On November 13, 2023 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported that it has signed a conditional Term Sheet to acquire QSAM Biosciences, Inc. (QSAM) and its lead asset, CycloSam (Samarium-153-DOTMP) (Press release, Telix Pharmaceuticals, NOV 13, 2023, View Source [SID1234637579]). QSAM is a United States (U.S.) based clinical stage company developing therapeutic radiopharmaceuticals for primary and metastatic bone cancer.

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CycloSam is highly synergistic with Telix’s existing therapeutic development activity in both prostate cancer and sarcoma. The proposed acquisition, subject to customary completion terms, will further enhance and differentiate Telix’s innovation position to provide a continuum of care to patients from diagnosis and staging, systemic treatment of metastatic disease, to palliative care.

With the broad success of immunotherapies and the more recent clinical impact of radiopharmaceutical therapies in diseases like prostate cancer, there is an unmet need to cost-effectively and safely manage pain from bone metastases in very late-stage patients. This unmet need is further exacerbated by the quality-of-life issues associated with metastatic pain management, particularly opioid administration. In the U.S. alone, there are an estimated 400,000 patients up-staged with malignant bone metastasis primarily from prostate, breast and lung cancer.[1]

In addition to near-term opportunities in metastatic disease management, the proposed acquisition may also broaden the Company’s pipeline depth in osteosarcoma, a disease that mostly affects children and young adults, where QSAM has received Orphan Drug[2] and Rare Pediatric[3] Disease Designations (RPDD) from the FDA. The RPDD designation may enable CycloSam to be brought to market more rapidly through additional incentives, including eligibility for a Priority Review Voucher (PRV) under which the FDA may reduce the review period for a drug candidate to six months.

Douglas Baum, QSAM CEO and Co-Founder said, "CycloSam is a novel, de-risked clinical asset that has the potential to deliver tangible improvements of prior bone-seeking agents with established efficacy, safety and commercial utility. By joining forces with Telix we are accessing a specialised commercial team, distribution network and development expertise, with the goal of realising the full potential of this asset."

Dr Christian Behrenbruch, Managing Director and Group CEO of Telix continued, "We are pleased to announce our intention to acquire QSAM. This acquisition will bring a validated therapeutic candidate with the potential to accelerate development under the Orphan Drug and Rare Pediatric Disease Designations, and a highly experienced team that has completed numerous FDA approvals.

With CycloSam we plan to leverage Telix’s extensive experience and success in distributing short-life radiopharmaceuticals using a cold kit product from a nuclear pharmacy. Given these factors, we see a strong pathway to commercialisation."

Deal terms and conditions
Upon signing of the Term Sheet, Telix has agreed to pay QSAM an upfront Collaboration and Option Fee of US$2 million (approximately AU$3.1 million)[4] to advance development efforts based on mutually agreed goals and to provide sixty days of exclusivity pending completion of diligence and execution of a definitive acquisition agreement (Purchase Agreement).

If the acquisition of QSAM proceeds, upon closing, Telix will pay a total purchase price of US$33.1 million in equity through the issue of fully paid ordinary Telix shares. Following closing, Telix will also pay up to US$90 million in contingent clinical and commercial milestone payments in cash or equity (at Telix’s election), subject to achievement and satisfactory completion of milestones, through a Contingent Value Rights structure. The Purchase Price constitutes approximately a 52% premium to QSAM’s fully diluted capitalisation for the 10 days prior to execution of the Term Sheet.

The execution of the Purchase Agreement and closing of the acquisition is subject to many conditions, including satisfactory completion of diligence by both parties and approval of QSAM shareholders. The material terms of the Term Sheet relating to the proposed acquisition of QSAM are subject to change.

If the proposed Acquisition of QSAM does not close, the Collaboration and Option Fee will be converted to QSAM common stock at US$6.70 per share.

About bone cancer
In the U.S., there are over 400,000 new patients diagnosed each year with metastatic bone cancer and 350,000 patient deaths.[5] The incidence of advanced malignant tumours with bone metastasis can be up to 70%, especially common in patients with advanced prostate and breast cancer.[6] Osteosarcoma and Ewing’s sarcoma are the most common malignancies of bone tissues in children. The current standard of care is aggressive and suboptimal, and has led to marginal success with significant side effects and poor long-term survival prognosis.

