On May 14, 2019 Phio Pharmaceuticals Corp. (NASDAQ: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported its financial results for the first quarter ended March 31, 2019 and provided a business update (Press release, Phio Pharmaceuticals, MAY 14, 2019, View Source [SID1234536286]).

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"Our financial results during the first quarter of the year continue to remain consistent and in line with our projections for 2019. With an approximate $2 million quarterly cash burn expected to continue for the remainder of the year, the Company expects to have funding into the second half of 2020 while also providing us with the ability to significantly advance and expand our pipeline projects. In parallel with progressing our lead pipeline products, we have expanded our collaborations to include a collaboration with Glycostem Therapeutics and have increased our data output supporting the broad applicability of our self-delivering RNAi platform in various immune cell types and immuno-oncology applications, such as adoptive cell therapy and direct intra-tumoral use of our products," said Dr. Gerrit Dispersyn, President and CEO of Phio Pharmaceuticals Corp. "By bringing new talent on board and implementing improved R&D processes, we look forward to further accelerating our R&D efforts within the financial expectations and projected cash runway."

Quarter in Review and Recent Corporate Updates

Leadership:
Appointed Gerrit Dispersyn, Dr. Med. Sc. as the Company’s President and Chief Executive Officer. Dr. Dispersyn succeeded Geert Cauwenbergh, Dr. Med. Sc., who retired as CEO of the Company and remains as a member of the Company’s Board of Directors.
Appointed John A. Barrett, Ph.D. as the Company’s Chief Development Officer. Dr. Barrett joins Phio from Ziopharm Oncology, Inc. where he served as the company’s Vice President of R&D and Translational Medicine. He has accumulated over 25 years of experience working in research and development and is an expert in developing cell-based immuno-oncology therapies. Dr. Barrett will lead the development and execution of the Company’s preclinical and clinical strategy for our product candidates.
Entered into a research collaboration with Glycostem Therapeutics BV to explore the potential synergies of using our sd-rxRNA in combination with Glycostem’s proprietary Natural Killer-cell (NK-cell) generation technology (oNKord). The goal of the collaboration is to develop cellular immunotherapies for cancer treatment with enhanced efficacy and/or safety, resulting in further improvement of Glycostem’s cellular immunotherapies for the treatment of cancer patients.
Presented a poster titled "Feasibility and efficacy using self-delivering RNAi against TGFB1 to reduce TME immunosuppression" at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019.
Data from the poster demonstrated that an sd-rxRNA targeting TGFB1 was efficiently taken up by cancer and immune cells resulting in downregulation of target gene expression. The data further showed that intra-tumoral injection of sd-rxRNA targeting TGFB1 can reduce the immunosuppressive tumor microenvironment and potentially boost immune effector cell activity. Future clinical development activities will explore the possible synergistic effects of this approach in combination with adoptive cell therapy or other oncology therapies.
On May 14, 2019, the Nasdaq Stock Market provided written notice and granted the Company an additional 180 calendar days, or until November 11, 2019, to regain compliance with the minimum bid price requirements set forth in the Nasdaq listing rules. The written notice has no effect on the listing of the Company’s common stock at this time.
Select Financial Results

Cash Position

At March 31, 2019, the Company had cash of $12.7 million as compared with $14.9 million at December 31, 2018. The Company expects its cash to provide funding into the second half of 2020.

Research and Development Expenses

Research and development expenses for the quarter ended March 31, 2019 were $1.1 million as compared with $1.4 million for the quarter ended March 31, 2018. The decrease was primarily due to a reduction in headcount and the payroll-related expenses, as well as the completion of the Company’s drug manufacture of RXI-762 in the prior year period.

General and Administrative Expenses

General and administrative expenses for the quarter ended March 31, 2019 were $1.1 million as compared with $0.9 million for the quarter ended March 31, 2018. The increase was primarily due to an increase in stock-based compensation expense related to the restricted stock issued to the Company’s former Chief Executive Officer in lieu of cash compensation.

