On November 14, 2023 Mustang Bio, Inc. ("Mustang") (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, reported financial results and recent corporate highlights for the third quarter that ended September 30, 2023 (Press release, Mustang Bio, NOV 14, 2023, View Source [SID1234637661]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "In the third quarter of 2023, Mustang continued to make meaningful progress in the development of our lead clinical candidate MB-106, a CD20-targeted, autologous CAR-T cell therapy to treat relapsed or refractory B-cell non-Hodgkin lymphomas ("B-NHL") and chronic lymphocytic leukemia ("CLL"). We announced the first data from our ongoing multicenter, open-label, non-randomized Phase 1/2 clinical trial evaluating MB-106’s safety and efficacy. The data were consistent with efficacy and safety results from the ongoing investigator-sponsored trial at Fred Hutchinson Cancer Center ("Fred Hutch") which have shown complete remission for as long as three years. In the multicenter trial, substantial clinical benefit was observed in all four indolent lymphoma patients treated at dose level 1 (3.3 x 106 cells/kg), including two complete responses in follicular lymphoma ("FL") patients, one of whom was previously treated with CD19 CAR-T cell therapy. A third patient, with a diagnosis of Waldenstrom macroglobulinemia ("WM"), who had nine prior treatments and high disease burden, achieved a very good partial response, which is generally the best response that can be achieved in this disease. No cytokine release syndrome greater than Grade 1 was observed in any of the four patients, and no occurrences of immune effector cell-associated neurotoxicity syndrome were reported. Additional safety and efficacy data from the multicenter trial will be reported at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting in December, including follow-up for the four dose-level-1 patients and data from all patients treated at dose level 2 (1.0 x 107 cells/kg) who have had at least 28 days of follow-up. At the End-of-Phase 1 meeting with the FDA expected in the first quarter of 2024, Mustang anticipates recommending dose level 2 as the Phase 2 dose for indolent lymphoma. Mustang further anticipates that results from the Phase 1 indolent lymphoma arm of the multicenter trial will support an accelerated Phase 2 registration strategy for WM, with the first pivotal Phase 2 WM patient to be treated potentially in mid-2024. Additionally, we plan to initiate a pivotal Phase 2 clinical trial in at least one additional B-cell malignancy in 2025."

Dr. Litchman continued, "Mustang also announced that the U.S. Food and Drug Administration ("FDA") accepted the Company’s Investigational New Drug ("IND") application to initiate a Phase 1 open label, multicenter clinical trial to assess the safety, tolerability and efficacy of MB-109, a novel combination of MB-101 (IL13Rα2‐targeted CAR-T cell therapy) and MB-108 (HSV-1 oncolytic virus), for the treatment of recurrent glioblastoma ("GBM") and high-grade astrocytoma. The FDA’s safe-to-proceed within 30 days of IND filing is testimony to the talent and resourcefulness of our team, in light of the complexity of the clinical trial, which involves the interplay of 2 complex biologic agents, each with its own unique safety profile."

Financial Results:

As of September 30, 2023, Mustang’s cash and cash equivalents and restricted cash totaled $10.3 million, compared to $16.1 million at June 30, 2023, and $76.7 million as of December 31, 2022, a decrease of $5.8 million for the quarter and a decrease of $66.4 million year-to-date. Subsequent to the end of the third quarter, Mustang raised approximately $4.4 million of gross proceeds in a registered direct offering completed in October 2023.
Research and development expenses were $9.5 million for the third quarter of 2023, compared to $15.5 million for the third quarter of 2022. Non-cash, stock-based expenses included in research and development were $(19) thousand for the third quarter of 2023, compared to $0.3 million for the third quarter of 2022.
Gain on the sale of property and equipment was $1.4 million, in connection with the sale of assets to uBriGene.
General and administrative expenses were $2.1 million for the third quarter of 2023, compared to $3.4 million for the third quarter of 2022. Non-cash, stock-based expenses included in general and administrative expenses were $0.1 million for the third quarter of 2023, compared to $0.2 million for the third quarter of 2022.
Net loss attributable to common stockholders was $10.1 million, or $1.23 per share, for the third quarter of 2023, compared to a net loss attributable to common stockholders of $19.0 million, or $2.42 per share, for the third quarter of 2022.
Recent Corporate Highlights:

General Corporate:

In July 2023, Mustang announced that the Company amended its previously announced asset purchase agreement with uBriGene, the U.S. subsidiary of uBriGene Group, a leading cell and gene therapy contract development and manufacturing organization, and closed the transaction. Per the terms of the amended asset purchase agreement, at closing, uBriGene acquired all of Mustang’s assets primarily relating to the manufacturing and production of cell and gene therapies at Mustang’s state-of-the-art clinical- and commercial-scale cell and gene therapy manufacturing facility in Worcester, Massachusetts, for upfront consideration of $6 million in cash. Mustang’s lease to the premises on which the facility is located (as well as related contracts and manufacturing personnel) did not transfer at closing because such transfer requires the consent of the landlord, which has requested an additional thirty business days to consider the proposed transfer following the landlord’s receipt of the final determination letter of the U.S. Committee on Foreign Investment in the United States ("CFIUS") regarding the transaction (as discussed further below) and a summary of Mustang’s and uBriGene’s reaction to such final determination. An additional $5 million contingent payment will be payable to Mustang upon (i) Mustang’s raising $10 million in gross proceeds from equity raises following the closing of the transaction and (ii) completion of the assignment of Mustang’s lease to uBriGene, which remains subject to landlord’s approval, within two years of the closing. Until the lease is transferred to uBriGene, Mustang will retain its facility lease and facility personnel, and will continue to occupy the leasehold premises and manufacture its lead product candidate, MB-106, at that site.

As previously disclosed, in connection with the sale of its manufacturing facility to uBriGene, Mustang and uBriGene previously submitted a voluntary notice with CFIUS to obtain clearance for the transaction, although obtaining such clearance was not a condition to closing the transaction. On November 13, 2023, CFIUS requested Mustang and uBriGene withdraw and re-file their joint voluntary notice to allow more time for review and discussion regarding the nature and extent of national security risk posed by the Transaction, and whether and to what extent mitigation of risk would be feasible. Upon CFIUS’s request, Mustang and uBriGene submitted a request to withdraw and re-file their joint voluntary notice. On November 13, 2023, CFIUS granted this request, accepted the joint voluntary notice and commenced a new 45-day review period commencing on November 14, 2023, which may be followed by a 45-day investigation period. Mustang and uBriGene have been and will continue to be actively engaged with CFIUS, and they remain fully committed to obtaining clearance from CFIUS and completing the full transfer of the manufacturing facility to uBriGene. There can be no assurance, however, that CFIUS will ultimately provide clearance with respect to the transaction, or what mitigating measures may be required in order to obtain such clearance.

In October 2023, Mustang completed a registered direct offering priced at-the-market for approximately $4.4 million in gross proceeds.

MB-106:

Mustang’s lead clinical candidate is MB-106, a CD20-targeted, autologous CAR-T cell therapy to treat a wide range of hematologic malignancies, including WM and FL. MB-106 continues to demonstrate a favorable safety and efficacy profile in both the Fred Hutch single institution and Mustang Bio multicenter Phase 1/2 clinical trials.
In August 2023, Mustang Bio announced the first data from its ongoing multicenter, open-label, non-randomized Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 CAR-T cell therapy. Initial data show substantial clinical benefit in four of four indolent lymphoma patients, including two complete responses in FL patients, one who was previously treated with CD19 CAR-T cell therapy. From a safety perspective, 3 patients experienced Grade 1 cytokine release syndrome and no occurrences of immune effector cell-associated neurotoxicity syndrome were reported. These data align with ongoing results from the investigator-sponsored trial at Fred Hutch that show ongoing complete remission for more than three years.
The FDA granted Orphan Drug Designation to MB-106 for the treatment of WM, and results from this arm of the multicenter trial are expected to support an accelerated Phase 2 registration strategy for WM, with the first pivotal Phase 2 patient with WM to be treated potentially in mid-2024. Mustang Bio will report additional safety and efficacy data from the multicenter trial at the 65th ASH (Free ASH Whitepaper) Annual Meeting, taking place December 9-12, 2023, in San Diego. Finally, Mustang Bio expects to initiate a pivotal Phase 2 clinical trial in at least one additional B-cell malignancy in 2025.
Mazyar Shadman, M.D., M.P.H., Study Chair, Associate Professor and physician at Fred Hutch and University of Washington also presented data from the ongoing Fred Hutch Phase 1/2 clinical trial, specific to two B-cell non-Hodgkin lymphoma cohorts, FL and WM. In the FL data cohort (n=20), an overall response rate ("ORR") of 95% was seen, of which 80% of patients experienced a complete response and 15% had a partial response. The complete response patients include a patient who was previously treated with a CD19-directed CAR-T cell therapy. Of the six patients who experienced cytokine release syndrome ("CRS"), only one had Grade 2, while the remaining five had Grade 1. Ten patients continue to experience complete response for more than 10 months, four patients have experienced complete response for more than two years (all ongoing), and the first patient enrolled has sustained complete response for more than 3 years. In the WM cohort (n=6), all of whom had received prior Bruton tyrosine kinase inhibitor, two patients experienced complete response, one of whom continues to be in complete response at more than 22 months. No patients experienced CRS or immune effector cell-associated neurotoxicity syndrome over Grade 2. None of the six patients with WM have needed to start new therapy for their disease.
MB-109:

In October 2023, Mustang Bio announced that the FDA accepted the Company’s IND to initiate a Phase 1 open label, multicenter clinical trial to assess the safety, tolerability and efficacy of MB-109, a novel combination of MB-101 (IL13Rα2‐targeted CAR-T cell therapy) and MB-108 (HSV-1 oncolytic virus), for the treatment of recurrent GBM and high-grade astrocytoma.

As previously reported, preclinical data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting 2022 supported this combination therapy to optimize results to treat recurrent GBM. The combination leverages MB-108 to make cold tumors "hot," thereby potentially improving the efficacy of MB-101 CAR-T cell therapy. Data presented separately on MB-101 and MB-108 showed infusions were well tolerated in recurrent GBM patients. Two patients treated solely with MB-101 who had high levels of intratumoral CD3+ T cells pre-therapy (i.e., "hot" tumors) achieved complete responses lasting 7.5 and 31+ months, respectively. Importantly, of the 53 City of Hope ("COH") Phase 1 patients disclosed at AACR (Free AACR Whitepaper) meeting in 2022, these two complete responses were observed in the two patients with the "hottest" tumors prior to treatment with MB-101.

On November 14, 2023 Alaunos Therapeutics, Inc. ("Alaunos" or the "Company") (Nasdaq: TCRT), reported financial results for the third quarter ended September 30, 2023 (Press release, Alaunos Therapeutics, NOV 14, 2023, View Source [SID1234637648]). As previously announced, the Company is exploring strategic alternatives with Cantor Fitzgerald & Co. as its strategic advisor. Alaunos continues to reduce spend and cost-savings measures taken to date are expected to extend its cash runway into the second quarter of 2024.

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Operational & Corporate Update

Clinical Data from TCR-T Library Phase 1/2 Trial: Eight patients were treated and evaluated in the Company’s TCR-T Library Phase 1/2 Trial. Patients with pancreatic (3), colorectal (4) and non-small cell lung cancer (1) were treated, with certain of the pancreatic and colorectal patients also having lung metastases. Overall, the trial showed the Company’s T cells were well-tolerated in all evaluable participants with no dose-limiting toxicities (DLTs) and no immune effector cell-associated neurotoxicity syndrome (ICANS) were observed. All cytokine release syndrome (CRS) events were within grades 1-3 and were self-limiting or resolved with standard clinical management and, in some cases, a single dose of tocilizumab.

One patient with non-small cell lung cancer (NSCLC) achieved an objective partial response with six months progression-free survival. Six other patients achieved best overall response of stable disease. The total overall response rate was 13% and disease control rate was 87% in evaluable patients with advanced, metastatic, refractory solid tumors (see attached figure). Disease control was measured by objective responses and stable disease. Increased secretion of interferon-gamma relative to baseline was detected in all patients’ serum post-cell transfer suggesting recognition of the tumor by the infused TCR-T cells. Persistence of TCR-T cells in peripheral blood was detected in all evaluable patients at their last follow-up, including up to six months in one patient. Infiltration of TCR-T cells into the tumor was also detected in three samples where a fresh biopsy was collected suggesting homing to the tumor microenvironment. All patients have progressed or withdrawn from the trial and long-term follow-up is ongoing for a subset of patients with no further intervention per the treatment protocol. This trial established proof-of-concept that Sleeping Beauty TCR-T cells can result in objective clinical responses and recognize established tumors in vivo.

Despite the encouraging TCR-T Library Phase 1/2 Trial data, based on the substantial cost to continue development and the current financing environment, Alaunos announced in August 2023 that it would not pursue any further development of its clinical programs.

hunTR TCR Discovery Platform Identifies Proprietary TCRs: Alaunos has discovered multiple proprietary TCRs targeting driver mutations through its hunTR TCR discovery platform. In addition to TCRs that recognize KRAS and TP53 mutations similar to those licensed from the National Cancer Institute, the Company identified additional TCRs that bind to other driver mutations and TCRs that are restricted to additional HLAs. Alaunos believes that the hunTR library has the potential to allow for the treatment of a large patient population.