On November 13, 2023 Theriva Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported financial results for the third quarter ended September 30, 2023, and provided a corporate update (Press release, Theriva Biologics, NOV 13, 2023, View Source [SID1234637574]).

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"We are encouraged by the growing clinical data that underscores the promise of our systemically administered oncolytic adenovirus and lead program, VCN-01, in key indications and combinations," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "Data from the ongoing study of VCN-01 in combination with durvalumab in patients with recurrent/metastatic squamous cell carcinoma of the head and neck (R/M HNSCC) were recently presented at the annual ESMO (Free ESMO Whitepaper) Congress. Results showed enhanced patient survival, which correlated with VCN-01 mediated increases in the CPS (combined positive score for PD-L1 staining), a key determinant of outcomes with anti-PD-(L)1 checkpoint inhibitor therapies. Together with data presented at this year’s SITC (Free SITC Whitepaper) meeting, these data further validate the feasibility of combining VCN-01 with immunotherapies."

Mr. Shallcross continued, "We continue to advance VIRAGE, our Phase 2b trial of VCN-01 in newly-diagnosed metastatic pancreatic ductal adenocarcinoma (PDAC), with patients dosed across sites in the U.S. and Spain. We have observed a consistent safety and tolerability profile and remain on track to complete enrollment for VIRAGE in the first half of 2024. As part of our commitment to transforming therapeutic approaches for devastating cancers, we will meet with the FDA before year-end to discuss the development pathway for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma. While our key area of focus is on advancing and maximizing the therapeutic potential of VCN-01, we continue to explore opportunities to bolster our pipeline with new oncolytic virus candidates from utilizing our Albumin Shield technology."

Recent Program Highlights and Anticipated Milestones:

VCN-01:

Dosing is underway and enrollment continues to progress for VIRAGE, the randomized, controlled, multicenter, open-label Phase 2b trial of VCN-01 in combination with standard-of-care chemotherapy (gemcitabine/nab-paclitaxel) as a first line therapy in newly diagnosed metastatic PDAC patients. Dosing at sites across the U.S. and Spain continues and VCN-01 has been well tolerated with a safety profile consistent with prior clinical trials. The trial is expected to enroll 92 patients and remains on track to complete enrollment in H1 2024.
The Institut Catala d’Oncologia (ICO) presented Phase 1 data from the investigator-sponsored study evaluating VCN-01 in combination with durvalumab for patients with R/M HNSCC. Encouraging survival was observed in patients progressing to anti-PD(L)-1 agents after systemic VCN-01 in combination with durvalumab. These data were featured in a poster presentation at the ESMO (Free ESMO Whitepaper) Congress, held both virtually and in Madrid, Spain from October 20-24, 2023.
Theriva hosted a virtual KOL event featuring expert oncologist Ricard Mesia, M.D. (Institut Català d’Oncologia / Catalan Institute of Oncology). In addition to reviewing key takeaways from the ESMO (Free ESMO Whitepaper) poster presentation, Dr. Mesia discussed the unmet medical needs in head and neck cancer, current treatment limitations, and the therapeutic potential of VCN-01 to elicit an extended anti-tumor immune response.
The University of Pennsylvania presented initial data from a Phase 1 study evaluating VCN-01 in combination with mesothelin-directed lentiviral transduced human chimeric antigen receptor modified T cells (huCART-meso) in patients with pancreatic and serous epithelial ovarian cancer. Initial results highlight the feasibility of administering VCN-01 with huCART-meso cells to treat solid tumors. These data were featured in a poster presentation at the SITC (Free SITC Whitepaper) Annual Meeting, held both virtually and in San Diego, November 1-5, 2023.
Additional anticipated milestones:
The Company will meet with the FDA before year-end to discuss the clinical development and potential registration pathway for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma.
SYN-004 (ribaxamase):

Dosing is underway for the ongoing Phase 1b/2a randomized, double-blinded, placebo-controlled clinical trial of SYN-004 (ribaxamase) in allogeneic hematopoietic cell transplant (HCT) recipients for the prevention of acute graft-versus-host-disease (aGVHD). SYN-004 appeared to be well tolerated in HCT patients treated with IV meropenem and SYN-004 was not detected in blood samples from the majority of the evaluable patients. The trial is on track to complete enrollment in the second cohort in H1 2024.
Third Quarter Ended September 30, 2023 Financial Results

