On September 9, 2024 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company, reported it will share new data for TEVIMBRA (tislelizumab) at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2024 Congress (ESMO 2024) in Barcelona, Spain, September 13-17, 2024 (Press release, BeiGene, SEP 9, 2024, View Source [SID1234646423]). BeiGene has seven abstracts accepted at ESMO (Free ESMO Whitepaper) 2024, with one selected for the special session revisiting the ESMO (Free ESMO Whitepaper) Virtual Plenary held in February 2024.

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New Data Add to Evidence for TEVIMBRA Across Multiple Disease States

As an encore to the ESMO (Free ESMO Whitepaper) plenary, interim results from the RATIONALE-315 study show the statistically significant event-free survival (EFS) and trend for overall survival (OS) benefit favoring neoadjuvant tislelizumab plus chemotherapy with adjuvant tislelizumab vs. placebo plus chemotherapy with adjuvant placebo for patients with resectable non-small cell lung cancer (NSCLC) (session #VP1-2024, Sept. 13 from 4:17-4:29 p.m. CEST). These results further reinforce the data presented at ESMO (Free ESMO Whitepaper) 2023 showing major pathologic response (MPR) and pathologic complete response (pCR) rate were significantly improved: 56.2% vs 15.0% (P<.0001) and 40.7% vs 5.7% (P<.0001), respectively. The safety profile of the tislelizumab arm was consistent with that of individual therapies, with 72.1% (vs. 66.4% in the placebo arm) of patients in the tislelizumab arm experiencing grade ≥3 treatment-related adverse events (TRAEs) and 15.5% (vs. 8.0% in the placebo arm) experiencing serious TRAEs. The most common TRAEs were decreased neutrophil count, decreased white blood cell count and alopecia. Improvement in symptomology from RATIONALE-315 will also be shown as patient-reported outcomes (poster #1213P, Sept. 14).
Three-year overall survival data from RATIONALE-305 continue to demonstrate the long-term efficacy and safety of tislelizumab plus chemotherapy in patients with first-line advanced or metastatic gastric cancer/gastroesophageal junction cancer (GC/GEJC) (poster #1437P, Sept. 16), as well as improvements in patient-reported outcomes (poster #1449P, Sept. 16).
Long-term outcomes in the ITT population as well as those receiving long-term exposure to tislelizumab plus chemotherapy as treatment for first line squamous NSCLC in RATIONALE-307 show a continued OS benefit with clinically promising four-year OS rates (poster #1323P, Sept. 14).
Relative effectiveness of tislelizumab vs. other anti-PD-1 treatments approved in the European Union and UK for second-line esophageal squamous cell carcinoma (ESCC) using anchored simulated treatment comparison of data from RATIONALE-302 and comparator clinical studies (poster #1417P, Sept. 16).
"TEVIMBRA has shown potential across multiple disease states, and the data at ESMO (Free ESMO Whitepaper) 2024 further supports its position as the foundational asset of our solid tumor portfolio," said Dr. med. Jan-Henrik Terwey, Vice President, Medical Affairs Europe at BeiGene. "As part of our commitment to bring innovative cancer medicines to more patients, we recently launched TEVIMBRA in EMA-approved indications in Germany, Austria and Norway, and we are working to make TEVIMBRA available across Europe."

TEVIMBRA in Europe

BeiGene recently launched TEVIMBRA in the first European countries following EU marketing authorizations for the treatment of eligible patients with ESCC and NSCLC. TEVIMBRA is also approved in the UK and Switzerland for eligible patients with advanced or metastatic ESCC.

"Advanced or metastatic ESCC and NSCLC are aggressive cancers with limited treatment options," said Markus Moehler, M.D., Ph.D., of the Johannes Gutenberg University Medical Center Mainz in Germany. "The availability of tislelizumab for these patients represents an important next step to advance the treatment landscape."

