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Onconova Therapeutics Provides Corporate Update and Reports First Quarter 2020 Financial Results

On May 14, 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 quarter ended March 31, 2020, and provided a business update (Press release, Onconova, MAY 14, 2020, View Source [SID1234558040]).

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"With enrollment completed in March, Onconova’s pivotal Phase 3 INSPIRE trial is advancing to the next catalyst. We are fortunate to have achieved full enrollment of INSPIRE prior to the pandemic-driven disruptions to research studies at hospitals and cancer centers across the globe," said Steven M. Fruchtman, M.D., President and Chief Executive Officer. "Based on survival trends in the INSPIRE trial, we continue to anticipate reporting topline survival data in the second half of 2020. We expect to present the results of this trial at a major medical meeting later this year."

Dr. Fruchtman continued, "Beyond INSPIRE, we are primed for additional progress, including the to be initiated Phase 1/2a study of rigosertib plus nivolumab in Stage IV KRAS mutated lung adenocarcinoma, following the renewal of clinical cancer research programs post their COVID-mandated stoppage, as well as additional planned studies of rigosertib and our pipeline programs. We are preparing for and look forward to multiple corporate milestones in the second half of 2020."

First Quarter 2020 Developments and Recent Highlights

Completed enrollment of the pivotal Phase 3 INSPIRE trial
Opened investigator-initiated study of rigosertib plus nivolumab in Stage IV KRAS mutated lung adenocarcinoma
Re-acquired rights to rigosertib in Greater China
Next generation CDK 4/6 + ARK5 inhibitor, ON123300, IND approved in China
Nominated life sciences industry veteran Terri Shoemaker to the Company’s Board of Directors
Additional Upcoming Company Milestones Expected

Pivotal survival data from the INSPIRE trial expected in 2H 2020
European Hematology Association Virtual Congress presentation in June 2020:
Mutations in RAS Pathway Genes Correlates with Type of Failure to Azacitidine: Genomic Analysis at Randomization onto the Inspire Trial (EHA-4044)
Type C meeting to be requested in 2Q 2020 for a randomized Phase 2/3 study of the combination of oral rigosertib plus azacitidine
Expansion of the rigosertib investigator-initiated 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 for 4Q 2020, and Phase 1 study commencement in China planned for 2H 2020
Anticipated launch of Early Access Program with Inceptua Medicines Group in 2H 2020
First Quarter 2020 Financial Results
Cash and cash equivalents as of March 31, 2020, totaled $31.0 million, compared to $22.7 million as of December 31, 2019. Common stock warrant exercises since our financing transaction in November 2019 have added $10.6 million to our balance sheet. Of the almost 29 million common stock warrants outstanding as of March 31, 2020, over 80% of them were in-the-money as of May 13th. Based on current projections, the Company continues to expect that its cash and cash equivalents as of March 31, 2020 will be sufficient to fund ongoing trials and operations into the third quarter of 2021.

Net loss was $5.1 million for the quarter ended March 31, 2020, compared to $7.6 million for the quarter ended March 31, 2019. Research and development expenses were $3.4 million for the quarter ended March 31, 2020 and $4.1 million for the comparable period in 2019. General and administrative expenses were $1.8 million for the quarter ended March 31, 2020 and $3.2 million for the comparable period in 2019.

Conference Call and Webcast Information
The Company will host a conference call today, May 14, 2020, at 4:30 p.m. Eastern Time, to provide a corporate update and discuss first quarter 2020 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 3488818. The call will also be webcast live. Please click here to access the webcast. A replay will be available following the live webcast.

To facilitate an on-time conference call start, Onconova recommends that participants dial in 15 minutes before the 4:30 p.m. ET start time.

Oncolytics Biotech® Announces Publication of Abstracts at the 2020 American Society of Clinical Oncology Virtual Annual Meeting

On May 14, 2020 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported the publication of two abstracts in connection with the upcoming ASCO (Free ASCO Whitepaper) Virtual Annual Meeting on May 29-31 (Press release, Oncolytics Biotech, MAY 14, 2020, View Source [SID1234558039]). The first abstract "("Multiple Myeloma Abstract"), which has been accepted as an electronic poster, reports on viral replication, tumor immune responses, and treatment safety in multiple myeloma patients treated with pelareorep in combination with carfilzomib (Kyprolis). The second abstract ("Pancreatic Cancer Abstract") reports on treatment tolerability and efficacy in pancreatic cancer patients treated with pelareorep, in combination with pembrolizumab (Keytruda).

