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Pharma Two B Appoints David S. Tierney, M.D. as the Company’s Chief Executive Officer

On February 18, 2020 Pharma Two B Ltd., a privately held company developing innovative therapeutics based on previously approved drugs for Parkinson disease, reported the appointment of David S. Tierney M.D. as Chief Executive Officer, effective immediately as well as the establishment of a fully owned U.S. subsidiary to serve as the marketing base of the Company’s activities (Press release, Pharma Two B, FEB 18, 2020, View Source [SID1234554416]).

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Pharma Two B’s main product, P2B001, is targeting patients with early stage Parkinson’s Disease. According to the Michael J. Fox Foundation for Parkinson’s Research (MJFF), Parkinson’s disease is estimated to affect nearly 1 million people in the United States and more than 6 million people worldwide. The Company estimates that approximately 15%-20% of these patients could be defined as early stage patients.

"We are excited to have David, a highly accomplished pharmaceutical executive with proven business development, medical and financial acumen, join Pharma Two B as CEO," said Ehud Marom, Chairman of Pharma Two B’s Board of Directors. "David has a successful track record in drug approvals, developing and executing plans of commercial organizations as well as raising equity capital raising from both private and VC investors, and the public markets, including the completion of an IPO."

Dr. Tierney stated, "I am delighted to lead Pharma Two B at this pivotal time and believe there are substantial opportunities to create significant value by registering P2B001 and building the commercial infrastructure to launch the product in the U.S. in 2021. This plan will include securing the needed financial resources for a successful launch through quality financings with high quality investors that will prepare the Company for an IPO and or strategic combination."

P2B001 is a low dose, sustained release combination of pramipexole and resagiline. The drug is orally dosed once daily with no titration. P2B001 is near the end of the Phase 3 pivotal study and the Company is preparing for an NDA submission next year via the 505(b)(2) regulatory pathway. Dr. Tierney added, "I look forward to working with our team and board members, completing the ongoing Phase 3 study and building the infrastructure to support a successful launch, including marketing and collaboration with potential partners."

About David S. Tierney, M.D.

Prior to David’s appointment as CEO of Pharma Two B, he was President and CEO of BioPharmX Corp (NYSE American: BPMX) and of Icon Bioscience, Inc., where he led a team that in 2018 received U.S. FDA approval of its New Drug Application (NDA) for DEXYCU (dexamethasone intraocular suspension), a dropless, long-acting therapeutic for treating inflammation associated with cataract surgery. Icon was merged with EyePoint Pharmaceutical, Inc (NASDAQ: EYPT) in March 2018.

Dr. Tierney served as President and COO of Oceana Therapeutics, Inc., a specialty therapeutic company he co-founded in 2008. Oceana quickly established a global commercial network and by 2011 was acquired by Salix Pharmaceuticals. In 2000, David was appointed President & CEO of Hydro Med Sciences (HMS), a research firm with a promising drug delivery platform. Under his leadership, HMS emerged as Valera Pharmaceuticals, a fully integrated, commercial, specialty pharma company that successfully completed an initial public offering in 2005. Valera has since been merged into Endo Pharmaceuticals (NASDAQ: ENDP). In total, Dr. Tierney has over 30 years of experience in executive and management positions in the pharmaceutical industry. Dr. Tierney received his medical degree from Royal College of Surgeons in Ireland.

David Tierney is the recipient of two Ernst & Young Entrepreneur of the Year awards, first in 2005 and again in 2011, respectively, reflecting his accomplishments at Valera and Oceana. He was also featured in the inaugural 2010 Irish Life Science 50, an honor presented by the President of Ireland in conjunction the Irish Voice and Irish America Magazine. Additionally, in 2005, 2006, and 2011, PharmaVOICE 100 named David one of the most inspiring people in health sciences.

Moleculin Presents Clinical Trial Data Demonstrating a Median Reduction of 56% in Skin Cancer Lesions

On February 18, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported the final data from its CTCL clinical trial of WP1220 for the treatment of cutaneous T-cell lymphoma (CTCL), which was published and presented by Dr. M. Sokolowska-Wojdylo in conjunction with the 4th Annual World Congress of Cutaneous Lymphomas in Barcelona, Spain on February 13, 2020 (Press release, Moleculin, FEB 18, 2020, View Source [SID1234554415]). The final results supported the safety of topical WP1220 and demonstrated a median improvement in the Composite Assessment of Index Lesion Severity (CAILS) score of 56% in treated (index) lesions for patients completing the study.

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

"WP1220 is part of our p-STAT3 inhibitor drug portfolio. Late last year, we announced the preliminary results of this proof of concept Phase 1 trial. For years, p-STAT3 (the activated form of STAT3) has been considered an ‘undruggable’ target because of the difficulty of reaching and affecting this cell-signaling protein," commented Walter Klemp, Moleculin’s Chairman and CEO. "We are pleased to conclude this trial and demonstrate a therapeutic effect from a p-STAT3 inhibitor which could be considered a significant breakthrough in cancer research."

