to treat an ultra-rare disease called severe leukocyte adhesion deficiency-I. → Meg Alexander has been promoted to president and COO of Ovid Therapeutics , which is on the verge of starting a Phase 2 study of its Graviton -partnered ROCK2 inhibitor in cerebral cavernous malformations. This is Alexander’s third appearance in as many years after she was named chief strategy officer last year and corporate affairs chief in 2022. “Her extensive experience, operational expertise, and business acumen position her well to lead the next phase of the company’s evolution,” Ovid CEO Jeremy Levin said in a statement . → Sana Biotechnology finance chief Nate Hardy will resign “for personal reasons” on Oct. 4, according to an SEC filing . Hardy was on the finance team at Juno Therapeutics with CEO Steve Harr before he took the CFO job at Sana in 2018. The Seattle-based biotech just hired UCB ’s Dhaval Patel as its CSO. → After five years on the job, Forge Biologics CEO Tim Miller is stepping down , making way for commercial chief John Maslowski to take over Oct. 1. Maslowski initially joined Forge as chief commercial officer in June 2021. Before that, he was president and CEO of Castle Creek Biosciences and Fibrocell Science . → Matt Shaulis is headed to BeiGene as general manager of North America on Sept. 25, while Josh Neiman — the chief commercial officer for North America and Europe — has stepped down “to pursue a new entrepreneurial opportunity at a healthcare technology startup,” according to a release . For the last year and a half, Shaulis has been chief commercial officer and US president for Swedish drug developer Hansa Biopharma , and he took on the role of president, inflammation and immunology for the international developed markets at Pfizer from 2018-21. At the end of his seven-year tenure with the pharma giant, he was elevated to SVP for global commercial and medical go-to-market model transformation. → Jeff Hartness has been named chief commercial officer at I&I biotech Apogee Therapeutics , which hit the Nasdaq with Sagimet Biosciences on July 14, 2023. Hartness spent more than 15 years with Sanofi before jumping to Bausch Health in 2016 as VP, strategic account management. Last year, he was promoted to EVP and head of Bausch Health’s market access, commercial operations, policy and government affairs teams, while serving as general manager of the neurology portfolio and generics businesses. Chaired by ex- Prometheus Biosciences chief and current Mirador Therapeutics CEO Mark McKenna , Apogee is trying to distinguish itself in the atopic dermatitis space with its monoclonal antibody APG777 . → The team at non-opioid pain biotech Latigo Biotherapeutics is quickly taking shape under new CEO Nima Farzan , who succeeded Westlake Village BioPartners ’ Desmond Padhi in late July. After bringing in medical chief Neil Singla last month, Latigo has tapped William Blair analyst Tim Lugo as CFO. Lugo joined William Blair as a senior associate in 2005 and would eventually become a partner, leading the biotechnology equity research team. Latigo also added Westlake managing director Beth Seidenberg and Foresite Capital ’s Jim Tananbaum to the board of directors. → Now that Rytelo ( imetelstat ) is being sold in the US for lower-risk myelodysplastic syndromes with transfusion dependent anemia, Geron Corporation has recruited Jim Ziegler as chief commercial officer. Ziegler had been EVP, commercial for Iovance , which secured the long-awaited FDA approval of its TIL therapy Amtagvi for melanoma in February. He also spent a decade in two separate stints with Gilead , culminating in his role as VP of the cardiopulmonary and inflammation business unit. → Alan List is retiring as CMO of Precision BioSciences , and 12-year Merck vet Murray Abramson has jumped on board to lead clinical development. Abramson left Merck in 2011 to become VP of global clinical operations at Biogen, a position he held for nine years. From 2020-22, Abramson was SVP of clinical innovation for Tempus . Precision also said in a release that ex- Aligos Therapeutics EVP John Fry will be a strategic clinical advisor for its hepatitis B program. Following his resignation as president and CEO of Moffitt Cancer Center over his connections to China, List reemerged as Precision’s medical chief in 2021. → Rami Daoud has made his way to gene therapy developer Affinia Therapeutics as chief business and financial officer. Daoud comes off a yearlong stint as president of GeneScience Pharmaceuticals USA and was a corporate development exec with Amarin . Rick Modi ’s crew was an Endpoints 11 winner in 2021 and had IPO aspirations that ultimately faded in