General and administrative expenses decreased to $212,000 for the three months ended September 30, 2023, from $2.4 million for the three months ended September 30, 2022. This decrease of 91% is primarily comprised of the decrease in the fair value of the contingent consideration of $1.6 million, along with lower salary and bonus costs, investor relations fees, audit fees, travel, and VCN administrative expenses not included in the prior year, offset by an increase in consulting fees. The charge related to stock-based compensation expense was $95,000 for the three months ended September 30, 2023, compared to $93,000 for the three months ended September 30, 2022.

Research and development expenses increased to $4.0 million for the three months ended September 30, 2023, from approximately $2.6 million for the three months ended September 30, 2022. This increase of 56% is primarily the result of higher clinical trial expenses related to our VIRAGE Phase 2 clinical trial of VCN-01 in PDAC, offset by decreased expenses related to our Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, Phase 1a clinical trial of SYN-020, and decreased manufacturing expenses related to our Phase 1a clinical trial of SYN-020. We anticipate research and development expenses to increase as we continue enrollment in our VIRAGE Phase 2 clinical trial of VCN-01 in PDAC and our ongoing Phase 1 clinical trial in retinoblastoma, expand GMP manufacturing activities for VCN-01, and continue supporting our VCN-11 and other preclinical and discovery initiatives. The charge related to stock-based compensation expense was $40,000 for the three months ended September 30, 2023, compared to $28,000 related to stock-based compensation expense for the three months ended September 30, 2022.

Other income was $388,000 for the three months ended September 30, 2023 compared to other income of $161,000 for the three months ended September 30, 2022. Other income for the three months ended September 30, 2023 is primarily comprised of interest income of $382,000 and an exchange gain of $6,000. Other income for the three months ended September 30, 2022 is primarily comprised of interest income of $170,000 offset by an exchange loss of $9,000.

Cash and cash equivalents totaled $31.2 million as of September 30, 2023, compared to $41.8 million as of December 31, 2022.

Conference Call

Theriva Biologics will host a conference call on Monday, November 13, 2023, at 8:30 a.m. ET to discuss its financial results for the quarter ended September 30, 2023 and provide a corporate update. Individuals may participate in the live call via telephone by dialing 1-877-451-6152 (domestic) or 1-201-389-0879 (international) and using the conference ID: 13741546. Participants are asked to dial in 15 minutes before the start of the call to register. Investors and the public can access the live and archived webcast of this call via the "News & Media" section of the company’s website, View Source, under "Events" or by clicking here, up to 90 days after the call.

On November 13, 2023 POINT Biopharma Global Inc. (NASDAQ: PNT) (the "Company" or "POINT"), a company accelerating the discovery, development, and global access to life-changing radiopharmaceuticals, reported financial results for the third quarter ended September 30, 2023, and provided a business update (Press release, Point Biopharma, NOV 13, 2023, View Source [SID1234637573]).

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"The founding mission of our company is to accelerate the discovery, development and global access to radiopharmaceuticals," said Joe McCann, Ph.D., CEO of POINT Biopharma. "I view Lilly’s agreement to acquire POINT as validation of the uniqueness of our team, infrastructure and pipeline. Together, we can become a global radiopharmaceutical leader, executing on our mission and transforming lives touched by cancer."

POINT to be Acquired by Lilly

On October 3, 2023, Eli Lilly and Company (NYSE: LLY) and POINT Biopharma Global Inc. (NASDAQ: PNT) announced a definitive agreement for Lilly to acquire POINT for a purchase price of $12.50 per share in cash (an aggregate of approximately $1.4 billion) payable at closing. The purchase price payable at closing represents a premium of approximately 87% to POINT’s closing stock price on Oct. 2, 2023, the last trading day before the announcement of the transaction, and 68% to the 30-day volume-weighted average price. The tender offer, which was previously scheduled to expire at one minute past 11:59 p.m., Eastern time, on Nov. 9, 2023, has been extended until 5:00 p.m., Eastern time, on Nov. 16, 2023, unless the tender offer is further extended or earlier terminated, in order for the parties to satisfy outstanding closing conditions. The proposed acquisition is expected to close near the end of 2023, subject to customary closing conditions, including the tender of at least a majority of the outstanding Shares as of the expiration of the tender offer.