The European Commission approvals were based on the results from four randomized Phase 3 studies in the RATIONALE program: RATIONALE-302 (NCT03430843) for ESCC and RATIONALE-307 (NCT03594747), RATIONALE-304 (NCT03663205) and RATIONALE-303 (NCT03358875) for NSCLC. The approved indications for TEVIMBRA in the EU are:

In combination with carboplatin and either paclitaxel or nab-paclitaxel for the first-line treatment of adult patients with squamous NSCLC who have locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or metastatic NSCLC.
In combination with pemetrexed and platinum-containing chemotherapy for the first-line treatment of adult patients with non-squamous NSCLC whose tumors have PD-L1 expression on ≥50% of tumor cells with no EGFR or ALK positive mutations and who have locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or metastatic NSCLC.
As monotherapy for the treatment of adult patients with locally advanced or metastatic NSCLC after prior platinum-based therapy. Patients with EGFR mutant or ALK positive NSCLC should also have received targeted therapies before receiving tislelizumab.
As monotherapy for the treatment of adult patients with unresectable, locally advanced or metastatic ESCC after prior platinum-based chemotherapy.
About TEVIMBRA (tislelizumab)

Tislelizumab is a uniquely designed humanized immunoglobulin G4 (IgG4) anti-programmed cell death protein 1 (PD-1) monoclonal antibody with high affinity and binding specificity against PD-1. It is designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors.

On September 9, 2024 Prelude Therapeutics Incorporated (Nasdaq: PRLD) ("Prelude" or the "Company"), a clinical-stage precision oncology company, reported the publication of an abstract regarding PRT3789 at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024 taking place in Barcelona, Spain September 13-17, 2024 (Press release, Prelude Therapeutics, SEP 9, 2024, View Source [SID1234646438]). The abstract can be found on the ESMO (Free ESMO Whitepaper) 2024 website Registration | ESMO (Free ESMO Whitepaper) Congress 2024

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"We are excited for the opportunity to share the first ever clinical data of a novel, highly-selective SMARCA2 degrader," stated Jane Huang, M.D., President and Chief Medical Officer of Prelude. "Patients whose cancer has a SMARCA4 mutation have limited treatment options and generally very aggressive disease. Although PRT3789 as a first-in-class molecule targeting a novel mechanism is early in its development, we are highly encouraged by the safety profile, target engagement and clinical activity we have seen to date."

PRT3789 is a potent and highly selective, first-in-class SMARCA2 degrader, in Phase 1 clinical development in biomarker selected SMARCA4 mutant patients. Enrollment remains on track, and the Company expects to conclude monotherapy dose escalation by year end 2024 and identify a recommended Phase 2 dose. In addition, enrollment of patients into back-fill cohorts enriched for NSCLC and SMARCA4 loss-of-function mutations is ongoing. Objectives for this first Phase 1 clinical study are to establish the safety and tolerability profile of PRT3789 as both monotherapy and in combination with docetaxel, evaluate activity, pharmacokinetics and pharmacodynamics and determine a dose and potential indications for advancement into a registrational clinical trial.

Oral presentation title: First Clinical Results from a Phase 1 Trial of PRT3789, a First-in-Class Intravenous SMARCA2 Degrader, in Patients with Advanced Solid Tumors with a SMARCA4 Mutation.

Observations in the abstract include:

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As of the March 7, 2024 data cutoff date, 40 pts had been enrolled (NSCLC [18], pancreatic [5], breast [3], esophageal [2], other [12]; 55% have loss-of-function mutations;

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Dose escalation had proceeded through 6 levels, from 24-212 mg, with 2 backfill cohorts opened;
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No DLTs or study drug-related SAEs have been reported;
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The most common AEs reported, of any grade or relatedness, are nausea (25%), constipation and dyspnea (each 17.5%), decreased appetite and fatigue (each 15%), and anemia (12.5%);
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Dose-related increases in AUC were observed;
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Dose-dependent decreases in SMARCA2 levels were seen at all doses with a trend for increasing depth and duration with increasing doses;
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Minimal effects on SMARCA4 levels were seen;
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Clinical activity of PRT3789 therapy noted to date includes RECIST partial responses, tumor shrinkage and prolonged stable disease (longer than response to most recent therapy) in patients with advanced, heavily pretreated esophageal cancer and NSCLC.

Updated data will be presented at ESMO (Free ESMO Whitepaper).

