On August 12, 2021 ALX Oncology Holdings Inc., ("ALX Oncology") (Nasdaq: ALXO) a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, reported financial results for the second quarter ended June 30, 2021 and provided clinical development and operational highlights (Press release, ALX Oncology, AUG 12, 2021, View Source [SID1234591861]).

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"We are pleased to report on the substantial progress we made since the prior quarter with our lead product candidate, ALX148, and that the United States Adopted Names (USAN) Council has approved ‘evorpacept’ as the nonproprietary (generic) name for ALX148," said Jaume Pons, Ph.D., Founder, President and Chief Executive Officer of ALX Oncology. "Our highlights include the presentation of additional positive Phase 1b data in second-line or greater HER2 positive gastric or gastroesophageal junction cancer from our ASPEN-01 trial during an oral session at the ESMO (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer; these results provide the basis for initiating a randomized Phase 2/3 trial in the same setting (ASPEN-06) scheduled to start in the second half of this year."

"We recently dosed the first patient in our Phase 2 ASPEN-04 trial in first line metastatic or unresectable recurrent head and neck squamous cell carcinoma in combination with KEYTRUDA and chemotherapy, are continuing enrollment in our Phase 2 ASPEN-03 trial in first line metastatic or unresectable, PD-L1 positive recurrent head and neck cancer in combination with KEYTRUDA, and plan to present full results of the Phase 1b study (ASPEN-01) in the fourth quarter of this year. In addition, we plan to present results from our Phase 1 study in myelodysplastic syndromes, to initiate a Phase 2 study in the same setting (ASPEN-02), as well as to initiate a Phase 1 study in acute myeloid leukemia (ASPEN-05)," Dr. Pons continued.

Recent Clinical Developments for Evorpacept (Also known as ALX148)

First Patient Dosed in Phase 2 ASPEN-04 Study
In July 2021, dosed first patient in the Phase 2 ASPEN-04 study evaluating the combination of evorpacept, a next generation CD47 blocker, with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, and standard 5-fluorouracil and platinum chemotherapy for the first line ("1L") treatment of patients with metastatic or unresectable, recurrent head and neck squamous cell carcinoma ("HNSCC"). In June 2021, the U.S. Food and Drug Administration ("FDA") informed ALX Oncology that it reviewed its standard non-clinical safety study and has lifted the previously set partial clinical hold and cap on patient enrollment.
First Patient Dosed in Phase 2 ASPEN-03 Study
In May 2021, dosed first patient in the Phase 2 ASPEN-03 study, which is also evaluating the efficacy of evorpacept in combination with KEYTRUDA (pembrolizumab) for the 1L treatment of patients with PD-L1 expressing metastatic or unresectable, recurrent HNSCC with a combined positive score ("CPS") ≥ 1. In June 2021, the FDA informed ALX Oncology that it reviewed its standard non-clinical safety study and has lifted the previously set partial clinical hold and cap on patient enrollment.
Data for Phase 1b ASPEN-01 Study Presented at 23rd ESMO (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer
In July 2021, updated clinical data from the Phase 1b ASPEN-01 trial evaluating evorpacept in combination with trastuzumab and CYRAMZA (ramucirumab) for the treatment of gastric or gastroesophageal junction cancer ("G/GEJ") were shared in an oral presentation at the 23rd ESMO (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer. Data showed that evorpacept in combination with trastuzumab and ramucirumab is highly active and well-tolerated in patients with second line ("≥2L") or greater HER2 positive G/GEJ cancer.
Collaboration and Supply Agreement Entered with Eli Lilly
In June 2021, ALX Oncology entered into a clinical trial collaboration and supply agreement with Eli Lilly and Company to evaluate the combination of evorpacept with CYRAMZA (ramucirumab), Lilly’s anti-VEGFR2 antibody, for the treatment of patients with HER2 positive G/GEJ. Under the terms of the agreement, ALX Oncology will conduct a Phase 2/3 study to evaluate the efficacy of evorpacept in combination with ramucirumab, trastuzumab, and paclitaxel for the treatment of patients whose tumors have progressed following treatment of HER2 targeted therapy and chemotherapy. Lilly will supply ramucirumab for this trial.
Anticipated Key Milestones for Remainder of 2021

