On April 26, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE), ("NeuBase"), a biotechnology company accelerating the genetic revolution with a new class of precision genetic medicines, reported the closing of its previously announced underwritten public offering of 9,200,000 shares of its common stock (inclusive of 1,200,000 shares that were sold pursuant to the underwriters’ full exercise of their option to purchase additional shares of NeuBase’s common stock), at a price to the public of $5.00 per share, generating gross proceeds of $46.0 million before deducting the underwriting discounts and commissions and offering expenses payable by NeuBase (Press release, NeuBase Therapeutics, APR 26, 2021, View Source [SID1234578571]). NeuBase intends to use the net proceeds from the offering for general corporate purposes, working capital and development of its product candidates and pipeline expansion.

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RBC Capital Markets, Oppenheimer & Co. Inc. and Chardan acted as the joint book-running managers for the offering, and National Securities Corporation acted as co-manager for the offering.

The securities described above were offered by NeuBase pursuant to a shelf registration statement on Form S-3 (File No. 333-254980) previously filed with the Securities and Exchange Commission (the "SEC") on April 1, 2021 and declared effective by the SEC on April 14, 2021. A final prospectus supplement and the accompanying prospectus relating to and describing the offering was filed with the SEC. Copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained by visiting the SEC’s website at www.sec.gov or by contacting RBC Capital Markets, Attention: Equity Syndicate, 200 Vesey Street, 8th Floor, New York, NY 10281, or by telephone at (877) 822-4089 or by e-mail at [email protected], Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, NY 10004, or by telephone at (212) 667-8055 or by e-mail at [email protected], or Chardan, 17 State Street, 21st floor, New York, New York 10004, by telephone at (646) 465-9032 or by e-mail at [email protected].

This press release does not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

On April 26, 2021 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that the European Medicines Agency (EMA) has validated the Company’s Type II Variation Marketing Authorization Application (MAA), which seeks to expand the currently authorized indication for NEXPOVIO in the European Union to include, in combination with Velcade (bortezomib) and low-dose dexamethasone, the treatment of adult patients with multiple myeloma who have received at least one prior therapy (Press release, Karyopharm, APR 26, 2021, View Source [SID1234578486]). Validation of the application confirms the submission is complete to begin the EMA’s review process. The MAA is supported by the positive results from the pivotal Phase 3 BOSTON study, which evaluated once-weekly selinexor in combination with once-weekly Velcade and low-dose dexamethasone (SVd) compared to standard twice-weekly Velcade plus low-dose dexamethasone (Vd) in patients with multiple myeloma who have received one to three prior lines of therapy. The results of the BOSTON study were published in The Lancet in November 2020 and were the basis for the U.S. Food and Drug Administration approval of XPOVIO’s expanded indication in December 2020.

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"The submission of a Type II Variation Marketing Authorization Application based on positive data from the Phase 3 BOSTON study represents an important step towards our goal of further expanding the treatment options available to patients with multiple myeloma in Europe," said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm. "We look forward to the EMA’s review of this supplemental data, which further reinforces the broader therapeutic potential of NEXPOVIO."

In March 2021, NEXPOVIO was granted conditional marketing authorization by the European Commission in combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents, and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy. The new MAA will be reviewed by Committee for Medicinal Products for Human Use (CHMP), which will issue an opinion to the European Commission regarding the potential approval for the expanded indication. Karyopharm expects this review to be completed in the fourth quarter of 2021. This application is also intended to fulfill Karyopharm’s obligation in the context of the conditional marketing authorization of NEXPOVIO granted in March 2021.

About Multiple Myeloma in Europe

Multiple myeloma (MM) is an incurable cancer with significant morbidity and the second most common hematologic malignancy. In 2020, there were approximately 51,000 new cases and 32,000 deaths from MM in Europe1. While the treatment of MM has improved over the last 20 years, and overall survival has increased considerably, the disease remains incurable, and nearly all adult patients will eventually relapse and develop disease that is refractory to all authorized anti-MM therapies. Therefore, there continues to be a high unmet medical need for new therapies, particularly those with novel mechanisms of action.