Investor Conference Call and Webcast Information

Prelude Therapeutics will host a conference call, live webcast with slides and a Q&A on Friday, September 13, 2024 at 12:00 PM EST. A live webcast of the presentation will be available at Events & Presentations – Prelude Therapeutics (preludetx.com). A replay of the webcast will be available shortly after the conclusion of the call and archived on the Company’s website for 60 days following the call. The Company will be posting its updated corporate presentation shortly after 10:00 AM EST on its website at Events & Presentations – Prelude Therapeutics (preludetx.com).

On September 9, 2024 NeoImmuneTech, Inc. (NIT or "NeoImmuneTech"), a global leader in T cell-based immunotherapy, reported that it will present interim results of the Phase 1b clinical trial (NIT-112) combining a CAR-T therapy with NT-I7 (efineptakin alfa) during the ‘Mini oral session 1: Haematological malignancies’ at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) in Barcelona, Spain, from September 13 to 17 (Press release, NeoImmuneTech, SEP 9, 2024, https://www.prnewswire.com/news-releases/neoimmunetech-presents-promising-interim-results-of-car-t-combination-with-its-nt-i7-asset-at-esmo-2024-302241828.html [SID1234646454]). These initial results present a promising approach to enhancing the potential of CAR-T therapies while maintaining a stable safety profile.

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The NIT-112 clinical trial involves patients with Large B-cell Lymphoma (LBCL) who received CAR-T therapies such as Kymriah, Yescarta, or Breyanzi, followed by NT-I7 administration 21 days after CAR-T infusion. The trial aims to evaluate safety, tolerability, the recommended Phase 2 dose (RP2D), and the potential for NT-I7 to enhance CAR-T expansion, persistence and stemness.

Interim results show that at dose levels 4 and 5, which are considered mid-level doses (360, 480 µg/kg), NT-I7 demonstrated a stable safety profile. No cases of Cytokine Release Syndrome (CRS) or Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), which are known as high-risk side effects of CAR-T therapies, were observed subsequent to NT-I7 administration. Only mild, manageable side effects (Grade 1-2), such as injection site erythema and swelling were observed in 6 out of 11 patients (54.5%). Overall, a strong safety profile was observed.

NT-I7 administration resulted in significant amplification of CAR-T cells, prolonged persistence and an increase in T cell stemness, characterized by a higher frequency of T cells with a stem-cell memory (Tscm) phenotype (CD45RA+CCR7+CD95+). The overall response rate (ORR) from the interim results was 81.1% (9 out of 11 patients), with 7 achieving a complete response (CR) and 2 achieving a partial response (PR). Kymriah is known to have an ORR of 52% in LBCL patients.

Dr Luke Oh, President and Chief Executive Officer of NeoImmuneTech, Inc., said: "We are thrilled by the preliminary results of study NIT-112 showing NT-I7’s ability to amplify CAR-T cells. This successful amplification of CAR-T cells is expected to translate into improved patient outcomes. NIT plans to actively pursue technology transfer related to the combination of NT-I7 with CAR-T therapies and we look forward to accelerating our discussions with the organizations already conducting preclinical studies combining NT-I7 with our own CAR-T technologies."

Title: Phase 1b study of NT-I7 (efineptakin alfa), a long-acting IL-7, post-CD19-directed CAR T cell therapy in diffuse large B-cell lymphoma (DLBCL)
Presentation Number: 806MO
Speakers: Armin Ghobadi (St. Louis, United States of America)
Lecture Time: 15:05 – 15:10 (Fri, 2024. 09. 13)
Session Name: Mini oral session 1: Hematological malignancies (ID 31)
Room: Toledo Auditorium – Hall 3

About NT-I7 (efineptakin alfa) (rhIL-7-hyFc)
NT-I7 (efineptakin alfa) is the only clinical-stage long-acting human IL-7 and is being developed in oncologic and immunologic indications, where T cell amplification and increased functionality may provide clinical benefit. IL-7 is a fundamental cytokine for naïve and memory T cell development and sustaining immune response to chronic antigens (as in cancer) or foreign antigens (as in infectious diseases). NT-I7 exhibits favorable PK/PD and safety profiles, making it an ideal combination partner. NT-I7 is being studied in multiple clinical trials in solid tumors and as vaccine adjuvant. Studies are being planned for testing in hematologic malignancies, additional solid tumors and other immunology-focused indications.

