On September 18, 2023 Leading CRISPR gene editing companies Integrated DNA Technologies and Aldevron reported to have inked a new global distribution agreement to expand key CRISPR solutions for cell and gene therapy customers (Press release, Aldevron, SEP 18, 2023, View Source [SID1234635225]). Effective today, IDT will stock and sell three additional research use only (RUO) and current good manufacturing practice (cGMP) CRISPR nucleases with unique performance characteristics, manufactured by Aldevron, to provide researchers with an accelerated path to the clinic.

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Orders for new CRISPR enzymes can be placed at www.idtdna.com/AldevronCRISPRnucleases.

"IDT and Aldevron are united in accelerating CRISPR research worldwide by working together to deliver key solutions for translational medicine, increasing our shared value to the entire CRISPR community," said Sandy Ottensmann, VP/GM, Gene Writing and Editing & Core/PCR Business Units at IDT. "Expanding access to best-in-class gene editing reagents and tools to enable customers to rapidly innovate and translate their projects from research breakthroughs to potential life-saving treatments is key to advancing the future of genomic medicine, and we’re proud to work alongside Aldevron to realize this important work."

"Successful clinical translation requires high quality reagents and consistent performance from proof-of-concept through patient dosing. We are thrilled to partner with IDT and bring together their expertise in CRISPR chemistry and oligo production with Aldevron expertise in cGMP CRISPR nuclease and ribonucleoprotein (RNP) manufacturing," said Tom Foti, Vice President and General Manager of Aldevron’s Protein Business Unit. "By combining manufacturing strengths, from day one, IDT clients now can access Aldevron CRISPR proteins which are designed for clinical translation."

Customers investigating genetic and inherited diseases can now purchase both research grade and full cGMP CRISPR nucleases from IDT. cGMP products accelerate researchers’ path to the clinic by including cGMP quality documentation required for regulatory filings.

The following Aldevron-manufactured enzymes, engineered to be used in a variety of applications including electroporation, transfection, and microinjection, are now available through IDT:

SpyFi Cas9 Nuclease—designed for greater editing efficiency and reduced off-target effects. When used in conjunction with an effective guide RNA, target site, and double-stranded DNA cut strategies, this can deliver higher-fidelity editing with less off-target activity than wild-type SpCas9 nuclease. A Drug Master File (DMF) is on file with the U.S. Food and Drug Administration to streamline regulatory filings. Aldevron’s SpyFi Cas9 Nuclease is sold under license of patents and/or patents pending from IDT.
Eureca-V Nuclease—a Class 2, Type V nuclease based on novel MAD7 protein licensed from Inscripta that targets a T-rich PAM domain and creates a staggered double-strand break at the target locus. This enzyme supports research and development in the allogeneic cell therapy space.
SpCas9 Nuclease—a wildtype SpCas9 nuclease for use in development work as well as standard cGMP products for clinical studies. SpCas9 is supported by an extensive QC panel at research grade and GMP product levels. A Drug Master File (DMF) is on file with the U.S. Food and Drug Administration to streamline regulatory filings.

On September 18, 2023 Lantern Pharma Inc. (NASDAQ: LTRN), an artificial intelligence (AI) company developing targeted and transformative cancer therapies using its proprietary AI and machine learning (ML) platform, RADR, with multiple clinical stage drug programs, reported that the United States Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for LP-284 (Press release, Lantern Pharma, SEP 18, 2023, View Source [SID1234635224]). LP-284 is being developed for the treatment of relapsed or refractory non-Hodgkin’s lymphoma (NHL), including mantle cell lymphoma (MCL) and double hit lymphoma (DHL) and other high-grade B-cell lymphomas (HGBL). Lantern expects to commence enrollment of patients for the first-in-human Phase 1 trial for LP-284 during the fourth quarter of 2023.