Strategic Alternatives: The Company continues to explore strategic alternatives, which may include but are not limited to, an acquisition, merger, reverse merger, sale of assets, strategic partnerships, capital raises or other transactions. In connection with the strategic reprioritization, the Company has reduced its workforce by approximately 80% to date in order to streamline the organization and to maximize its cash runway.

Third Quarter Ended September 30, 2023, Financial Results

Collaboration Revenue: Collaboration revenue was $0 for the third quarter of 2023, compared to $2.9 million for the third quarter of 2022. The decrease was due to revenue earned under the Solasia License and Collaboration Agreement in 2022 that did not recur in 2023.

Research and Development Expenses: Research and development expenses were $3.7 million for the third quarter of 2023, compared to $7.9 million for the third quarter of 2022, a decrease of approximately 54%. The decrease was primarily due to lower program expenses of $0.8 million as a result of our wind-down of clinical activities, a $0.6 million decrease in employee-related expenses due to our reduced headcount, an accrual adjustment related to our de-prioritized clinical programs of $0.3 million and a $2.5 million milestone payment to MD Anderson in 2022 under the terms of our patent and technology license agreement that did not recur in 2023.

General and Administrative Expenses: General and administrative expenses were $3.6 million for the third quarter of 2023, compared to $3.3 million for the third quarter of 2022, an increase of approximately 9%. The increase was primarily due to higher consulting and professional services expenses of $0.9 million related to increased legal costs, partially offset by a $0.4 million decrease in employee-related expenses due to our reduced headcount and a $0.2 million decrease in insurance fees.

Restructuring Costs: Restructuring costs were $0.4 million for the third quarter of 2023, compared to $0 for the third quarter of 2022 due to severance expenses for terminated employees related to our strategic reprioritization announced in August 2023.

Property and Equipment and Right-of-Use Asset Impairment: Property and equipment and right-of-use asset impairment charges were $1.0 million for the third quarter of 2023, compared to $0 for the third quarter of 2022 due to changes in the intended use of our property and equipment and lease right-of-use asset following the announcement of our strategic reprioritization in August 2023.

Net Loss: Net loss was $8.5 million, or $(0.04) per share, for the third quarter of 2023, compared to a net loss of $8.9 million, or $(0.04) per share, for the third quarter in 2022.

Cash, Cash Equivalents and Restricted Cash: As of September 30, 2023, Alaunos had approximately $11.9 million in cash balances. The Company expects to have sufficient cash resources to fund operations into the second quarter of 2024 as a result of its ongoing strategic reprioritization.

On November 14, 2023 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported financial results and a business update for the third quarter ended September 30, 2023 (Press release, VolitionRX, NOV 14, 2023, View Source [SID1234637647]). Volition management will host a conference call tomorrow, November 15 at 8:30 a.m. U.S. Eastern Time to discuss these results. Conference call details can be found below.

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Cameron Reynolds, President and Group Chief Executive Officer at Volition, commented:

"I am pleased to report that in the first nine months of 2023 revenue for the Nu.Q Vet Cancer Test grew almost five-fold over the prior year period, reflecting sales of the reference kits through our global, regional and local supply agreements. Additionally, total year-to-date revenue was $531,000, a 185% increase compared to the comparable period of 2022. We believe that these results demonstrate steady growth, but not yet the "ramp" in revenues that we anticipate. We expect revenues to accelerate as we close out 2023 and into the early part of 2024 as several additional distributors come online with our Nu.Q Vet Test.

"We continued to make considerable progress with our Nu.Q NETs pillar. Most recently, we completed the Q-Sub process with the U.S. Food and Drug Administration, and we are happy to announce that they have agreed to the regulatory pathway of a traditional 510(k) with an adjudicated clinical comparator. We are grateful for the opportunity to participate in the Q-Sub process and appreciate the feedback we have received. The interactions were engaging and helpful and we feel gave us a clear regulatory path we need to follow for this potentially exciting opportunity.

"We hosted our first Key Opinion Leader Roundtable Event focused on sepsis in September and published an expert-led report. We also presented new data at the ESICM congress in Milan in October and were featured in a peer-reviewed publication from one of our Centers of Excellence in Europe.

"In October, we unveiled what we believe to be an entirely new cancer detection method at ESMO (Free ESMO Whitepaper) 2023, the annual congress of the European Society for Medical Oncology, and hosted a webinar detailing this transformational method. The results to date are exciting and we are now developing a range of cancer-specific assays which we expect to be more accurate than these preliminary results, and look forward to sharing our progress beginning in the first quarter of 2024. We believe that this opportunity is significant, and we have been encouraged by the level of interest shown by a range of leading diagnostic and liquid biopsy companies following ESMO (Free ESMO Whitepaper).