Multiple Myeloma Abstract
Abstract ID: 8535
Session: Hematologic Malignancies-Plasma Cell Dyscrasia
Poster ID: 435
Abstract Title: Oncolytic virus pelareorep plus carfilzomib phase I trial in carfilzomib-refractory patients (NCI 9603): Responses with cytokine storm
Presenter: Craig Hofmeister, M.D., MPH, Associate Professor, Department of Hematology and Medical Oncology Emory University School of Medicine

•Reported selective infection of cancerous cells with pelareorep, with associated CD8+ and natural killer (NK) cell recruitment, PD-L1 upregulation, activated caspase-3 expression, and increased pelareorep protein production within myeloma cells.
•Two patients achieved unconfirmed partial responses, two patients had stable disease, and two patients had progressive disease. All patients had advanced and challenging to treat carfilzomib-refractory disease.
•Of the two responding patients, one patient developed a fever and grade 4 thrombocytopenia that resolved, and the other patient developed a cytokine storm, associated with rapid tumor lysis.
•Data supports the potential of future studies investigating pelareorep, carfilzomib, and immune checkpoint inhibitor combination therapy in multiple myeloma patients.
•Additional data to be presented at the poster session on May 29, 2020.

Pancreatic Cancer Abstract
Abstract ID: e16789
Abstract Title: Pembrolizumab in combination with the oncolytic virus pelareorep in patients progressing on systemic chemotherapy for advanced pancreatic adenocarcinoma, a Phase II study
Presenter: Devalingam Mahalingam, MD, Ph.D., Associate Professor of Medicine at Northwestern University Feinberg School of Medicine and a member of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University

•Preliminary data indicate that the combination of pelareorep and pembrolizumab resulted in tumor-specific replication, a high degree of T cell repertoire turnover, and the creation of new T cell clones in the peripheral blood during treatment.
•The treatment was found to be well tolerated, with most treatment-related adverse events being grade 1 or 2.
•One patient achieved a partial response and three achieved stable disease, with an overall disease control rate of 30% in evaluable patients.
•The study will not proceed to stage 2 in unselected patients, however further evaluation of the anti-tumor activity of pelareorep and anti-PD-1 therapy is now planned in biomarker defined pancreatic patients in a subsequent study.
•Detailed translational and biomarker data from this study will be published later in the year.

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.

Achieve Reports Financial Results for First Quarter 2020 and Provides Update on Cytisinicline Development Program

On May 14, 2020 Achieve Life Sciences, Inc. (Nasdaq: ACHV), a clinical-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine addiction, reported first quarter 2020 financial results and provided an update on the cytisinicline clinical development program (Press release, OncoGenex Pharmaceuticals, MAY 14, 2020, View Source [SID1234558038]).

Recent Highlights

Submitted required non-clinical supportive data for longer cytisinicline treatment to the U.S. Food & Drug Administration (FDA) in preparation for Phase 3 cytisinicline ORCA-2 trial initiation

Closed financing for gross proceeds of approximately $1.9 million, prior to deducting placement agent commissions and estimated offering expenses

Presented new findings from the Phase 2b ORCA-1 trial evaluating cytisinicline in U.S. smokers at the Society for Research on Nicotine & Tobacco (SRNT) Annual Conference

Established agreement with the FreeMind Group to assist in securing non-dilutive funding to evaluate cytisinicline in vapers and e-cigarette users

"The COVID-19 pandemic has revealed an increased risk for smokers due to the known relationship between smoking and respiratory illnesses and we see an even greater need to do all that we can to help them quit," commented Rick Stewart, Chairman and Chief Executive Officer of Achieve. "We expect to initiate the 750 patient Phase 3 ORCA-2 trial in the second-half of 2020, subject to financing and the ability to conduct the trial safely in light of the COVID-19 pandemic."

