On August 15, 2022 Novartis reported that the Phase III CANOPY-A study evaluating adjuvant treatment with canakinumab (ACZ885), an inhibitor of interleukin-1beta (IL-1β), in adult patients with stages II-IIIA and IIIB (T>5cm N2) completely resected (R0) non-small cell lung cancer (NSCLC) did not meet its primary endpoint of disease-free survival (DFS) versus placebo1 (Press release, Novartis, AUG 15, 2022, View Source [SID1234618375]). No unexpected safety signals were observed.1 Findings from the trial will be presented at an upcoming medical meeting.

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"We made an investment in the CANOPY program based on signals of reduced lung cancer incidence and mortality observed in the CANTOS study. These positive signals supported the study of canakinumab as adjuvant treatment for early lung cancer," said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development, Novartis. "While we are disappointed CANOPY-A did not show the benefit we hoped for, every trial generates scientific evidence that supports future research and development, and we look forward to continuing to pursue new therapeutic options for people living with lung cancer, whose needs remain urgent and significant. We thank the patients and clinical investigators whose time and commitment made this research possible."

CANOPY-A is a Phase III, multicenter, randomized, double blind study that is evaluating the efficacy and safety of canakinumab as adjuvant treatment in patients with NSCLC stages II-IIIA and IIIB (T>5cm N2), per American Joint Committee on Cancer/The Union for International Cancer Control (AJCC/UICC) 8th edition staging, whose margins are free of cancer following surgery2. In the trial, 1,382 patients were randomized 1:1 to canakinumab, 200 mg subcutaneously every three weeks, or matching placebo for up to one year2. Patients completed standard-of-care adjuvant cisplatin-based chemotherapy and radiation therapy, if applicable, prior to randomization2.

About canakinumab (ACZ885)
Canakinumab is a human monoclonal antibody that binds with high affinity and selectivity to human IL-1β and inhibits IL-1β activity by blocking its interaction with its receptors3-5. By inhibiting IL-1β, preliminary evidence suggests that canakinumab may suppress Pro-Tumor Inflammation to 1) enhance anti-tumor immune response; 2) reduce tumor cell proliferation, survival and invasiveness; and 3) impair angiogenesis5. Pro-Tumor Inflammation enables tumor development by driving cancer-causing processes and suppressing anti-tumor immune responses6,7.

About the CANOPY program
Novartis launched the CANOPY study program after observing significantly lower than expected rates of lung cancer mortality among patients in the Phase III cardiovascular CANTOS trial. The CANTOS trial evaluated canakinumab as a secondary prevention measure for cardiovascular events in patients following a heart attack5,8. Patients in the CANTOS trial also were at high risk for inflammatory cancers like lung cancer due to advanced age, smoking history, and other clinical risk factors5,8. Based on these findings, Novartis launched three large-scale, randomized, Phase III clinical trials and a Phase II clinical trial to investigate canakinumab as a potential treatment option in NSCLC.

Novartis and lung cancer
Lung cancer is one of the most common cancers worldwide, accounting for more than 2 million new cases diagnosed each year9. More people die of lung cancer every year than any other cancer9. There are two main types of lung cancer—small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC)10. NSCLC accounts for approximately 85% of lung cancer diagnoses11, and 30-55% of patients with early NSCLC develop recurrence despite resection12.

Novartis is committed to working with the scientific and medical communities to reimagine the treatment of lung cancer and pursue advances in medicine that could extend the survival of people living with lung cancer. Novartis is developing experimental therapies that block cancer growth; learning more about ways to activate the body’s immune system; increasing understanding of the relationship between chronic inflammation and tumor growth and progression; and exploring the potential for advanced nuclear medicine to fight the disease.

On August 15, 2022 Nuvo Pharmaceuticals Inc. (TSX:MRV; OTCQX:MRVFF) d/b/a Miravo Healthcare (Miravo or the Company), a Canadian-focused healthcare company with global reach and a diversified portfolio of commercial products, reported its financial and operational results for the three and six months ended June 30, 2022 (Press release, Nuvo Pharmaceuticals, AUG 15, 2022, View Source [SID1234618374]). For further details on the results, please refer to Miravo’s Management, Discussion and Analysis (MD&A) and Condensed Consolidated Interim Financial Statements for the three and six months ended June 30, 2022, which are available on the Company’s website (www.miravohealthcare.com). All figures are in Canadian dollars, unless otherwise noted.

