On November 14, 2024 Flashpoint Therapeutics, a clinical-stage therapeutics company pioneering structural nanomedicine, reported significant expansion of its therapeutic pipeline through strategic acquisitions and a new partnership valued at $50M with The King Abdullah International Medical Research Center (KAIMRC) in Saudi Arabia (Press release, Flashpoint Therapeutics, NOV 14, 2024, View Source [SID1234648422]).

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Structural nanomedicine is an innovative therapeutic approach that enables the creation of precisely engineered nanostructures combining multiple drug components in optimized configurations. This platform technology significantly enhances target cell delivery, therapeutic potency, and safety profiles compared to conventional treatment methods.

"Complex diseases remain inadequately addressed by traditional drugs," said Chad Mirkin, PhD, scientific founder of Flashpoint Therapeutics. "Structural nanomedicines allow us to arrange medicinally active components into configurations that can treat previously undruggable diseases. This represents a fundamental shift in therapeutic development."

The newly announced partnership with KAIMRC establishes the state-of-the-art Flashpoint-KAIMRC Center of Excellence (COE) in Structural Nanomedicine. The Flashpoint-KAIMRC COE is expected to receive funding up to $50M in the next five years to conduct clinical trials on structural nanomedicine candidates and advance structural nanomedicine drug discovery programs targeting diseases prevalent in Saudi Arabia and globally.

Through strategic licensing and acquisition deals with Northwestern University, Holden Pharmaceuticals, and Exicure, Flashpoint has assembled over 150 issued patents and patent applications covering nucleic acid, protein, and CRISPR gene editing therapeutics.

Flashpoint’s lead program, FLASH-001, is a structural nanomedicine incorporating toll-like receptor 9 agonists for immuno-oncology applications. In phase 1b/2 trials, FLASH-001 has demonstrated a curative effect in a small subset of Merkel Cell Carcinoma patients. Flashpoint plans to expand the study in 2025 to larger patient cohorts and additional cancer types. The company’s pipeline includes clinical stage programs targeting skin disorders and multiple preclinical programs in immuno-oncology, neurology, and other indications.

"The establishment of our Center of Excellence with KAIMRC and our growing clinical pipeline demonstrate the momentum behind Flashpoint’s structural nanomedicine platform," said Adam Margolin, PhD, Chief Executive Officer of Flashpoint Therapeutics. "We’re executing a dual strategy of internal development and pharmaceutical partnerships to advance first-in-class structural nanomedicines that could fundamentally change how we treat complex diseases."

On November 14, 2024 Mabwell (688062.SH), an innovative biopharmaceutical company with entire industry chain, reported that its novel Nectin-4 targeting ADC (R&D code: 9MW2821) has received IND approval for two clinical studies (Press release, Mabwell Biotech, NOV 14, 2024, View Source [SID1234648421]). The first will explore its use in combination with a PD-1 monoclonal antibody in the treatment of perioperative urothelial carcinoma, while the second will investigate its combination with other antitumor agents for the treatment of advanced solid tumors.

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The recent approval of a multi-drug combination treatment program for cervical cancer and esophageal squamous cell carcinoma, along with the expansion of 9MW2821’s indication to include the perioperative stage for urothelial carcinoma, is anticipated to significantly broaden the therapeutic reach of 9MW2821, potentially benefiting a larger patient population.

9MW2821 is a novel Nectin-4 targeting ADC developed by Mabwell. It stands out as the first clinical-stage drug candidate among Chinese companies for this specific target. Clinical studies for 9MW2821 are underway across a range of tumor indications:

Urothelial Carcinoma (UC):

Monotherapy has advanced to Phase III clinical trials and is included in the CDE’s Breakthrough Therapy Designation (BTD) list.
Combination therapy with PD-1 has entered Phase III clinical trials.
Combination therapy with PD-1 for perioperative UC has been approved for clinical research.
Cervical Cancer (CC):

The first drug candidate targeting Nectin-4 to enter Phase III clinical trials globally.
Combination therapy with PD-1 and other drugs has been approved for clinical research.
Received FDA Fast Track Designation (FTD).
Triple-Negative Breast Cancer (TNBC):

Monotherapy (post-chemotherapy and topoisomerase ADC treatment) has entered Phase II clinical trials.
Combination therapy with PD-1 has entered Phase II clinical trials.
Received FDA FTD.
Esophageal Cancer (EC):

Monotherapy has entered Phase II clinical trials.
Combination therapy with PD-1 and other drugs has been approved for clinical research.
Received FDA FTD for esophageal squamous cell carcinoma (ESCC); received FDA Orphan Drug Designation (ODD).