late 2022. → HC Bioscience has recruited Simon Tsang to replace Christine Foster as CBO. Tsang is an Amgen corporate development alum who was VP, business development for Tesaro from 2016-19. After GSK bought Tesaro for $5.1 billion, he landed at Pivotal bioVenture Partners as an operating partner. HC Bioscience is in a small-but-emerging group of biotechs working on tRNA-based drugs, along with Flagship ’s Alltrna and Tevard Biosciences . → Rivus Pharmaceuticals , which claimed a Phase 2a win last month with its drug for obesity-related heart failure with preserved ejection fraction, has appointed Tom O’Neil as CFO and ex- ProfoundBio COO/CFO Erin Lavelle to the board of directors. As finance chief, O’Neil helped lead Satsuma Therapeutics to its IPO in 2019 and its sale to Shin Nippon Biomedical Laboratories last year. Lavelle orchestrated ProfoundBio ’s $112 million Series B in February, and Genmab would agree to buy the ADC biotech less than two months later. She also picked up another board seat this week at Aviceda Therapeutics. → We have an impressive lineup of CMO appointments: First, Eli Lilly’s obesity and metabolic disease partner HAYA Therapeutics has named Jordan Shin as CMO. Shin had been SVP of clinical development and translational science (cardiology) for gene therapy player Lexeo Therapeutics , which went public last November with a $100 million IPO. Earlier, he worked for Renovacor as SVP of clinical development and translational science until it was sold to Rocket Pharmaceuticals . Up to $1 billion is on the line with HAYA and Lilly’s recent drug discovery pact . → Canadian psychedelic biotech Reunion Neuroscience has brought in Mark Pollack as medical chief. Pollack briefly worked for Sage as SVP for global medical affairs and was part of a leadership shakeup that also involved the departures of CSO Al Robichaud and development chief Jim Doherty . Reunion went private last summer with the help of MPM BioImpact , and a $103 million Series A followed in May from MPM, Novo Holdings and several other investors. → A week after Reunion’s Series A financing, San Diego upstart Aardvark Therapeutics raised $85 million in a Series C to bankroll late-stage trials for its Prader-Willi syndrome asset ARD-101 . And like Reunion, Aardvark has found a new medical chief. Manasi Sinha Jaiman was promoted to the same position at stem cell therapy maker ViaCyte before it was sold to Vertex in 2022 for $320 million , and she would stay at Vertex as a VP. In the same release, Aardvark included the appointment of regulatory affairs leader Tom Bicsak , who came on board in July. → Milan-based AAVantgarde Bio is picking up Jayashree Sahni as CMO. Sahni is taking over the reins from Naveed Shams , who had served in the role since 2022. Sahni joins the team from Novartis , where she was senior global program clinical lead. Earlier in her career, Sahni was with Roche , where she worked on the Phase 1 and 2 clinical trials for Vabysmo . → Elsewhere, RA Capital -backed Tyra Biosciences has recruited Doug Warner as CMO. Warner joins the California-based crew from eFFECTOR Therapeutics , where he served in the same role. Previously, Warner had an 18-year stint at Amgen, where he led development of drugs like Vectibix , Xgeva and Prolia . Warner’s appointment comes after Tyra picked up $200 million in PIPE financing in February to progress its oral FGFR3-selective inhibitor, TYRA-300 . → Longwood ’s Solu Therapeutics , which debuted in August 2023 with a $31 million seed round, has welcomed Sergio Santillana as CMO. Peer Review told you about Santillana’s resignation from the same position at Ikena Oncology in February, and he’s also the ex-CMO at Merrimack Pharmaceuticals and Ariad Pharmaceuticals . Earlier in his career, Santillana had a string of roles at Eli Lilly, GSK and Takeda . → Maud Brandely is retiring after eight years as CMO of French cancer biotech Transgene , and Sanofi Genzyme vet Emmanuelle Dochy has replaced her . Dochy just completed a seven-year run at Bayer , where she recently served as VP of global medical affairs for the tyrosine kinase inhibitor (TKI) oncology franchise and scientific partnerships head. Meanwhile, Transgene has also enlisted Roche oncology alum Maurizio Ceppi as CSO. → KalVista Pharmaceuticals has named Brian Piekos as CFO. Far from his first run as finance chief, Piekos served in the role at Elicio Therapeutics , Gemini Therapeutics and AMAG Pharmaceuticals . In May, KalVista announced that it was dropping its preclinical program in hereditary angioedema in favor of focusing on its HAE pill sebetralstat . At the time, KalVista said it would find a partner for its dropped