"I am also delighted in the progress we are making to ensure our Nu.Q Vet Cancer Test is accessible worldwide, with our recent launch in the UK and Ireland through two leading local companies; NationWide Laboratories and Veterinary Pathology Group. We anticipate launches in additional markets in Asia in the coming months."

An interview with Cameron Reynolds, President and Group Chief Executive Officer of Volition, Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development LLC, and Terig Hughes, Group Chief Financial Officer of Volition.

View Source

Financial Highlights

·

Cash and cash equivalents as of September 30, 2023, totaled approximately $10.8 million compared with $10.9 million at the end of 2022.
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Subsequent to quarter end, a Walloon institutional fund and regional government bodies of the Walloon Region of Belgium approved providing additional funding to the company aggregating approximately $6 million. We expect the funding transactions to close this month.
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Expect to receive a further $13 million in milestone payments from Heska Corporation in the coming months.

Event: VolitionRx Limited Third Quarter 2023 Earnings and Business Update Conference Call

Date: Wednesday, November 15, 2023

Time: 8:30 a.m. U.S. Eastern Time

U.S. & Canada Dial-in: 1-877-407-9716 (toll free)

U.K. Dial-in: 0 800 756 3429 (toll free)

Toll/International: 1-201-493-6779

Conference ID: 13742634

Cameron Reynolds, President and Group Chief Executive Officer of Volition, will host the call along with Terig Hughes, Group Chief Financial Officer, Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development LLC, Dr. Andrew Retter, Medical Consultant to Volition and Scott Powell, Executive Vice President, Investor Relations. The call will provide an update on important events which have taken place in the third quarter of 2023 and upcoming milestones.

A live audio webcast of the conference call will also be available on the investor relations page of Volition’s corporate website at View Source In addition, a telephone replay of the call will be available until November 29, 2023. The replay dial-in numbers are 1-844-512-2921 (toll-free) in the U.S. and Canada and 1-412-317-6671 (toll) internationally. Please use replay pin number 13742634.

On November 14, 2023 Vincerx Pharma, Inc. (Nasdaq: VINC)("Vincerx"), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported financial results for the third quarter ended September 30, 2023 and provided a corporate update (Press release, Vincerx Pharma, NOV 14, 2023, View Source [SID1234637646]).

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"This has been a standout quarter for Vincerx, with progress across all three clinical programs, including VIP943, our first ADC from the VersAptx Platform, our versatile and adaptable, next-generation bioconjugation platform, and VIP236, our first-in-class SMDC," said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx. "The Vincerx team has done an outstanding job delivering ahead of expectations. Not only were we able to dose our first VIP943 patient within a matter of weeks of FDA clearance, but we have quickly moved through our first cohort and are beginning enrollment in our second cohort. This momentum is a testament to the promising safety profile seen in the first cohort and the enthusiasm from investigators participating in the trial. We expect to report preliminary data from this study in mid-2024."

"We also continued to make excellent progress with our other clinical programs. VIP236, our first-in-class SMDC for advanced solid tumors, is dosing patients under an optimized dosing schedule of once every three weeks, and we are pleased with the early safety profile we are seeing. We expect to report preliminary data in early 2024. In addition, enitociclib, our CDK9 inhibitor, continues to show promise. The transformed follicular lymphoma patient in our monotherapy study remains on treatment and has achieved long-term stable disease for more than 16 months with a 51% reduction in target lesions. Furthermore, our collaboration with the National Institute of Health (NIH) on a Phase 1 dose-escalation study of enitociclib with venetoclax and prednisone continues to make progress, with four patients dosed and an overall favorable safety profile. Even more exciting, the first patient on the second dose level remains on study with a partial response with evidence of an 80% reduction in the pulmonary lesion and resolution of skin lesions from their peripheral T-cell lymphoma," added Dr. Hamdy.

"We continue to take a very disciplined approach to our portfolio, focusing our resources on our two lead programs, VIP943 and VIP236. Concurrently, we are exploring business development opportunities that can advance all of our clinical programs as well as leverage the power of our VersAptx Platform. The therapeutic and safety potential of ADCs and other conjugates is being widely recognized, as evidenced by the overall excitement and activity in the ADC space, including collaborations, acquisitions, and positive clinical results. We believe that our next-generation ADC technology represents a significant step forward in the treatment of cancer by potentially overcoming the safety and efficacy challenges associated with many ADCs. As a result, we feel we are well-positioned to capitalize on industry enthusiasm for bioconjugates," concluded Dr. Hamdy.