Introduction & Objectives: Mycosis Fungoides or MF, the most common variant of CTCL, is a disease with symptomatic, disfiguring skin lesions. STAT3, an oncogenic transcription factor, has been identified as a critical regulator of MF, whereby the activation of STAT3 through phosphorylation (p-STAT3) has been linked to tumor proliferation and suppression of immune responses. WP1220, a synthetic compound, potently inhibits the activity of p-STAT3 and the growth of CTCL cell lines. This Phase 1b study was designed to demonstrate the safety and efficacy of WP1220 after topical treatment of MF.

Results: Of 5 subjects enrolled, 9 lesions were assessed according to the CAILS scoring system. The only adverse event (AE) was mild contact dermatitis in one subject felt not to be related to the drug. 4 of the 5 subjects had significant improvement in CAILS scores on index lesions, with a median reduction of 56% (range 25%-94%). Improvement was noted within 7 days of treatment initiation and maintained 1 month after discontinuation. Independent dermatologic review based on photographic documentation was conducted and corroborated these findings.

Conclusions: WP1220, an inhibitor of p-STAT3, shows safety and significant efficacy in MF after topical treatment. We believe this is the first demonstration in humans that inhibition of p-STAT3 with topical therapy has efficacy in CTCL. A larger Phase 2 study is now being planned.

Karyopharm to Participate at 9th Annual SVB Leerink Global Healthcare Conference

On February 18, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, reported that Michael Kauffman, MD, PhD, Chief Executive Officer, reported that it will participate in a fireside chat at the 9th Annual SVB Leerink Global Healthcare Conference on Tuesday, February 25, 2020 at 11:30 a.m. ET in NYC (Press release, Karyopharm, FEB 18, 2020, View Source [SID1234554414]).

A live webcast of the fireside chat can be accessed on the "Events & Presentations" in the Investor section of the Company’s website, View Source A replay of the webcast will be archived on the Company’s website for 90 days following the fireside chat.

HOOKIPA Pharma to Present at SVB Leerink 9th Annual Global Healthcare Conference

On February 18, 2020 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics targeting infectious diseases and cancers based on its proprietary arenavirus platform, reported that HOOKIPA’s management team will present and host one-on-one meetings at the SVB Leerink 9th Annual Global Healthcare Conference, taking place February 25 – 27, 2020 in New York (Press release, Hookipa Pharma, FEB 18, 2020, View Source [SID1234554413]):

Presentation: Tuesday, February 25, 2020 at 10:00 a.m. ET, Lotte New York Palace, Room Kennedy I
A live audio webcast of the presentation held at the SVB Leerink Healthcare Conference will be available within the Investors & Media section of HOOKIPA’s website at View Source An archived replay will be accessible for 30 days following the event.

Analysis of RNA sequences and modifications by mass spectrometry published in Nature Communications

On February 17, 2020 STORM Therapeutics, the biotechnology company focused on the discovery of small molecule therapies modulating RNA epigenetics, reported that it has published a scientific paper in the peer reviewed journal Nature Communications (Press release, STORM Therapeutics, FEB 17, 2020, View Source [SID1234561052]).

The paper is entitled: ‘A computational platform for high-throughput analysis of RNA sequences and modifications by mass spectrometry’ and showcases breakthrough analysis of RNA sequences and modification using STORM’s mass spectrometry. The work, which was carried out in collaboration with the University of Cambridge, University of Tübingen, Germany, and the University of Pennsylvania, US describes a novel algorithm that allows the quantitative analysis of RNA modifications in the context of their RNA sequence using mass spectrometry. See link to paper: View Source, DOI: 10.1038/s41467-020-14665-7

Dr Oliver Rausch, CSO of STORM Therapeutics, said: "STORM is a pioneer in RNA epigenetics and we are very pleased to see the publication of this paper in Nature Communications, validating the use of STORM’s algorithm, created to extend the use of mass spectrometric data to RNA epigenetics in order to boost the discovery power of teams investigating the role of RNA modifications in disease."

The study describes a novel computational algorithm named Nucleic Acid Search Engine (NASE) that allows sequencing of RNA molecules by mass spectrometry including positional identification of chemical modifications on the RNA molecules. In particular, the algorithm enables the mapping of RNA fragmentation patterns obtained by mass spectrometry to RNA sequence databases, thus identifying the originating RNA molecules

Dr Hendrik Weisser, Principal Scientist at STORM and senior author on the paper commented: "I am delighted to see the publication of this new algorithm in a world leading journal. Our algorithm represents a novel approach for characterizing modifications on many different RNA species. Unbiased quantitative analysis of RNA modification on specific RNAs is one of the key challenges in the novel field of RNA Epigenetics and resources like NASE are critical to rapid innovation and advancement in the discovery of RNA modifications. By making this approach available publicly we are providing a new tool to the community that will accelerate overall progress in the field."