On May 15, 2023 Manhattan BioSolutions, Inc. ("Manhattan Bio"), an emerging biotechnology company developing new classes of precision biologics for the treatment of advanced cancers, reported that it has entered into an exclusive option agreement with the Wisconsin Alumni Research Foundation (WARF) for the innovative pan-RNA degrader technologies (pRNAD) in oncology (Press release, Manhattan BioSolutions, MAY 15, 2023, View Source [SID1234631728]). The exclusive option agreement involves the intellectual property rights to technology developed in the laboratory of Ronald T. Raines at the University of Wisconsin-Madison.

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Ronald T. Raines, Ph.D., is currently the Roger and Georges Firmenich Professor of Natural Products Chemistry at the Massachusetts Institute of Technology (MIT), an Extramural Member of the Koch Institute for Integrative Cancer Research at MIT, and an Associate Member of the Broad Institute of MIT and Harvard. Professor Raines has also joined Manhattan Bio’s Board of Directors.

The pRNAD technology is based on an engineered derivative of a naturally occurring nuclease protein involved in host defense to selectively degrade RNA in both tumors and their microenvironment. In preclinical studies, pRNAD has shown a favorable tolerability profile and potent single agent activity against a variety of cancer models such as ovarian, prostate, non-small cell lung and colon cancers. The lead drug candidate has been previously evaluated in a Phase 1 human clinical trial conducted in patients with advanced refractory solid tumors, showing promising results.

Dr Borys Shor, Chairman and CEO, Manhattan Bio, commented: "We are excited to announce this agreement and associated data acquisition, which adds potentially first-in-class clinic-ready program to our pipeline of differentiated biologics. This agent was designed to specifically target and disrupt key vulnerabilities in tumors, including their dependence on extracellular RNA and hyperactive RNA metabolic pathways. Our strategy has been to build a portfolio of world-class development candidates through a combination of in-licensing or in-house discovery. The RNAD transaction is in line with this vision."

Pending completion of the investigational new drug (IND) application process and subsequent regulatory activation of the IND, Manhattan Bio plans to initiate a phase 1b clinical trial in selected tumor indications. The company is also advancing innovative technologies capable of potentially generating multiple Investigational New Drug Applications (INDs), including novel linker/payload technologies for antibody-drug conjugates.

On May 15, 2023 Lipella Pharmaceuticals Inc. (Nasdaq: "LIPO") ("Lipella," "our, "us" or the "Company"), a clinical-stage biotechnology company addressing serious diseases with significant unmet need, reported a corporate update and announces its first quarter 2023 financial results (Press release, Lipella Pharmaceuticals, MAY 15, 2023, View Source [SID1234631727]).

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Dr. Jonathan Kaufman, CEO of Lipella, said, "Lipella has had a strong start to 2023 with the release of positive top line results from our Phase 2a clinical trial evaluating the safety and efficacy of LP-10 as a treatment for hemorrhagic cystitis. The results have been presented at prestigious meetings including the 2023 Biotech Showcase and as part of a late-breaking presentation at the Annual Meeting of the American Urological Association. We expect to schedule a Type B meeting with the FDA regarding this program in the coming months.

"We were also delighted to enter a manufacturing collaboration agreement with Cook MyoSite to facilitate Chemistry, Manufacturing, and Control documentation for LP-10 in anticipation of Lipella’s forthcoming IND application."

Operational Highlights

● In March 2023, Lipella created a Scientific Advisory Board in oral health to focus on the development of LP-310, the company’s product candidate for the treatment of symptomatic oral lichen planus. The five-member board is made up of a group of highly regarded experts in oral medicine.
● In January 2023, Lipella presented successful top line results of its Phase 2a clinical trial for LP-10 in hemorrhagic cystitis at the 2023 Biotech Showcase.

