On May 17, 2022 Idera Pharmaceuticals, Inc. ("Idera," "we," and "our") (Nasdaq: IDRA) reported that positive interim results from Amsterdam UMC, Vrije Universiteit Amsterdam, the Netherlands, regarding its investigator-sponsored trial, INTRIM 1, involving tilsotolimod, Idera’s synthetic Toll-like receptor 9 agonist. Based on these results, the trial has been stopped early (Press release, Idera Pharmaceuticals, MAY 17, 2022, View Source [SID1234614737]).

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INTRIM 1 is a randomized, double-blind, placebo-controlled Phase 2 trial among patients with localized, excised melanoma (pathological tumor stage 3-4) with no regional metastases detected and no evidence of distant metastasis. The trial involved a single, intradermal injection of 8 mg tilsotolimod or saline placebo given at the primary tumor excision site, followed by re-excision and sentinel lymph node (SLN) biopsy 7-10 days later. Noting that there were more patients with ulcerated lesions in the placebo arm compared to the tilsotolimod arm, topline interim results of the respective SLN-positivity rates showed a 70% lower SLN+ rate among patients injected with tilsotolimod as compared to those injected with placebo; the placebo SLN+ rate was in the mid-40%s. Statistical significance exceeded the pre-specified p-value of 0.008. Adverse reactions included injection site reactions, malaise, fever and flu-like symptoms.

"This is an exciting result from tilsotolimod, and we are pleased for the patients and their families to whom it offers hope for the future," said Vincent Milano, Idera’s Chief Executive Officer. "These results, together with data supporting tilsotolimod’s mechanism of action and encouraging safety profile from across the array of earlier pre-clinical and clinical work, reinforce the potential of tilsotolimod to offer benefit to patients with certain cancers. As a result, we plan to actively pursue a strategic partnership for tilsotolimod so that its full potential for patients may continue to be explored."

This interim result validates previously reported results from INTRIM 1 that showed immune activation, including elevated frequencies of key dendritic cells, in early analysis by flow cytometry of the SLN biopsies. The trial will continue to relapse-free survival (RFS) and overall survival (OS) at 5 and 10 years after SLN biopsy.

"Currently, there are limited adjuvant treatments available to improve survival after surgical excision of a primary melanoma," said Prof. Tanja de Gruijl of Amsterdam UMC. "We are delighted with the results we have seen in this study, which suggest that tilsotolimod administered at the excision site lowers the extent of tumor-positive lymph nodes and, if it improves overall survival, offers early melanoma patients a potential new treatment option." Amsterdam UMC investigators plan to present the full data set at an upcoming medical meeting.

About Melanoma

Melanoma is a cancer that begins in a type of skin cell called melanocytes. While melanoma is the least common type of skin cancer, it has a poor prognosis when not detected and treated early. Early stages of melanoma are treated by removing, or excising, the tumor surgically. However, even after surgery, nearly one-third of all melanoma patients will experience disease recurrence and most relapses eventually will progress to metastatic disease. When regional lymphatic metastases are present, the 10-year survival rate is 68% – 24%. SLN biopsy is a useful prognostic tool for the assessment of melanoma relapse and mortality risk. Ulceration is associated with more SLN positivity.

In many cases, additional cancer treatment, referred to as adjuvant therapy, is given after the primary excision of the melanoma. The goal of adjuvant therapy is to reduce the risk of melanoma returning and potentially metastasizing. The adjuvant market size for melanoma excision cases is over 1 million cases globally and about 300,000 in the US annually. As is the case in many forms of cancer, melanoma becomes more difficult to treat once the disease has spread, or metastasized, beyond the skin to other parts of the body.

About INTRIM 1

INTRIM 1 is a multi-center investigator-sponsored trial conducted by UMC Amsterdam among patients with pT3-4 cN0M0 melanoma. The study is a randomized, double-blind, placebo-controlled Phase 2 trial of a single, intradermal injection of 8 mg tilsotolimod or of saline placebo given at the primary tumor excision site 7-10 days prior to SLN biopsy.

