On January 29, 2024 enGene Holdings Inc. (Nasdaq: ENGN) (or "enGene" or the "Company"), a clinical-stage genetic medicines company whose non-viral lead program is in a pivotal study for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), reported its financial results for the fiscal year ended October 31, 2023 and provided a corporate update (Press release, enGene, JAN 29, 2024, View Source [SID1234639693]).

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"During 2023, enGene made substantial progress advancing our lead program, EG-70 (detalimogene voraplasmid), in our ongoing pivotal-stage LEGEND study in BCG-unresponsive non-muscle-invasive bladder cancer with carcinoma in situ. In addition, we were pleased to build out an accomplished leadership team and transition into a public company," said Jason Hanson, Chief Executive Officer of enGene. "Based on the highly differentiated attributes of our non-viral DDX genetic medicine platform, we believe that EG-70 may become a powerful and heavily utilized therapy in the practicing urologists’s armamentarium, providing both therapeutic benefits for patients as well as ease-of-use advantages as compared to existing NMIBC therapeutics and those in late-stage development. We continue to enroll patients in our pivotal study and look forward to reporting interim clinical results in mid-2024."

Ryan Daws, Chief Financial Officer of enGene commented: "Together with our $50M expanded debt facility with Hercules Capital, the financing transactions completed concurrent with the reverse merger provide support for the ongoing LEGEND study of EG-70 in BCG-unresponsive and BCG-naïve NMIBC patients with Cis, BLA-enabling activities, and additional clinical applications. The Company is in a strong financial position, with cash on hand expected to support operations into Q2 2025, beyond the anticipated completion of the interim EG-70 clinical data and additional expected corporate milestones."

Recent Business Highlights:

Public Company Launch and EG-70 Clinical Program Progress: On November 1, 2023, enGene Holdings Inc launched as a publicly traded company focused on developing non-viral gene therapies based on its novel and proprietary dually derived chitosan, or "DDX", gene delivery platform, which allows localized delivery of multiple gene cargos directly to mucosal tissues and other organs. The Company’s lead product candidate, EG-70, is an intravesically administered, non-viral, polymeric nanoparticle comprising a DNA plasmid encoding three genes designed to generate a local immune reaction in proximity to tumors. enGene is enrolling patients in a combined Phase 1/2 open label registrational study of EG-70, referred to as "LEGEND" (ClinicalTrials.gov identifier NCT04752722) with interim data expected mid-year.

Achieved Primary and Secondary Endpoints in Phase 1 Portion of LEGEND Study in NMIBC: The Phase 1 portion enrolled 22 BCG-unresponsive patients, exhibited an encouraging safety profile, a 3-month complete response rate of 68% and supported dose selection for the Phase 2 portion of the study.

Expanded Hercules Capital Debt Facility: On December 22, 2023, enGene announced the expansion of its existing debt facility with Hercules to up to $50 million, subject to certain milestones, with $22.5 million advanced at closing of which approximately $8.6 million was applied to refinance in full the term loans outstanding under the prior loan agreement with Hercules.

Key Leadership Hires: enGene continued to invest in its senior leadership and add experienced members to its Board of Directors. On February 2, 2023, Alex Nichols, Ph.D., joined as President and Chief Operating Officer, on September 7, 2023, Richard Bryce, M.D. was hired as Chief Medical Officer and on November 29, 2023, Ryan Daws joined as Chief Financial Officer. Additionally, on December 19, 2023, Lota Zoth, CPA, was appointed to the Company’s Board of Directors and serves as the Company’s Audit Committee Chair.
Past and Upcoming Anticipated Milestones

Presented clinical data from LEGEND Phase 1 trial at 24th Annual Meeting of the Society of Urologic Oncology in November 2023.

Announce interim readout from the pivotal Phase 2 LEGEND study mid-2024.
Full Year 2023 Financial Results

Cash and cash equivalents, as of October 31, 2023, were $81.5 million, compared to $20.4 million as of October 31, 2022. The Company expects that its existing cash and cash equivalents will fund operating expenses and capital expenditures into the second quarter of 2025.

Full Year 2023

Total operating expenses were $26.0 million for 2023, compared to $19.4 million for 2022. Research and development expenses increased by $1.0 million, mainly due to increasing CMC and clinical costs relating to our pivotal EG-70 study. General and administrative expenses increased by $5.6 million, primarily driven by share-based compensation and professional fees associated with accounting and audit related fees.