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Key Developments

Three months ended June 30, 2022 include the following:

Total revenue was $20.9 million, an increase of 6% compared to $19.8 million for the three months ended June 30, 2021. Adjusted total revenue(1) was $23.3 million, an increase of 17% compared to $19.9 million for the three months ended June 30, 2021.
Net income was $18.3 million compared to net income of $9.1 million for the three months ended June 30, 2021. Adjusted EBITDA(1) was $10.3 million, an increase of 40% compared to $7.4 million for the three months ended June 30, 2021.
Revenue related to the Blexten franchise, Cambia and Suvexx was $12.8 million, an increase of 34% compared to revenue of $9.5 million for the three months ended June 30, 2021. Total Canadian prescriptions of Blexten, Cambia and Suvexx increased by 17%, 6% and 85%, respectively compared to the three months ended June 30, 2021.
The Company repaid $3.2 million (US$2.5 million) of the Amortization Loan to Deerfield Management Company, L.P. (Deerfield).
As at June 30, 2022, cash and cash equivalents were $28.6 million.
Six months ended June 30, 2022 include the following:

Total revenue was $36.6 million, an increase of 7% compared to $34.2 million for the six months ended June 30, 2021. Adjusted total revenue(1) was $39.1 million, an increase of 13% compared to $34.4 million for the six months ended June 30, 2021.
Net income was $20.3 million compared to net loss of $8.8 million for the six months ended June 30, 2021. Adjusted EBITDA(1) was $13.4 million, an increase of 15% compared to $11.7 million for the six months ended June 30, 2021.
Revenue related to the Blexten franchise, Cambia and Suvexx was $20.9 million, an increase of 36% compared to revenue of $15.4 million for the six months ended June 30, 2021. Total Canadian prescriptions of Blexten, Cambia and Suvexx increased by 18%, 4% and 100%, respectively compared to the six months ended June 30, 2021.
The Company repaid $6.7 million (US$5.3 million) of the Amortization Loan to Deerfield.
(1)

Non-IFRS financial measure. These measures are not recognized under IFRS and do not have standardized meanings prescribed by IFRS. See the Non-IFRS Measures section for definitions, reconciliations and the basis of presentation of the Company’s non-IFRS measures.

Business Update

In August 2022, Miravo announced the appointment of Anthony Snow to its Board of Directors. Mr. Snow has over twenty years of experience investing in and advising public and private companies. Mr. Snow is currently the President and Co-Portfolio Manager of Red Oak Partners, LLC. He also serves as President and a director of CBA Florida, Inc. (previously known as Cord Blood America, Inc.). Prior to joining Red Oak Partners, Mr. Snow worked at Soros Fund Management where he was part of a two-person team that managed a global long/short equity portfolio. Prior to Soros, he also focused on global equities at both Ardea Capital Management, as part of the founding team, and Wyper Capital Management. Previously, Mr. Snow was an Associate at private equity firm Lindsay Goldberg. Mr. Snow began his career at Merrill Lynch & Co. as an Analyst in the Mergers & Acquisitions group. Mr. Snow received a B.B.A. with high distinction from the University of Michigan, concentrating in finance and accounting, and an M.B.A. from Harvard Business School.
In August 2022, Miravo’s U.S. partner for Pennsaid 2% announced it would be winding down the business segment that currently promotes and sells Pennsaid 2% in the U.S. in response to the market erosion resulting from an at-risk launch of a generic version of Pennsaid 2% in May 2022. The Company has conducted a thorough evaluation of its manufacturing operations based in Varennes, Québec, where it manufactures Pennsaid 2%, and has determined that its continued operation of its manufacturing facility is no longer viable as a result of this lost revenue stream. Miravo is exploring strategic alternatives to monetize its manufacturing facility and related intellectual property, while winding down its manufacturing operations. Miravo estimates that prior to any recoveries, its restructuring charges related to the wind-down of its manufacturing operations will be $2.0 million – $2.5 million. The Company anticipates that a wind-down of its manufacturing operations may take 9 – 12 months, depending on various factors, some of which are beyond the Company’s control.
In June 2022, Miravo’s European partner, Orion Corporation (Orion) received marketing authorization for Suvexx packaged in bottles in Denmark, Estonia, Finland, Hungary, Lithuania, Latvia and Sweden (collectively the EU Markets). Orion intends to launch Suvexx in the EU Markets throughout 2023 once marketing authorization for a blister pack format has been obtained in each jurisdiction.
In May 2022, Miravo filed U.S., Canadian, European and PCT patent applications for a reformulated and improved version of Resultz. This new formulation maintains the original treatment claims, but is now enhanced with a 100% effectiveness claim for killing nits (the lice eggs) in addition to head lice. The Company believes this enhanced efficacy against nits adds value to existing Resultz partners, as well as other companies active in the head lice category globally who may be interested in licensing the technology. The Company commenced its partnering process for this new intellectual property during Q2 2022. Additional basic development work is anticipated to be conducted to support the new product.
"Our second quarter was marked by a number of notable events. First, this was the first time our Adjusted EBITDA was more than $10 million in a quarter. This financial milestone was driven by the continued growth of our Commercial Business segment and the continued prescription and sales growth of our promoted products Blexten, Cambia and Suvexx. Second, our European partner, Orion, received marketing authorizations for Suvexx in 7 of the 9 territories they have licensed, and we anticipate the final two authorizations to be obtained in the coming months. This marks the first ex-North American approvals for Suvexx and is a testament to the strength of the regulatory submission prepared by our scientific affairs team. We look forward to a commercial launch of Suvexx in Europe during the first half of 2023," said Jesse Ledger, Miravo’s President & CEO.

