On March 24, 2020 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with an initial focus on myelodysplastic syndromes (MDS), reported financial results for the year ended December 31, 2019 and provided a corporate update (Press release, Onconova, MAR 24, 2020, View Source [SID1234555787]).

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"Following significant progress throughout 2019, our team’s immediate focus remains to advance our pivotal Phase 3 INSPIRE trial to the next milestone," said Steven M. Fruchtman, M.D., President and Chief Executive Officer. "We are excited to announce the completion of the planned enrollment of 360 patients in the INSPIRE trial in higher-risk (HR) MDS patients. INSPIRE represents one of the largest studies ever conducted in this patient population. Based on survival trends on the INSPIRE trial to date, we anticipate reporting topline survival data in the second half of 2020 and presenting the results at a medical meeting later this year. We continue to analyze deep genomic sequencing, including mutations of the Ras pathway, on these patients. Important genomic results were presented at ASH (Free ASH Whitepaper) 2019 and additional data is expected to be presented at future medical meetings."

Dr. Selina Luger, M.D., Professor of Medicine at the Hospital of the University of Pennsylvania Division of Hematology Oncology, commented, "in second line HR MDS, following progression or failure of patients to respond to hypomethylating agents, there is no standard of care and currently no health authority approved agent in the world. There is a tremendous unmet medical need for these patients. The INSPIRE trial, which has now reached full accrual, is studying the experimental agent rigosertib to determine if it can significantly prolong the survival of these patients when compared to physician’s choice of therapy. We eagerly look forward to the results of this pivotal trial."

Dr. Fruchtman continued, "We are also pleased about the progress of our plans for additional investigator-initiated studies – including the Phase 1/2a study of rigosertib plus nivolumab for the treatment of Stage IV KRAS mutated lung adenocarcinoma. We anticipate the first patient to be entered onto the trial once the COVID-19 environment improves sufficiently."

Fourth Quarter 2019 and Additional Achievements

Knight Therapeutics License for Rigosertib in Canada
Specialised Therapeutics License for Rigosertib in Australia and New Zealand
Inceptua Medicines Pre-approval Access Collaboration for Rigosertib in Selected Countries Outside the US
Re-acquired Rigosertib Rights in Greater China
Next generation CDK 4/6 + ARK5 inhibitor, ON123300, IND approved in China
Additional Milestones for Rigosertib and Pipeline Products

Clinical trial protocol progressing for a randomized Phase 2/3 study of the combination of oral rigosertib plus azacitidine
Expansion of the rigosertib investigator-initiated studies program to include KRAS mutated non-small cell lung cancer, melanoma and other RAS mutated-driven cancers
Next generation CDK 4/6 + ARK5 inhibitor, ON123300, US IND submission planned in 4Q 2020
Pivotal survival data from the INSPIRE trial expected in 2H 2020
Full Year 2019 Financial Results

Cash and cash equivalents as of December 31, 2019, totaled $22.7 million, compared to $17.0 million as of December 31, 2018. Besides the $9 million of net proceeds from the financing closed in early 2020, common stock warrant exercises since 12/31/19 have added $5.7 million to the Company’s cash balance resulting in a cash balance at February 29, 2020 of $32.6 million. Based on current projections, the Company expects that cash and cash equivalents will be sufficient to fund ongoing trials and operations into the third quarter of 2021.

Net loss was $21.5 million for the year ended December 31, 2019, compared to $20.4 million for the year ended December 31, 2018. Research and development expenses were $15.5 million for the year ended December 31, 2019 and $16.9 million for the comparable period in 2018. General and administrative expenses were $8.3 million for the year ended December 31, 2019 and $7.6 million for the comparable period in 2018.

Conference Call and Webcast Information

The Company will host a conference call today, March 24, 2020, at 4:30 p.m. Eastern Time, to provide a corporate update and discuss year-end 2019 financial results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the U.S., or internationally (210) 229-8823 and using conference ID 3267259. The call will also be webcast live. Please click here to access the webcast. A replay will be available following the live webcast.