Completed FDA Submission

In preparation for initiation of the Phase 3 ORCA-2 clinical trial of cytisinicline, the Company has now submitted to FDA all requested non-clinical data to allow 6 and 12 weeks of cytisinicline treatment in the Phase 3 trials. No new safety or toxicology signals were observed in the non-clinical data evaluating either 13 or 26 weeks of cytisinicline treatment.

Closed private placement financing for gross proceeds of $1.9 million

In April 2020, Achieve announced it entered into definitive agreements for a private placement of its securities for gross proceeds of approximately $1.9 million, prior to deducting placement agent commissions and estimated offering expenses. The Company intends to use the proceeds to fund clinical research and development, and for general working capital.

Additional ORCA-1 Results at SRNT Annual Conference

An oral presentation featuring new ORCA-1 Phase 2b trial analyses was presented at the SRNT Annual Meeting in March 2020. In addition to previously reported data indicating a statistically significant improvement in quit rates, new analyses demonstrate cytisinicline biochemical efficacy via measurement of serum cotinine as well as the previous carbon monoxide efficacy. Additionally, further analyses confirm that cytisinicline benefit was observed across all clinical sites, baseline

characteristics, and attributes. Thus, regardless of trial site location, patient demographics, smoking history, or prior treatments, all subjects treated with cytisinicline showed similar smoking cessation benefit.

Agreement with the FreeMind Group to secure non-dilutive financing for vaping trials

In the first quarter of 2020, Achieve retained the FreeMind Group, an international consulting firm dedicated to assisting life science organizations secure non-dilutive funding. Achieve and FreeMind are conducting a strategic assessment of potential non-dilutive funding opportunities from various public and private sources, followed by anticipated grant production and submission, to further the clinical development of cytisinicline in vaping or e-cigarette cessation.

Financial Results

As of March 31, 2020, the company’s cash, cash equivalents, and restricted cash was $12.2 million. Total operating expenses for the quarter ended March 31, 2020 were $3.4 million. Total net loss for the quarter ended March 31, 2020 was $3.3 million.

As of March 14, 2020, Achieve had 37,515,408 shares outstanding.

Conference Call Details

Achieve will host a conference call at 8:00 a.m. Eastern time today, Thursday, May 14, 2020. To access the webcast, log on to the investor relations page of the Achieve website at View Source Alternatively, access to the live conference call is available by dialing (877) 472-9809 (U.S. & Canada) or (629) 228-0791 (International) and referencing conference ID 4477265. A webcast replay will be available approximately two hours after the call and will be archived on the website for 90 days.

Novartis data at ASCO and EHA showcase bold approaches to reimagine cancer and blood disorders through multiple therapeutic platforms

On May 14, 2020 Novartis reported that data from more than 110 abstracts, including Novartis-sponsored and investigator-initiated trials, will be presented during the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program and the European Hematology Association (EHA) (Free EHA Whitepaper) EHA (Free EHA Whitepaper)25 Virtual Congress (Press release, Novartis, MAY 14, 2020, View Source [SID1234558037]). The ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) meetings will be held May 29-31, and June 11-14, respectively.

"We are living in a world of uncertainty, but cancer won’t wait. Now, more than ever, we need to continue to be bold together. Our data at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) highlight our unique approach to harnessing the power of multiple treatment platforms to deliver transformative medicines to people living with cancer and blood disorders," said Susanne Schaffert, PhD, President, Novartis Oncology. "We look forward to ‘seeing’ everyone virtually at the congresses and helping participants access key data and information through our dedicated congress portals."