INTRIM 1 is intended to examine the ability of tilsotolimod to induce loco-regional and systemic immune stimulation, and therefore, improve survival among the targeted patient population. Outcome measures of the trial include the following:

· Primary endpoint: rate of tumor positive SLN at the time of biopsy.
·Secondary endpoints:
·Immune response in the SLN and peripheral blood 7-10 days post-biopsy, as measured by frequency and activation state of lymph node resident (LNR) conventional dendritic cells (DC) and melanoma antigen-specific T cell responses in the SLN and peripheral blood.
·RFS, determined by the length of time from intradermal injection of tilsotolimod to first documentation of recurrence (RFS), measured at 5 and 10 years after SLN biopsy.
·OS at 5 and 10 years after SLN biopsy, determined by the length of time from intradermal injection of tilsotolimod to death from any cause.

While enrollment for the primary endpoint has been halted early as a result of the primary endpoint being met, the study will continue to its secondary endpoints. For more information on the INTRIM 1 trial, please visit www.ClinicalTrials.gov (NCT04126876).

About Tilsotolimod (IMO-2125)

Tilsotolimod is an investigational, synthetic Toll-like receptor 9 agonist. Tilsotolimod has been shown to promote both innate (Type-I IFN, antigen presentation) and adaptive (T cells) immune activation and may, in turn, contribute to tumor suppression and regression. For more information on tilsotolimod trials, please visit www.ClinicalTrials.gov.

On May 17, 2022 QSAM Biosciences Inc. (OTCQB: QSAM), a company developing next-generation therapeutic radiopharmaceuticals, including Samarium-153 DOTMP (CycloSam), for the treatment of bone cancer and related diseases, reported that financial results for the first quarter ended March 31, 2022, as filed with the SEC on May 16, 2022 in the Company’s Form 10-Q, and provides a corporate update (Press release, QSAM Biosciences, MAY 17, 2022, View Source [SID1234614736]).

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Recent Corporate Highlights:

●Dosed first patient in its Phase 1 clinical trial for CycloSam for the treatment of metastatic bone cancer in April. Completion of the first cohort of patients in the trial is expected this quarter. The Phase 1 study consists of 17 patients divided into four dose-escalating cohorts in an open-label trial to evaluate the safety, tolerability, dosimetry and preliminary efficacy of CycloSam.
●Advanced the onboarding process of three additional clinical trial sites for CycloSam; expects to report further definitive information and details over following months.
●Awarded Rare Pediatric Disease designation from the FDA for CycloSam in the treatment of osteosarcoma in children. This designation qualifies a future study for a transferable Priority Review Voucher that is inclusive of fast-track FDA review process.
●Added two distinguished healthcare industry veterans with significant finance, public company and operational experience to the Board of Directors .
●Postponed contemplated NASDAQ "uplisting" and public offering due to adverse market conditions.

"As previously highlighted, the recent dosing of our first patient with CycloSam was a milestone event for QSAM. We are very pleased with the progress we have made in under 24 months – advancing from a preclinical asset to a drug candidate treating patients with metastatic bone cancer, receiving both Orphan and Rare Pediatric Disease designations from the FDA, performing a successful single patient study at a world-renowned medical institution, and establishing our supply chain, manufacturing and executive team – efficiently utilizing under $2 million," stated Douglas R. Baum, CEO and co-founder of the Company.

"Given highly challenging market conditions, in particular for small cap biotechnology companies, we made the prudent decision in May to suspend our planned equity raise and NASDAQ uplisting. We did not believe the valuation we would have received at this time reflects the real intrinsic value of our technology and market opportunity. We are committed to bringing CycloSam to market, and this will require additional capital; but we are also committed to raising such capital in a manner that limits dilution and provides a clear path to increasing shareholder value. In line with this, management has agreed to defer over 50% of the cash component of our salaries so that our current capital and that which we raise in the short-term can primarily be used to advance our clinical trial and provide data that supports our strong belief that CycloSam can have a meaningful impact on children and adults suffering from bone cancer," added Baum.

Financial Results for the Quarter Ended March 31, 2022:

Net loss attributable to common stockholders for the quarter ended March 31, 2022 was $1,873,879, compared to approximately $4,956,458 for the quarter ended March 31, 2021.

Operating expenses were $1,770,109 for the three months ended March 31, 2022, as compared to $3,333,857 for the three months ended March 31, 2021. The majority of the $1,563,748 decrease in operating expenses was due to other income and expenses related to the loss on debt extinguishment of $744,505, loss on conversion of debt to equity of $390,067, and non-cash stock-based compensation expense related to stock issued for services, all in the 2021 period.