Full results of a Phase 1b study of evorpacept in combination with Merck’s KEYTRUDA (pembrolizumab) and chemotherapy for the treatment of patients with HNSCC (ASPEN-01) are planned to be presented in the fourth quarter of 2021.
Initiation of a Phase 1b clinical trial with evorpacept in combination with Zymeworks’ zanidatamab in patients with advanced HER2-expressing breast cancer and other solid tumors is expected in the second half of 2021.
Initiation of a Phase 1 clinical trial evaluating evorpacept in combination with azacitidine and venetoclax in patients with acute myeloid leukemia ("AML") (ASPEN-05) is planned in the second half of 2021.
Initiation of a randomized Phase 2 trial of evorpacept in combination with Herceptin (trastuzumab), CYRAMZA (ramucirumab) and paclitaxel in second- or third-line treatment of patients with HER2-positive G/GEJ cancer (ASPEN-06) is expected in the second half of 2021.
Results of a Phase 1 clinical trial of evorpacept in combination with azacitidine in patients with myelodysplastic syndromes ("MDS") and the initiation of the Phase 2 clinical trial in MDS (ASPEN-02) are expected in the fourth quarter of 2021.
Second Quarter 2021 Financial Results:

Cash and Cash Equivalents: Cash and cash equivalents as of June 30, 2021, were $410.0 million. ALX Oncology continues to believe its cash and cash equivalents is sufficient to fund planned operations through 2024.
Net Loss: Generally accepted accounting principles (GAAP) net loss attributable to common stockholders was $16.3 million, or $0.40 per basic and diluted share and $14.0 million, or $4.41 per basic and diluted share for the three months ended June 30, 2021 and 2020, respectively. Non-GAAP net loss attributable to common stockholders was $14.0 million for the three months ended June 30, 2021, as compared to $10.7 million for the three months ended June 30, 2020. A reconciliation of GAAP to non-GAAP financial results can be found at the end of this news release.
Related-party Revenue: Related-party revenue for the three months ended June 30, 2021 was nil compared to $0.5 million for the prior-year period. The decrease in related-party revenue relates to the termination of the Tollnine Agreement as of July 1, 2020.
Research and Development ("R&D") Expenses: R&D expenses consist primarily of pre-clinical, clinical and manufacturing expenses related to the development of evorpacept. These expenses for the three months ended June 30, 2021 were $11.2 million, compared to $7.7 million for the prior-year period. The increase of $3.5 million was primarily attributable to an increase of $3.7 million in clinical and development costs due to higher expenses associated with increased pre-clinical, clinical and other research costs in advancement of our current lead product candidate, evorpacept, an increased personnel-related costs of $1.1 million primarily due to headcount growth, and an increase of $0.3 million other research and development costs primarily driven by milestone payments triggered by the initiation of our Phase 2 trials, offset by a decrease of $1.6 million in stock-based compensation expense primarily resulting from the modification of stock options in the second quarter of 2020 whereas there was no such modification in 2021.
General and Administrative ("G&A") Expenses: G&A expenses consist primarily of administrative employee-related expenses, legal and other professional fees, patent filing and maintenance fees, and insurance. These expenses for the three months ended June 30, 2021 were $5.1 million, compared to $3.2 million for the prior-year period. This increase of $1.9 million was primarily attributable to an increase in stock-based compensation expense of $1.0 million primarily resulting from additional stock option award grants at higher fair values, an increase in personnel-related costs of $0.5 million due to headcount growth, and a $0.4 million increase in other general and administrative costs related to being a public company, including directors and officers liability insurance premiums.

