On May 10, 2022 SQZ Biotechnologies (NYSE: SQZ), focused on unlocking the full potential of cell therapies for multiple therapeutic areas, reported first quarter 2022 financial results and recent portfolio updates (Press release, SQZ Biotech, MAY 10, 2022, View Source [SID1234614125]).

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"Our year is off to an exciting start with the FDA IND clearance to initiate clinical trials of our eAPCs – our multifunctional mRNA-based investigational cell therapy that targets solid tumors by engineering multiple T cell signals simultaneously," said Armon Sharei, Ph.D., Chief Executive Officer and Founder at SQZ Biotechnologies. "We were also delighted by the recent FDA Fast Track Designation for our lead APC clinical candidate, which had promising clinical results in 2021, providing the potential to accelerate its registrational path. We look forward to continued progress on the evolution of our technology platform and unique therapeutic pipeline with anticipated clinical data across our APC, eAPC, and AAC trials in the second half of this year."

First Quarter 2022 and Recent Portfolio Updates

SQZ Antigen Presenting Cell ("APC") Platform in Oncology

Granted FDA Fast Track Designation for lead cell therapy candidate SQZ-PBMC-HPV; the designation is designed to accelerate the development and review of treatments for serious or life-threatening diseases
Published peer reviewed preclinical research supporting the development of SQZ APC cancer vaccine therapeutic programs in the Journal of Immunology
Continued enrollment of high dose monotherapy and combination with checkpoint inhibitors in the Phase 1/2 trial of SQZ-PBMC-HPV
SQZ Enhanced Antigen Presenting Cell ("eAPC") Platform in Oncology

Received FDA IND clearance to initiate a Phase 1/2 clinical trial (COMMANDER-001) of the first eAPC therapeutic candidate in patients who have HPV16+ solid tumors
Initiated enrollment of monotherapy stage of the COMMANDER-001 trial
SQZ Activating Antigen Carriers ("AAC") Platform in Oncology

Continued enrolling and opening additional sites for the monotherapy stage of the ENVOY-001 Phase 1/2 (SQZ-AAC-HPV-101) trial
SQZ Tolerizing Antigen Carriers ("TAC") Platform in Immune Tolerance

Published peer reviewed preclinical research in Frontiers in Immunology supporting potential SQZ TAC platform therapeutic development across a variety of complex autoimmune diseases
Progressed studies supporting anticipated TAC IND submission for celiac disease in the first half of 2023; company’s point-of-care manufacturing system intended to produce clinical batches
SQZ Potential Pipeline Expansion Research

Awarded $2 million grant from the National Institutes of Health that will support the development of cell engineering methods to reprogram immune cells directly into dopamine-producing neurons as a potential novel therapeutic approach for the treatment of Parkinson’s disease
First Quarter 2022 Financial Highlights

Revenue for the quarter ended March 31, 2022, was $2.9 million compared to $5.5 million for the same period in 2021
Research and development expenses for the quarter ended March 31, 2022, were $17.0 million compared to $14.7 million for the same period in 2021; the increase was primarily due to higher development and manufacturing costs associated with our clinical-stage product candidates, as well as increased personnel-related costs to support continued progress with the Company’s pipeline
General and administrative expenses for the quarter ended March 31, 2022, were $6.9 million compared to $6.1 million for the same period in 2021; the increase was primarily due to higher personnel and other corporate-related costs, including stock-based compensation expense and other costs related to operating as a public company
Net loss for the quarter ended March 31, 2022, was $21.0 million, compared to $15.4 million for the same period in 2021
As of March 31, 2022, the Company had cash and cash equivalents of $122.9 million and anticipates this will be sufficient to fund operating expenses and capital expenditure requirements into the second half of 2023

On May 10, 2022 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported its business update for the quarter ended March 31, 2022 (Press release, Transgene, MAY 10, 2022, View Source [SID1234614123]).

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Over the first quarter of 2022, all of Transgene’s preclinical and clinical assets progressed in line with expectations. All clinical-stage immunotherapies are slated to deliver data by the end of the year.

Key events of the period and upcoming news flow

TG4050

At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2022 annual meeting, held in New Orleans, LA, April 8-13, Transgene discussed new preliminary positive data on TG4050, its individualized cancer vaccine. These immunological and clinical data highlight the potential of this highly innovative neoantigen vaccine.

A poster on the progress of the two ongoing Phase I trials of TG4050 has been accepted for presentation at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, taking place in Chicago, IL, June 3-7, 2022. More information will be provided on May 27, 2022, after the release of the abstracts by ASCO (Free ASCO Whitepaper).

An article on the two ongoing trials with TG4050 was also published in the Journal for ImmunoTherapy of Cancer [[1]]. The publication demonstrated that it is possible to develop a patient specific vaccine within a few weeks for patients with a low to moderate tumor mutational burden.