On September 9, 2024 BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX: BCT) ("BriaCell" or the "Company"), a clinical-stage biotechnology company that develops novel immunotherapies to transform cancer care, reported a poster presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 39th Annual Meeting, held November 6-10, 2024, in Houston, TX (Press release, BriaCell Therapeutics, SEP 9, 2024, View Source [SID1234646424]).

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"We are thrilled to be invited to present our data at this prestigious conference," stated Miguel Lopez-Lago, PhD, BriaCell’s Chief Scientific Officer. "We look forward to continuing our investigations of novel targeted immunotherapy candidates in clinical studies with the goal of making a positive contribution to the lives of breast cancer and prostate cancer patients."

The details about the presentation and session Information are as follows:

Location: Exhibit Halls A B George R. Brown Convention Center, Houston, TX
Date and Time: Friday, Nov. 8, 2024, 9:00 am -7:00 pm CST

Following the presentation, a copy of the poster will be posted on View Source

On September 9, 2024 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported completion of the first cohort of the Phase 2 STARBORN-1 trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of pediatric patients with lymphatic malformations (LMs) (Press release, Protara Therapeutics, SEP 9, 2024, https://ir.protaratx.com/news-releases/news-release-details/protara-therapeutics-announces-completion-first-cohort-phase-2 [SID1234646439]). Enrollment is now underway in additional cohorts.

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"The initial data is compelling and reflective of the significant benefit observed in previous studies with OK-432, the predecessor of TARA-002," said Nancy Bauman, MD, Children’s National Medical Center: Children’s Research Institute, Washington DC, and investigator for the STARBORN-1 trial. "There is a pressing need for an effective therapeutic option for LMs, a rare condition mainly affecting children for which there are currently no U.S. FDA approved agents. I remain excited about the potential for TARA-002 to play a meaningful role in the treatment of these patients and look forward to future learnings from this important study."

Of three patients treated in the first cohort, which enrolled individuals six years to less than 18 years of age, two patients treated with TARA-002 achieved a complete response after receiving one dose of TARA-002; the responses were seen in a patient with a macrocystic lymphatic malformation and a patient with a ranula. The safety and tolerability seen in this cohort was consistent with that of the historical experience with OK-432 and included treatment emergent adverse events (TEAEs) of pain, swelling, fatigue, and body temperature increases. All TEAEs were mild to moderate and resolved.

"We are pleased with the progress of our Phase 2 STARBORN-1 trial and the encouraging results we have seen thus far," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We have received positive feedback from our investigators and already have a number of patients on waiting lists for our subsequent cohorts, and expect to share initial results from our next cohort in the first half of 2025."

STARBORN-1 is a Phase 2 single-arm, open-label, prospective clinical trial evaluating the safety and efficacy of intracystic injection of TARA-002 for the treatment of macrocystic and mixed cystic LMs (≥ 50% macrocystic disease) in participants six months to less than 18 years of age. The trial will enroll approximately 30 patients including age de-escalation safety lead-in cohorts of children ages six years to less than18 years, two years to less than six years, and six months to less than two years, who will receive up to four injections of TARA-002 spaced approximately six weeks apart.

The primary endpoint of the trial is the proportion of participants with macrocystic and mixed cystic LMs who demonstrate clinical success, defined as having either a complete response (90% to 100% reduction from baseline in total LM volume) or substantial response (60% to less than 90% reduction in total LM volume) as measured by axial imaging.

About TARA-002 in LMs

TARA-002 is an investigational cell therapy based on the broad immunopotentiator, OK-432, which was originally granted marketing approval by the Japanese Ministry of Health and Welfare as an immunopotentiating cancer therapeutic agent. This cell therapy is currently approved in Japan and Taiwan for LMs and has been used to successfully treat thousands of pediatric patients with this rare condition. In addition, OK-432 was studied in the largest ever conducted Phase 2 trials in LMs, in which the therapy was administered via a now-closed compassionate use program led by the University of Iowa.

TARA-002 has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of LMs.

About Lymphatic Malformations

Lymphatic malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of three years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection; and cosmetic and other functional disabilities.