On June 3, 2022 Sapience Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery and development of peptide therapeutics to address difficult-to-treat cancers, reported data from a Phase 1 study demonstrating efficacy proof-of-concept of the Company’s lead program, ST101, in patients with refractory solid tumors (Press release, Sapience Therapeutics, JUN 3, 2022, View Source [SID1234615517]). The data will be presented in a poster discussion session at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL on June 5, 2022.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

ST101, a first-in-class peptide antagonist of C/EBPβ, is currently being evaluated in the Phase 2 portion of an ongoing Phase 1-2 clinical study in patients with advanced unresectable and metastatic solid tumors (NCT04478279). The primary objectives of the Phase 1 clinical study were to evaluate the safety and tolerability of ST101 and to determine the recommended Phase 2 dose. Secondary and exploratory objectives included pharmacokinetics (PK), preliminary efficacy (RECIST 1.1), and pharmacodynamic (PD) evaluation. The study used a 3+3 dose-escalation design, with once-weekly IV infusion dosing of ST101 at 0.5, 1, 2, 4, 6, 9 mg/kg or a flat dose of 500 mg.

Phase 1 Study Results:

As of May 5, 2022, 25 patients with multi-metastatic disease that were refractory to standard therapy received a median of 8 weeks of treatment
Three patients show ongoing clinical benefit:
One confirmed PR lasting more than 51 weeks in a patient with cutaneous melanoma
Eight patients with varying histologies had stable disease lasting 9-81+ weeks (2 ongoing)
No DLTs, dose modifications, or serious adverse events (SAEs) related to ST101
Pharmacokinetics and pharmacodynamics support the selection of a 500 mg flat dose as the recommended Phase 2 dose
Dr. Alice Bexon, Sapience’s Chief Medical Officer, commented, "We remain extremely pleased with the safety and efficacy ST101 has demonstrated in Phase 1, with evidence of clinical benefit across the whole dose range explored, particularly at higher doses and in patients with melanoma. The Phase 2 expansion portion of the study is going very well, with all cohorts enrolling, and a confirmed partial response in a patient with recurrent glioblastoma, which is very exciting. We believe that ST101’s unique mechanism of action targeting C/EBPß represents a potentially transformative approach to treating cancer."

+indicates ongoing treatment

SD=stable disease
PR=partial response

Details of the poster presentation at ASCO (Free ASCO Whitepaper) 2022 are as follows:

Abstract Number: 3014
Title: Efficacy proof-of-concept from a phase 1 study of a novel therapeutic peptide, ST101, targeting the oncogenic transcription factor C/EBPβ in patients with refractory solid tumors
Session Title/Track: Developmental Therapeutics – Molecularly Targeted Agents and Tumor Biology
Session Type: Poster Discussion Session
Date/Time: June 5, 2022, 2:30 PM CDT

Abstracts and full session details can be accessed through the ASCO (Free ASCO Whitepaper) meeting planner: Abstracts | ASCO (Free ASCO Whitepaper) Annual Meeting

About ST101 and the Phase 1-2 Study
ST101, a first-in-class antagonist of C/EBPβ, is currently being evaluated in the Phase 2 portion of an ongoing Phase 1-2 clinical study in patients with advanced unresectable and metastatic solid tumors (NCT04478279). ST101-101 is an open-label, two-part, Phase 1-2 dose-finding study designed to determine the safety, tolerability, PK, PD, and proof-of-concept efficacy of ST101 in patients with advanced solid tumors. The study consists of two phases: Phase 1 dose escalation/regimen exploration and Phase 2 expansion. In the ongoing dose escalation study, ST101 has demonstrated clinical proof-of-concept with a durable RECIST 1.1-confirmed partial response (PR) in a patient with cutaneous melanoma and evidence of long-lasting stable disease in several additional patients. In the ongoing Phase 2 dose expansion part of the study, ST101 has demonstrated clinical proof-of-concept with a confirmed partial response in a patient with recurrent GBM. Sapience is actively enrolling patients with GBM, metastatic cutaneous melanoma, locally advanced or metastatic hormone-receptor positive breast cancer and castration-resistant prostate cancer. ST101 has been granted Fast Track designation for recurrent GBM and advanced cutaneous melanoma in patients who have disease progression on or after anti-PD-1/anti-PD-L1 therapy, as well as orphan designations from the FDA for advanced melanoma, glioma and AML, and from the European Commission for the treatment of glioma.