Mr. Ledger went on to state, "We remain committed to enhancing profitability and cash flow generation while advancing our strategic growth objectives. After careful review of the cost structure and contribution provided by operating an under-utilized manufacturing facility and considering the recent entry of generic Pennsaid 2% in the U.S., we concluded that the continued operation of our manufacturing facility does not align with our strategic growth objectives. Exiting our involvement with direct manufacturing will improve our gross profit margins and better focus the Company’s efforts and resources on advancing the key growth engines of our business: our Commercial Business and our international Licensing and Royalty Business segments. On behalf of our entire Company, I would like to thank all the employees involved in our manufacturing operations for their significant contributions over the years. The in-house development of both the Pennsaid 1.5% and Pennsaid 2% products and the launch of these products in various territories around the world would not have been possible without the hard work of this very resourceful and resilient team."

Second Quarter 2022 Financial Results
Adjusted total revenue was $23.3 million and $39.1 million for the three and six months ended June 30, 2022 compared to $19.9 million and $34.5 million for the three and six months ended June 30, 2021. The $3.4 million increase in adjusted total revenue in the current quarter was primarily attributable to a $2.4 million increase in revenue from the Commercial Business segment and an increase of $2.3 million (US$1.8 million) for Yosprala-related milestone revenue billed to the Company’s Japanese licensee, offset by a decrease of $1.3 million of revenue from the Production and Service Business segment.

Revenue attributable to the Commercial Business segment increased during the three months ended June 30, 2022 due to a $3.3 million increase in sales of the Company’s promoted products (Blexten, Cambia, Suvexx and Neovisc), offset by a $0.8 million decrease in sales of the Company’s mature products. Revenue attributable to the Commercial Business segment increased during the six months ended June 30, 2022 due to a $5.5 million increase in sales of the Company’s promoted products, slightly offset by a decrease in sales of the segment’s mature products.

The Production and Service Business segment revenue decreased during the three and six months ended June 30, 2022, primarily due to a decrease in the Company’s Pennsaid 2% product sales.

For the three months ended June 30, 2022, the $2.3 million increase in adjusted total revenue in the Licensing and Royalty Business segment was the result of an increase of $2.3 million (US$1.8 million) for Yosprala-related milestone revenue billed to the Company’s Japanese licensee.

Adjusted EBITDA was $10.3 million for the three months ended June 30, 2022 compared to $7.4 million for the comparative quarter. During the three months ended June 30, 2022, a $2.1 million increase in gross profit from the Commercial Business segment and a $2.2 million increase in amounts billed to customers for existing contract assets was offset by a $0.5 million increase in sales and marketing expenses and a $0.8 million decrease in gross profit contribution from the Company’s Production and Service Business segment.

Adjusted EBITDA was $13.4 million for the six months ended June 30, 2022 compared to $11.7 million for the six months ended June 30, 2021. During the six months ended June 30, 2022, a $3.0 million increase in gross profit from the Commercial Business segment and a $2.2 million increase in amounts billed to customers for existing contract assets was offset by a $0.3 million decrease in the contribution from the License and Royalty Business segment, a $1.6 million decrease in gross profit contribution from the Production and Service Business segment, a $0.5 million increase in sales and marketing expenses and a $1.0 million increase in general and administrative expenses.

Non-IFRS Measures
The Company discloses non-IFRS financial measures (adjusted total revenue, adjusted EBITDA, and cash value of loans) and non-IFRS ratios (adjusted EBITDA per share and net debt leverage ratio) that are not recognized under and do not have standardized meanings prescribed by IFRS. Accordingly, such measures are not necessarily comparable and may not have been calculated in the same way as similarly named financial measures presented by other companies. These measures should be considered as supplemental in nature and not as a substitute for related financial information prepared in accordance with IFRS. The Company believes that shareholders, investment analysts and other readers find such measures and ratios helpful in understanding and assessing the Company’s financial performance. We utilize these measures in managing our business, including as means of performance measurement, cash management, debt compliance and assessing leverage and borrowing capacity. Because non-IFRS financial measures and non-IFRS ratios do not have standardized meanings prescribed under IFRS, securities regulations require that such measures be clearly defined, identified, and for non-IFRS financial measures, reconciled to their nearest IFRS measure. The applicable definition, calculation and reconciliation of each such measure used in this press release is provided below.

Adjusted Total Revenue
The Company defines adjusted total revenue as total revenue, plus amounts billed to customers for existing contract assets, less revenue recognized upon recognition of a contract asset. Management believes adjusted total revenue is a useful supplemental measure to determine the Company’s ability to generate cash from its customer contracts used to fund its operations.

The following is a summary of how adjusted total revenue is calculated, reconciled to the nearest IFRS measure

Adjusted EBITDA
EBITDA refers to net income (loss) determined in accordance with IFRS, before depreciation and amortization, net interest expense (income) and income tax expense (recovery). The Company defines adjusted EBITDA as EBITDA, plus amounts billed to customers for existing contract assets, inventory step-up expenses, stock-based compensation expense, loss on fair value of derivative liabilities, loss on fair value of contingent and variable consideration, impairment loss, foreign currency loss, other losses less revenue recognized upon recognition of a contract asset, stock-based compensation recovery, gain on fair value of derivative liabilities, gain on fair value of contingent and variable consideration, impairment recovery, foreign currency gain and other income. Management believes adjusted EBITDA is a useful supplemental measure to determine the Company’s ability to generate cash available for working capital, capital expenditures, debt repayments, interest expense and income taxes.