About NEXPOVIO (selinexor)

NEXPOVIO, which is marketed as XPOVIO in the U.S., is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) medicine. NEXPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). NEXPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion. NEXPOVIO (selinexor) has been granted conditional marketing authorization by the European Commission in combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents, and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy.

On April 26, 2021 Scopus BioPharma Inc. (Nasdaq: "SCPS") reported the submission of an investigational new drug application ("IND") to the United States Food and Drug Administration ("FDA") for its immuno-oncology RNA therapy for the treatment of multiple cancers (Press release, Scopus BioPharma, APR 26, 2021, View Source [SID1234578505]). The IND filing is a key milestone for the planned Phase 1 clinical trial for B-cell non-Hodgkin lymphoma to be initiated at City of Hope.

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Scopus is a biopharmaceutical company developing transformational therapeutics based on groundbreaking scientific and medical discoveries. City of Hope is a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases near Los Angeles, California.

Joshua R. Lamstein, Chairman of Scopus BioPharma, stated, "The IND submission to the FDA for a Phase 1 clinical trial for our lead drug candidate is a key milestone for Scopus. We are excited about the prospects of entering the clinic. We believe this, and other imminent developments, will be significant drivers of shareholder value."

Scopus’ lead drug candidate encompasses both RNA therapy and immunotherapy by synthetically linking siRNA to an oligonucleotide TLR9 agonist, creating the potential for targeted gene silencing with simultaneous TLR stimulation and immune activation in the tumor microenvironment. This highly-distinctive drug candidate was developed in the City of Hope laboratories of Hua Yu, Ph.D. and Marcin Kortylewski, Ph.D. Yu is co-leader of the Cancer Immunotherapeutics Program and Billy and Audrey L. Wilder Professor in Tumor Immunotherapy. Kortylewski is an associate professor in the Department of Immuno-Oncology.

Mr. Lamstein added, "We are privileged to be working with City of Hope. The IND submission is testament to the respective efforts and close cooperation of the Scopus and City of Hope teams. We have developed strong working relationships with Dr. Yu and Dr. Kortylewski, both preeminent researchers in immuno-oncology, and other outstanding City of Hope professionals. City of Hope’s collective scientific, manufacturing, clinical, regulatory and operational capabilities are being coordinated by City of Hope’s Translational Development Center (TDC). The IND submission to the FDA was accomplished on time and on budget. This was made possible by the commitment and efforts of the exceptional TDC team brought together by Ashley Baker Lee, Senior Vice President, Research Operations. We are extremely grateful to Ashley and the entire superb research operations team."

On April 26, 2021 Pulse Biosciences, Inc. (Nasdaq: PLSE), a novel bioelectric medicine company progressing Nano-Pulse Stimulation (NPS) technology, reported it will report financial results for the first quarter of 2021 after market close on Monday, May 10, 2021 (Press release, Pulse Biosciences, APR 26, 2021, View Source [SID1234578572]). Company management will host a corresponding conference call beginning at 1:30pm PT.

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Investors interested in listening to the conference call may do so by dialing 1-877-705-6003 for domestic callers or 1-201-493-6725 for international callers. A live and recorded webcast of the event will be available at View Source

On April 25, 2021 Sirnaomics, Inc., a biopharmaceutical company engaged in the discovery and development of RNAi therapeutics against cancer and fibrotic diseases, reported the initiation of the Phase 2b study of the company’s lead drug candidate, STP705, for the treatment of squamous cell skin cancer (Press release, Sirnaomics, APR 25, 2021, View Source [SID1234578436]).

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The randomized, double-blind, placebo-controlled study will evaluate the safety and efficacy of intralesional injection of STP705 in 100 adult patients with squamous cell carcinoma in situ (isSCC). This is a two-part dose escalation trial; in the run-in period, the 30 ug and 60 ug dosing regimens from the Phase 2a study of STP705 will be further evaluated, in addition to a third new dose level. The second part of the trial will further evaluate the two most efficacious dosing regimens. The primary endpoint of this trial is the proportion of participants with histological clearance of treated isSCC lesion at the end of treatment. Histological clearance will be defined as the absence of detectable evidence of isSCC tumor cell nests as determined by central pathology review.