Business Highlights and Upcoming Milestones

Pipeline Updates

PNT2002: 177Lu-labelled PSMA-targeted radioligand therapy

Enrollment in PNT2002’s phase 3 SPLASH trial (NCT04647526) is complete and topline data is expected in the fourth quarter of 2023.

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PNT2004: fibroblast activation protein-α (FAP-α) targeted radioligand therapy

The PNT2004 program leverages the D-ala-boroPro FAP targeting warhead, which is a potent and selective FAP inhibitor. PNT6555 is the current clinical lead in the PNT2004 program.

The phase 1 FRONTIER trial (NCT05432193) utilizing 68Ga/177Lu-labelled PNT6555 closed enrollment after the dosing of cohort 3 (12 GBq) in October. There are no plans for expansion of the third cohort, and there were no dose limiting toxicities. Analysis of the dosimetry and clinical data from the FRONTIER trial is underway and includes pancreatic ductal adenocarcinoma, colorectal cancer, cholangiocarcinoma, and esophageal cancer subjects.

Presentation of data from the FRONTIER study is anticipated in the first half of 2024, and based on initial promising data we expect to advance at least one 2nd-generation D-ala-boroPro-based lead into the clinic next year.

‍

PNT2001: 225Ac-labelled next-generation PSMA-targeted radioligand therapy

For the phase 1 portion of ACCEL, the first-in-human phase 1/2 clinical trial for PNT2001’s actinium-225 program, we expect the first patient dosed in this trial to be in the first quarter of 2024. The trial was designed to enable the parallel exploration of PNT2001 in two populations: patients with later-stage mCRPC and patients with earlier-stage BCR or PSMA-positive oligorecurrent disease.

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Manufacturing & Supply Chain Updates

In July 2023, we announced an expanded agreement with ITM Isotope Technologies Munich SE (ITM), which broadens the supply of ITM’s no-carrier-added lutetium-177 (n.c.a. 177Lu) to POINT to enable its usage in the clinical and potential future commercial development of the 177Lu-based molecules in POINT’s development pipeline.

In September 2023, we signed a supply agreement with Eckert & Ziegler AG for no-carrier-added lutetium-177 (n.c.a. 177Lu).

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Discovery Updates

In September 2023, we announced a collaboration and license agreement to develop and commercialize DARPin-targeted radioligands ("Radio-DARPins"). The collaboration gives POINT exclusive access to Athebio’s intellectual property and capabilities in DARPin development in the radioligand therapy field. Together, the parties will collaborate in discovery, candidate selection and preclinical development of Athebody DARPins for use as Radio-DARPin drug entities. POINT will be solely responsible for the clinical development and commercialization of Radio-DARPins translated from the discovery collaboration.

Third Quarter 2023 Financial Results

Cash, Cash Equivalents, and Investments: As of September 30, 2023, POINT had approximately $399.0 million in cash, cash equivalents, and investments, which is anticipated to fund operations into 2026.

Net Loss: Net loss was $24.8 million and $66.7 million, or $0.23 and $0.63 net loss per share, for the three and nine months ended September 30, 2023, respectively, as compared to a net loss of $24.0 million and $65.0 million, or $0.26 and $0.71 net loss per share, respectively, for the same periods in 2022.

Research and Development Expenses: Research and development expenses were $26.9 million and $85.1 million for the three and nine months ended September 30, 2023, respectively, as compared to $20.8 million and $54.1 million, respectively, for the same periods in 2022.

General and Administrative Expenses: General and administrative expenses were $5.5 million and $15.6 million for the three and nine months ended September 30, 2023, respectively, as compared to $3.8 million and $11.7 million for the same periods in 2022.

On November 13, 2023 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for the third quarter of 2023 (Press release, Y-mAbs Therapeutics, NOV 13, 2023, View Source [SID1234637567]).

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"We delivered $20 million in DANYELZA (naxitamab-gqgk) net product sales in the third quarter of 2023, reflecting 59% growth compared to the same period in 2022, as sales continued trending upward since our initial launch," said Mike Rossi, President and Chief Executive Officer. "Our U.S. commercial team has made excellent progress by penetrating more high-volume Children’s Oncology Group ("COG") sites while our ex-U.S. partners continue to gain further traction with physicians prescribing DANYELZA across Europe and China for relapsed or refractory high-risk neuroblastoma patients."