(1) Income tax expense for the three and six months ended June 30, 2022, includes $1.7 million and $2.0 million for deferred income tax due to the utilization of loss carry forwards that were previously recognized [$1.1 million and $1.4 million for the three and six months ended June 30, 2021]

(2) The Company’s derivative liabilities are measured at fair value through profit or loss at each reporting date. As a result of the decrease in the share price in the current quarter and a decrease in the volatility of the Company’s shares, amongst other inputs, the value of the Company’s derivative liabilities decreased and the Company recognized net non-cash gains of $19.3 million on the change in fair value of derivative liabilities for the three months ended June 30, 2022 [$6.9 million net non-cash gains for the three months ended June 30, 2021]. During the six months ended June 30, 2022 as a result of the decrease in the share price and a decrease in the volatility of the Company’s shares, amongst other inputs, the value of the Company’s derivative liabilities decreased and the Company recognized net non-cash gains of $22.6 million on the change in fair value of derivative liabilities [$11.5 million net non-cash loss for the six months ended June 30, 2021].

(3) In the three and six months ended June 30, 2022, the impairment loss of $0.9 million represented a $46 write-down of certain mature intangible assets and $0.3 million for Resultz goodwill in the Commercial Business segment and a $0.6 million write-down of certain intangible assets in the Licensing and Royalty Business segment.

Management to Host Conference Call/Webcast
Management will host a conference call to discuss the results today (Monday, August 15, 2022) at 11:00 a.m. ET. To participate in the conference call, please dial (416) 764-8646 or 1 (888) 396-8049 / Conference ID: 64633261. Please call in 15 minutes prior to the call to secure a line. You will be put on hold until the conference call begins.

A live audio webcast and replay webcast of the conference call will be available through:

View Source

On August 15, 2022 RenovoRx, Inc. (Nasdaq: RNXT), a biopharmaceutical company focused on the localized treatment of difficult-to-treat solid tumors through its proprietary RenovoRx Trans-Arterial Micro-Perfusion (RenovoTAMPTM) therapy platform, reported its financial results for the quarter ended June 30, 2022 (Press release, Renovorx, AUG 15, 2022, View Source [SID1234618373]).

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"Over the past decade, our team has developed and refined a unique therapy platform for difficult-to-treat solid tumors by localizing and targeting chemotherapy to minimize systemic side-effects, improve quality of life, and potentially extend life. We started with one of the most aggressive tumor types – pancreatic cancer," said Shaun Bagai, CEO of RenovoRx. "Innovation and success in treatment options for pancreatic cancer are sparse. Through our Phase 3 TIGeR-PaC clinical study, we have the potential to revolutionize the treatment of pancreatic cancer, shifting patients’ focus from coping with the treatment and its side-effects, to enjoying more time with their family and loved ones."

Mr. Bagai continued, "The most significant milestone to date for our therapy platform is expected during the fourth quarter of this year: the first prospective interim analysis for this pivotal Phase 3 study. This data will provide us with important insight into the potential for RenovoTAMP at a juncture in the trial equivalent to a robust Phase 2 oncology study, with the rigor of having it randomized. We have also submitted a protocol for a Phase 2/3 clinical trial in extrahepatic (or outside the liver) cholangiocarcinoma to FDA – advancing our therapy platform to other indications. We plan to commence the study and enroll the first patient during the fourth quarter of 2022, assuming the protocol is acceptable to FDA."

"To support our growth and infrastructure, in July, we added James Ahlers, an accomplished life sciences finance leader, as Chief Financial Officer, and expanded our finance team with the addition of Ron Kocak, a seasoned financial reporting and accounting professional, as Vice President & Controller," said Mr. Bagai. "With the addition of James and Ron, we continue to build the foundation to support the evolution of our clinical pipeline."

Financial Highlights for the Quarter Ended June 30, 2022

As of June 30, 2022, the Company had cash and cash equivalents and short-term marketable securities of $10.8 million.
Research and development expenses were $1.4 million for the quarter ended June 30, 2022, compared to $0.5 million for the quarter ended June 30, 2021. The $0.9 million increase was primarily due to a $0.5 million increase in preclinical research and development and regulatory expenses, and a $0.2 million increase in clinical consulting to support the ongoing Phase 3 trial.
General and administrative expenses were $1.2 million for the quarter ended June 30, 2022, compared to $0.3 million for the quarter ended June 30, 2021. This $0.9 million increase was primarily due to a $0.4 million increase in professional and consulting services related to post-IPO support, and a $0.2 million increase for higher personnel related costs.
Net loss was $2.6 million for the quarter ended June 30, 2022, compared to net loss of $1.3 million for quarter ended June 30, 2021.
As of June 30, 2022, the Company had 9,066,863 common shares outstanding.
About the Phase 3 TIGeR-PaC Clinical Trial

TIGeR-PaC is a randomized multi-center Phase 3 study using RenovoRx’s innovative therapy platform, RenovoTAMPTM (RenovoRx Trans-Arterial Micro-Perfusion). The study is evaluating the Company’s first product candidate, RenovoGemTM, to treat locally advanced pancreatic cancer (LAPC) through the intra-arterial delivery of gemcitabine (FDA-approved chemotherapy). The study has a primary endpoint of overall survival and several secondary endpoints, including quality of life.