On November 14, 2024 Azurity Pharmaceuticals reported that the U.S. Food and Drug Administration (FDA) has approved Danziten, the first and only nilotinib with no mealtime restrictions indicated for adult patients with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase and adult patients with chronic phase (CP) and acute phase (AP) resistant or intolerant to prior therapy that included imatinib (Press release, Azurity Pharmaceuticals, NOV 14, 2024, View Source [SID1234648420]).

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"Danziten offers a new nilotinib treatment option with the equivalent efficacy to Tasigna, but without the fasting requirements of Tasigna," said Richard Blackburn, CEO of Azurity Pharmaceuticals, Inc. "Unlike Tasigna, the boxed warning on the Danziten label has no requirement for patients to take their medication in a fasted state, liberating CML patients from mealtime restrictions."

Tasigna has established efficacy in adults with newly diagnosed Ph+ CML-CP and resistant or intolerant Ph+ CML-CP and CML-AP.1 However, Tasigna has variable bioavailability that considerably increases when taken with food. In a concentration dependent manner, Tasigna may significantly prolong QT interval on surface electrocardiogram (ECG) when inappropriately taken with food. As such, strict fasting with Tasigna is crucial to avoid cardiotoxicity.2

Danziten is a re-engineered formulation of nilotinib without mealtime restrictions that offers equivalent efficacy to Tasigna but with improved bioavailability, allowing for a lower dose.1,3,4 Danziten demonstrates consistent pharmacokinetics, with no clinically significant differences in nilotinib exposure regardless of fasting state or meal type, while offering the proven efficacy expected from nilotinib.1,5,6 With optimal tyrosine kinase inhibitor (TKI) therapy, patients can achieve deep molecular responses, and some may even attain treatment-free remission.7 The life expectancy of newly diagnosed CP-CML patients who have responded to appropriate treatment is now approaching that of the general population. However, challenges remain, including patient adherence to treatment. Danziten has the potential to improve adherence due to the removal of fasting requirements.7,8

Danziten will be available in the coming weeks through Biologics by McKesson and Limited Specialty Distribution. For full prescribing information including boxed warning, please visit www.Danziten.com.

Azurity will offer patient support through DanzitenCONNECT, a comprehensive program, subject to terms and conditions, that includes Prior Authorization support and Benefits Investigation, a free first month of Danziten, a co-pay of as little as $0, and a Patient Assistance Program (PAP).

On November 14, 2024 Allarity Therapeutics, Inc. ("Allarity" or the "Company") (NASDAQ: ALLR), a Phase 2 clinical-stage pharmaceutical company dedicated to developing personalized cancer treatments using its proprietary, drug-specific patient selection technology, reported financial results for the third quarter ended September 30, 2024, and provided an update on recent operational highlights (Press release, Allarity Therapeutics, NOV 14, 2024, View Source [SID1234648419]).

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"This quarter’s progress marks a steady period of advancement for Allarity as we have maintained a strong cash position, achieved record patient duration on stenoparib treatment, and welcomed new members to our leadership team who bring the expertise needed to shape a successful future for the Company," said Thomas Jensen, CEO of Allarity Therapeutics. "We continue to drive the development of our promising, novel dual PARP/tankyrase inhibitor, stenoparib, forward, and we remain optimistic that our efforts will ultimately bring new hope to ovarian cancer patients, especially those who currently have few or no treatment options."