Net Loss

Net loss for the quarter ended March 31, 2019 was $2.1 million, compared with $2.2 million for the quarter ended March 31, 2018. The decrease was primarily due to changes in operating expenses, as discussed above

On May 14, 2019 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer, and through our license to Santen Pharmaceutical Co. Ltd., wet age‐related macular degeneration, reported financial results for the first quarter ended March 31, 2019 (Press release, Tracon Pharmaceuticals, MAY 14, 2019, View Source [SID1234536341]).

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Recent Corporate Highlights

In April, we announced termination of further enrollment into company sponsored trials of TRC105 due to lack of efficacy in the Phase 3 TAPPAS trial evaluating TRC105 in combination with Votrient (pazopanib) in patients with advanced or metastatic angiosarcoma.
"While we were disappointed in the outcome of the TAPPAS interim analysis, we have several other active clinical programs and look forward to developing multiple bispecific antibodies through our broad and long term partnership with I-Mab Biopharma," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "We are poised to initiate first-in-human dosing of the CD73 antibody TJ004309 and look forward to 2020 when we expect to begin clinical development of the first of up to five bispecific antibodies. We also continue to evaluate companies with first-in-class or best-in-class clinical stage assets who would benefit from accessing our product development platform, which we believe offers a rapid and capital-efficient U.S. drug development solution."

Expected Upcoming Milestones

Dosing of the first patient in a Phase 1 study of TJ004309 as a single agent and in combination with Tecentriq (atezolizumab), a PD-L1 checkpoint inhibitor marketed by Roche, in patients with advanced solid tumors is expected mid-2019.

Publication of TRC253 Phase 1 data in patients with metastatic castrate resistant prostate cancer is expected in the second quarter of 2019.

Top-line data from the randomized Phase 2 AVANTE trial of DE-122 in patients with wet age-related macular degeneration (AMD) are expected in the first half of 2020.
First Quarter 2019 Financial Results

Cash, cash equivalents and short-term investments were $32.1 million at March 31, 2019, compared to $39.1 million at December 31, 2018. We expect our current cash, cash equivalents and short-term investments to fund operations into the third quarter of 2020.

Collaboration revenue was $0 for the first quarter of 2019 compared to $3.0 million for the first quarter of 2018. The decrease was due to the $3.0 million non-refundable upfront payment received in connection with our prior agreement with Ambrx, which was recorded as revenue in the first quarter of 2018.

Research and development expenses for the first quarter of 2019 were $5.2 million compared to $9.4 million for the first quarter of 2018. The decrease was primarily attributable to lower manufacturing expenses for TRC105 in the first quarter of 2019 as compared to the 2018 period.

General and administrative expenses for the first quarter of 2019 were $1.9 million compared to $1.8 million for the first quarter of 2018.

Net loss for the first quarter of 2019 was $7.2 million compared to $8.4 million for the first quarter of 2018.
Investor Conference Call

The Company will hold a conference call today at 4:30 p.m. EST / 1:30 p.m. PST to provide an update on corporate activities and to discuss the financial results of its first quarter 2019. The dial-in numbers are (855) 779‑9066 for domestic callers and (631) 485-4859 for international callers. Please use passcode 9290299. A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About DE-122 (carotuximab)

DE-122, a novel ophthalmic formulation of carotuximab, is active in preclinical choroidal neovascularization (CNV) models and designed to enhance the effect of approved VEGF inhibitors used to treat wet AMD. DE-122 is being investigated in the Phase 2 randomized AVANTE trial assessing the efficacy and safety of intravitreal injections in combination with Lucentis (ranibizumab) compared to Lucentis monotherapy in patients with wet AMD.