On March 24, 2020 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported a favourable assessment from the Safety Committee following review of data from the window of opportunity study in early-stage breast cancer, known as AWARE-1 (Press release, Oncolytics Biotech, MAR 24, 2020, View Source [SID1234555786]). Consistent with the safety run-in with patients receiving pelareorep and Tecentriq, Cohort 1 demonstrated widespread viral replication in the majority of tumors with the creation of a pro-inflammatory effect in the tumor microenvironment. No negative effects to healthy tissue were noted.

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The Committee evaluated safety parameters from patients participating in the safety run-in phase of the trial, consisting of select patients from cohorts 2 and 3, along with the fully enrolled cohort 1, and determined there were no safety concerns. The Committee also approved an amendment of the study to reduce the dose of Tecentriq to be consistent with the currently approved breast cancer dose of 840mg. The study will continue to enroll patients and the Safety Committee will meet again for an additional pre-planned meeting. Cohorts 1 and 2 represent our target tumor type of HR+ / HER2- and data from these patients will inform the design of the planned phase 3.

"After reviewing the totality of safety data, including patients receiving pelareorep plus the standard of care and those also receiving Tecentriq, the Safety Committee for AWARE-1 confirmed no significant toxicity resulting from treatment," said Dr. Rita Laeufle, Chief Medical Officer at Oncolytics Biotech. "The study is continuing as planned, recruiting additional patients and examining the combination of pelareorep, plus the standard of care plus Tecentriq. We look forward to presenting updated data at the ESMO (Free ESMO Whitepaper) Breast Cancer conference in May, which will describe meaningful changes to the tumor microenvironment, evidence of tumor infection, and of course, our biomarker correlated to immunogenic response and viral replication."

About AWARE-1
AWARE-1 is an open label window-of-opportunity study in early stage breast cancer enrolling 38 patients into five cohorts:

Cohort 1 (n=10), HR+ / HER2- (pelareorep + letrozole)

Cohort 2 (n=10), HR+ / HER2- (pelareorep + letrozole + atezolizumab)

Cohort 3 (n=6), TNBC (pelareorep + atezolizumab)

Cohort 4 (n=6), HR+ / HER2+ (pelareorep + trastuzumab + atezolizumab)

Cohort 5 (n=6), HR- / HER2+ (pelareorep + trastuzumab + atezolizumab)

The study combines pelareorep with the standard of care according to breast cancer subtype and atezolizumab. Patients are biopsied on day one followed immediately by treatment, then again on day three, and a final biopsy after three weeks, on the day of their mastectomy. Data generated from this study is intended to confirm that the virus is acting as a novel immunotherapy and to provide comprehensive biomarker data by breast cancer sub-type. The primary endpoint of the study is overall CelTIL (a measurement of cellularity and tumor-infiltrating lymphocytes). Secondary endpoints for the study include CelTIL by breast cancer subtype, safety and tumor, and blood-based biomarkers.

The study is being coordinated by Dr. Aleix Prat, Head of Medical Oncology at the Hospital Clínic of Barcelona, Associate Professor of the University of Barcelona and the Head of the Translational Genomics and Targeted Therapeutics in Solid Tumors Group at August Pi i Sunyer Biomedical Research Institute (IDIBAPS) and member of Oncolytics’ Scientific Advisory Board.

About Breast Cancer
Breast cancer is the most common cancer in women worldwide, with over two million new cases diagnosed in 2018, representing about 25 percent of all cancers in women. Incidence rates vary widely across the world, from 27 per 100,000 in Middle Africa and Eastern Asia to 85 per 100,000 in Northern America. It is the fifth most common cause of death from cancer in women globally, with an estimated 522,000 deaths.
Breast cancer starts when cells in the breast begin to grow out of control. These cells usually form a tumor that can often be seen on an x-ray or felt as a lump. The malignant tumor (cancer) is getting worse when the cells grow into (invade) surrounding tissues or spread (metastasize) to distant areas of the body.

About Pelareorep
Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.

On March 24, 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 that Walter Klemp, Chairman and Chief Executive Officer, will present at the Life Sciences Investor Forum being held online at LifeSciencesInvestorForum.com on March 26th (Press release, Moleculin, MAR 24, 2020, View Source [SID1234555785]).