Key highlights of data accepted by ASCO (Free ASCO Whitepaper):

Kisqali overall survival subgroup analysis from MONALEESA-3 and -7 trials, and results on Piqray plus fulvestrant in ABC patients with a PIK3CA mutation from the BYLieve study:
Overall survival in patients with ABC with visceral metastases (mets), including those with liver mets, treated with ribociclib plus endocrine therapy in the MONALEESA-3 and -7 trials [Abstract # 1054; poster 139]
Alpelisib + fulvestrant in patients with PIK3CA-mutated HR+/HER2- ABC previously treated with cyclin-dependent kinase 4/6 inhibitor + aromatase inhibitor: BYLieve Study Results [Abstract #1006; oral presentation]

Five-year Tafinlar+Mekinist data in the adjuvant treatment of BRAFV600-mutated melanoma, and updated data in combination with immune checkpoint inhibitor spartalizumab (PDR001):
Long-term benefit of adjuvant dabrafenib+trametinib in patients with resected stage III BRAF V600–mutant melanoma: 5-year analysis of COMBI-AD [Abstract #10001; oral presentation]
The anti-PD-1 antibody spartalizumab in combination with dabrafenib and trametinib in advanced BRAF V600-mutant melanoma: efficacy and safety findings from Parts 1 and 2 of the Phase III COMBI-i trial [Abstract #10028; poster 377]

Tabrecta data updates from multiple analyses from the GEOMETRY study among patients with METex14-mutated and MET-amplified advanced non-small cell lung cancer (NSCLC):
Capmatinib in patients with METex14-mutated advanced NSCLC who received prior immunotherapy: results from the Phase 2 GEOMETRY Mono-1 study [Abstract #9509; oral presentation]
Capmatinib in patients with METex14-mutated or high MET-amplified advanced NSCLC: results from Cohort 6 of the phase 2 GEOMETRY mono-1 study [Abstract #9520; poster 286]

Safety data from a US expanded access program with radioligand therapy Lutathera (lutetium Lu 177 dotatate)*** in patients with advanced neuroendocrine tumors (NETs):
Safety of 177Lu‑DOTATATE in patients with advanced NETs: data from a US expanded access program [Abstract #4604; poster 212]

Learnings from the inclusion of patient insights in the research and development process, through the Novartis Global Oncology Patient Insight Panels (GOPIPs):
Patient engagement in clinical trial design and implementation: A pragmatic approach to valued insights [Abstract #e14084; online publication]
In addition, TheraP, sponsored by the Australian & New Zealand Urogenital and Prostate (ANZUP) Cancer Trials Group, comparing investigational radioligand therapy 177Lu-PSMA-617 to cabazitaxel in patients with metastatic castration resistant prostate cancer (mCRPC) progressing after docetaxel, will be presented:

TheraP: A randomised phase II trial of LuPSMA theranostic versus cabazitaxel in mCRPC progressing after docetaxel: Initial results (ANZUP protocol 1603) [Abstract #5500; oral presentation]
Key highlights of data accepted by EHA (Free EHA Whitepaper):

Efficacy and safety data on the investigational anti-TIM-3 monoclonal antibody MBG453, which targets both immune and myeloid cells in patients with high-risk myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML):
Anti-TIM-3 antibody MBG453 in combination with hypomethylating agents in patients with high-risk MDS and AML: a Phase 1 study [Abstract #S185; oral presentation; Sunday, June 14, 8:00 AM CEST]

A longer term Phase I safety and efficacy analysis of asciminib, an investigational treatment specifically targeting the BCR-ABL myristoyl pocket (STAMP), in heavily pre-treated patients with chronic myeloid leukemia (CML):
Asciminib in heavily pretreated patients with Philadelphia chromosome-positive (Ph+) CML in chronic phase sensitive to TKI therapy [Abstract #S170; oral presentation; Friday, June 12, 8:30 AM CEST]

Efficacy results in patients with acute graft-versus-host disease (GvHD) treated with Jakavi (ruxolitinib)****:
Ruxolitinib versus best available therapy in patients with steroid-refractory acute GvHD: overall response rate by baseline characteristics in the randomized Phase 3 REACH2 trial [Abstract #S255; oral presentation; Friday, June 12, 8:00 AM CEST]
More information, including the list of Novartis-sponsored abstracts, and access to the presentations for registered participants will be available on View Source, starting on May 28, and on View Source, by June 11.

NeuBase Therapeutics Reports Financial Results for the Second Fiscal Quarter of 2020

On May 14, 2020 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies using its scalable PATrOL platform to address genetic diseases, reported its financial results for the three and six month periods ended March 31, 2020 (Press release, NeuBase Therapeutics, MAY 14, 2020, View Source [SID1234558036]).

