saridegib oral (IPI 926 oral) / Infinity Pharma, Mundipharma - LARVOL DELTA

Phase 2 Trial of Topical Application of the Hedgehog Inhibitor Patidegib in Patients With Gorlin Syndrome. (PubMed, Br J Dermatol) - "Patidegib Topical Gel warrants further clinical development."

Journal • P2 data • Basal Cell Carcinoma • Oncology • PTCH1

A phase 3, randomized controlled trial of topical patidegib gel 2% in patients with Gorlin syndrome (EADV 2024) - "A new Phase 3 trial designed to investigate patidegib 2% gel in PTCH1+/- GS patients with a high burden of facial BCCs at baseline has been opened at Sites in the EU, UK, and USA. 25 SEPTEMBER - 28 SEPTEMBER 2024 POWERED BY M-ANAGE.COM"

Clinical • Late-breaking abstract • P3 data • Basal Cell Carcinoma • Genetic Disorders • Non-melanoma Skin Cancer • Oncology • PTCH1

Topical hedgehog inhibitors for basal cell carcinoma: how far away are we? (PubMed, Expert Opin Pharmacother) - "These agents such as vismodegib and sonidegib are associated with short-lived responses as well as significant adverse effects including myalgias, dysgeusia, and alopecia. Patidegib and itraconazole are two topical Hh inhibitors agents emerging as alternatives to oral Hh inhibiton for difficult-to-treat BCCs...Ongoing and recent clinical studies on topical Hedgehog inhibitors show great promise for the development of an agent with a high therapeutic index and limited adverse effects. If patidegib continues to show clinical efficacy in randomized controlled trials, it may become a universal therapy for all subtypes of difficult-to-treat BCC."

Journal • Alopecia • Basal Cell Carcinoma • Musculoskeletal Pain • Non-melanoma Skin Cancer • Oncology • Pain • Solid Organ Transplantation • Transplantation

Hedgehog pathway inhibition by topical patidegib to reduce BCC burden in patients with basal cell nevus (Gorlin) syndrome. (ASCO 2018) - P2; "Topical patidegib gel has the potential to prevent and mitigate facial BCCs in Gorlin patients. These results will be confirmed in a Phase 3 trial enrolling approximately 150 patients in the United States and Europe."

Clinical • Oncology

Infinity completes enrollment in phase 2 trial of IPI-926, an oral smoothened antagonist, in pancreatic cancer and provides additional program updates (Market Watch) - P2, N=122; IPI-926-03; Infinity Pharmaceuticals announced that it has completed enrollment of its P2 trial of IPI-926 in pts with previously untreated pancreatic cancer; The primary endpoint of the trial is overall survival; Additional data from the trial will be presented at a future medical meeting; Preclinical data of IPI-926 in combination with nab-paclitaxel in a xenograft model of pancreatic cancer will also be presented

Anticipated data • Enrollment completion • Pancreatic Cancer

A phase Ib trial of IPI-926, a hedgehog pathway inhibitor, plus gemcitabine in patients with metastatic pancreatic cancer (2012 Gastrointestinal Cancers Symposium) - P1/2; N=16; IPI-926-03; The most common AEs occurring were fatigue (75% total, 6% ≥ G3), thrombocytopenia (63%, 25%), anemia (56%, 13%), nausea (50%, 0%), diarrhea (44%, 0%), vomiting (44%, 0%), peripheral edema (44%, 0%), pyrexia (44%, 0%), and AST increase (44%, 13%); Dose reductions of IPI-926 occurred in 3 (19%) pts, and of gemcitabine occurred in 11 (69%) pts, primarily for thrombocytopenia (9 pts)

P1b data • Pancreatic Cancer

Pilot study of cetuximab and the hedgehog inhibitor IPI-926 in recurrent head and neck cancer (clinicaltrials.gov) - P1, N=24; Recruiting -> Active, not recruiting.

Enrollment closed • Head and Neck Cancer • Oncology

Phase shift: P1 completed for the indication solid tumors (Infinity Pharmaceuticals) - IPI926 / Infinity

Corporate Pipeline Update

Infinity Pharma stops phase 2 trial of saridegib drug; Shares plunge (Wall Street Journal Online) - P1/2, N=130; IPI-926-03; Drug developer Infinity Pharmaceuticals Inc said it is stopping the P2 trial of saridegib to treat pancreatic cancer, after it was found that the trial wouldn't meet the company's goals; Infinity said SR when compared with a placebo plus gemcitabine showed rates favoring those receiving the placebo, due partly to more pts with a progressive form of the disease ending up in the saridegib arm of the study

Trial terminated • Pancreatic Cancer

ANTI-BCC EFFICACY OF A TOPICAL HEDGEHOG INHIBITOR IN BCNS PATIENTS IN A PHASE 2 RANDOMIZED CONTROLLED TRIAL (EADO 2018) - "Importantly, unlike oral HH inhibitors, topical patidegib did not produce hair loss, taste loss, or muscle cramps and produced circulating blood levels at least 500-fold lower those found in patients treated with oral patidegib. In , patidegib is a promising new topical approach to treatment and prevention of BCC and phase III studies are now planned"

Clinical • P2 data • Oncology

A phase Ib trial of FOLFIRINOX plus saridegib, an oral hedgehog (Hh) inhibitor, in pts with advanced pancreatic cancer (PDAC) (ASCO 2012) - Presentation time: Mon, Jun 4; 8:00 AM - 12:00 PM; Anticipated presentation at ASCO 2012

Anticipated P1 data presentation • Pancreatic Cancer

Q2 '11 Results (Infinity Pharmaceuticals) - IPI-926 / Infinity Pharma; Anticipated enrollment completion of P2 trial in pancreatic cancer in H2 '11

Anticipated enrollment completion • None • Pancreatic Cancer

Development of a topical Hedgehog inhibitor for prevention of new surgically eligible basal cell carcinomas in patients with Gorlin Syndrome: Phase 3 trial (SID 2019) - P3; "...We have incorporated the cyclopamine derivative patidegib into a gel that in two Phase 2 trials has demonstrated efficacy potential vs. BCCs - complete response (12 of 45 tumors in patidegib topical gel arms vs. 0 of 16 in vehicle) and fewer new BCCs (3 out of 12 subjects in patidegib topical gel arms vs. 3 out of 5 in vehicle; ITT one-sided P=0.05); with none of the significant oral HH inhibitor class adverse events observed...Therefore, we have initiated a Phase 3 Trial in North America and Europe with the aim of enrolling 150 Gorlin subjects randomized 1:1 for application of active vs. placebo gel to the face for a year, testing prevention of the development of new surgically eligible BCCs (ClinicalTrials.gov Identifier: NCT03703310). Patients interested in participating and clinicians interested in more details can contact us at clinical@pellepharm.com."

Clinical • P3 data