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Details of the presentations are below:

Event: Life Sciences Investor Forum
Date: March 26, 2020
Time: 11:30 – 12:00 PM ET
Link: View Source
This will be an interactive online event where investors are invited to ask the company questions in real-time. If attendees are not able to join on the day of the conference, an archived webcast will also be made available after the event.

It is recommended that investors pre-register and run the online system check to expedite participation and receive event updates.

On March 24, 2020 Gossamer Bio, Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics in the disease areas of immunology, inflammation and oncology, reported its financial results for the fourth quarter and year ended December 31, 2019 and provided a business update (Press release, Gossamer Bio, MAR 24, 2020, View Source [SID1234555784]).

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"Our hearts are with the patients, families, caregivers and medical professionals suffering and sacrificing in the ongoing Covid-19 viral pandemic. We are monitoring the situation on a daily basis to understand the impact on Gossamer and our programs and are taking the necessary actions now to do what is best for our patients, employees and company," said Sheila Gujrathi, M.D., Co-Founder and Chief Executive Officer of Gossamer Bio.

"2019 was a year of execution for Gossamer Bio, as we continued to advance all four of our clinical-stage product candidates in our target areas of immunology, inflammation and oncology. Notwithstanding the Covid-19 pandemic, we expect to continue our momentum in 2020, with data from all of our candidates expected this year. We are committed to advancing our product candidates and the field of medicine for the betterment of patients and their families, and we look forward to providing updates on these efforts throughout the year."

Clinical-Stage Product Candidate Updates

GB001: Oral DP2 Antagonist for Eosinophilic Asthma and Chronic Rhinosinusitis (CRS)

Gossamer has made available three poster presentations from its GB001 program for patients with asthma. All three posters, which Gossamer had planned to present at the now cancelled 2020 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting, can be found in the Posters and Publications section of Gossamer’s website:

"Effect of the DP2 Antagonist GB001 on Asthma Worsening in Patients with Mild-Moderate Asthma" showed that GB001 treatment is associated with longer time to asthma worsening / exacerbation and observed treatment effects were greater in the populations with higher baseline FeNO and / or eosinophils.

"In Vitro and In Vivo Profile of GB001, a Potent and Selective DP2 Antagonist for the Treatment of Moderate-Severe Asthma" demonstrates that, in pre-clinical studies,

GB001 is an insurmountable antagonist and compares favorably to other DP2 antagonists in functional residence time and prolonged pharmacodynamic effects, while inhibiting immune cell infiltration and improving airway function.

"Corticosteroid Use Across Asthma Healthcare Providers: A Real-world Experience." The widespread use of OCS revealed by this claims analysis underscores the high level of unmet need for these patients and the need for new therapies.

Enrollment in the ongoing Phase 2b LEDA study in patients with moderate-to-severe eosinophilic asthma has been completed. We are on track to conduct an interim analysis in the second quarter of this year, following study completion by approximately two thirds of patients. Topline results are expected in the second half of this year.

Enrollment in the ongoing Phase 2 TITAN proof-of-concept study in chronic rhinosinusitis, both with and without nasal polyps has been completed. Topline data from the TITAN study are expected in the second half of this year.

We continue to evaluate the possibility of initiating a Phase 2 study in chronic spontaneous urticaria and expect to make this decision in the second half of the year following a review of available data and the competitive landscape.

GB002: Inhaled PDGFR Inhibitor for Pulmonary Arterial Hypertension (PAH)

Enrollment is underway in the Phase 1b study of GB002 in patients with PAH. Gossamer expects to report initial topline results from the study in the second quarter of this year.

Due to the ongoing Covid-19 viral pandemic, the Phase 2 study in patients with PAH will likely commence in the second half of this year. This trial will enroll functional class II and III PAH patients. Patients will remain on their background therapies throughout the study. The primary endpoint for this 24-week study will be change in PVR from baseline. A key secondary endpoint will be change from baseline in 6-minute walk distance at week 24.

GB004: Oral HIF-1α Stabilizer for Inflammatory Bowel Disease

Enrollment is complete in the Phase 1b study of GB004 in patients with active mild-to-moderate ulcerative colitis (UC). The primary goals of the study are to assess safety, tolerability, PK, PD and target engagement in patients with active disease. Gossamer expects to report topline results from this Phase 1b study in the second quarter of this year.