Financial Highlights

● Lipella had approximately $4.0 million in cash, cash equivalents and marketable securities as of March 31, 2023.
● Net loss for the quarter ended March 31, 2023 was approximately $1.1 million.
● Investors are encouraged to read the Company’s Quarterly Report on Form 10-Q when it is filed with the Securities and Exchange Commission (the "SEC"), which will contain additional details about Lipella’s financial results as of and for the period ended March 31, 2023.

On May 15, 2023 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported its financial results for first quarter ended March 31, 2023 (Press release, Leap Therapeutics, MAY 15, 2023, View Source [SID1234631726]).

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Leap Highlights:

· Presenting new long-term follow-up data from Part A of the Phase 2 DisTinGuish study of DKN-01 plus tislelizumab and chemotherapy in first-line patients with advanced gastroesophageal adenocarcinoma (GEA) at the upcoming 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting

· Enrollment completed in Part A of the Phase 2 DeFianCe study of DKN-01 in combination with standard of care bevaiczimab and chemotherapy as a second-line treatment for patients with advanced colorectal cancer (CRC)

· Acquired Flame Biosciences, adding FL-301, a clinical stage anti-Claudin18.2 antibody, and preclinical antibody programs targeting Claudin18.2/CD137 and GDF15 to Leap’s pipeline, along with approximately $50 million in cash.

"We continued to execute extremely well on our DKN-01 program during the first quarter of 2023 with the completion of enrollment in Part A of the DeFianCe second-line CRC study and excellent progress in enrolling our randomized, controlled Part C of the DisTinGuish first-line GEA study," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "We look forward to presenting long-term follow-up data from Part A of the DisTinGuish study at ASCO (Free ASCO Whitepaper) in June, including updated response and overall survival data. With the acquisition of Flame Biosciences at the beginning of the year, we are in a strong financial position to develop our pipeline of personalized medicines for cancer patients."

DKN-01 Development Update

· Updated data from Part A of the DisTinGuish Study of DKN-01 plus tislelizumab and chemotherapy in gastric cancer patients to be presented at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting. The Company will be presenting new long-term follow-up data in first-line patients with advanced GEA from Part A of the DisTinGuish study (NCT0436380), a Phase 2 clinical trial evaluating Leap’s anti-Dickkopf-1 (DKK1) antibody, DKN-01, in combination with tislelizumab and chemotherapy at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting, being held in Chicago, IL on June 2-6, 2023. Details of the presentation are below:

Title: A phase 2 study (DisTinGuish) of DKN-01 in combination with tislelizumab + chemotherapy as first-line (1L) therapy in patients with advanced gastric or GEJ adenocarcinoma (GEA).

Presenter: Samuel J. Klempner, Harvard Medical School

Session Type: Poster Discussion Session

Session Title: Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary

Date and Time: Monday, June 5, 2023, at 11:30 a.m. ET

Abstract Number: 4027

Poster Number: 335

· Announced completion of enrollment in Part A of the DeFiance Study of DKN-01 for the treatment of colorectal cancer patients. The DeFianCe study (NCT05480306) is a Phase 2, randomized, open-label, multicenter study of DKN-01 in combination with standard of care bevacizumab and chemotherapy in patients with advanced CRC who have received one prior systemic therapy for advanced disease. The study began with an initial Part A cohort that has enrolled 33 patients and is designed to expand into a 130-patient Part B randomized controlled trial. The primary objective is progression free survival. Secondary objectives include overall response rate, duration of response, and overall survival. Leap expects to report initial data from Part A of the study in mid-2023.

Selected First Quarter 2023 Financial Results

Net Loss was $41.9 million for the first quarter 2023, compared to $10.4 million for the same period in 2022. The increase was primarily due to in-process research & development (IPR&D) expense of $29.6 million associated with the acquisition of Flame Biosciences.