As of March 31, 2022, the Company had cash of approximately $872,066. The Company believes this is sufficient to support operations at the current pace into the third quarter of 2022. The Company expects to raise additional capital through equity or debt offerings in 2022.

As of May 16, 2022, the Company had 1,686,587 common shares outstanding. In the first quarter of 2022, the Company issued 34,219 shares of common stock to a service provider and a director for prior services, and to retire the remaining outstanding convertible debentures. At the end of the first quarter of 2022 there were 488,235 shares of common stock that could be issued upon the conversion of preferred stock, warrants and convertible debt, excluding employee stock options.

On May 17, 2022 Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") reported that it has received clearance from the FDA for its investigational new drug application (IND) for Abivertinib (Fujovee) for the Phase 2 MAVERICK study to be conducted in participants with metastatic castrate resistant prostate cancer (mCRPC) at multiple centers in the United States (Press release, Sorrento Therapeutics, MAY 17, 2022, View Source [SID1234614735]). The MAVERICK study will be conducted in a partnership with the Prostate Cancer Clinical Trials Consortium and will enroll participants with both abiraterone-naïve and abiraterone-progressing mCRPC. The MAVERICK trial will be conducted as an open-label study of Abivertinib with abiraterone in up to 100 participants harboring the adrenal-permissive HSD3β1 allele (heterozygous or homozygous). The primary objective of the study is to evaluate the efficacy of Abivertinib with abiraterone via an assessment of 6-month radiographic progression-free survival (rPFS) and the primary endpoint is the 6-month rPFS defined as a percent of subjects alive and without progression by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 for measurable disease and Prostate Cancer Working Group 3 (PCWG3) criteria for bone metastases. Participants will remain on treatment until radiographic progression, unacceptable toxicity, intercurrent illness or other reasons (such as subject withdrawal).

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Extragonadal androgen production requires the activity of 3β-hydroxysteroid dehydrogenase isoenzyme-1 (3β-HSD1) encoded by the HSD3β1 allele and an identified single nucleotide polymorphism (SNP) can create an adrenal-permissive phenotype that allows for more rapid development of mCRPC resulting in earlier castration resistance and shortened overall survival. Increased BTK expression has been observed in various solid tumors, including prostate cancer, and it is believed that the interplay between BTK inhibition and 3βHSD is responsible. BTK inhibition has been shown to inhibit androgen production, particularly from extragonadal precursor steroids, in preclinical models. In addition to selectively targeting both mutant forms of EGFR (T790M and exon 19 and 21 mutations), Abivertinib irreversibly binds to the BTK receptor, preventing phosphorylation of the receptor. Abivertinib is an oral capsule taken twice daily and has been well tolerated across multiple cancer types in doses up to 300 mg twice daily.

The global market for CRPC for 2021 was $2.7 billion, with the U.S. accounting for 67% ($1.83 billion), and a CAGR of 4.15%, projected to reach $4.56 billion, with the U.S. projected to account for 68% ($3.08 billion) by 2031(1).

On May 17, 2022 Arbutus Biopharma Corporation (Nasdaq: ABUS), a clinical-stage biopharmaceutical company leveraging its extensive virology expertise to develop novel therapeutics that target specific viral diseases, reported that William Collier, Arbutus’ President and Chief Executive Officer, will present at the H.C. Wainwright Global Investment Conference on Tuesday, May 24, 2022, at 11:30 am ET in Miami, FL (Press release, Arbutus Biopharma, MAY 17, 2022, https://investor.arbutusbio.com/news-releases/news-release-details/arbutus-present-hc-wainwright-global-investment-conference [SID1234614732]).

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A live webcast of the presentation can be accessed through the Investors section of Arbutus’ website at www.arbutusbio.com. An archived replay of the webcast will be available on the Arbutus website after the event.

On May 17, 2022 PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to discovering, developing and commercializing highly differentiated medicines for devastating diseases, reported that George Farmer, Ph.D., Chief Financial Officer, will present at the UBS Global Healthcare Conference on Tuesday, May 24, 2022, at 10:45am EDT (Press release, PureTech Health, MAY 17, 2022, View Source [SID1234614731]). A webcast of the presentation will be available at View Source

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