TIGeR-PaC is currently enrolling unresectable LAPC patients at several sites across the US. To learn more about the study and the participating clinical trial sites, visit View Source

On August 15, 2022 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease through the inhibition of SEMA4D, reported financial results for the second quarter ended June 30, 2022 and provided a corporate update on key events since April, 2022 (the last 5 months) (Press release, Vaccinex, AUG 15, 2022, View Source [SID1234618372]).

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"Vaccinex has made excellent progress this year in its clinical programs to evaluate the potential use of our proprietary SEMA4D inhibitor, pepinemab, in oncology and neurodegenerative disease," said Maurice Zauderer, Ph.D., President and Chief Executive Officer of Vaccinex. "We have previously reported several promising responses in the open label, Phase 1b/2 KEYNOTE oncology B84 trial to evaluate pepinemab and KEYTRUDA (pembrolizumab, a PD-1 inhibitor) in patients with advanced recurrent or metastatic head and neck cancer (R/M HNSCC). An expanded interim analysis of study data is planned in Q4 2022. With continued positive results, we are hopeful that this combination could be a promising treatment option for patients with R/M HNSCC who have limited treatment choices,"

Dr. Zauderer continued, "Vaccinex is also very pleased to announce the recent publication of the results of the Phase 2 SIGNAL study of pepinemab in Huntington’s Disease (HD) in Nature Medicine. This is an important milestone for the neurodegenerative disease program. While, as previously reported, the study did not meet its pre-specified primary endpoints, we believe the results provide compelling signals of cognitive benefit, evidenced by multiple exploratory and post-hoc efficacy assessments, and support further development in HD and other neurodegenerative indications including Alzheimer’s disease. Importantly, treatment resulted in a statistically significant increase in brain metabolic activity (measured by FDG-PET) in 15 of 26 brain regions of patients with Early Manifest HD. Multiple studies have shown that reduced FDG-PET signal correlates with cognitive decline and clinical progression in Alzheimer’s Disease (AD). Based on these observations, we are excited to have initiated the randomized, double-blind, Phase 1/2a SIGNAL-AD study in 40 subjects with early AD. We expect this study will complete enrollment by Q1 2023."

Dr. Zauderer continued, "Vaccinex’s clinical programs in oncology and neurodegenerative disease are poised to yield important data over the next twelve to eighteen months. We look forward to updating the clinical community and investors on our progress and thank all of the patients, caregivers, and participating clinical sites and investigators for their continued support of these promising clinical programs."

Recent Milestones:

Oncology:

Initial data from the ongoing Phase 1b/2 KEYNOTE-B84 study in R/M HNSCC was published in the ASCO (Free ASCO Whitepaper) proceedings (May 26, 2022) and at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (AACR 2022) (April 11, 2022)
Neurodegenerative Disease:

Results of the Phase 2 SIGNAL-HD study were published in Nature Medicine (Feigin et al., August 2022)
Posters related to the ongoing Phase 1/2a SIGNAL-AD study was presented at the 2022 American Academy of Neurology (AAN) Annual Meeting (April 2022) and at the Alzheimer’s Association International Conference (July 2022)
ActivMAb Platform Technology:

Advances in use of the ActivMAb platform to select antibodies against difficult multi-pass membrane targets (e.g. GPCR and ion channels) were reported at the PEGS Boston Conference & Expo (May 2022)
The company is engaged in multiple biopharmaceutical collaborations employing this technology for drug discovery. An IND for the first such clinical product is expected to be filed in 2023.
Upcoming Milestones:

Head and Neck Cancer: Phase 1b/2 KEYNOTE B84 study

Interim analysis: Expected Q4 2022
Completion of enrollment: Expected by H1 2023
Alzheimer’s Disease: Phase 1/2a SIGNAL-AD study

Completion of enrollment: Expected by Q1 2023
Topline data: Expected H2 2023
Pepinemab Program Overview:

Oncology: Head and Neck Cancer

Rationale: Multiple prior studies suggest that inhibition of SEMA4D increases immune infiltration and alters the balance of cytotoxic and immunosuppressive cells in the tumor microenvironment. As SEMA4D is highly expressed in head and neck cancer, there is strong rationale for development in this indication.

Status: Enrollment is underway in the Phase 1b/2 KEYNOTE B84 clinical study evaluating pepinemab in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in recurrent or metastatic head and neck cancer. The study was designed to enroll up to 65 subjects across 18 U.S. trial sites to assess safety and efficacy of the combination pepinemab/pembrolizumab. Key endpoints of the study will include objective response, duration of response and overall survival.

