Moleculin Announces Completion of Second Single Ascending Dose (SAD) Cohort in Phase 1a Clinical Trial of WP1122 in the UK

On July 8, 2022 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 and viruses, reported preliminary results from the second cohort of the Company’s first-in-human Phase 1a study of WP1122 (Press release, Moleculin, JUL 8, 2022, View Source [SID1234616561]). This cohort consisted of 8 subjects dosed with 16 mg/kg or placebo in the dose escalation trial evaluating the safety and pharmacokinetics (PK) of WP1122 in healthy volunteers in the United Kingdom (UK). Based on the overall results in Cohort 2, the Company deemed the cohort dose safe and well-tolerated and began its SAD Cohort 3 with a dose escalation to 32 mg/kg.

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The Phase 1a, first-in-human, randomized, double-blind, placebo-controlled, overlapping SAD and MAD is investigating the effects of WP1122 administered as an oral solution in healthy human volunteers. It is the first step in a planned investigation of WP1122 for the treatment of COVID-19. Dose escalation will take place in sequential SAD cohorts, and MAD will start as soon as SAD has successfully completed at least 3 dosing cohorts. This study in healthy volunteers will explore safety and PK, and subsequent antiviral clinical development is expected to be in patients infected with SARS-CoV-2 to further evaluate safety and establish a favorable risk/benefit profile. The Company expects to enroll approximately 80 subjects in this trial.

Walter Klemp, Chairman and Chief Executive Officer of Moleculin commented, "We continue to be encouraged by the safety data demonstrated by WP1122 in the SAD portion of our first-in-human Phase 1a study of WP1122. With two of the SAD cohorts now completed with promising preliminary results, we are working to enroll and complete Cohort 3 at 32 mg/kg in order to proceed to the MAD phase of the trial toward establishing a maximum tolerated dose. To date, WP1122 has demonstrated no dose escalating stopping criteria, and we look forward to taking the next step in studying the potential of WP1122 for the treatment of certain viral diseases, including COVID-19, and cancers."

During the SAD portion of this study, dose escalation will proceed up to a maximum dose of 64 mg/kg as a single dose. Dosing of WP1122 began in SAD at 8 mg/kg as a single dose and has escalated in two-fold increments (i.e., to 16 and now to 32 mg/kg as single doses, etc.) in subsequent cohorts. Dosing of WP1122 in the MAD cohorts will start after a dose of 32 mg/kg has been shown to be safe in the single dose cohort. The first dose administered in MAD will be 16 mg/kg every 12 hours (32 mg/kg/day) for 7 days and dosing in the second MAD cohort will escalate to 32 mg/kg every 12 hours (64 mg/kg/day) for 7 days.

For more information about the study, please visit clinicaltrials.gov and reference identifier NCT05195723. Moleculin is also in the process of identifying additional countries where potential future Phase 2 COVID-19 clinical studies could occur.

About WP1122
WP1122 was developed as a 2-DG prodrug to provide a more favorable pharmacological profile and was found to have greater potency than 2-DG alone in preclinical models where tumor cells require higher glycolytic activity than normal cells. WP1122 has also been shown to have a greater antiviral effect than 2-DG against SARS-CoV-2 in MRC-5 cells in culture. The improved pharmacokinetic and pharmacodynamic (PK/PD) profile of WP1122 compared to 2-DG was noted in female mice following oral dosing at equimolar (i.e., equivalent levels of 2-DG) doses.

While the Company is in the process of identifying additional countries where potential future Phase 2 COVID-19 clinical studies might occur, the volatility and unpredictability of COVID-19 incidence in various countries may limit the ability to recruit certain subjects and could make it infeasible to conduct a Phase 2 clinical trial in a given country. Additionally, Moleculin recently received IND clearance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1 study of WP1122 for the treatment of Glioblastoma Multiforme (GBM). The Company is seeking collaborators with the intent to commence clinical trials of WP1122 in cancer indications including GBM, pancreatic cancer and others.

Seagen to Host Conference Call and Webcast Discussion of Second Quarter 2022 Financial Results on July 28, 2022

On July 8, 2022 Seagen Inc. (Nasdaq: SGEN) reported that it will report its second quarter 2022 financial results on Thursday, July 28, 2022 after the close of U.S. financial markets (Press release, Seagen, JUL 8, 2022, View Source [SID1234616563]). Following the announcement, Company management will host a conference call and webcast at 4:30 p.m. Eastern Time to discuss the results and provide a business update.

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Conference call and webcast information:

Telephone 844-763-8274 (U.S.) or +1 412-717-9224 (international); conference ID 10167970
Webcast with slides can be accessed at investor.seagen.com. A webcast replay will be archived on the Company’s website.