Mr. Rossi continued, "Supported by a solid financial foundation, we have advanced our novel SADA radioimmunotherapy platform with the continued execution of our Phase 1 GD2-SADA trial and the recent Investigational New Drug ("IND") clearance of our CD38-SADA program. With existing cash and cash equivalents anticipated to support our business operations as currently planned into 2027, a growing commercial product in DANYELZA, and a differentiated radioimmunotherapy platform in SADA, we believe Y-mAbs is on a path to potentially transform the treatment paradigm for a variety of cancers and improve patients’ lives."

Third Quarter 2023 and Recent Corporate Developments

· On October 18, 2023, Y-mAbs announced that its Board of Directors appointed Mr. Rossi as President and Chief Executive Officer, effective November 6, 2023. Thomas Gad, who founded Y-mAbs in 2015 and has served as Interim Chief Executive Officer since 2022, has transitioned to the role of Vice Chairman of the Board of Directors and Chief Business Officer.
· On October 17, 2023, the U.S. Food & Drug Administration ("FDA") cleared Y-mAbs’ IND for CD38-SADA, marking the second clinical development program utilizing the Company’s novel SADA technology platform.
· On October 16, 2023, Y-mAbs announced the publication of the study of naxitamab-based chemoimmunotherapy in patients with chemoresistant high-risk neuroblastoma ("HR-NB") in the journal Cancers. The study investigated the HITS combination in patients with high-risk neuroblastoma who did not respond well to induction therapy. Patients who received HITS immediately after induction had higher response rates (47% vs. 18%) and superior estimated three-year overall survival (85% vs. 29%) compared with those who received the same combination regimen later in the course of treatment. The publication is entitled, "Early Salvage Chemo-Immunotherapy with Irinotecan, Temozolomide and Naxitamab Plus GM-CSF (HITS) for Patients with Primary Refractory High-Risk Neuroblastoma Provide the Best Chance for Long-Term Outcomes."

· On October 11, 2023, Y-mAbs showcased three poster presentations, in addition to an online publication, of DANYELZA at the 55th Congress of the International Society of Pediatric Oncology in Ottawa, Canada.
· On September 28, 2023, Y-mAbs received approval of its Mexican Marketing Authorization Application ("MAA") by COFEPRIS for DANYELZA, marking the Company’s second approval in Latin American with its distribution partner Adium Pharma S.A.

Financial Results

Revenues

DANYELZA net product revenues were $20.0 million and $61.0 million for the third quarter and nine months ended September 30, 2023, which represented increases of 59% and 86%, respectively, over $12.5 million and $32.8 million in the comparable periods of 2022.

The DANYELZA net product revenues of $20.0 million in the third quarter of 2023, represented a marginal decline compared to the second quarter of 2023, primarily driven by modest unevenness in international revenues after a series of inventory stocking orders from the Company’s international partners as reported in recent quarters.

As of September 30, 2023, Y-mAbs has delivered DANYELZA to 57 centers across the U.S. since initial launch, with nine new accounts added so far in 2023. During the third quarter ended September 30, 2023, approximately 63% of the vials sold in the U.S. were sold outside of Memorial Sloan Kettering Cancer Center ("MSK"), compared to 61% in the second quarter ended June 30, 2023.

Y-mAbs reported total revenues of $20.5 million and $61.5 million for the third quarter and nine months ended September 30, 2023, which represented increases of 63% and 82%, respectively, over $12.5 million and $33.8 million in the comparable periods of 2022. Total revenues in the third quarter and nine months ended September 30, 2023 included $0.5 million of license revenue recognized upon the September 2023 achievement of marketing authorization for DANYELZA in Mexico under the Company’s sublicense agreement with Adium. There was no license revenue in the third quarter ended September 30, 2022, and license revenue for the nine months ended September 30, 2022 was $1.0 million.