Third Quarter 2023 Corporate Highlights:

VIP943: CD123-KSPi ADC for leukemias and myelodysplastic syndrome

VIP943, the first ADC from our VersAptx Platform, consists of an anti-CD123 antibody, a unique linker cleaved intracellularly by legumain, and a novel kinesin spindle protein inhibitor (KSPi) payload enhanced with our CellTrapper technology. Our proprietary effector chemistry (linker + payload) was designed to reduce non-specific release of the payload and ensure payload accumulation in cancer cells versus healthy cells. The increased therapeutic index has the potential to address challenges associated with many ADCs by improving efficacy and reducing severe toxicities.

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VIP943 is in a Phase 1 dose-escalation trial evaluating patients with relapsed/refractory acute myeloid leukemia, myelodysplastic syndrome, and B-cell acute lymphoblastic leukemia who have exhausted standard therapeutic options (NCT06034275).

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The VIP943 IND was approved by the FDA within 30 days, and we dosed our first patient in less than three weeks of FDA clearance. In addition, enrollment has moved quickly (Cohort 1, n=3), and we are now enrolling our second cohort. We are pleased with the early safety profile.

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We will present new data on robust preclinical activity at the 65th American Society for Hematology Meeting (ASH 2023) in December.

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We expect to report preliminary clinical trial data in mid-2024.

VIP236: SMDC with αvb3 integrin binder linked to optimized CPT payload for solid tumors

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VIP236, the first-in-class SMDC from our VersAptx Platform, consists of an αvb3 integrin binder, a neutrophil elastase linker cleaved in the tumor microenvironment, and a camptothecin (CPT) payload optimized for high permeability and low efflux. VIP236 was designed to deliver its payload to advanced/metastatic tumors that express αvb3.

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Preclinical data show enhanced efficacy, independent of HER2 status, in patient-derived and cell line-derived gastric cancer models compared with ENHERTU, an approved ADC.

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VIP236 is being evaluated in a Phase 1 dose-escalation trial treating patients with advanced or metastatic solid tumors (NTC05371054). As VIP236 is a first-in-class drug, the Phase 1 trial is evaluating various dosing schedules.

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To date, 10 patients with advanced or metastatic disease that has relapsed or is refractory to standard of care have received VIP236; the early safety profile of once every three weeks dosing is promising.

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We expect to report preliminary clinical trial data in early 2024.

VIP924: CXCR5-KSPi ADC for B-cell malignancies

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VIP924, the second ADC from our VersAptx Platform, consists of an anti-CXCR5 antibody, a unique linker cleaved intracellularly by legumain, and a novel KSPi payload enhanced with our CellTrapper technology. Our proprietary effector chemistry (linker + payload) was designed to reduce non-specific release of the payload and ensure payload accumulation in cancer cells versus healthy cells. The increased therapeutic index has the potential to address challenges associated with many ADCs by improving efficacy and reducing severe toxicities.

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VIP924 preclinical data demonstrate significant tumor growth inhibition in a panel of lymphoma cell lines and cell line- and PDX-derived lymphoma models. VIP924 also induces sustained tumor regression in mantle cell lymphoma (MCL) and diffuse large B-cell lymphoma (DLBCL) models, including ibrutinib-refractory MCL models.

LOGO

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At ASH (Free ASH Whitepaper) 2023, preclinical data will be presented showing that our first-in-class anti-CXCR5 ADC compares favorably to the approved ADCs, Polivy and Zynlonta.

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Despite compelling preclinical data, we continue to judiciously pace our investment in VIP924 to focus resources on VIP236 and VIP943.

Enitociclib: CDK9 inhibitor for hematologic malignancies and solid tumors

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Enitociclib, a highly selective CDK9 inhibitor, prevents activation of RNA polymerase II, resulting in reduction of known oncogenes MYC and MCL1.

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Enitociclib is in a dose-escalation Phase 1 trial (NTC05371054) in collaboration with the NIH evaluating the combination of enitociclib, venetoclax and prednisone in DLBCL and peripheral T-cell lymphoma (PTCL). The first dose level completed enrollment with no drug-related safety signal (n=3; 1=DLBCL, 2=PTCL). The first patient on the second dose level (n=1; 1=PTCL) remains on study with a partial response due to an 80% reduction in the pulmonary lesion on computerized tomography (CT) scan and resolved skin lesions. Investigators are pleased with the safety profile of this novel combination and continue with enrollment.

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Additional combination studies will be determined based on financing/partnering support.