World Orphan Drugs Congress, Boston MA July 12-13, 2022

On July 8, 2022 Simbec-Orion reported that We are excited to be attending the World Orphan Drug Congress USA in Boston (Press release, Simbec-Orion, JUL 8, 2022, View Source [SID1234616564])

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The World Orphan Drug Congress brings together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers. The conference is a place to meet and brainstorm ways to advance orphan drug development and improve access to life-saving therapies.

Driving complex rare and orphan clinical development programs forward on a successful path has been our focus for more than 25 years, and we would love to learn more about your needs. Connect with Romillie Cruz, MD and Maria Frane during the meeting at booth 636, or by scheduling your meeting today https://bit.ly/3O3vfXb #raredisease #WorldOrphanUSA

Neuren appoints Chief Medical Officer in United States

On July 8, 2022 Neuren Pharmaceuticals (ASX: NEU) reported the appointment of Liza A. Squires M.D. to the new position of Chief Medical Officer, based in the United States (Press release, Neuren, JUL 8, 2022, View Source;[email protected] [SID1234616565]).

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Dr Squires is a board certified physician in General Pediatrics and Neurology with Special Competence in Child Neurology. Over the past 20 years, she has held positions of increasing responsibilities in both early and late-stage development at Johnson and Johnson, Shire Pharmaceuticals, Lumos Pharma, Aevi Genomic Medicine and Origin Biosciences. She has led and contributed to multiple New Drug Applications resulting in global regulatory approvals and has extensive experience in orphan drug development.

Dr Squires trained in general pediatrics at Yale University and did her residency in Child Neurology at Massachusetts General Hospital.

Neuren CEO Jon Pilcher commented: "I am very pleased to welcome Liza at such an exciting time for Neuren. Her ideal skills and experience in pediatric neurology and orphan drug development will make a big difference to the team as we advance our ambitious plans for NNZ-2591 in multiple indications."

ADC Therapeutics Announces Exclusive License Agreement with Sobi to Develop and Commercialize ZYNLONTA® (loncastuximab tesirine-lpyl) in Europe and Select International Territories

On July 8, 2022 ADC Therapeutics SA (NYSE: ADCT) reported it has entered into an exclusive license agreement with Swedish Orphan Biovitrum AB (Sobi) for the development and commercialization of ZYNLONTA for all hematologic and solid tumor indications outside of the United States, greater China, Singapore and Japan (Press release, ADC Therapeutics, JUL 8, 2022, View Source [SID1234616566]). The Marketing Authorization Application (MAA) for ZYNLONTA was validated by the European Medicines Agency (EMA) at the end of October 2021, and orphan drug designation was granted for ZYNLONTA for the treatment of diffuse large B-cell lymphoma (DLBCL) in Europe. ADC Therapeutics and Sobi intend to make ZYNLONTA available following a regulatory decision that is expected by the first quarter of 2023.

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Under the terms of the agreement, ADC Therapeutics will receive an upfront payment of $55 million, and is eligible to receive $50 million upon regulatory approval of ZYNLONTA in third-line DLBCL by the European Commission and up to approximately $330 million in additional regulatory and sales milestones. ADC Therapeutics will also receive a percentage of royalties ranging from the mid-teens to the mid-twenties based on net sales of ZYNLONTA in Sobi’s territories. Sobi will share a portion of select global ZYNLONTA clinical trial costs.

"We are thrilled to establish this important partnership with Sobi to continue expanding our global reach to bring ZYNLONTA to as many patients as possible worldwide," said Ameet Mallik, Chief Executive Officer of ADC Therapeutics. "Sobi has a strong global commercial infrastructure, proven capabilities in the areas of hematology and rare diseases, and importantly, shares our passion for improving the lives of patients."

Guido Oelkers, Chief Executive Officer of Sobi, said, "ADC Therapeutics has an innovative and validated technology platform and is a leader in the evolving field of antibody-drug conjugates. We are delighted about this collaboration to bring loncastuximab tesirine to Europe and beyond to serve an unmet need in debilitating orphan diseases in haematology."

In April 2021, the U.S. Food and Drug Administration (FDA) granted accelerated approval to ZYNLONTA as the first and only CD19-targeted ADC as a single-agent treatment for adult patients with relapsed or refractory DLBCL after two or more lines of systemic therapy. ADC Therapeutics has an exclusive license agreement with Mitsubishi Tanabe Pharma Corporation (MTPC) for the development and commercialization of ZYNLONTA for all hematologic and solid tumor indications in Japan. In addition, Overland ADCT BioPharma, a joint venture formed by Overland Pharmaceuticals and ADC Therapeutics, is working to develop and commercialize ZYNLONTA in greater China and Singapore. Overland ADCT BioPharma is now conducting a registrational pivotal Phase 2 clinical trial of ZYNLONTA in relapsed or refractory DLBCL in China.