Third Quarter 2024 and Recent Operational Highlights

Encouraging Patient Outcomes in Phase 2 Stenoparib Trial: On September 16, 2024, the Company announced that two patients in its Phase 2 trial for stenoparib in advanced ovarian cancer had exceeded one year on treatment, demonstrating durable clinical benefit in this heavily pre-treated population and highlighting the potential of stenoparib as a meaningful treatment option for patients with limited or no alternatives.
New Leadership to Drive Clinical Development: The Company, led by President and Chief Development Officer Jeremy R. Graff, Ph.D., and Consultant Chief Medical Officer Jose Iglesias, M.D., both appointed in October 2024, is working closely with its scientific advisors to prepare a follow-on trial aimed at FDA regulatory intent. Dr. Graff—a distinguished oncology expert with over 25 years in drug development who previously served as a consultant to Allarity—and Dr. Iglesias—a seasoned leader in oncology clinical trials and former Celgene executive—bring invaluable experience and strategic insight to advance Allarity’s clinical trial efforts.
Appointment of New CFO: In September 2024, Allarity appointed Alexander Epshinsky as Chief Financial Officer. With nearly a decade of financial leadership experience in the biotech sector, Mr. Epshinsky brings valuable expertise to support the Company’s financial strategy as it advances the development of stenoparib.
European Patent Secured for Stenoparib DRP: The Company was granted a European patent in October 2024 for its proprietary DRP companion diagnostic specific to stenoparib, strengthening Allarity’s market position by securing critical IP protection for this unique diagnostic in an important market. Patent applications are also pending in other key regions, including the U.S., Japan, and China.
Regained NASDAQ Compliance: In October 2024, the Company received formal notice from The Nasdaq Stock Market LLC confirming that it had regained compliance with the minimum bid price requirement under Nasdaq Listing Rule 5550(a)(2) for continued listing on the Nasdaq Capital Market, effectively concluding the previously disclosed listing matter.
Third Quarter 2024 Financial Highlights

Cash Position: As of September 30, 2024, Allarity had cash and cash equivalents of $18.5 million, compared to $0.2 million at December 31, 2023. The Company maintains a financial runway extending into 2026.

R&D Expenses: Research and development expenses for the third quarter of 2024 were $1.0 million, compared to $1.9 million for the third quarter of 2023. Additionally, the Company recorded a $9.7 million intangible asset impairment charge (non-cash) for the third quarter of 2024.

G&A Expenses: General and administrative expenses for the third quarter of 2024 were $1.6 million, compared to $2.5 million for the third quarter of 2023.

Net Loss: Net loss attributable to common stockholders for the third quarter of 2024 was $12.2 million (primarily due to the aforementioned $9.7 million intangible asset impairment charge), compared to a net loss of $5.6 million for the third quarter of 2023.

About the Drug Response Predictor – DRP Companion Diagnostic
Allarity uses its drug-specific DRP to select those patients who, by the gene expression signature of their cancer, are found to have a high likelihood of benefiting from a specific drug. By screening patients before treatment, and only treating those patients with a sufficiently high, drug-specific DRP score, the therapeutic benefit rate may be significantly increased. The DRP method builds on the comparison of sensitive vs. resistant human cancer cell lines, including transcriptomic information from cell lines combined with clinical tumor biology filters and prior clinical trial outcomes. DRP is based on messenger RNA expression profiles from patient biopsies. The DRP platform has proven its ability to provide a statistically significant prediction of the clinical outcome from drug treatment in cancer patients dozens of clinical studies (both retrospective and prospective). The DRP platform, which can be used in all cancer types and is patented for more than 70 anti-cancer drugs, has been extensively published in the peer-reviewed literature.

On November 14, 2024 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported financial results for the third quarter ended September 30, 2024, and recent corporate updates (Press release, Abeona Therapeutics, NOV 14, 2024, View Source [SID1234648417]).

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"With the acceptance of our Biologics License Application (BLA) resubmission for pz-cel, we are ramping up our commercial readiness efforts, especially with respect to onboarding potential pz-cel treatment sites and continuing discussions with payors," said Vish Seshadri, Chief Executive Officer of Abeona.