On June 3, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported encouraging preliminary safety and efficacy results from its Phase 1B study of unesbulin (PTC596) in advanced leiomyosarcoma (LMS) patients (Press release, PTC Therapeutics, JUN 3, 2022, View Source [SID1234615516]). The results demonstrated that treated patients achieved an 18.2 percent objective response rate and a 51.5 percent disease control rate. In addition, unesbulin was generally well tolerated. The results from the dose escalation study which evaluated unesbulin in combination with dacarbazine (DTIC), will be presented on Saturday, June 4, during the Sarcoma Oral Abstract Session beginning at 1:15 p.m. CDT at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The results demonstrated with unesbulin in LMS patients are very promising," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "Leiomyosarcoma is an aggressive soft tissue sarcoma that has significant high unmet medical need, especially for those patients who have relapsed or are refractory to current treatments. We are excited to bring a new chemical entity to the fight against cancer."

The primary objectives of the study were to determine the recommended Phase 2 dose (RP2D) of unesbulin in combination with DTIC and to characterize the safety profile of the combination. In addition, the secondary objectives included progression free survival and duration of response. Unesbulin 300 mg taken twice a week in combination with DTIC 1,000 mg/m2 every 21 days was established as the RP2D.

"Leiomyosarcoma is a particularly aggressive tumor type that desperately requires new treatment options for patients," said Brian Van Tine, M.D., Ph.D., Sarcoma Program, Washington University and lead clinical investigator. "The preliminary results demonstrate promising early efficacy in patients with advanced leiomyosarcoma."

Based on the preliminary data from the Phase 1B study, PTC has initiated the SUNRISE LMS study. SUNRISE LMS is an international, placebo-controlled, registration-directed study comparing the efficacy and safety of unesbulin and DTIC versus placebo and DTIC in patients with advanced LMS who have received ≥1 prior line of systemic therapy. The study is ongoing (ClinicalTrials.gov identifier: NCT05269355).

About Unesbulin (PTC596)
Unesbulin is an investigational oral tubulin binding agent that arrests tumor cells in G2/M phase, including cancer stem cells, through the action of inhibiting tubulin polymerization. Unesbulin was discovered through PTC’s proprietary discovery platform.

About Leiomyosarcoma
Leiomyosarcoma (LMS) is a rare and aggressive cancer found in smooth muscle tissue. LMS is one of the more aggressive sarcoma subtypes representing 10 to 28 percent1 of all soft tissue sarcomas and has a high risk of recurrence leading to decreased disease-specific survival.2 Approximately 4,000 patients are diagnosed each year in the United States and there is a 2:1 incidence in females compared to males.3

On June 3, 2022 Fate Therapeutics, Inc. (the "Company" or "Fate Therapeutics") (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, reported that the Company will present at the 2022 Jefferies Global Healthcare Conference on Friday, June 10, 2022 at 9:00 AM ET in New York, New York (Press release, Fate Therapeutics, JUN 3, 2022, View Source [SID1234615515]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast, if recorded, of the presentation can be accessed under "Events & Presentations" in the Investors section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website shortly after the event.

On June 3, 2022 Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported that John Lee, chief development officer, and Lis Leiderman, M.D., MBA, chief financial officer, will participate in a fireside chat at the Jefferies Global Healthcare Conference on Friday, June 10, 2022 at 9:00 a.m. ET (Press release, Decibel Therapeutics, JUN 3, 2022, View Source [SID1234615514]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the fireside chat may be accessed by visiting the Investors section of the Decibel Therapeutics website at View Source An archived replay of the webcast will be available on the Company’s website for approximately 90 days following the fireside chat.