On August 12, 2021 Cardinal Health reported an agreement with TerraPower that will help advance the next generation of cancer treatment (Press release, Cardinal Health, AUG 12, 2021, View Source [SID1234591439]). Working together, the companies will develop and produce Actinium-225, which will be utilized in drug trials involving targeted alpha therapy for diseases such as breast, prostate, colon and neuroendocrine cancers, melanoma and lymphoma. Learn more in today’s press release

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On August 12, 2021 Brooklyn ImmunoTherapeutics, Inc. (NYSE American: BTX) ("Brooklyn"), a biopharmaceutical company focused on exploring the role that gene editing/cell and cytokine therapy can have in treating patients with cancer, blood disorders, and monogenic diseases, reported financial results for the quarter ended June 30, 2021 (Press release, Brooklyn ImmunoTherapeutics, AUG 12, 2021, View Source [SID1234591393]).

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Financial and corporate highlights for the quarter ended June 30, 2021 and subsequently include the following:

Completed the acquisition of Novellus Therapeutics Limited ("Novellus") in July 2021. Novellus is developing next‑generation engineered mesenchymal stem cell ("MSC") therapies using patented mRNA-based cell reprogramming and gene editing technologies licensed from Factor Bioscience ("Factor").

Raised nearly $51 million through equity line sales of common stock for general corporate purposes, including working capital to be used to enhance the development of the mRNA gene editing and cell therapies technology recently licensed from Factor.

Appointed Jay Sial as chief administrative officer and Kevin D’Amour, Ph.D. as chief scientific officer.

Established a research and development center in Cambridge, Massachusetts, to pursue its mRNA-based gene editing and cellular therapies, co-locating with Factor.

Howard Federoff, M.D., Ph.D., Brooklyn’s President and Chief Executive Officer, commented, "The second quarter advanced the evolution of Brooklyn ImmunoTherapeutics from being a cytokine-focused immunotherapeutics company to a platform company with a pipeline of next-generation engineered cellular, gene editing and cytokine products. This was in no small measure due to the acquisition of Novellus, which enables us to utilize the full range of the MSCs they have developed with no restriction on fields of use. By combining these with our licensed mRNA-based cell reprogramming and gene editing technology from Factor Bioscience, we believe we can create a platform technology that will lead to a family of product candidates in varying stages of development from target selection to research and preclinical, including one in the respiratory area that is in the IND-enabling stage."

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"Further, we believe we are positioned to develop a variety of gene-modified products using this platform, which ultimately could help solve problems of treating certain conditions that to date have confounded science, including potential treatments for a set of solid tumors, autoimmune disorders where inflammation is a prominent feature, and addressing multiple issues in the liver, the brain and the eye, as well as applying that technology again to iPSCs for a multitude of applications," continued Dr. Federoff.

"The quarter also resulted in creating a strong balance sheet and a growing in-licensed intellectual property portfolio," said Dr. Federoff. "Following the Novellus acquisition, we had approximately $25 million of cash on hand, which we estimate will fund our operations and expansion through the end of 2023," Dr. Federoff continued. "We are awaiting the readout of our Phase 2b trial for neoadjuvant head and neck cancer with our original asset, IRX-2 human-derived cytokines, during the first half of 2022. Additionally, we have multiple investigator-driven trials in a number of additional cancer types, and additional planned studies in 2022 and 2023."

"We have made a considerable investment in ensuring that we have the right people in place, and with the addition of our new chief scientific officer Dr. Kevin D’Amour and new chief administrative officer Jay Sial. Now it’s time for us to begin to execute on these ambitious plans that this next-generation version of Brooklyn ImmunoTherapeutics promises," concluded Dr. Federoff.

Financial Results for Quarter Ended June 30, 2021

Operating expenses for the quarter ended June 30, 2021 were $10.1 million, as compared to operating expenses for the quarter ended June 30, 2020 of $2.0 million.