GB1275: Oral CD11b Modulator for Oncology Indications

Enrollment for the KEYNOTE-A36 Phase 1/2 study to evaluate GB1275 as a monotherapy and in combination with either KEYTRUDA (pembrolizumab) or chemotherapy in patients with selected advanced solid tumors is underway, and we expect to report initial Phase 1 data in the second half of this year.

Financial Results for Quarter and Full Year Ended December 31, 2019

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of December 31, 2019, were $401.8 million. In response to the ongoing Covid-19 viral pandemic and anticipated potential challenges to clinical trials globally, Gossamer has planned a series of cost-optimization initiatives. As a result, we currently expect cash, cash equivalents and marketable securities, and access to our debt facility will be sufficient to fund operating and capital expenditures to the middle of 2022.

Research and Development (R&D) Expenses: For the quarter ended December 31, 2019, R&D expenses were $42.6 million, compared to R&D expenses of $25.9 million for the same period in 2018. R&D expenses for the full year ended December 31, 2019, were $143.4 million compared to $55.3 million for the full year ended December 31, 2018. The increases were primarily due to an increase in expenses for GB001, GB002, GB004 and GB1275 and increased headcount.

In-Process Research and Development (IPR&D) Expenses: For the quarter ended December 31, 2019, IPR&D expenses were $1.6 million, compared to $0.0 million for the same period in 2018. IPR&D expenses for the full year ended December 31, 2019, were $3.6 million compared to $49.7 million for the full year ended December 31, 2018.

General and Administrative (G&A) Expenses: For the quarter ended December 31, 2019, G&A expenses were $11.6 million, compared to $13.9 million for the same period in 2018. G&A expenses for the full year ended December 31, 2019, were $39.1 million compared to $44.1 million for the full year ended December 31, 2018. The decreases were primarily attributable to a decrease in stock-based compensation costs, which was partially offset by increases in personnel-related costs, professional and legal fees, costs associated with insurance, and facility and office-related costs.

Net Loss: Net loss for the three months ended December 31, 2019, was $54.7 million, or $0.89 per share, compared to a net loss of $38.8 million, or $4.92 per share, for the same period in 2018. Net loss for the full year ended December 31, 2019, was $180.3 million, or $3.29 per share compared to a net loss of $147.0 million, or $22.59 per share, for the full year ended December 31, 2018.

On March 24, 2020 Laekna Therapeutics Shanghai Co., Ltd. (Laekna) reported a new agreement with Novartis giving the China-based biotech company exclusive global rights to Novartis’ anti-PD-L1 Antibody (FAZ053).

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FAZ053 has been involved in a phase I clinical trial in patients with advanced cancer. The trial demonstrated positive proof-of-concept results for a tolerable safety profile and clinical efficacy of FAZ053 in diverse solid tumors. Laekna, a company dedicated to the research and development of small-molecule and antibody-targeted drugs, now has exclusive rights to develop and commercialize the PD-L1 oncology asset. With this move, the company aims to advance clinical research and introduce pioneering new drugs to patients, including the rapidly growing cancer patient population in China.

"A combination of PD-L1 Antibody with chemotherapy, or targeted therapy can greatly improve treatment response and prolong survival," said Dr. Yong Yue, CMO of Laekna. "We have mapped a number of combination strategies to treat a variety of cancers that do not respond or are resistant to checkpoint inhibitor treatments."

Laekna plans to start trials on combination therapies as soon as possible in order to address the unmet needs of people with cancer and bring innovative, effective treatments to more cancer patients across China.

This is the third in a recent series of agreements between Laekna and Novartis. "Novartis is a global leader in oncology drug innovation and we believe that the three oncology assets we have acquired from Novartis have great synergistic anti-cancer effects," said Dr. Chris Lu, founder and CEO of Laekna. "This synergy will allow us to apply various combinations to treat many cancer indications, including triple negative breast cancer and prostate cancer."

As part of this agreement, Novartis will receive an upfront payment, with additional payments at key development milestones as well as royalties on future sales.