"We have generated a solid foundation of data that we expect will enable us to produce mutation-specific genetic medicines for a multitude of diseases. The positive non-human primate pharmacokinetic (biodistribution) and patient-derived cell line pharmacodynamic (activity) data released on March 31st illustrate our ability to create a new class of mutation silencing therapies. In addition, our proprietary targeting technology enables our PATrOL compounds to achieve therapeutically relevant drug concentrations throughout the body, including in the brain," said Dietrich A. Stephan, Ph.D., chief executive officer of NeuBase.

"Our therapeutic development programs continue to drive forward, despite the difficult macroenvironment, as evidenced by the recent positive platform validation data and well-received public offering. As we look ahead, our initial focus is to continue advancing our Huntington’s disease ("HD") and myotonic dystrophy type 1 ("DM1") programs through lead optimization and IND-enabling studies. We expect to announce the lead candidate for the NT0100 Program for HD by the end of calendar year 2020, followed by the initiation of IND-enabling studies during the first half of calendar year 2021. Finally, our unique biodistribution profile allows us the opportunity to develop therapies against targets and organ systems that we believe other antisense companies cannot currently reach in the body, including into the brain after systemic delivery, which is one of the grand challenges in drug delivery of macromolecules. We believe this unique ability validates our large opportunity in the antisense space," continued Dr. Stephan.

Second Fiscal Quarter of 2020 and Recent Operating Highlights

·Announced positive, preclinical pharmacokinetic ("PK") and pharmacodynamic ("PD") data validating the first-in-class genetic therapy PATrOL platform:
oPK studies of the PATrOL-enabled compound in non-human primates (NHPs) demonstrated, among other things, rapid uptake of the compound out of the body’s circulation after systemic intravenous administration, penetration by the compound into every organ and tissue system studied, and retention of therapeutically relevant doses for greater than one week after single-dose injection;
oPD studies in patient-derived cell lines demonstrated, among other things, activity in engaging target disease-causing transcripts and knocking-down resultant malfunctioning mutant protein levels preferentially over normal protein knock-down; and dose-limiting toxicities were not observed relative to a control either at or above the doses demonstrating activity in human cells in vitro; and
oPATrOL enabled compounds were generally well-tolerated in vivo after systemic administration, both after single dose administration in NHPs and multi dose administration in mice for over a month.

Financial Results for the Fiscal Quarter Ended March 31, 2020:

·At March 31, 2020, the Company had cash and cash equivalents of approximately $5.8 million, compared with cash and cash equivalents of approximately $10.3 million at September 30, 2019. Subsequent to the end of the fiscal second quarter of 2020, the Company completed a public equity offering that generated net proceeds of approximately $33.3 million. The Company believes that its current cash balance will provide sufficient capital to fund operations into the second calendar quarter of 2022;
·For the three month period ended March 31, 2020, the Company reported a net loss of approximately $4.4 million, or a net loss of $0.26 per share, compared with a net loss of approximately $2.0 million, or a net loss of $0.33 per share, for the same period last year; and
·For the three month period ended March 31, 2020, total operating expenses were approximately $4.4 million, consisting of approximately $2.8 million in general and administrative expenses and $1.6 million of research and development expenses. This compares with total operating expenses of $1.9 million for the same period last year, which was comprised of approximately $1.9 million in general and administrative expenses and $0.03 million in research and development expenses.

Financial Results for the Six Month Period Ended March 31, 2020:

·For the six month period ended March 31, 2020, the Company reported a net loss of approximately $8.9 million, or a net loss of $0.52 per share, compared with a net loss of approximately $3.5 million, or a net loss of $0.59 per share, for the same period last year; and
·For the six month period ended March 31, 2020, total operating expenses were approximately $8.1 million, consisting of approximately $5.3 million in general and administrative expenses and $2.8 million of research and development expenses. This compares with total operating expenses of $3.4 million for the same period last year, which was comprised of approximately $2.3 million in general and administrative expenses and $1.1 million in research and development and research and development- license acquired expenses.