Research and development expenses were $38.9 million for the first quarter 2023, compared to $7.8 million for the same period in 2022. The increase in research and development expenses was due to IPR&D expense associated with the Flame acquisition of $29.6 million, increased headcount and compensation expense of $0.8 million, increased manufacturing costs of $0.8 million, increased stock based compensation expense of $0.1 million, partially offset by decreased clinical trial costs of $0.2 million.

General and administrative expenses were $3.8 million for the first quarter 2023, compared to $2.8 million for the same period in 2022. The increase in general and administrative expenses was due to increased finance and legal fees, primarily associated with the Flame acquisition, of $0.7 million and increased headcount and compensation expense of $0.3 million.

Cash and cash equivalents totaled $102.0 million at March 31, 2023. Research and development incentive receivables totaled $2.3 million at March 31, 2023.

On May 15, 2023 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported that new preclinical data on its IPH6501 tetra-specific ANKET (Antibody-based Natural Killer cell Engager Therapeutics) have been selected for oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2023 congress, being held 8 to 15 June 2023 in Frankfurt, Germany (Press release, Innate Pharma, MAY 15, 2023, View Source [SID1234631725]).

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ANKET is Innate’s proprietary platform for developing next-generation, multi-specific natural killer (NK) cell engagers to treat certain types of cancer. This fit-for-purpose technology is creating an entirely new class of molecules to induce synthetic immunity against cancer. Innate’s latest innovation, the tetra-specific ANKET, is the first NK cell engager technology to engage activating receptors (NKp46 and CD16), a tumor antigen and the interleukin-2 receptor (via a non-alpha IL-2 variant, IL-2v) via a single molecule. By providing proliferation and activation signals targeted to NK cells, tetra-specific ANKET leverages the advantages of harnessing NK cell effector functions against cancer cells.

IPH6501, the first tetra-specific ANKET, continues toward a Phase 1 clinical trial in 2023.

Presentation details

Presentation: S258

Title: IPH6501 is a first-in-class tetraspecific antibody-based natural killer cell engager therapeutic developed for the treatment of B-cell non-Hodgkin’s lymphomas

Session: s425 Gene therapy and immunotherapy – Biology & translational research

Date and time: 10/06/2023, 11:30 – 12:45

Location: Fantasie

Speaker: Olivier Demaria, PhD, R&D Director, Science Leader at Innate Pharma

On May 15, 2023 Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (Inhibikase or Company), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson’s disease ("PD"), Parkinson’s-related disorders and other diseases of the Abelson Tyrosine Kinases, reported financial results for the first quarter ended March 31, 2023 and highlighted recent developments (Press release, Inhibikase Therapeutics, MAY 15, 2023, View Source [SID1234631724]).

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"With a clear clinical path ahead, we are focused on executing across our pipeline portfolio in 2023," said Dr. Milton H. Werner, President and Chief Executive Officer of Inhibikase. "Our recent industry presentations and publications have highlighted the exciting promise of our lead IkT-148009 program in neurodegenerative disease. We began screening patients in April at 7 sites for our Phase 2 ‘201’ trial for Parkinson’s disease and anticipate up to fourteen sites to be screening patients by the end of May. In addition, we are rapidly advancing our ‘501’ bioequivalence study for IkT-001Pro for Stable-Phase CML and anticipate commencing the confirmatory analysis portion of the study in June 2023. We look forward to providing updates on both our clinical and preclinical efforts later this year."

Recent Developments and Upcoming Milestones:

Actively screening patients across multiple sites in the Phase 2 ‘201’ Clinical Trial of IkT-148009 for the Treatment of Parkinson’s Disease: The ‘201’ trial is a 1:1:1:1 randomized, double-blind, twelve-week dosing trial intended to assess the safety, tolerability and steady-state pharmacokinetics of IkT-148009 as primary endpoints. Inhibikase plans to enroll 120 patients with untreated Parkinson’s Disease (Hoehn & Yahr < 3.0) who have yet to require symptomatic therapy. The study will evaluate three doses of IkT-148009 on a staggered schedule with 50 and 100 mg doses preceding 200 mg enrollment. The trial will also measure a hierarchy of fifteen Parkinson’s-related disease assessments in the brain and gut as secondary or exploratory endpoints. Thirty-six clinical sites have now been selected with 27 fully contracted. Up to 14 sites could be actively screening patients by the end of May 2023.