Research and development expenses increased to $5.4 million for the quarter ended June 30, 2021 compared to $1.0 million in the quarter ended June 30, 2020. Research and development expenses increased due to upfront payments associated with licensed technology, increased clinical trial expenses, and stock-based compensation for the issuance of equity awards. Brooklyn expects research and development expenses to continue to grow as it expands its clinical trial activities.

General and administrative expenses increased to $4.6 million in the second quarter of 2021 compared to $1.0 million during the same period in 2020. The quarter-over-quarter increase in general and administrative expense was primarily related to increased legal, accounting and consulting fees associated with merger and acquisition activity, costs associated with being a publicly traded company, and increased stock-based compensation resulting from the issuance of equity awards. Brooklyn expects general and administrative expenses to continue to increase in future periods as it increases its business activities and incurs costs associated with being a publicly traded company.

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Net loss for the quarter ended June 30, 2021 was $(27.8) million, as compared to $(3.1) million for the quarter ended June 30, 2020.

As of June 30, 2021, Brooklyn ImmunoTherapeutics had $50.2 million in cash, of which approximately $23.0 million was paid as partial consideration for the acquisition of Novellus on July 16, 2021.

On August 12, 2021 A Buffalo-based biotechnology company reported that it has received a $2.2 million federal grant to begin clinical trials on a treatment for brain cancer using light therapy (Press release, Photolitec, AUG 12, 2021, View Source [SID1234587063]).

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Photolitec LLC, which spun off from Roswell Park Comprehensive Cancer Center in 2010, received the three-year grant from the National Institutes for Health through the Small Business Innovation Research (SBIR) program, along with approvals from the Food & Drug Administration to begin phase one human trials.

The trials will allow the company to use its Photobac compound in combination with photodynamic therapy (PDT) to treat glioblastoma, a type of brain cancer.

This next step in the drug development process follows orphan drug status approval for Photobac by the FDA in late 2017; and an initial SBIR grant of $1.9 million awarded earlier that year.

Ravindra Pandey, founder and chief scientific officer, is director of pharmaceutical chemistry at Roswell Park and first developed the compounds there in his lab. They were later licensed back to the company and have been further developed in collaboration with global partners AMI-Organics in India and HISUN Pharma in China.

With no approved treatments yet for these types of brain tumors and the recurrence rates, the orphan drug status will definitely help speed up the process, Pandey said. The process helps surgeons be more precise in attacking tumors with better specificity, he said.

"No treatment works except PDT and this compound can treat tumors not only with the margins that you want, but deep-seated tumors. So the chances are we’ll have a much better response," he said.

Scott Friedman, general counsel at Photolitec and chairman at Lippes Mathias Wexler Friedman LLP, said though the process is taking longer than initially projected, the company is making great progress.

"Ravi is continuing to make great progress," he said. "There are hoops that early-stage companies without necessarily hundreds of millions or billions of dollars are being asked to jump through, so it’s a challenging environment to succeed in and it does occasionally take more time than might be expected. But I believe great companies figure out how to move forward."

Though PDT is not new, Photolitec’s approach applies light to a photosensitizer, which destroys tumor cells when combined with the oxygen present in the cancer. The company’s Photobac allows for deeper penetration into the tumor, which also limits the impact on surrounding tissue and reduces long-term skin phototoxicity.

Plans call for submitting clinical protocols to Roswell Park’s review board for approval, with a goal of beginning trials soon thereafter. Pandey is hopeful that process will be hastened with the FDA approval already in hand.

"Generally what happens is we send it for IRB first and then FDA but in this case it was the other way around," he said. "We hope that will make it faster. We already have the drug formulations and we’re all ready to go."

Pandey said the new funding from NIH, plus a score of "outstanding" on the research by peer reviewers also helps boost the chances the drug will attract lots of attention from big pharma when it comes time to manufacture and market Photobac. Whether investors will keep manufacturing here in Western New York is another question.

"We’re waiting on phase one data first, and that could take a couple of years," he said.

Pfizer has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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