For the full year 2023, net loss attributable to common shareholders was $104.7 million, or $151.22 per share, compared to $29.0 million, or $44.30 per share, for the same period in 2022. The increase in net loss is mainly attributed to the increase in other expense. In 2023, within other expense are non-cash charges of $80.7 million arising from fair value adjustments on our convertible debentures and related derivative liabilities and a loss on extinguishment upon conversion of our convertible debentures; partially offset by non-cash fair value gain of $10.8 million from the cancellation of Series C preferred share warrants liabilities upon the Reverse Recapitalization on October 31, 2023.

On January 29, 2024 Oricell Therapeutics (Oricell), a clinical-stage biotechnology company, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for OriCAR-017 for patients with relapsed/refractory multiple myeloma (R/R MM) (Press release, OriCell Therapeutics, JAN 29, 2024, View Source [SID1234639692]). OriCAR-017 is a chimeric antigen receptor (CAR) T cell therapy targeting GPRC5D. The therapy leverages Oricell’s proprietary platforms including OriAb antibodies, OriCAR construct and unique CMC know-how to achieve optimal binding and superior persistence and anti-tumor efficacy out of rejuvenated CAR-T cells. The IND enables Oricell to initiate the clinical development for OriCAR-017 in the US immediately.

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The FDA IND approval for OriCAR-017 follows its NMPA IND approval in 2023 and the publication of clinical results from an Investigator Initiated Trial (POLARIS study) at the 2022 ASCO (Free ASCO Whitepaper), 2022 EHA (Free EHA Whitepaper) and The Lancet Haematology. The data showed that all ten patients with R/R MM responded to the therapy per IMWG criteria, achieving a 100% overall response rate (ORR), 80% stringent complete response. 100% minimal residual disease (MRD) negative rate was detected at day 28 and further confirmed at month 3. The therapy was well-tolerated, with no Immune effector cell-associated neurotoxicity syndrome (ICANS), no cerebellar disorder, no delayed infections, and only Grade 1/Grade 2 CRS that resolved rapidly. Of the ten R/R MM patients, 40% had extramedullary disease (EMD), 50% received prior BCMA CAR-T treatment(s), 70% had high-risk cytogenetics, and 70% with ECOG 2, 80% at ISS stage II & III.

"The evidenced superior safety, efficacy and durability profile of OriCAR-017 is truly exciting and will significantly benefit multiple myeloma patients on a global scale. Ten years’ R&D cumulates not only OriCAR-017 but also the robust and integrated technology platforms that generate one-of-its-kind CAR-T products for liquid and solid tumors," said Peter He, Co-founder and Chief Scientific Officer of Oricell.

"Exceptional teamwork is what Oricell relies upon. The fact that we were able to complete technical transfer from China to the U.S. in five months speaks for the standard of our teamwork." Helen Yang, Co-founder and Chief Executive Officer of Oricell further stated that, "With a great team and support from our stakeholders, we are confident in our ability to delivering best-in-class cell therapies to patients, providing them new hope and possibilities."

On January 29, 2024 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a clinical-stage biotechnology company focused on the treatment and prevention of cancer, reported its participation in the 2024 NeauxCancer Conference organized by the Cancer Advocacy Group of Louisiana (CAGLA), being held February 29 – March 2, 2024 in New Orleans (Press release, Anixa Biosciences, JAN 29, 2024, View Source [SID1234639691]). Anixa Chairman and CEO Dr. Amit Kumar will present and meet one-on-one with investors throughout the day on Friday, March 1st.

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Dr. Kumar’s remarks will feature Anixa’s use of the body’s immune system to take multiple approaches in fighting cancer. The Company’s vaccines focus on immunizing against "retired" proteins found to be expressed in certain forms of cancer, a mechanism that has never before been utilized for cancer vaccine development.

Attendees at the conference will include oncologists and other clinicians interested in learning and discussing the latest practices for treating and taking care of cancer patients. The investment track will enable the medical professionals attending the conference, as well as regional investment professionals, to learn about emerging technologies for cancer treatment and prevention.

Registration and attendance at the conference for investors is complimentary. Investors can register via the link below.