BT-001

Promising preclinical data with BT-001 were presented at AACR (Free AACR Whitepaper) 2022 and published in the Journal for ImmunoTherapy of Cancer [[2]] demonstrating the broad and robust antitumor activity of this Invir.IO oncolytic virus.

Transgene and BioInvent will provide an update on the progress of the clinical trial of BT-001 in Q2 2022. Initial Phase I results will be presented at a scientific conference in H2 2022. These first results aim to establish the tolerability of BT-001 and to determine the dose and administration schedule for further development.

Invir.IO collaboration with PersonGen BioTherapeutics

Transgene announced the launch of a preclinical collaboration with PersonGen BioTherapeutics. This collaboration aims to evaluate the feasibility and efficacy of a combination therapy against solid tumors, combining PersonGen’s CAR-T cell injection with an oncolytic virus from the Invir.IO platform.

Governance

Transgene plans to reinforce its corporate governance by separating the roles of Chairman and CEO. Transgene’s Board of Directors has proposed Dr. Alessandro Riva, MD, as the Non-executive Chairman of the Company. If this nomination is accepted at the upcoming General Shareholder Meeting (May 25, 2022), the roles of Chairman and CEO will be separated. With 30 years of experience in the Life Sciences industry, Dr. Riva will be working closely with Transgene’s CEO Hedi Ben Brahim to realize the potential of the Company’s technology platforms and products to benefit cancer patients.

Transgene’s Board of Directors also proposed the appointment of Prof. Jean‑Yves Blay (subject to the authorization of the public authority to which he reports) and Laurence Espinasse as directors.

Steven Bloom joined Transgene as Vice President, Chief Business Officer (CBO). In this position, he has become a member of the executive committee, leading global business development strategy, alliance management and program management. In particular, he is focused on building the profile of Transgene in the US, where he is based, as part of establishing the Company as a world leader in virus-based immunotherapies.

Transgene also announced that the date of the release of the first half 2022 financial results and of the interim report has been advanced to September 7, 2022.

Summary of key ongoing clinical trials and expected milestones

myvac

TG4050

Phase I

NCT03839524

Targets: tumor neoantigens

ü Codeveloped with NEC

ü New positive data in first patients demonstrating the immunogenicity of the vaccine as well as first signs of clinical activity presented at AACR (Free AACR Whitepaper) 2022

Ü Additional data on the 2 trials expected at ASCO (Free ASCO Whitepaper) (June 2022) and H2 2022

Ovarian cancer – after surgery and first-line chemotherapy

ü Trial ongoing in the USA and in France

ü Patient enrollment progressing in line with forecast

TG4050

Phase I

NCT04183166

HPV-negative head and neck cancer – after surgery and adjuvant therapy

ü Trial ongoing in the UK and in France

ü Patient enrollment progressing in line with forecast

TG4001

+ avelumab
Phase II

NCT03260023

Targets: HPV16 E6 and E7 oncoproteins

Recurrent/metastatic anogenital HPV-positive – 1st (patients ineligible for chemotherapy) and 2nd lines

ü Randomized Phase II trial comparing the combination of TG4001 with avelumab versus avelumab alone

ü Active patient enrollment in Europe (France and Spain), trial initiated in the USA

Ü Results of the interim analysis expected in Q4 2022 (N≈50)

Invir.IO

BT-001

Phase I/IIa

NCT04725331

Payload: anti-CTLA4 antibody and GM-CSF cytokine

Solid tumors

ü Co-development with BioInvent

ü Very encouraging preclinical results presented at AACR (Free AACR Whitepaper) 2022

ü Trial ongoing in France, Belgium and approved in the USA

Ü Update on clinical trial expected in Q2 2022

Ü First Phase I clinical results to be presented at a scientific congress in H2 2022

TG6002

Phase I/IIa

NCT03724071

Payload: FCU1 for the local production of a 5-FU chemotherapy

Gastro-intestinal cancer (colorectal cancer for Phase II) – Intravenous (IV) administration

ü Multicenter trial ongoing in Belgium, France and Spain

ü Proof-of-concept data of the IV administration presented in 2021 (ESMO & AACR (Free AACR Whitepaper))

ü Dose escalation completed to the maximum projected dose (3×109 pfu), confirming the good safety profile. Assessment of this intensified administration schedule ongoing

Ü End of Phase I expected mid-2022

TG6002

Phase I/IIa

NCT04194034

Colorectal cancer with liver metastasis – Intrahepatic artery (IHA) administration

ü Multicenter trial ongoing in the UK and in France

ü Ongoing enrollment of patients from the latest dose escalation cohort (109 pfu)

Ü First data expected mid-2022

During the first quarter of 2022, revenue from collaborative and licensing agreements was mainly composed of revenue from the collaboration with AstraZeneca.