In March 2023, Inhibikase completed an evaluation of safety and steady-state pharmacokinetic (PK) profile of the 200 mg dose of IkT-148009 in six healthy volunteers and submitted the data to the FDA in April 2023. The Company is completing an ethics committee review at all sites to add this dose into the ‘201’ trial.

Completed Dose escalation portion of the ‘501’ bioequivalence study of IkT-001Pro: IkT-001Pro is the Company’s prodrug formulation of imatinib mesylate intended to enhance the safety and efficacy of imatinib (marketed as Gleevec) in patients with Chronic Myelogenous Leukemia (CML). In May 2023, Inhibikase completed dosing of the four dose escalation cohorts evaluating 300, 400 and 500 and 600 mg IkT-001Pro. As the Company completes the pharmacokinetic analysis of these four cohorts, Inhibikase anticipates identifying the dose of IkT-001Pro that delivers the equivalent dose of commercial 400 mg imatinib mesylate. IkT-001Pro has shown a favorable safety profile, with fewer adverse events observed relative to 400 mg imatinib mesylate and none of clinical significance. Inhibikase anticipates commencing the confirmatory analysis of the bioequivalent dose of IkT-001Pro in thirty-two additional healthy volunteers using a two-period crossover design in June 2023. The Company expects to complete this confirmatory analysis by the end of the second quarter 2023. Inhibikase is also considering the addition of a cohort that will measure bioequivalence for high-dose imatinib delivered by prodrug that is equivalent to 600 mg imatinib mesylate to further explore the safety benefit of IkT-001Pro over standard-of-care, subject to agreement with the FDA.

Advancing preclinical development of IkT-148009 in MSA: In March 2023, Inhibikase announced that the Investigational New Drug (IND) application for the Phase 2 trial of IkT-148009 in MSA was opened. An ongoing MSA animal model study has shown that pre-exposure prophylaxis of IkT-148009 precludes loss of function in a transgenic model of MSA; an independent model is running concurrently with the transgenic model to confirm the apparent functional benefit of IkT-148009 treatment. These studies will form the basis for determining the timing for initiation of a planned Phase 2 clinical study of IkT-148009 in MSA.
First Quarter Financial Results

Net Loss: Net loss for the quarter ended March 31, 2023 was $4.5 million, or $0.16 per share, compared to a net loss of $4.6 million, or $0.18 per share in the quarter ended March 31, 2022.

R&D Expenses: Research and development expenses were $2.9 million for the quarter ended March 31, 2023 compared to $3.0 million in the quarter ended March 31, 2022. The decrease was primarily due to the company restarting its Phase 2 ‘201’ clinical trial.

SG&A Expenses: Selling, general and administrative expenses for the quarter ended March 31, 2023 were $1.9 million compared to $1.7 million for the quarter ended March 31, 2022. The increase was primarily the result of legal, consulting fees and promotional related costs.

Cash Position: Cash and cash equivalents and marketable securities were $25.7 million as of March 31, 2023. This includes the net proceeds from the Company’s $10 million January 2023 concurrent registered direct offering and private placement. The Company expects that existing cash and cash equivalents will be sufficient to fund operations into the fourth quarter of 2024.

Conference Call Information
The conference call is scheduled to begin at 8:00am ET on May 16, 2023. Participants should dial 1-844-825-9789 (United States) or 1-412-317-5180 (International) with the conference code 8552474. A live webcast may be accessed using the link here, or by visiting the investors section of the Company’s website at www.inhibikase.com. After the live webcast, the event will be archived on Inhibikase’s website for approximately 90 days after the call.