About TRC253

TRC253 is a novel, orally bioavailable small molecule that is a potent, high affinity competitive inhibitor of the androgen receptor (AR) and AR mutations, including the F877L mutation. The AR F877L mutation results in an alteration in the AR ligand binding domain that confers resistance to therapies for prostate cancer. Therapies targeting the AR have demonstrated clinical efficacy by extending time to disease progression, and in some cases, the survival of patients with metastatic castration-resistant prostate cancer. However, resistance to these agents is often observed and several molecular mechanisms of resistance have been identified, including gene amplification, overexpression, alternative splicing, and point mutation of the AR. TRC253 is currently being studied in a Phase 1/2 clinical trial in prostate cancer. For more information about the clinical trial, please visit TRACON’s website at www.traconpharma.com/clinical_trials.php

About TJ004309

TJ004309 is a novel, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine, which is highly immunosuppressive. TJ004309 is currently being studied in a Phase 1 trial to assess safety and preliminary efficacy as a single agent and when combined with the PD-L1 checkpoint inhibitor Tecentriq in patients with advanced solid tumors.

On May 14, 2019 Bio-Techne Corporation (NASDAQ:TECH) reported that Chuck Kummeth, President and Chief Executive Officer, will present at the UBS Global Healthcare Conference on Wednesday, May 22nd, 2019, at 9:00 a.m. EDT (Press release, Bio-Techne, MAY 14, 2019, View Sourcenews/detail/138/bio-techne-to-present-at-the-ubs-global-healthcare-conference" target="_blank" title="View Sourcenews/detail/138/bio-techne-to-present-at-the-ubs-global-healthcare-conference" rel="nofollow">View Source [SID1234536925]). The conference will be held at the Grand Hyatt Hotel in New York City.

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A live webcast of the presentation can be accessed via Bio-Techne’s Investor Relations website at View Source or through the following link https://protect-us.mimecast.com/s/H5PbCR6MPEIgkMNru9JEaM?domain=cc.talkpoint.com.

On May 14, 2019 Sosei Group Corporation ("the Company"); (TSE: 4565) reported encouraging progress and initial success of its strategic multi-target drug discovery collaboration with Pfizer (Press release, Sosei Heptares, MAY 14, 2019, https://www.prnewswire.com/news-releases/sosei-heptares-reports-progress-with-pfizer-as-multi-target-collaboration-delivers-first-candidate-for-clinical-advancement-300849503.html [SID1234552772]). The research phase of the collaboration has delivered several milestones leading to the advancement of new potential candidate programs against G protein-coupled receptor (GPCR) targets nominated by Pfizer in major disease areas.

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These targets have clinical and biological validation as key points for therapeutic intervention targeting metabolic and inflammatory diseases but have proved challenging to address with conventional discovery approaches because of inherent technical challenges. To address these challenges, Sosei Heptares and Pfizer scientists have worked closely together to leverage their complementary expertise in enabling GPCR-focused structure-based drug design (SBDD) and development initially directed towards up to ten GPCR targets nominated by Pfizer.

In addressing these targets, Sosei Heptares has delivered stabilized receptors (StaR proteins), X-ray structures and biophysical data on certain programs, triggering milestone payments from Pfizer, including US$3 million announced today. Further milestones payments are contemplated under the agreement, with potential for royalties also payable provided the criteria under the agreement are satisfied. Pfizer also made a US$33 million equity investment in Sosei Heptares in 2015. In the future, Pfizer and Sosei Heptares anticipate publication of select research findings from their collaboration.

Dr. Malcolm Weir, Executive VP and Chief R&D Officer of Sosei Heptares, said: "Pfizer has a real appreciation of the potential value that structural studies can bring to drug design and discovery. Sosei Heptares is delighted with the relationship and interaction that has developed between our respective scientists since starting the collaboration and particularly with the achievement announced today. This was made possible, in part, through the collaborative application of our SBDD platform, which is also being used in other promising programs. We look forward in the coming years to the continued progress of these programs."