Investment Track Details:

Anixa presentation:

Friday, March 1, 2024, at 11:30 AM CST

Conference registration:

Available on the conference website

1×1 meetings:

Open to investors upon conference registration

Chadwick Landry, President and Executive Director of CAGLA stated, "We are pleased to be adding an investment track to our annual conference. We are also pleased that Anixa will be presenting at this inaugural year of the investment track. We plan to make the investment track a regular event at this well-attended conference to enable regional investors and medical professionals to gain firsthand exposure to some of the most exciting emerging companies developing technologies to address cancer."

The 2024 NeauxCancer Conference has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of Ochsner Clinic Foundation and CAGLA. The Ochsner Clinic Foundation is accredited by the ACCME to provide continuing medical education for physicians.

On January 29, 2024 Phanes Therapeutics, Inc. (Phanes), a clinical stage biotech company focused on innovative drug discovery and development in oncology, reported that the U.S. Patent and Trademark Office has granted the registration of trademarks for its proprietary technology platforms PACbody (Reg. No. 7,080,972), SPECpair (Reg. No. 7,279,821) and ATACCbody (Reg. No. 7,075,638) (Press release, Phanes Therapeutics, JAN 29, 2024, View Source [SID1234639690]). The three proprietary platforms were developed for bispecific antibodies with native IgG-like structure.

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PACbody is a proprietary approach for constructing bispecific antibodies with native IgG-like structure, which have been shown to have increased stability and high expression levels. The PACbody technology does not involve any protein fusion approach.

SPECpair enables the use of the conventional monoclonal antibody (mAb) manufacturing process in the production of native IgG-like bispecific antibodies with lower CMC and clinical risks.

ATACCbody is a technology for targeting solid tumors using immuno-oncology molecules with modulated anti-CD3 activities designed to minimize the risk of cytokine release syndrome.

Both PACbody and SPECpair were employed in the design of PT886, a first-in-class bispecific antibody targeting claudin 18.2 and CD47. It was granted orphan drug designation (ODD) by the FDA for the treatment of pancreatic cancer in 2022 and is currently in phase 1 clinical trial.

"Phanes is very pleased about the granted registrations of trademarks for our technology platforms," said Dr. Ming Wang, Founder and CEO of Phanes. "We will continue to leverage these technologies in the discovery of innovative therapies for patients."

On January 29, 2024 Asieris Pharmaceuticals (Stock Code: 688176.SH), a global biopharmaceutical leader in the discovery, development, and commercialization of innovative drugs for genitourinary tumors and related diseases, reported the results of the Phase III clinical trial and real-world study for APL-1706, a drug designed for the diagnosis and management of bladder cancer (Press release, Asieris Pharmaceuticals, JAN 29, 2024, View Source [SID1234639689]). This significant announcement was made at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO-GU), featuring a poster presentation (Abstract No. 593).

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The study incorporated 158 patients in a randomized controlled trial (RCT) and 19 patients in a real-world study (RWS). Notably, the use of Blue Light Cystoscopy (BLC) in conjunction with APL-1706 significantly improved the detection of bladder cancer lesions compared to traditional white light cystoscopy (WLC). In the RCT, 43.3% of patients (42 out of 97) diagnosed with Ta, T1, or CIS and 33.3% in the RWS (4 out of 12 patients) had at least one lesion detected by BLC that was not identified by WLC (p<0.0001). Additionally, in the RCT, 9.6% (11 out of 114) and in the RWS, 7.1% (1 out of 14) of CIS patients had additional lesions detected under BLC that were not found using WLC.

The detection rates for PUNLMP, CIS, Ta, T1, and T2 ~ T4 tumors in the RCT were notably high with BLC, reaching 94.7%, 100%, 98.2%, and 100% respectively, compared to the WLC detection rates of 42.1%, 76.1%, 91.2%, and 100%. Similarly, in the RWS, BLC detection rates for these tumor stages were 100%, compared to WLC rates of 50%, 81%, and 100%. These findings confirm that APL-1706 combined with BLC offers superior detection of bladder cancer in the Chinese population, especially in CIS patients, while maintaining good tolerability.

Following these encouraging results, the New Drug Application (NDA) for APL-1706 was accepted by the National Medical Products Administration (NMPA) in November 2023.