Operating Costs and Expenses

Cost of Goods Sold

Cost of goods sold was $2.6 million and $2.5 million for the third quarter ended September 30, 2023 and 2022, respectively. Cost of goods sold was $9.3 million and $5.4 million for the nine months ended September 30, 2023 and 2022, respectively. The increase in cost of goods sold in both periods was primarily driven by increased product revenue in the three and nine months ended September 30, 2023, and inventory write-downs of $0.4 million and $0.8 million in the three and nine months ended September 30, 2023, respectively, partially offset by a $1.2 million charge related to an inventory batch that did not meet the Company’s quality specifications during the three and nine months ended September 30, 2022.

Excluding the above inventory charges, the Company’s gross margin decreased slightly in the three and nine months ended September 30, 2023, compared to the same periods in 2022, due to an increase in ex-U.S. revenues, which were at lower gross margins.

Research and Development

Research and development expenses were $15.4 million for the third quarter ended September 30, 2023, a reduction of 32% compared to $22.5 million for the third quarter ended September 30, 2022. The $7.1 million decrease was primarily due to decreased spending on deprioritized programs, which resulted in a $5.7 million decrease in outsourced manufacturing, decreased personnel-related costs, inclusive of stock-based compensation, of $2.0 million, a $2.0 million decrease in outsourced research and supplies, and a $0.3 million decrease in clinical trials, partially offset by a $4.1 million increase in milestones and license acquisition costs related to the Company’s SADA License Agreement, as the Company determined that certain time-based clinical milestones within the agreement are probable of achievement based on the availability of necessary data and the assessment of clinical progress in the third quarter of 2023.

For the nine months ended September 30, 2023, research and development expenses were $40.8 million, a reduction of 43% compared to $71.8 million for the nine months ended September 30, 2022. The $31.0 million decrease was primarily due to decreased spending on deprioritized programs as described above, resulting in a $17.9 million decrease related to outsourced manufacturing, a $6.8 million decrease in outsourced research and supplies, a $3.1 million decrease in clinical trials and a $4.1 million decrease in personnel-related costs, partially offset by a $4.1 million increase in milestones and license acquisition costs related to the Company’s SADA License Agreement, as the Company determined that certain time-based clinical milestones within the agreement are probable of achievement based on the availability of necessary data and the assessment of clinical progress in the third quarter of 2023.

The $2.0 million and $4.1 million decreases in personnel-related costs during the three and nine months ended September 30, 2023, respectively, were driven by the headcount reduction as part of Company’s restructuring plan announced in January 2023, partially offset by severance charges recognized in conjunction with the restructuring plan.

Selling, General, and Administration

Selling, general, and administrative expenses were $10.2 million for the third quarter ended September 30, 2023, a reduction of 25% compared to $13.6 million for the third quarter ended September 30, 2022. The $3.4 million decrease in selling, general and administrative expenses was primarily attributable to a $1.9 million decrease in commercialization expenses, incurred in 2022 in anticipation of the potential omburtamab launch.

For the nine months ended September 30, 2023, selling, general, and administrative expenses were $33.7 million, a reduction of 33% compared to $50.1 million for the nine months ended September 30, 2022. The $16.4 million decrease in selling, general and administrative expenses was primarily attributable to a $10.9 million charge in the nine months ended September 30, 2022 related to contractual severance-related benefits for the Company’s former Chief Executive Officer, and, to a lesser extent, a $2.9 million decrease in commercialization expenses, incurred in 2022 in anticipation of a potential omburtamab launch.

Net Loss

Y-mAbs reported a net loss for the third quarter ended September 30, 2023, of $7.7 million, or ($0.18) per basic and diluted share, compared to a net loss of $27.5 million, or ($0.63) per basic and diluted share, for the quarter ended September 30, 2022. For the nine months ended September 30, 2023, the Company reported a net loss of $20.4 million, or ($0.47) per basic and diluted share, compared to a net loss of $96.7 million, or ($2.21) per basic and diluted share, for the nine months ended September 30, 2022. The favorable decrease in net loss was primarily driven by an increase in DANYELZA U.S. and international product revenues in the third quarter and nine months ended September 30, 2023, decreased research and development cost, and decreased selling, general and administration cost.

Cash and Cash Equivalents

As of September 30, 2023, Y-mAbs had approximately $86.6 million in cash and cash equivalents which, together with anticipated DANYELZA product revenues, is expected to support operations as currently planned into 2027. This estimate reflects the Company’s current business plan that is supported by assumptions that may prove to be inaccurate, such that Y-mAbs could use its available capital resources sooner than it currently expects.