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Proof-of-concept monotherapy efficacy has been generated in early-stage clinical studies:

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Hematologic malignancies: two patients with double-hit DLBCL achieved complete metabolic responses after 10 cycles of treatment and remain in remission, off treatment, for over four years; one patient with transformed follicular lymphoma remains on treatment, achieving long-term stable disease for over 16 months with a 51% reduction in target lesions.

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Solid tumors: 13 responses of stable disease, including durable disease control, in patients with ovarian, pancreatic, and salivary gland cancer.

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Strong preclinical evidence for additional indications (eg, multiple myeloma) and pediatric indications.

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Collaborators from the University of Calgary will present a poster at ASH (Free ASH Whitepaper) 2023 showing preclinical activity in pediatric leukemia.

Additional Corporate Highlights

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Our next-generation bioconjugation platform is now known as the VersAptx Platform. This name reflects the versatile and adaptable features and strengths of this innovative technology.

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We received a one-year extension of Small and Medium-Sized Enterprise (SME) status by the European Medicines Agency’s (EMA) Micro, Small and Medium-Sized Enterprise, enabling us to become eligible for EMA fee reductions and waiver and other financial incentives.

Third Quarter 2023 Financial Results

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Vincerx had approximately $20.8 million in cash, cash equivalents and marketable securities as of September 30, 2023, as compared to approximately $52.5 million as of December 31, 2022. Based on its current business plans and assumptions, Vincerx believes its available capital will be sufficient to meet its planned operating requirements into late 2024.

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Research and development expenses for the three- and nine-months ended September 30, 2023 were approximately $6.8 million and $25.3 million, as compared to approximately $11.1 million and $40.8 million for the same periods in 2022. The decrease for the three months ended September 30, 2023 compared with the same period in 2022 is primarily the result of decreases in manufacturing services associated with our ADC program of approximately $4.1 million and clinical services of approximately $0.9 million, partially offset by the $1.0 million development milestone in connection with Vincerx’s IND filing for VIP943. The decrease for the nine months ended September 30, 2023 compared with the same period in 2022 is primarily the result of decreases in manufacturing services associated with our ADC program of approximately $5.5 million, stock-based compensation expense of approximately $5.1 million, clinical services of approximately $2.7 million, and payroll related costs of approximately $1.7 million as a result of our headcount reduction in June 2022.

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General and administrative expenses for the three- and nine-months ended September 30, 2023 were approximately $3.5 million and $11.8 million, as compared to approximately $4.5 million and $14.9 million for the same periods in 2022. These decreases are primarily due to decreases in stock-based compensation expense of approximately $0.5 million and $2.3 million for the three- and nine-months ended September 30, 2023, respectively, as well as a decrease in professional services of $0.5 million and $0.8 million for the three and nine months ended September 30, 2023, respectively.

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For the quarter ended September 30, 2023, Vincerx reported a net loss of approximately $9.7 million, or $0.46 per share, compared to a net loss of approximately $16.9 million, or $0.80 per share, for the quarter ended September 30, 2022.

On November 14, 2023 Werewolf Therapeutics, Inc. (the "Company" or "Werewolf") (Nasdaq: HOWL), an innovative biopharmaceutical company pioneering the development of conditionally activated therapeutics engineered to stimulate the body’s immune system for the treatment of cancer, reported a business update and announced financial results for the third quarter ended September 30, 2023 (Press release, Werewolf Therapeutics, NOV 14, 2023, View Source [SID1234637644]).

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"Werewolf has made tremendous strides since last quarter, most notably with the presentation of promising first-in-human data from our lead clinical program, WTX-124. Preliminary data presented at SITC (Free SITC Whitepaper) indicate that WTX-124 is well-tolerated and elicits monotherapy biomarker and clinical activity, including two patients with ongoing unconfirmed partial responses in the 12 mg cohort," said Daniel J. Hicklin, Ph.D., President and Chief Executive Officer of Werewolf. "We look forward to sharing additional data to inform our recommended dose to proceed into monotherapy expansion arms in the first half of 2024. We are also pleased to announce the addition of WTX-518, an IL-18 INDUKINE molecule, as our newest pipeline candidate and expect to present preclinical data on this molecule in the first half of 2024."

Recent Highlights and Upcoming Milestones

WTX-124: a systemically delivered, conditionally activated Interleukin-2 (IL-2) INDUKINE molecule being developed as monotherapy and in combination with checkpoint inhibitor therapy in multiple solid tumor types.