On June 3, 2022 Bicara Therapeutics, a clinical-stage biotechnology company developing dual-action biologics designed to modulate the tumor microenvironment to elicit a potent and durable anti-tumor response, reported the presentation of new clinical data on BCA101, a bifunctional antibody designed to target the TGF-β trap to EGFR+ tumors (Press release, Bicara Therapeutics, JUN 3, 2022, View Source;utm_medium=rss&utm_campaign=bicara-therapeutics-presents-data-from-dose-escalation-portion-of-ongoing-phase-1-1b-trial-of-lead-bifunctional-program-bca101-at-the-american-society-of-clinical-oncology-2022-annual-meeting [SID1234615513]). Data from the dose escalation phase of the ongoing Phase 1/1b trial of BCA101 as a monotherapy and in combination with the immune checkpoint inhibitor pembrolizumab will be presented in a poster discussion session at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We observed durable responses across three different indications in a heavily pretreated and advanced cancer population, including a partial response in one patient with relapsed squamous cell lung cancer who was treated with BCA101 as monotherapy. Additionally, observed biomarkers support the mechanisms of action for BCA101 and demonstrate clear remodeling of the tumor microenvironment," said Liviu Niculescu, M.D., Chief Medical Officer of Bicara Therapeutics. "The findings from the dose escalation phase of the ongoing Phase 1/1b trial are compelling and support our view that BCA101 offers a potentially more efficacious and durable treatment option for patients with several types of solid tumors, particularly in combination with other immunotherapies."

"BCA101 is a first-in-class antibody with a unique mechanism of anti-tumor activity that has demonstrated encouraging safety, pharmacokinetic, pharmacodynamic and efficacy profiles," said Philippe L. Bedard, M.D., Staff Medical Oncologist at Princess Margaret Cancer Centre in Toronto.

BCA101 Data Highlights:

A confirmed partial response was observed in one patient with squamous-cell lung cancer (SQCLC) (refractory to chemo and PD-1) who received BCA101 monotherapy treatment. The patient continues in the study.
Durable confirmed responses were achieved with four patients treated with BCA101 in combination with pembrolizumab: two patients with head and neck squamous cell carcinoma (HNSCC) (one refractory to cetuximab, PD-1 and chemotherapy) and two patients with squamous cell carcinoma of the anal canal (SCAC).
BCA101 was safe at all tested dose levels as monotherapy and in combination with pembrolizumab.
BCA101 achieved dose-proportional PK and demonstrated definitive target engagement in both plasma and tumor tissue for both EGFR and TGF-b.
The recommended dose was declared at 1500 mg once weekly for BCA101 as monotherapy and in combination with pembrolizumab.
BCA101 is currently being evaluated in a Phase 1/1b study as monotherapy and in combination with pembrolizumab as a first-line therapy in patients with unresectable, recurrent or metastatic HNSCC and as a second-line therapy in patients with advanced SCAC who have received prior chemotherapy. A third cohort of patients with advanced or incurable cutaneous squamous cell carcinoma who have received previous anti-PD-1 therapy will be treated with BCA101 as a monotherapy. Bicara initiated the dose expansion arm of this study in February 2022. Primary results are expected in the second half of 2022.

ASCO Presentation Details

Title: A phase 1 trial of the bifunctional EGFR/TGF-β fusion protein BCA101 alone and in combination with pembrolizumab in patients with advanced solid tumors
Abstract: 2513
Session Type/Title: Poster Session/Developmental Therapeutics – Immunotherapy
Session Date and Time: Sunday, June 5, 2022, 12:30 – 2:00 p.m. EDT
About BCA101

BCA101 is a first-in-class EGFR / TGF-β-trap bifunctional antibody designed to modulate the tumor microenvironment by binding to the well-validated EGFR antigen and disabling TGF-β, a signaling molecule that plays a key role in suppressing the immune response within the tumor microenvironment. Promising preclinical data suggest that BCA101 is superior to the anti-EGFR antibody cetuximab and single-agent TGF-b inhibitors in preventing tumor recurrence, as well as in remodeling the tumor microenvironment and restoring immune activation. An ongoing Phase 1/1b dose-escalation clinical trial of BCA101 was initiated in July 2020 and has enrolled patients with various advanced solid tumors both as a single agent, as well as in combination with pembrolizumab, a PD-1 inhibitor. A recommended dose for expansion has been declared and the expansion phase of the study is currently enrolling. For more information, please visit study number NCT04429542 at www.clinicaltrials.gov.