Protara Therapeutics, Inc.

Good afternoon, everyone. My name is Jim, and I will be your conference operator today. At this time, I would like to welcome everyone to the Protara Therapeutics Lymphatic Mouth Formations KOL webinar. All lines have been placed on a mute to prevent any background noise. And please note that all questions for the Q&A discussion at the end should be entered into the chat box in the bottom left of our screen. And now, for opening remarks and introductions, I am pleased to turn the floor to our host, Senior Vice President of Investor Relations, Justine O'Malley. Welcome.

Thank you, Jim. Good afternoon. Thank you all for joining us today for a review of Protara's Lymphatic Malformations Program. Before we begin, I would like to remind you that during today's call, we will be making certain forward-looking statements. These statements represent our views as of today only and should not be relied upon as representing our views as of any subsequent date. Actual results may differ from our forward-looking statements due to various factors, including those described in the risk factors section of our most recent annual report and subsequently filed quarterly reports that are on file with the SEC. Except as required by law, we disclaim any obligation to update these statements, even if our views change. I will now turn the call over to Jesse Shepperman, co-founder, director, and chief executive officer.

Thank you, Justine, and thank you all for joining us this afternoon. Good afternoon from the World Congress of the International Society for the Study of Vascular Anomalies, or ISFAs. We are excited to provide an overview of our lymphatic malformations program today, including a recap of our recent FDA engagements and our plans to submit a BLA in the second half of 2027 for TARA-002 in lymphatic malformations. There is a significant unmet need in this rare disease, and we believe that TARA-002 has the potential to be the first approved therapy for patients with macrocystic and macrodominant mixed lymphatic malformations. I am joined today by Dr. Jackie Zumo, co-founder and chief R&D officer at ProTara, who will walk us through an overview of TARA-002 as a potential treatment for LMs. We're also privileged to have Starborn One investigators Dr. Naeem Nasiri and Jesse Jones here to provide their perspective on the disease and how TARA-002 may fit into the treatment armamentarium. Dr. Nasiri is a board-certified vascular and endovascular surgeon leading the vascular care group in Stanford, Connecticut, and is also an adjunct faculty member of the Yale School of Medicine and Yale New Haven Hospital. Dr. Jones is a board-certified interventional radiologist and an associate professor of medicine in the Department of Neurosurgery and Radiology at the University of Alabama. We're also joined by our Chief Commercial Officer, Bill Conkling, who will provide an overview of the LM's market and our views on commercialization. Finally, our CFO, Pat Fabio, will join us for the Q&A. At the end of the call, we will answer questions submitted via the chat box on the lower left-hand corner of the screen. For those of you who are new to Protara, we are a clinical stage biotechnology company developing transformative therapies for the treatment of cancer and rare diseases. Our lead asset is TARA-002, a genetically distinct strain of Streptococcus pyogenes that drives an immunologic identification and attack of cells that carry mutational burden. We are studying OO2 in non-muscle invasive bladder cancer, or NMIBC, where we recently announced positive data at the American Urological Association Conference last week. Today, however, we will focus on TARA-002 in LMs. In addition, we have a program in patients dependent on parental support where we are studying IV choline chloride, an investigational phospholipid substrate replacement therapy, and an ongoing registrational phase three trial. While the focus of today's call will be exclusively on LMs, we remain excited about all three of our pivotal programs and look forward to sharing additional milestones across the portfolio throughout the next 12 months. Now I'd like to talk about TARA-002, our investigational genetically distinct strain of strep pyogenes that is fully inactivated while retaining its immune-stimulating properties. OO2 is supported by an enormous amount of clinical data from its predecessor compound, OK-432, which is approved and marketed under the brand name Pasivinil in Japan by Chugai Pharmaceuticals, where it has been the standard of care in lymphatic malformations for 30 years. TARA-002 builds on the efficacy and safety demonstrated by OK432 NLMs over the years and is now in development by Pertara using modern manufacturing techniques, which have led to potential improvements in the product's profile. The clear unmet need and the potential for 002 as a treatment option in NLMs is reflected in the multiple regulatory designations granted by the FDA, including rare pediatric disease designation, orphan drug designation, breakthrough therapy and fast-track designation, and OO2 is eligible for a priority review voucher, or PRV, upon approval. We expect to complete enrollment of our pivotal STAR or N1 trial in LMs in the second half of this year and intend to submit a BLA based on these data in the second half of 2027. Looking ahead, OO2 has potential use beyond LMs. There is extensive historical literature on the use of OK432 in patients with multiple other maxillofacial cyst types. And indeed, there is direct patient experience with TARA002 and STARBORN1 in two ranula patients, suggesting that this investigational therapeutic can treat other cyst types, providing strong supportive evidence for expanding the opportunity for 002. As we announced last week, we've had productive, ongoing dialogue with the FDA on our LMS program. We received confirmation that the review of OO2 in LMS has been moved from the Office of Vaccines Research and Review to the Office of Therapeutic Products, or OTP. OTP is a division that has significant experience in pediatric rare diseases. It is also the review division for OO2 in NMIBC. The FDA has stated that they will evaluate the risk-benefit profile of 002 and LMs based on the results of Starborn 1. They have not requested any changes to the sample size or the endpoints of the trial. They have not requested an additional pivotal study and have confirmed that our non-clinical package is complete and that no additional reactogenicity or immunogenicity studies are required. We are pleased to have actionable feedback from the FDA on the path to registration for OO2 and LNs and look forward to submitting a BLA in the second half of 2027. I will now turn the call over to Dr. Nasiri to talk about lymphatic malformations and the unmet need in this patient population.