Vaccinex has exclusive global commercial and development rights to pepinemab and is a sponsor of the KEYNOTE-B84 study which is being performed in collaboration with Merck Sharp & Dohme Corp, a subsidiary of Merck and Co, Inc. Kenilworth, NJ, USA. KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co. Inc., Kenilworth, NJ, USA.

Other Trials. Pepinemab is also being evaluated in multiple investigator-sponsored trials (ISTs) being conducted by the Winship Cancer Institute of Emory University to evaluate pepinemab in combination with checkpoint inhibitors in "Window of Opportunity" biomarker studies of head and neck cancer and melanoma.

Neurodegenerative Disease:

Rationale: SEMA4D appears to be upregulated on damaged neurons in the brains of people with either Huntington’s Disease (HD) or Alzheimer’s Disease (AD), leading to physiological changes in the structure and function of the major inflammatory cells of the brain, astrocytes and microglia, that express receptors for SEMA4D. Preclinical studies conducted by Vaccinex have shown that pepinemab can inhibit SEMA4D and reverse neuroinflammation and restore normal functions associated with astrocytes.

Alzheimer’s Disease: The Phase 1/2a SIGNAL-AD Study. Enrollment is underway in this randomized, double-blind, placebo-controlled, multi-center safety and biomarker study of pepinemab in early AD. The study is planned to enroll 40 subjects across 15 U.S. trial sites. The trial is being funded in part by the Alzheimer’s Drug Discovery Foundation and by the Alzheimer’s Association under their 2020 Part the Cloud Program.

Financial Results for the Three Months Ended June 30, 2022:
Cash and Cash Equivalents and Marketable Securities. Cash and cash equivalents and marketable securities on June 30, 2022 were $11.4 million, as compared to $8.6 million as of December 31, 2021.

Research and Development Expenses. Research and development expenses for the quarter ended June 30, 2021 were $3.8 million as compared to $4.1 million for the comparable period in 2021.

The slight reduction in Research and Development expenses in the period ended June 30, 2022 compared to 2021 is primarily attributed to reduced clinical trial costs as a result of the completion of the CLASSICAL-Lung and SIGNAL studies phase 2 trials, partially offset by setup expenses for the SIGNAL-AD and HNSCC KEYNOTE-B84 studies.

General and Administrative Expenses. General and administrative expenses for the quarter ended June 30, 2022 were $1.6 million as compared to $1.6 million for the comparable period in 2021.

Essentially flat level of general and administrative expenses reflects careful cost control measures.

Comprehensive loss/Net loss per share. The Comprehensive Loss and Net loss per share for the quarter ended June 30, 2022 was $5.4 million and $(0.13) compared to $6.0 million and $(0.21) for the comparable period in 2021.

Full financial tables are included below. For further details on Vaccinex’s financials, refer to its Form 10-Q filed August 15, 2022 with the S.E.C.

About Pepinemab
Pepinemab is a humanized IgG4 monoclonal antibody that inhibits SEMA4D, which regulates the actin cytoskeleton of cells that plays an important role in tumor immunity and in inflammatory reactions in the brain. Preclinical and clinical data show that by preventing inflammatory reactivity pepinemab during disease progression, pepinemab preserves normal function of astrocytes and microglia, two types of glial cells that play a crucial role in the development and maintenance of neurons in the brain. Additional data show that pepinemab promotes infiltration and activation of dendritic cells and CD8+ T-cells and reverses immunosuppression within the tumor microenvironment. Pepinemab is being evaluated in several studies in oncology and neurodegenerative disease.

About ActivMAb
Vaccinex has developed a proprietary mammalian cell-based antibody discovery platform with unique multi-pass membrane target capabilities. The ActivMAb technology now has four main applications: complex membrane antigen presentation, antibody or antigen discovery, and protein optimization. Vaccinex has entered into an antibody license with Surface Oncology (Cambridge, MA) and into Material Transfer Agreements for drug discovery or process development with two major pharma utilizing this technology.

On August 15, 2022 AIM ImmunoTech Inc. (NYSE: American AIM) ("AIM" or the "Company"), an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases, including COVID-19, the disease caused by the SARS-CoV-2 virus, reported its financial results for the second quarter 2022 and provided a business update (Press release, AIM ImmunoTech, AUG 15, 2022, View Source [SID1234618371]).

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"As the second quarter results demonstrate, we believe we are closer than ever to unlocking Ampligen’s commercial potential," commented Thomas K. Equels, Chief Executive Officer of AIM. "Notably, we have recently seen the publication of positive clinical data regarding several unmet medical needs in highly lethal malignancies. We have a strong clinical development program, sufficient operating capital and enough Ampligen to support new clinical trials. Despite ongoing headwinds for the biotech sector, we believe we are well-positioned to take advantage of the value-driving catalysts across our pipeline and look forward to generating near- and long-term shareholder value. Finally, we remain focused on delivering sufficient data on Ampligen in oncology, which we believe could contribute to significant value creation."