Financial Guidance

The Company is updating and reiterating its full-year 2023 financial guidance, as follows:

· Reiterating anticipated DANYELZA net product revenues of between $80 million and $85 million;
· Lowering anticipated operating expenses to between $110 million and $115 million from previous guidance of between $115 million and $120 million;
· Lowering anticipated total annual cash burn to between $27 million and $32 million from previous guidance of between $40 million and $50 million; and
· Cash and cash equivalents now anticipated to support operations as currently planned into 2027 compared to previous cash runway guidance into 2026.

Webcast and Conference Call

Y-mAbs will host a conference call on Tuesday, November 14, 2023, at 9:00 a.m. ET. To participate in the call, please use the following dial-in information.

Investors (domestic): 877-407-0792
Investors (international): 201-689-8263
Conference ID: 13741478

To access a live webcast of the update, please use this link. Prior to the call and webcast, a slide presentation pertaining to Y-mAbs’ quarterly earnings will be made available in the investor relations section of the Company’s website, www.ymabs.com, shortly before the call begins.

On November 13, 2023 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating neurodegenerative disease and cancer through the inhibition of SEMA4D, reported financial results for the third quarter ended September 30, 2023 and provided a corporate update on progress in key programs (Press release, Vaccinex, NOV 13, 2023, View Source [SID1234637566]).

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Vaccinex achieved several important clinical milestones for pepinemab in both Alzheimer’s disease and Head and Neck Cancer.

Alzheimer’s Disease (AD):

Completed enrollment in the randomized, double-blind, Phase 1b/2a SIGNAL-AD trial of pepinemab in patients with mild Alzheimer’s disease (NCT04381468), funded in part by the Alzheimer’s Drug Discovery Foundation and by a grant from the Alzheimer’s Association.
Anticipate completing 12-months treatment in June 2024 at which time we will evaluate the impact of treatment on brain metabolic activity, a key biomarker of clinical progression in AD, as well as treatment effects on cognition employing several validated, clinically meaningful Alzheimer’s cognitive scales.
An improving AD-drug development environment, based on FDA’s recent full approval of LEQEMBI, enables the pathway to reimbursement and supports further investment in Alzheimer’s Disease drug development.
As previously reported, pepinemab has a differentiated mechanism of action, blocking SEMA4D, which is upregulated in neurons during stress of Alzheimer’s and Huntington’s disease and triggers the transformation of astrocytes and microglia from normal homeostatic functions to neuroinflammatory activity. Blockade of SEMA4D restores healthy astrocyte and neuronal functions while reducing neuroinflammation (Nature Medicine 2022).
We believe that the prevalence of AD (6 million people diagnosed with AD in the US alone) and current concerns about the limitations of anti-Aβ amyloid antibodies would make pepinemab attractive as a potential alternative to anti-Aβ antibodies or possibly for use in combination with an anti-Aβ for greater efficacy.
The potential impact of the AD program on Vaccinex valuation and financial resources make this Vaccinex’s most important near-term catalyst.
Head and Neck Cancer:

As previously reported, analysis of interim data from the first 36 patients in the single-arm, Phase 2 KEYNOTE B-84 study (NCT04815720) evaluating pepinemab in combination with KEYTRUDA in patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) suggests that the combination of pepinemab and KEYTRUDA resulted in an approximately 2X increase in objective responses (ORR) and progression free survival (PFS) in the subset of patients with hard-to-treat PD-L1-low tumors compared to historical response rates for checkpoint monotherapy in this population.
Biomarker data indicate that treatment induced the formation of highly organized lymphoid aggregates in tumor that correlate with disease control and have previously been shown to be important for positive response to checkpoint inhibitors.
Vaccinex and Merck are currently in the design stages for an expansion of the KEYNOTE-B84 study that may extend benefits to more patients.
Recent Milestones and News

Clinical Trials in Alzheimer’s Disease (CTAD) Conference Presentation:

In a highlighted podium presentation at the CTAD Conference on September 28, 2023. Vaccinex’s Senior Vice President for Clinical Development, Terrence Fisher, PhD, described the many physiological parallels between neurodegenerative processes in Alzheimer’s and Huntington’s disease (HD). A key common feature is the contribution of astrocyte activation (astrogliosis) to brain inflammation and damage. Vaccinex scientists have demonstrated that the stress of disease in both AD and HD leads to upregulation of SEMA4D in neurons and that this can trigger astrocytes to switch from their normal supportive physiological functions to inflammatory activity. This transition is marked by release of glial fibrillary acidic protein (GFAP), a characteristic astrocyte protein, into blood. Importantly, treatment with pepinemab was shown to result in a significant reduction in plasma GFAP levels in HD patients. Elevated plasma GFAP levels have also been reported to correlate with Aβ amyloid deposits in brain and to be associated with higher risk of dementia and faster rates of cognitive decline in AD.