As of March 31, 2022, government financing for research expenditures mainly consisted of accrual of 25% of the research tax credit expected for 2022 (€1.7 million in the first quarter of 2022 compared to €1.5 million for the same period in 2021).

Cash, cash equivalents and other financial assets

Cash, cash equivalents and other financial assets stood at €46.8 million as of March 31, 2022, compared to €49.6 million as of December 31, 2021. In the first quarter of 2022, Transgene’s net cash burn was €2.8 million, compared to €7.2 million for the same period in 2021. This decrease is notably linked to the receipt in January 2022 of the $8 million payment from AstraZeneca following the exercise of a first license option in December 2021 for an oncolytic virus developed by Transgene.

The Company holds shares of Tasly BioPharmaceuticals valued at €18.9 million at the end of December 2021.

The Company has a financial visibility through the end of 2023.

On May 10, 2022 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a clinical-stage precision oncology company designing and developing novel targeted therapies for cancer treatment, reported the U.S. Food and Drug Administration (FDA) granted an eighth regulatory designation, and third Breakthrough Therapy designation, to lead drug candidate repotrectinib (Press release, Turning Point Therapeutics, MAY 10, 2022, View Source [SID1234614122]).

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Breakthrough Therapy designation (BTD) was granted for the treatment of patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC) who have been previously treated with one ROS1 tyrosine kinase inhibitor and who have not received prior platinum-based chemotherapy. The efficacy analyses supporting the BTD application included approximately 50 patients pooled from the Phase 1 and Phase 2 portions of the TRIDENT-1 study. Efficacy evaluations in Phase 2 patients were by physician assessment.

"We are excited to receive our third BTD and eighth overall FDA regulatory designation for repotrectinib in an indication where there are no approved targeted therapies," said Mohammad Hirmand, M.D., Chief Medical Officer. "We are encouraged by the continued momentum in TRIDENT-1 with enrollment targets achieved in cohorts EXP-1, EXP-4 and EXP-6. We look forward to continuing to progress repotrectinib toward registration with our first pre-NDA meeting with the FDA to discuss the topline data by blinded independent central review from the ROS1-positive advanced NSCLC cohorts of the TRIDENT-1 study expected later this quarter."

BTD is granted by the FDA to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition. The criteria for BTD require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

Repotrectinib was previously granted two BTDs in: ROS1-positive metastatic NSCLC patients who have not been treated with a ROS1 tyrosine kinase inhibitor, and patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with one or two prior TRK tyrosine kinase inhibitors, with or without prior chemotherapy, and have no satisfactory alternative treatments. Repotrectinib was also previously granted four Fast-Track designations in: ROS1-positive advanced NSCLC patients who are ROS1 TKI naïve; ROS1-positive advanced NSCLC patients who have been previously treated with one prior line of platinum-based chemotherapy and one prior ROS1 TKI; ROS1-positive advanced NSCLC patients pretreated with one prior ROS1 TKI without prior platinum-based chemotherapy; and NTRK-positive patients with advanced solid tumors who have progressed following treatment with at least one prior line of chemotherapy and one or two prior TRK TKIs and have no satisfactory alternative treatments. Repotrectinib was also granted an Orphan Drug designation in 2017.

Enrollment across all six cohorts of the study remains open and continues to progress steadily.

On May 10, 2022 EDAP TMS SA (Nasdaq: EDAP), the global leader in robotic energy-based therapies, reported that it will release its financial results for the first quarter ended March 31st, 2022 after the markets close on Tuesday, May 17th, 2022 (Press release, EDAP TMS, MAY 10, 2022, View Source [SID1234614121]).

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An accompanying conference call and webcast will be hosted by Marc Oczachowski, Chairman of the Board and Chief Executive Officer, Ryan Rhodes, CEO of EDAP U.S., and François Dietsch, Chief Financial Officer. The call will be held at 8:30am EDT on Wednesday, May 18th, 2022. Please refer to the information below for conference call dial-in information and webcast registration.

On May 10, 2022 Pacira BioSciences, Inc. (NASDAQ: PCRX) reported that it will participate in an analyst-led fireside chat at the following two healthcare conferences (Press release, Pacira Pharmaceuticals, MAY 10, 2022, View Source [SID1234614120]):

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2022 RBC Capital Markets Global Healthcare Conference in New York, on Tuesday, May 17th at 8:00AM ET.
H.C. Wainwright Global Virtual Investment Conference on Tuesday, May 24th at 7:00AM ET.
Live audio of the events can be accessed by visiting the "Events" page of the company’s website at investor.pacira.com. A replay of the webcasts will also be available for two weeks following the events.