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"This is now our second novel drug candidate to receive IND clearance from the FDA in the past 100 days, further validating our approach of leveraging AI and machine learning to accelerate the development of our pipeline," stated Panna Sharma, Lantern’s President and CEO. "LP-284 holds blockbuster potential, and we have been able to expedite its journey from a concept to a first-in-human clinical trial in a highly efficient and cost-effective manner – less than 2.5 years and under approximately $2.7 million – underscoring the power and potential of our AI platform RADR to accelerate oncology drug discovery and development. RADR was used to unravel the mechanism of action of LP-284, prioritize its cancer indications, and generate machine-learning biomarker signatures that may be pivotal in selecting patients for future phases of the clinical trials. Our success-to-date with LP-284 reaffirms our commitment to harnessing the power of AI to transform cancer treatment and save lives."

LP-284 is a novel small molecule with a synthetically lethal mechanism of action that preferentially damages cancer cells that harbor mutations in DNA damage repair pathways. Lantern’s LP-284 program has been accelerated and focused using AI insights and biological modeling powered by RADR.

NHL is the leading hematological malignancy in the US and remains one of the leading causes of cancer deaths globally, with an estimated 500,000 new cases annually worldwide. Despite advances in NHL using combination and targeted therapies, nearly 20% to 40% of patients with certain subtypes still relapse after treatment. In aggressive subtypes of NHL, like MCL, nearly all patients relapse from standard-of-care (SOC) therapies. Globally, the annual market potential of LP-284 in NHL is estimated to be approximately $4 billion USD.

On September 18, 2023 Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, reported to have rescheduled its participation in Sidoti’s Small-Cap Virtual Conference to November 2023 due to funeral services for the late Howard Safir, a long-time member of the Citius Board (Press release, Citius Pharmaceuticals, SEP 18, 2023, View Source [SID1234635223]). Mr. Mazur was originally scheduled to present at the Sidoti Small-Cap Virtual Conference on Wednesday, September 20, 2023 at 2:30pm. Additional details regarding participation in the Sidoti conference in November will be shared closer to the event.

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On September 18, 2023 Kintara Therapeutics, Inc. (NASDAQ: KTRA) ("Kintara" or the "Company"), a biopharmaceutical company focused on the development of new solid tumor cancer therapies, reported financial results for its fiscal year ended June 30, 2023 and provided a corporate update (Press release, Kintara Therapeutics, SEP 18, 2023, View Source [SID1234635222]).

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RECENT CORPORATE DEVELOPMENTS

Awarded a $2.0 million Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH) to support the clinical development of REM-001, a second-generation photodynamic therapy (PDT) photosensitizer agent for the treatment of cutaneous metastatic breast cancer (CMBC). (June 2023)
Hosted a Key Opinion Leader (KOL) event featuring Patrick Y. Wen, M.D. (Harvard Medical School) and John de Groot, M.D. (UCSF Health) who discussed the current treatment landscape for patients suffering from glioblastoma (GBM), the most common and lethal form of brain cancer, along with Kintara’s potential treatment solution with VAL-083, a potential first-in-class small molecule chemotherapeutic. (August 2023)
Announced that the Company will be presenting a poster at the 2023 European Association of Neuro-Oncology (EANO) Annual Meeting taking place in Rotterdam, Netherlands, September 21-24, 2023. The presentation will include data from its lead program, VAL-083, for the treatment of recurrent GBM. (September 2023)
"We are looking forward to presenting additional compelling VAL-083 data at the EANO Annual Meeting later this month and continue to anticipate announcing top-line data in the international registrational GBM AGILE Study before the end of calendar 2023," commented Robert E. Hoffman, Kintara’s President and Chief Executive Officer. "We were thrilled to be awarded a $2.0 million grant from the NIH to support the further development of REM-001 in CMBC and expect to enroll the first patient in our planned 15-patient study in the fourth quarter of calendar year 2023."

SUMMARY OF FINANCIAL RESULTS FOR FISCAL YEAR 2023 YEAR ENDED JUNE 30, 2023

As of June 30, 2023, Kintara had cash and cash equivalents of approximately $1.5 million.