"In the recently concluded Phase 2a clinical trial of STP705 in isSCC, a high rate of patients achieved histological clearance in a dose dependent manner, which is the gold standard for skin cancer," said Patrick Lu, Ph.D., the founder, President and CEO of Sirnaomics. "As we initiate the Phase 2b trial, we are hopeful to learn more about the potential of this non-surgical, non-invasive treatment for common non-melanoma skin cancer, and more broadly the promise of RNAi therapeutics in oncology."

"isSCC continues to be a disease with high unmet therapeutic need where surgery is still considered the only viable treatment option for many patients," said Michael Molyneaux M.D., Chief Medical Officer of Sirnaomics. "We hope to build on the success we have seen in the Phase 2a study, where we achieved 90% histological clearance rates in the 30 ug and 60 ug dosing groups. We anticipate having an interim data readout late second half of 2021 that will guide our clinical development for this indication."

Additional information about this clinical trial is available at clinicaltrials.gov using the identifier: NCT04844983.

About Non-melanoma Skin Cancer and Squamous Cell Carcinoma In Situ
Skin cancer is the most common type of all cancers diagnosed each year in the United States. It is estimated that nearly half of cancers diagnosed every year will be skin cancers. Over the past decade, the incidence of skin cancers has increased dramatically. According to the JAMA Dermatology paper (Rogers, et. al. JAMA Dermatol. 2015151(10):1081-1086), an estimated 3.3 million people in the US suffer from non-melanoma skin cancer (NMSC) along with 5.43 million people that are currently living with cancer lesions. Data on specific types of NMSC were 2.55 million cases for basal cell carcinoma (47%): 2.57 million cases for squamous cell carcinoma including squamous cell carcinoma in situ (46.7%), plus another 332,000 cases of unspecified type of skin cancers.

A World Health Organization authorized report from "International Agency for Research on Cancer" (2019) has demonstrated that the number of deaths in 2018 globally for both men and women from NMSC is 65,155, where the mortality of Asia NMSC patients represents 41.9% of the global total, significantly more than other individual areas.

Squamous cell carcinoma in situ, also called Bowen disease, is the earliest form of squamous cell skin cancer (SCC). Along with basal cell carcinoma, SCC is one of two major subtypes of NMSC. The key driver for development of SCC is ultraviolet rays from the sun. It is believed that development of SCC is linked closely to genomic perturbations, genetic mutations, and altered expression of key molecules (e.g., overexpression of TGF-β1 and COX-2) that impacts squamous cell lineage commitment and terminal differentiation.

Surgery is the currently the most common treatment option for the treatment of NMSC. The various forms of surgical modalities carry significant cutaneous adverse events, risk of scar, infection and bleeding. Surgery can also have a significant recurrence rate. As a result, there is a high unmet need for an FDA approved local injection therapy that is safe and effective.

About STP705
Sirnaomics’ leading product candidate, STP705, is a siRNA (small interfering RNA) therapeutic that takes advantage of a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expression. The product candidate has received multiple IND approvals from both the US FDA and Chinese NMPA, including treatments of cholangiocarcinoma, nonmelanoma skin cancer and hypertrophic scar. STP705 has also received Orphan Drug Designation for treatment of cholangiocarcinoma and primary sclerosing cholangitis. STP705 is currently in four clinical trials for different indications. A Phase 2a study of STP705 for treatment of squamous cell skin cancer (isSCC) in adult patients demonstrated positive efficacy and safety results, with 76% of all patients (19/25) achieving complete histologically clearance and the two optimal dosing ranges achieving 90% histological clearance of tumor cell in the lesion. No significant or serious adverse events, including no significant cutaneous skin reactions, were reported in the study, and the company was able to define a clear therapeutic window in advance of later stage studies.