program. → Beverley Carr has taken the CBO post at London-based Charm Therapeutics , a startup co-founded by the University of Washington’s David Baker that uses its 3D deep learning tech DragonFold to discover new drugs. Carr is a GSK business development vet who had been chief business and operating officer for Amphista Therapeutics and then stepped into the role of interim CEO when Nicki Thompson left. → In July, Cue Biopharma announced that it was prioritizing its autoimmune pipeline and slashing 25% of its staff. Now, J&J vet Lucinda Warren has joined the Boston team as CBO. Warren’s 10-year stint at J&J culminated in her role as VP of business development for neuroscience and Japan regionally. Earlier in her career, Warren was with Janssen Cilag Australia and Centocor / Janssen Biologics . → Verily , the healthcare company spun out of Google parent Alphabet , has rolled out the welcome mat for Michael Radwin as its new head of data science and AI. Radwin joins from the CDC, where he was a founding member of the Office of Public Health Data, Surveillance, and Technology. Radwin’s résumé also includes roles at Intuit and Yahoo . Earlier in June, Verily announced that it was jumping into the GLP-1 business with the launch of a new virtual program to help patients manage cardiometabolic diseases with care teams and medications. → Dutch biotech VarmX , which is developing a reversal agent for blood thinners, has promoted Martijn Negen to the role of COO. Negen joined VarmX last September as SVP of global commercial strategy & business development from AM-Pharma . He also has experience from Portola Pharmaceuticals , Teva Global Specialty Medicines and Genentech. → Opthea has announced a slew of changes to its leadership team. First, the company is losing commercial chief Judy Robertson , CFO Peter Lang and SVP, global clinical operations Bruno Gagnon , effective Sept. 9. Filling in for Lang and Robertson in the interim will be Danforth Advisors managing director Daniel Geffken and Genentech and Novartis vet Mike Campbell , respectively. Meanwhile, Jen Watts will succeed Gagnon. That’s not all: Opthea is also picking up Dayong Li as SVP, biometrics and Anthony Bonifazio as VP, market access. → UK respiratory disease biotech Enterprise Therapeutics has introduced Annabella Amatulli as head of regulatory affairs. The Janssen vet was the global regulatory sciences leader for Alfasigma , the Italian company that bought Ocaliva maker Intercept Pharmaceuticals . → Tonix Pharmaceuticals has brought aboard Thomas Englese as EVP of commercial operations. Englese was formerly chief commercial officer of Tris Pharmaceuticals and Aziyo Biologics . Before that, he had an 11-year run with Mallinckrodt , culminating in his role as SVP and general manager of North America hospital therapies. → A handful of VCs have been making some moves. First up: Abingworth has turned to SFJ Pharmaceuticals founder and executive chair Robert DeBenedetto to tackle the role of managing director, clinical co-development; Sofinnova Partners has added a new partner. Karl Naegler just completed a four-year run as managing partner with Wellington Partners Life Sciences in June; and finally, Frazier Healthcare Partners has named Willis Chandler as an executive-in-residence on the growth buyout team. Chandler left Cencora (formerly AmerisourceBergen ) in June after nearly three years as president of global pharma services and six years overall. → Co-founded by Jim Wilson , Passage Bio has elected Ultragenyx CBO Tom Kassberg to the board of directors. One of Wilson’s new companies, GEMMA Biotherapeutics , licensed a trio of gene therapy candidates from Passage Bio in early August. → Enoch Kariuki has joined the board of directors at Cyrus Mozayeni ’s ADC shop Pheon Therapeutics , which is chaired by ex- Blueprint Medicines chief Jeff Albers . Kariuki became president of Endeavor BioMedicines this year and ran Lengo Therapeutics until it was purchased by Blueprint in 2021. → Yoshiharu Mizui now has a seat on the board of directors at SEED Therapeutics , the biotech near Philadelphia that struck a “molecular glue” deal with Eisai last month. Mizui is the president of Eisai’s VC arm, Eisai Innovation , and he also juggles two other roles as head of collaboration & incubation in the DHBL (Deep Human Biology Learning) office and head of academic & industry alliance. → Creyon Bio has made room for DCVC Bio operating partner Serge Messerlian and Lux Capital principal Shaq Vayda on the board of directors. Messerlian has board seats at Auron Therapeutics and Plexium , and Vayda’s numerous board credits include Atavistik Bio and Enveda Biosciences .