Charlotte Allerton, Head of Medicine Design at Pfizer said: "We are very pleased with the progress achieved through our collaboration with Sosei Heptares and are optimistic about our future work together. Sosei Heptares’ GPCR-focused structure-based drug design provides us with a valuable new approach to potentially enable the design of small molecules to modulate important disease targets."

Additional information about the collaboration with Pfizer

Sosei Heptares and Pfizer entered into a strategic drug discovery collaboration in November 2015 to research and develop potential new medicines directed at up to ten G protein-coupled receptor (GPCR) targets across multiple therapeutic areas.

Sosei Heptares will use its proprietary GPCR structure-guided platform to help deliver stabilised GPCRs (StaR proteins), high-resolution crystal structures and other technologies to support the discovery of potential novel agents directed to the GPCR targets selected by Pfizer. Pfizer will be responsible for developing and commercialising any potential therapeutic agents (small molecules or biologics) for each target and will have exclusive global rights to any potential resulting agents.

Sosei Heptares is eligible to receive research, development, regulatory and commercial milestone payments and tiered royalties on the net sales of any products that are commercialized by Pfizer. In addition, Pfizer made an equity investment in Sosei Heptares upon signing.

On May 14, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with an initial focus on Myelodysplastic Syndromes (MDS), reported financial results for the first quarter of 2019 ended March 31, 2019 (Press release, Onconova, MAY 14, 2019, View Source [SID1234536271]).

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"As we progress through 2019, we are advancing the clinical development of our pipeline and executing on our collaboration strategy, as highlighted by the recently announced license agreement for investigational rigosertib in Greater China with HanX Biopharmaceuticals (HanX)," said Steven M. Fruchtman, M.D., President and Chief Executive Officer. "2019 is an important year for Onconova and we are working diligently to execute on our objectives. If successful, we believe rigosertib could be the first new treatment for higher-risk MDS (HR-MDS) in more than 15 years."

Dr. Fruchtman added, "Enrollment in our Phase 3 intravenous (IV) rigosertib trial in second-line HR-MDS patients is continuing and is our top priority. We exceeded 75 percent enrollment of the study during the first quarter and are focused on completing enrollment in the second half of 2019 and reporting top-line data following full enrollment and 288 death events. We believe the addition of sites in Brazil and China later this year could contribute significantly to achieving our goal of completing enrollment by year end. We also look forward in the future to initiating a Phase 3 trial with oral rigosertib in combination with azacitidine in first-line HR-MDS patients and to filing an IND in the U.S. for ON 123300, a first-in-class CDK4/6 + ARK5 inhibitor for the treatment of a variety of advanced tumors."

First Quarter 2019 and Recent Highlights

Entered into rigosertib license agreement with HanX for the development and commercialization of rigosertib in Greater China, which, together with an equity investment at a premium, brings $4 million in cash to the Company. This new HanX license expands the existing collaboration between the two companies. Onconova and HanX previously signed a separate agreement in December 2017 for the pipeline compound ON 123300, Onconova’s novel CDK 4/6 inhibitor, which is expected to enter a Phase 1 clinical trial in the U.S. during the second half of 2019.
Achieved over 75 percent enrollment in the INSPIRE study during the first quarter, and remain focused on completing enrollment in the second half of 2019 and reporting top-line data following full enrollment and 288 death events.
More than 140 trial sites in 23 countries across four continents are open, including 21 sites in Japan. Opened 19 new clinical trial sites in 8 already participating countries to support completion of enrollment of 360 patients in the Phase 3 INSPIRE study. Additional geographies are being opened during the coming months to add approximately 25 more sites.
Attended MDS Symposium in Copenhagen May 8-11, 2019, for which five posters related to rigosertib were accepted for presentation.
Oral Rigosertib in Combination with Azacitidine for First-Line HR MDS Trial Progress and Near-Term Milestones