ActivMAb Platform Technology:

The first clinical candidate selected through use of this technology (SRF114, a fully human monoclonal antibody targeting CCR8 for the potential treatment of solid tumors), is in a Phase 1/2 study sponsored by our licensee, Surface Oncology, recently acquired by Coherus Biosciences, Inc. (transaction closed September 8, 2023). The technology and its potential applications for drug discovery against complex membrane protein targets including the "hard to drug" class of membrane-associated G protein-coupled receptors (GPCRs) and ion channels is also being utilized in multiple Vaccinex antibody discovery collaborations with leading biopharmaceutical companies.

Financial Results for the Three Months Ended September 30, 2023:

Cash and Cash Equivalents and Marketable Securities. Cash and cash equivalents and marketable securities on September 30, 2023 were $0.1 million, as compared to $6.4 million as of December 31, 2022.

During Q3 2023, the Company raised finances of $1.3 million. On August 22, 2023, Vaccinex completed a Private Placement and issued approximately 0.20 million shares1 of common stock for aggregate proceeds of $0.7 million. Similarly, on September 22, 2023, Vaccinex completed a Private Placement and issued approximately 0.25 million shares of common stock for aggregate proceeds of $0.6 million. Vaccinex (Rochester) L.L.C., which is majority owned and controlled by Dr. Maurice Zauderer, the Company’s President, Chief Executive Officer, and a member of its board of directors purchased approximately 0.14 million shares of common stock for gross proceeds of $0.3 million.

In addition, on October 3, 2023, the Company issued and sold to certain investors (i) 7,600,000 shares of the Company’s common stock together with common warrants to purchase up to 7,600,000 shares of common stock and (ii) 2,000,000 pre-funded warrants to purchase up to 2,000,000 shares of common stock together with common warrants to purchase up to 2,000,000 shares of common stock, at a purchase price of $1.00 and $0.999, respectively, for aggregate gross proceeds of $9.60 million. FCMI, which is controlled by Albert D. Frieberg, the chairman of the Company’s board of directors, and Vaccinex (Rochester) L.L.C. purchased 3,000,000 and 500,000 shares of our common stock and accompanying common warrants, respectively, for an aggregate purchase price of $3.50 million.

Finally, in Q3 2023, the Company recorded a receivable of $0.9 million for the Employee Retention Credit. The Company expects to receive the cash proceeds by the end of 2023 or early Q1 2024.

Research and Development Expenses. Research and development expenses for the quarter ended September 30, 2023 were $4.4 million as compared to $3.4 million for the comparable period in 2022.

The increase in research and development expenses is primarily attributable to increased patient enrollment in the SIGNAL-AD study and the Phase 1b/2 KEYNOTE B84 study in HNSCC.

General and Administrative Expenses. General and administrative expenses for the quarter ended September 30, 2023 were $1.5 million as compared to $1.4 million for the comparable period in 2022.

The increase was attributable to increased legal and patent related services.

Comprehensive loss/Net loss per share. The Comprehensive Loss and Net loss per share for the quarter ended September 30, 2023 was $4.9 million and $(1.09) compared to $4.8 million and $(1.67) for the comparable period in 2022.

Full financial tables are included below. For further details on Vaccinex’s financials, refer to its Form 10-Q filed November 13, 2023 with the S.E.C.

About Pepinemab
Pepinemab is a humanized IgG4 monoclonal antibody designed to block SEMA4D, which can trigger collapse of the actin cytoskeleton and loss of homeostatic functions of astrocytes and glial cells in the brain and dendritic cells in immune tissue. Pepinemab has been administered to more than 400 patients and appears to be well-tolerated and to have a favorable safety profile.