•In November 2023, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 38th Annual Meeting, Werewolf presented preliminary clinical data from the first four monotherapy dose escalation cohorts of Study WTX-124×2101, its Phase 1/1b, multi-center, open-label clinical trial evaluating WTX-124 in patients with immunotherapy sensitive advanced or metastatic solid tumors who have failed standard of care treatment, including checkpoint inhibitor therapy. The preliminary data established proof of mechanism for WTX-124 and proof of concept for Werewolf’s INDUKINE design, with highlights as follows, as of the data cut-off date of October 18, 2023:

◦WTX-124 was generally well-tolerated across 16 patients, with no Grade 3 or higher treatment-related adverse events, no related serious adverse events and no evidence of vascular leak syndrome, at doses up to and including 12 mg.
◦Early evidence of antitumor activity was observed in monotherapy dose escalation cohorts 3 (6 mg) and 4 (12 mg), including two patients with ongoing unconfirmed partial responses (PR) dosed at 12 mg. An additional patient dosed at 12 mg showed evidence of anti-tumor activity.
◦These WTX-124 data are consistent with key INDUKINE pharmaceutical properties, including systemic delivery of the WTX-124 prodrug with preferential activation in the tumor microenvironment while preserving a wide therapeutic index supportive of continued dose escalation.
•Werewolf is progressing Study WTX-124×2101, and dose escalation is ongoing in both the monotherapy and combination therapy arms of the study. The Company expects to report additional interim data from monotherapy dose escalation cohorts, declaration of a recommended dose for expansion, and opening of the monotherapy expansion arms in the first half of 2024.
•In September 2023, Werewolf hosted a virtual event reviewing the IL-2 landscape and opportunity for WTX-124 among next-generation therapeutic approaches. The event featured key opinion leader in cytokines and cancer immunotherapy, Michael Atkins, M.D., William M. Scholl Professor and Vice Chair of the Department of Oncology at Georgetown University.

WTX-330: a systemically delivered, conditionally activated Interleukin-12 (IL-12) INDUKINE molecule being developed in refractory and/or immunologically unresponsive tumors.

•Werewolf is progressing Study WTX-330×2101, its Phase 1, multi-center, open-label trial evaluating WTX-330 as a monotherapy in patients with immunotherapy insensitive or resistant advanced or metastatic solid tumors or non-Hodgkin lymphoma. Enrollment is ongoing in dose-escalation.

Additional Updates:
•Werewolf is announcing the addition of WTX-518 as a pipeline candidate for preclinical development. WTX-518 is a conditionally activated IL-18 INDUKINE molecule in development for the treatment of cancer and is wholly owned by Werewolf. IL-18 is designed to promote activation of immune cells in the tumor microenvironment resulting in antitumor immunity. Werewolf expects to present preclinical data regarding this molecule in the first half of 2024.
•At SITC (Free SITC Whitepaper), Werewolf also presented five posters with preclinical and translational data supporting PREDATOR platform capabilities; pipeline programs, including both clinical candidates as well as WTX-712 (IL-21); and the potential of INDUKINE molecules as a complement to other anti-cancer approaches, such as checkpoint inhibitors and cell therapy. All posters are available at investors.werewolftx.com/news-and-events/scientific-resources.

Financial Results for the Third Quarter of 2023:
•Cash position: As of September 30, 2023, cash and cash equivalents were $130.1 million, compared to $137.5 million as of June 30, 2023. The Company also had restricted cash and cash equivalents of $21.2 million as of September 30, 2023 and June 30, 2023, respectively. The Company expects that its existing cash and cash equivalents will be sufficient to fund its operational expenses and capital expenditure requirements through at least the fourth quarter of 2024.
•Collaboration revenue: Collaboration revenue was $5.9 million for the third quarter of 2023, compared to $5.0 million for the same period in 2022. Collaboration revenue consists of revenue recognized from the Company’s licensing agreement with Jazz Pharmaceuticals (Jazz) and includes fixed payments received from Jazz, plus costs incurred for research services to be reimbursed by Jazz.
•Research and development expenses: Research and development expenses were $10.8 million for the third quarter of 2023, compared to $13.1 million for the same period in 2022. The decrease in research and development expenses was primarily due to a decrease in contract manufacturing costs associated with WTX-124 and WTX-330. The decline in contract manufacturing costs was partially offset by an increase in clinical trial costs for WTX-124 and WTX-330.
•General and administrative expenses: General and administrative expenses were $4.3 million for the third quarter of 2023, compared to $4.4 million for the same period in 2022. The decrease in general and administrative expenses was primarily due to a reduction in insurance premiums, which was offset in part by an increase in costs incurred to protect the Company’s intellectual property.
•Net loss: Net loss was $8.3 million for the third quarter of 2023, compared to $11.9 million for the same period in 2022.