In December 2018, Onconova submitted an application for a SPA to the FDA for a Phase 3 Trial of oral rigosertib in combination with azacitidine for treatment of first-line higher-risk MDS adult patients. The Company is currently in discussions with the FDA regarding the SPA. Upon agreement regarding the SPA, Onconova hopes to initiate the Phase 3 study with the support of a partnership.
Business Development Progress for Rigosertib and Pipeline Products

Onconova entered into a new license agreement with HanX to develop and commercialize rigosertib in Greater China. HanX is a China-based pharmaceutical company focused on the development, registration, and commercialization of therapeutics for China. Under the terms of the agreement, Onconova has granted to HanX an exclusive license to develop and commercialize rigosertib in Greater China. In exchange for these rights, HanX will pay a $2 million up-front payment and make an additional $2 million equity investment in Onconova stock at a premium to market. In addition, HanX will initially dedicate $2 million in local currency to fund rigosertib development in China over the next two years and will be responsible for future development costs of the product in China pursuant to a joint development plan. HanX will make additional regulatory, developmental, and sales-based milestone payments to Onconova of up to $45.5 million and pay Onconova tiered royalties up to double digits on net sales in Greater China. If approval is received, Onconova will supply the finished product to HanX for development and commercialization. HanX also will support Onconova’s other clinical trial initiatives in Greater China.
ON 123300, an investigational first-in-class dual inhibitor of CDK4/6 + ARK5 with the potential to treat a variety of cancers, continues to make progress toward clinical development in the U.S. and China in partnership with HanX. HanX has conducted toxicology studies to support an Onconova IND filing in the U.S., anticipated in the second quarter of 2019.
Collaboration with the National Cancer Institute is ongoing for preclinical studies of rigosertib for treatment of pediatric cancer associated RASopathies.
Scientific presentations related to rigosertib development and clinical trials were made at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, the MDS Symposium in Copenhagen, and the Acute Leukemia Forum in Newport, CA; upcoming presentations will be made at the Acute Leukemia Forum in China, and the European Hematology Association (EHA) (Free EHA Whitepaper) Congress. Onconova will also attend the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Annual Meeting in June.
First Quarter 2019 Financial Results

Cash and cash equivalents as of March 31, 2019, totaled $10.4 million compared to $17.0 million at December 31, 2018. The Company expects that cash and cash equivalents at March 31, 2019 will be sufficient to fund ongoing trials and operations into the fourth quarter of 2019. After receiving the upfront proceeds from the HanX rigosertib transaction, the Company expects that its cash and cash equivalents will be sufficient to fund ongoing trials and operations into the first quarter of 2020.

Net loss was $7.6 million for the quarter ended March 31, 2019, compared to $5.1 million for the first quarter ended March 31, 2018. Research and development expenses were $4.1 million for the quarter ended March 31, 2019, and $4.6 million for the comparable period in 2018. General and administrative expenses were $3.2 million for the quarter ended March 31, 2019, and $1.9 million for the comparable period in 2018.

Conference Call and Webcast Information

The Company will host a conference call today, May 14, at 9 a.m. Eastern Time, to provide a corporate update and discuss first quarter 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: 4275108. The call will also be webcast live. Please click here to access the webcast. A replay will be available following the live webcast.

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become dysfunctional and thus produce an inadequate number of circulating blood cells. It is frequently associated with the presence of blasts or leukemic cells in the marrow. This leads to low numbers of one or more types of circulating blood cells, and to the need for blood transfusions. In MDS, some of the cells in the bone marrow are abnormal (dysplastic) and may have genetic abnormalities associated with them. Different cell types can be affected, although the most common finding in MDS is a shortage of red blood cells (anemia). Patients with higher-risk MDS may progress to the development of acute leukemia.

About Onconova Therapeutics, Inc.

Onconova Therapeutics, Inc. is a Phase 3-stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS). Using a proprietary chemistry platform, Onconova has created a pipeline of targeted agents designed to work against specific cellular pathways that are im