For the year ended June 30, 2023, Kintara reported a net loss of approximately $14.6 million, or $9.27 per share, compared to a net loss of approximately $22.7 million, or $25.80 per share, for the year ended June 30, 2022. The decreased net losses for the year ended June 30, 2023 compared to the year ended June 30, 2022 was largely due to lower research and development expenses, primarily lower clinical development costs. General and administrative costs were also lower during the same period primarily due to reduced level of staffing.

Selected Balance Sheet Data (in thousands)

June 30,

2023

June 30,
2022

$

$

Cash and cash equivalents

1,535

11,780

Working capital

188

9,268

Total assets

3,979

15,948

Total stockholders’ equity

731

11,795

Selected Statement of Operations Data (in thousands, except per share data)

For the year ended

June 30,

June 30,

2023

2022

$

$

Research and development

9,311

15,173

General and administrative

5,485

7,509

Other income

(147)

(21)

Net loss for the year

(14,649)

(22,661)

Series A Preferred cash dividend

(8)

(8)

Series C Preferred stock dividend

(362)

(2,462)

Net loss for the period attributable to common stockholders

(15,019)

(25,131)

Basic and fully diluted weighted average number of shares

1,620

974

Basic and fully diluted loss per share

(9.27)

(25.80)

Kintara’s financial statements as filed with the U.S. Securities Exchange Commission can be viewed on the Company’s website at: View Source

On September 18, 2023 Cantex Pharmaceuticals, Inc., a clinical stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions for which new treatments are urgently needed, and Lenox Hill Hospital’s Department of Neurological Surgery reported the initiation of a Phase 2 study to assess the safety and effectiveness of azeliragon in combination with radiation therapy as a treatment for newly diagnosed unmethylated glioblastoma (Press release, Cantex, SEP 18, 2023, View Source [SID1234635221]).

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Glioblastoma is a highly malignant primary brain tumor for which current therapeutic options provide a limited life extension benefit. Unmethylated glioblastoma does not respond well to chemotherapy, further limiting the available treatment options for patients with this severe form of cancer. New treatments for glioblastoma are urgently needed.

"We are pleased to announce the initiation of patient enrollment at Lenox Hill Hospital, a world-class provider of neurosurgical and cancer treatment, into this important Phase 2 clinical study," said Stephen G. Marcus, M.D., Cantex’ Chief Executive Officer. "Azeliragon inhibits activation of a receptor known as ‘RAGE’ on the surface of glioblastoma cells. RAGE has been implicated in the progression of glioblastoma and its resistance to radiation therapy. In pre-clinical studies, azeliragon enhanced the effectiveness of radiation therapy. With this study, we seek to enhance the effectiveness of radiation therapy and delay or prevent disease progression."

"Through the initiation of this Phase 2 study at Lenox Hill Hospital, we seek to explore the safety and potential efficacy of azeliragon as a treatment of unmethylated glioblastoma, which is less responsive to chemotherapy. This potential therapy may be an option for patients who lack many other treatment alternatives," said John Boockvar, MD, Vice Chair and Professor of Neurosurgery at Lenox Hill Hospital, and adjunct Professor at Cold Spring Harbor Laboratory.

About Azeliragon

Azeliragon is an orally administered capsule, taken once daily, that inhibits interactions of the receptor for advanced glycation end products (known as RAGE) with certain ligands, including HMGB1 and S100 proteins in the tumor microenvironment. Azeliragon was discovered by and originally under development for Alzheimer’s disease by vTv Therapeutics Inc. (NASDAQ: VTVT) from whom Cantex licensed worldwide rights to azeliragon. Clinical safety data from these trials, involving more than 2000 individuals dosed for periods up to 18 months, indicate that azeliragon is very well tolerated. Cantex also has ongoing clinical trials in newly diagnosed glioblastoma, brain metastasis, neoadjuvant therapy of breast cancer, metastatic pancreatic cancer, and hospitalized COVID patients to prevent acute kidney injury. These trials are based on robust pre-clinical data as well as the extensive clinical safety information from randomized placebo-controlled clinical trials.