Third Quarter and Recent Progress

Pz-cel for RDEB

Abeona completed a Type A meeting in August 2024 where it aligned with the FDA on the content for the resubmission of the Company’s BLA for pz-cel, its investigational first-in-class, autologous cell-based gene therapy currently in development for RDEB, including additional information to satisfy all Chemistry Manufacturing and Controls (CMC) requirements noted in the Complete Response Letter (CRL) issued in April 2024. The CRL required that certain CMC issues be addressed in the BLA resubmission, and did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA. The FDA did not request any new clinical trials or clinical data to support the approval of pz-cel.
Also in August 2024, the Centers for Medicare and Medicaid Services (CMS) granted a product-specific procedure code ICD-10-PCS (International Classification of Diseases, 10th Revision, Procedure Coding System) for pz-cel. Also, as part of the Inpatient Prospective Payment System (IPPS) Final Rule for fiscal year 2025, CMS assigned Medicare reimbursement of pz-cel to Pre-Major Diagnostic Category, Medicare Severity Diagnosis Related Group 018 (Pre-MDC MS-DRG 018), which is among the highest available inpatient hospital reimbursement levels for cell and gene therapies. The favorable Medicare decisions support efficient hospital billing, reimbursement and patient access.
In October 2024, Abeona resubmitted its BLA for pz-cel to the FDA, seeking approval of pz-cel as a potential new treatment for patients with RDEB.
Also in October 2024, Abeona entered into a lease agreement for additional facility space in Cleveland, Ohio to enable manufacturing capacity expansion beyond the current planned manufacturing footprint.
Also in October 2024, the United States Patent and Trademark Office issued a new patent (U.S. Patent No. 12,110,504) ("the ’504 Patent") and allowed the claims of a second patent (based on U.S. Patent Application No. 16/066,253) that is expected to issue in the coming weeks. Both patents are entitled "Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa Using Genetically Corrected Autologous Keratinocytes," and include claims that cover the use of pz-cel for the treatment of RDEB. The ’504 Patent has an expiration date of January 3, 2037, subject to any applicable patent term extension.
In November 2024, the FDA accepted for review the resubmission of Abeona’s pz-cel BLA and set a PDUFA target action date of April 29, 2025.
In preparation for potential commercialization, Abeona continues to make progress on several key initiatives, including onboarding high-volume epidermolysis bullosa treatment centers in the U.S. for pz-cel treatment, engaging payers to ensure patient access, and educating key stakeholders.
In preparation for potential pz-cel launch, Abeona has hired and trained personnel to support commercialization, manufacturing, supply chain and quality.
Pipeline and partnered programs

In July 2024, Abeona announced a non-exclusive agreement with Beacon Therapeutics, under which Beacon Therapeutics will evaluate Abeona’s patented AAV204 capsid for its potential use in AAV gene therapies for select ophthalmology indications.
In October 2024, Ultragenyx participated in a successful pre-BLA meeting with the FDA during which Ultragenyx aligned on the details of its BLA for partnered program UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA) that is expected to be filed around the end of 2024.
Third Quarter Financial Results and Cash Runway Guidance

Cash, cash equivalents, short-term investments and restricted cash totaled $110.0 million as of September 30, 2024. As of June 30, 2024, cash, cash equivalents, short-term investments and restricted cash totaled $123.0 million.

Abeona estimates that its current cash and cash equivalents, short-term investments and restricted cash, as well as its credit facility, are sufficient resources to fund operations into 2026, before accounting for any potential revenue from commercial sales of pz-cel, if approved, or proceeds from the sale of a Priority Review Voucher (PRV), if awarded by the FDA.

Research and development expenses for the three months ended September 30, 2024 were $8.9 million, compared to $7.1 million for the same period of 2023. General and administrative expenses were $6.4 million for the three months ended September 30, 2024, compared to $4.2 million for the same period of 2023. The increase in general and administrative expenses is primarily due to commercial and launch preparation costs. Net loss for the third quarter of 2024 was $30.3 million, including a $15.2 million loss resulting from the quarterly remeasurement of the fair value of warrant and derivative liabilities. In the third quarter of 2023, net loss was $11.8 million, including a $1.1 million loss resulting from the quarterly remeasurement of the fair value of warrant liabilities.

Conference Call Details

The Company will host a conference call and webcast on Thursday, November 14, 2024, at 8:30 a.m. ET, to discuss the quarter results. To access the call, dial 877-545-0320 (U.S. toll-free) or 973-528-0002 (international) and Entry Code: 500590 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at View Source The archived webcast replay will be available for 30 days following the call.