Recent Highlights

Reported positive follow-on patient data from a Single-Center Named Patient Program evaluating Ampligen as maintenance therapy for advanced pancreatic cancer indicating additional progression-free and overall survival over previously published data.
Provided a summary of Ampligen data supporting synergistic potential with checkpoint blockade therapies. See: "Combined loco-regional and systemic, triple agent chemoimmunotherapy increases biomarkers of T cell chemotaxis in ovarian cancer."
Provided an update on advancement of Ampligen clinical development program for the treatment of pancreatic cancer and announced the engagement of Amarex Clinical Research LLC, a world-renowned CRO, to conduct the upcoming Phase 2 study.
Reported positive data from Phase 2a study evaluating Ampligen as a component of a chemokine modulatory (CKM) regimen for the treatment of colorectal cancer metastatic to the liver.
Reported positive data from a Phase 1 study evaluating Ampligen for the treatment of stage 4 metastatic triple negative breast cancer.
Reported positive preliminary pilot study data from its ongoing Expanded Access Program (AMP-511) evaluating Ampligen as a therapeutic for "Long COVID." The preliminary data from this uncontrolled clinical trial found that patients reported significant improvements in fatigue symptoms after treatment with Ampligen compared to baseline, which the investigators considered a clinically significant decrease in fatigue-related measures. Based on these early results, AIM is working to move forward with a Phase 2 controlled trial.
Secured new state-of-the-art facility for product development and testing to advance research and development of Ampligen to treat multiple types of cancers, immune disorders, and viral diseases.
Bolstered intellectual property portfolio for Ampligen with issuance of new Netherlands utility patent covering Ampligen and other AIM-developed dsRNA products for use in COVID-19 treatment or prevention.
Clinical Program Update

Ampligen (rintatolimod): dsRNA being developed for globally important cancers, viral diseases and disorders of the immune system

Ampligen has demonstrated in the clinic the potential for standalone efficacy in a number of solid tumors. Additionally, Ampligen has shown therapeutic synergy with checkpoint inhibitors, including increasing survival rates and efficacy, in the treatment of animal tumors when used in combination with checkpoint blockade therapies. The first detection of Ampligen’s synergistic potential with checkpoint blockade therapeutics was witnessed in pre-clinical mouse models of melanoma and pancreatic cancers. Additionally, the Company now has data from two clinical studies – in advanced recurrent ovarian cancer and triple negative breast cancer – that indicate that the drug may have similar anti-tumor activity in humans.

Ampligen is being evaluated as a combinational therapy for the treatment of a variety of solid tumor types in multiple clinical trials – both underway and planned – at major cancer research centers around the U.S.. Ampligen is also being used to treat pancreatic cancer patients in an Early Access Program (EAP) approved by the Inspectorate of Healthcare in the Netherlands at Erasmus Medical Center.

Immuno-Therapy Targeting Multiple Cancers with High Unmet Need

Locally Advanced Pancreatic Cancer ("LAPC") – The Company recently reported new, positive data following evaluation of the initial data reported from the single-center named patient program at Erasmus for both metastatic and LAPC patient populations, analyzing the subset of patients with LAPC. While the predominance of the data collected by Erasmus is in metastatic cancer and those data show high statistical significance, a small cohort of five (5) LAPC patients also exhibited marked improvement with the Ampligen maintenance therapy following FOLFIRINOX. The overall survival from the start of FOLFIRINOX therapy of two (2) of the patients was 34 and 43 months and one patient was still surviving at the last reported checkup in April 2022 at 54 months. The Company’s Phase 2a study Investigational New Drug ("IND") application was cleared by the U.S. Food and Drug Administration ("FDA") and is on track to commence in Q3 2022. The study will compare the efficacy of Ampligen following FOLFIRINOX versus a control group that previously received FOLFIRINOX but no Ampligen for subjects with locally advanced pancreatic adenocarcinoma. Approximately 90 subjects expected to be enrolled across up to 30 centers in the U.S. and Europe.
Advanced Recurrent Ovarian Cancer – Phase 1/2 study of intraperitoneal chemo-immunotherapy in advanced recurrent ovarian cancer. Phase 1 portion was completed. The Phase 2 portion of the study is planned to be conducted in the future. ClinicalTrials.gov: NCT02432378
Advanced Recurrent Ovarian Cancer – A follow-up Phase 2 study of advanced recurrent ovarian cancer using cisplatin and pembrolizumab, plus Ampligen; up to 45 patients to be enrolled; numerous patients have commenced treatment. ClinicalTrials.gov: NCT03734692
Stage 4 Colorectal Cancer Metastatic to the Liver – Phase 2a study of Ampligen as a component of a chemokine modulatory regimen on colorectal cancer metastatic to liver was completed and met primary endpoint, evidenced by increased CD8a expression post-treatment (p=0.046).; 15 patients were treated and 12 patients were evaluable for the primary endpoint. Data suggest that chemokine modulatory (CKM) regimen with Ampligen may be useful to enhance effectiveness of immunotherapies. The data from the Phase 2a study was presented in April 2022 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022. ClinicalTrials.gov: NCT03403634
Stage 4 Metastatic Triple Negative Breast Cancer – Phase 1 study of metastatic triple-negative breast cancer using CKM therapy, including Ampligen and pembrolizumab, successfully met primary endpoint. Positive data from this proof-of-concept study demonstrate that short-term systemic CKM followed by pembrolizumab is well-tolerated and selectively enhances local cytotoxic T-lymphocyte (CTL) infiltration in the tumor microenvironment (TME). The data from the Phase 1 study was presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 in April 2022. ClinicalTrials.gov: NCT03599453
Early-Stage Prostate Cancer – Phase 2 study investigating the effectiveness and safety of aspirin and Ampligen with or without interferon-alpha 2b (Intron A) compared to no drug treatments in a randomized three-arm study of patients with prostate cancer before undergoing radical prostatectomy. Patient enrollment has been initiated in this study designed for up to 45 patients. ClinicalTrials.gov: NCT03899987
Early-Stage Triple Negative Breast Cancer – Phase 1 study of chemokine modulation plus neoadjuvant chemotherapy in patients with early-stage triple negative breast cancer has received FDA authorization. The objective of this study is to evaluate the safety and tolerability of a combination of Ampligen and celecoxib with or without Intron A, when given along with chemotherapy. The goal of this approach is to increase survival. Investigators are currently analyzing data. ClinicalTrials.gov: NCT04081389
Refractory Melanoma – Phase 2 study that will evaluate polarized dendritic cell vaccine, interferon alpha-2, Ampligen and celecoxib for the treatment of HLA-A2+ refractory melanoma at Roswell Park. Up to 24 patients to be enrolled. ClinicalTrials.gov: NCT04093323
Advanced Ovarian Cancer – AIM plans to develop a Phase 2 Cisplatin Resistant Advanced Recurrent Ovarian Cancer Clinical Study utilizing Ampligen at the University of Pittsburgh.
Broad-Spectrum Immune System Response Against SARS-CoV-2 (COVID-19)

