On April 26, 2021 Mission Therapeutics ("Mission"), a drug discovery and development company focused on selectively inhibiting deubiquitylating enzymes (DUBs), reported that Paul Thompson, Chief Scientific Officer, will present at the Mitochondria-Targeted Drug Development Digital Summit (27-29 April) (Press release, Mission Therapeutics, APR 26, 2021, View Source [SID1234578445]).

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Paul will present a seminar titled: USP30 Inhibitors: Releasing the Brakes to Improve Mitochondrial Quality Control During Disease, on 28 April at 14:15 EST / 20.15 CEST. He will explore multiple disease pathologies and detail how they can be prevented by inhibiting Mission Therapeutics’ in-house DUB target, USP30.

The Mitochondria-Targeted Drug Development Digital Summit is an industry-led meeting focused on end-to-end mitochondria targeting. The virtual event will be attended by over 80 senior CSOs, CEOs, research scientists, academics and investors, who share a commitment to tackling mitochondrial and age-related diseases. Over the three days, a series of presentations, workshops and networking sessions will focus on success stories, common obstacles, and opportunities in the field of mitochondria-targeted drug development.

On April 26, 2021 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported that Ellen Kim, MD, Medical Director, Dermatology Clinic, Perelman Center for Advanced Medicine, Professor of Dermatology at the Hospital of the University of Pennsylvania, and the Lead Principal Investigator for the Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study, delivered a presentation at the American Academy of Dermatology (AAD) Association Virtual Meeting Experience, held April 23-25, 2021 (Press release, Soligenix, APR 26, 2021, View Source [SID1234578485]). The presentation, which was designated "Top 12 Late-Breaking Research," expanded on data related to the efficacy and safety of HyBryte (SGX301) in the treatment of cutaneous T-cell lymphoma (CTCL).

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Oral Presentation:

Visible Light Activated Topical Hypericin Ointment in CTCL: Phase 3 FLASH Study Results Dr. Kim’s presentation and Q&A session is archived on the AAD Virtual Meeting site and will be accessible via registration. Attendees can register here.

Key Highlights:

In addition to its demonstrated, statistically significant efficacy in the placebo controlled portion of the trial, HyBryte was also shown to improve its treatment response with longer treatment durations, resulting in a response rate of 40% after 12 weeks of therapy (p<0.0001) and 49% after 18 weeks of therapy (p<0.0001).
Compared to other, second-line, approved drugs for the treatment of CTCL, this response rate was shown to be as good or better than other treatments, with significantly less safety concerns. In the FLASH study, the rate of serious adverse events was only 2.4%, with none related to the use of HyBryte, and the rate of severe adverse events was only 4%.
Skin related events such as pruritus, erythema, hyperpigmentation, burning sensation, dermatitis, occurred in only 16% of HyBryte treated patients vs 10% of the placebo patients and were generally mild or moderate in severity.
"This trial was the largest multicenter, randomized, double-blind, placebo-controlled skin directed therapy study in MF/CTCL (mycosis fungoides/cutaneous T-cell lymphoma) to date," noted Dr. Kim. "It is so exciting to be able to tell the world about the efficacy and safety we saw HyBryte demonstrate in this trial. Based on its non-mutagenic mechanism of action, HyBryte should not be associated with long-term actinic skin damage or increased risk of skin cancer, which would be an advantage over traditional ultraviolet light based phototherapy. This study was a tremendous multicenter, collaborative effort and I would like to thank all site investigators, and especially our patients, for their contributions to developing this promising new therapy."

About the American Academy of Dermatology Virtual Meeting

The AAD meeting is an annual meeting, dedicated to a broad range of dermatology related topics, as described here.

About HyBryte

HyBryte (SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light 16 to 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).

The Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions. Follow-up visits were completed in Q4 2020, and the clinical study report to support the NDA is in the process of being finalized.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte’s mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.

On April 26, 2021 Himalaya Therapeutics ("Himalaya"), a clinical-stage biopharmaceutical company focused on development and commercialization in Greater China of a novel class of investigational antibody therapeutics for the treatment of solid tumor cancer, which are based on the Conditionally Active Biologics ("CAB") technology platform, reported the appointment of Brian Zhang, Ph.D., as chief executive officer (Press release, Himalaya Therapeutics, APR 26, 2021, View Source [SID1234578504]).

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"We are thrilled to welcome Dr. Zhang to our growing team. His extensive experience will help drive Himalaya towards its ambitious goals," said Carolyn Short, co-founder and president of Himalaya Therapeutics since its founding in 2014. "Dr. Zhang is our second executive hire this quarter as we ramp up, and will provide great leadership as we continue to build this world-class team."

Dr. Zhang is a global pharmaceutical veteran, with decades of experience on the R&D side, overseeing products from discovery to commercialization, on the corporate side, leading and founding healthcare companies, and on the investing side, as a venture partner and an angel investor. While at Hoffmann-La Roche, he helped lead the development of Xenical and the discovery of Dorzagliatin, among others, and helped establish its bioinformatics group. More recently, he cofounded several biotechnology startups and invested in biopharmaceuticals. He received his Ph.D. from the UCLA School of Medicine and his B.S. from Nanjing University. He also conducted brain research at the Chinese Academy of Sciences, earning a grant for further study abroad.

"I am excited to join the Himalaya team and look forward to taking the impressive pipeline of a dozen drug candidates to move into the Chinese market as well as globally. The CAB platform has the potential to be truly groundbreaking and applicable to some of the most challenging problems we have faced in our battle against cancer."

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 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.