Previous animal studies yielded positive results utilizing Ampligen in Western Equine Encephalitis Virus, Ebola, Vaccinia Virus (which is used in the manufacture of smallpox vaccine) and SARS-CoV-1. The Company has conducted experiments in SARS-CoV-2 showing Ampligen has a powerful impact on viral replication. The prior studies of Ampligen in SARS-CoV-1 animal experimentation may predict similar protective effects against SARS-CoV-2. AIM is currently evaluating the safety and effectiveness of intravenous Ampligen to reduce replication of SARS-CoV-2 virus from upper airway in patients in an ongoing Phase 1/2 study for the treatment of COVID-19 cancer patients. The Company plans to conduct an intranasal study of Ampligen to potentially enhance and expand natural immunity.

The FDA has authorized Ampligen in a clinical trial of patients with COVID-19 who have a pre-existing cancer. That Phase 1/2a study utilizing Ampligen is underway in the investigator-sponsored Phase 2 trial at the Roswell Park Comprehensive Cancer Center. ClinicalTrials.gov: NCT04379518

Immune System Disorders (ISD): Myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) / COVID-19 Long Hauler

The Company is currently sponsoring an ongoing, FDA-authorized AMP-511 (See: ClinicalTrials.gov: NCT00215813) expanded access program (EAP) for ME/CFS patients in the United States. AIM has enrolled four post-COVID patients with new onset ME/CFS following acute COVID-19. Following at least 12 weeks of Ampligen treatment, each of these four patients indicated they had experienced a reduction in fatigue, as measured via Patient-Reported Outcomes questionnaires. A statistical analysis of these data indicated that the decrease in fatigue compared to baseline was statistically significant (p<0.002), despite the small number of patients. Based in part on these early positive data, AIM is working toward filing an IND application with the FDA for a Phase 2 study of Ampligen for the treatment of post-COVID conditions.

Recent Ampligen Data Publications

Presented data at the prestigious American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022:
Negative impact of paclitaxel on human breast tumor microenvironment and its reversal by the combination of interferon-α with TLR3 agonist rintatolimod
Initial results of a phase II study evaluating a chemokine-modulatory (CKM) regimen in patients with colorectal cancer metastatic to the liver
Systemic Rintatolimod and Interferon-α2b selectively reprogram local tumor microenvironment in patients with metastatic triple negative breast cancer for enhanced influx of cytotoxic T-lymphocytes but not regulatory T-cells
Combined loco-regional and systemic, triple agent chemoimmunotherapy increases biomarkers of T cell chemotaxis in ovarian cancer
Presented Rintatolimod: a potential therapeutic molecule for human pancreatic cancer cells expressing Toll-Like Receptor 3 at the 15th Annual International Hepato-Pancreato-Biliary Association (IHPBA) World Congress.
Summary of Financial Highlights for Second Quarter 2022

As of June 30, 2022, AIM reported cash and cash equivalents of $34.5 million, compared to $32.1 million as of December 31, 2021.
Research and development expenses for the three months ended June 30, 2022 were $2.5 million, compared to $XX1.3 million for the same period in 2021.
General and administrative expenses were $2.2 million for the three months ended June 30, 2022, compared to $2.1 million for the same period in 2021.
The net loss from operations for the three months June 30, 2022 was $4.9 million, or $0.10 per share, compared to $5.9 million, or $0.12 per share, for the three months ended June 30, 2021.