On November 14, 2019 Trovagene, Inc. (Nasdaq: TROV), a clinical-stage, Precision Cancer Medicine oncology therapeutics company developing drugs that target cell division (mitosis), for the treatment of various cancers including prostate, colorectal and leukemia, reported new positive data from its Phase 2 trial evaluating onvansertib in combination with Zytiga (abiraterone acetate – Johnson & Johnson)/prednisone in patients with metastatic Castration-Resistant Prostate Cancer (mCRPC) (Press release, Trovagene, NOV 14, 2019, View Source [SID1234551282]). The data are being presented today at the European Multidisciplinary Congress on Urological Cancers (EMUC) in Vienna, Austria.

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"The new data shared today builds upon the encouraging clinical response seen to date when onvansertib is added to treatment in patients who have developed resistance to androgen receptor signaling inhibitor (ARSi), Zytiga," said Dr. Mark Erlander, Chief Scientific Officer of Trovagene. "Of particular significance are the positive results we are observing in patients who harbor the highly aggressive, resistant variant of the androgen receptor (AR-V7). These patients are resistant to ARS inhibitors including Zytiga and Xtandi (enzalutamide – Pfizer) and their therapeutic options are not only limited, but often ineffective. We believe the addition of onvansertib has the potential to deliver transformative benefit to patients with mCRPC by extending the duration of response to treatment with ARS inhibitors."

Data Summary

The newly reported data include efficacy and safety assessments as of the October 28, 2019 data cut-off date for 15 patients that completed 3 months of treatment and were eligible for evaluation of the primary efficacy endpoint of disease control. Response to treatment was evaluated based on a decrease or stabilization in prostate specific antigen (PSA) values (primary endpoint) and confirmed by radiographic scans.

Efficacy
Overall, across both arms (A and B), a 60% response (SD + PR) was observed in patients who were evaluable for efficacy (completed 3 months of treatment); 72% of patients had a decrease in PSA following one cycle of treatment with onvansertib; 6 patients remain on treatment for ≥4 months.

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All 5 patients who tested positive for the highly-aggressive, resistant AR-V7 variant had decreases in PSA following one cycle of treatment with onvansertib; the primary efficacy endpoint (SD + PR) was achieved in 3 out of 4 evaluable patients (completing 12 weeks of treatment).
Safety
In both arms (A and B) onvansertib in combination with abiraterone was safe and well-tolerated. The most frequent adverse events (AEs) were expected, on-target (based on mechanism of action of onvansertib) hematologic (anemia, neutropenia, thrombocytopenia and white blood cell (WBC) decrease). All hematologic AEs were easily and effectively managed and resolved by delaying or reducing the dose and/or adding growth factor support. No unexpected or off-target AEs have been reported to-date.

About the Phase 2 Trial of Onvansertib in mCRPC
The trial is a Phase 2 open-label multi-center study of onvansertib in combination with Zytiga (abiraterone acetate)/prednisone in patients with mCRPC, showing signs of disease progression and resistance to Zytiga demonstrated by two rising PSA values separated by at least one week, while on Zytiga(NCT03414034). The primary efficacy endpoint is the proportion of patients achieving disease control after 12 weeks of study treatment, as defined by lack of prostate specific antigen (PSA) progression. The trial is being conducted by Beth Israel Deaconess Medical Center (BIDMC), Dana-Farber Cancer Institute (Dana-Farber), and Massachusetts General Hospital Cancer Center (MGH). David Einstein, MD, Genitourinary Oncology Program at BIDMC, is the principal investigator for the trial.
About Onvansertib
Onvansertib is a first-in-class, third-generation, oral and highly-selective adenosine triphosphate (ATP) competitive inhibitor of the serine/threonine polo-like-kinase 1 (PLK1) enzyme, which is over-expressed in multiple cancers including leukemias, lymphomas and solid tumors. Onvansertib targets the PLK1 isoform only (not PLK2 or PLK3), is orally administered and has a 24-hour half-life with only mild-to-moderate side effects reported. Trovagene believes that targeting only PLK1 and having a favorable safety and tolerability profile, along with an improved dose/scheduling regimen will significantly improve on the outcome observed in previous studies with a former panPLK inhibitor in AML.
Onvansertib has demonstrated synergy in preclinical studies with numerous chemotherapies and targeted therapeutics used to treat leukemias, lymphomas and solid tumor cancers, including irinotecan, FLT3 and HDAC inhibitors, taxanes and cytotoxins. Trovagene believes the combination of onvansertib with other compounds has the potential to improve clinical efficacy in acute myeloid leukemia (AML), metastatic castration-resistant prostate cancer (mCRPC), non-Hodgkin lymphoma (NHL), colorectal cancer and triple-negative breast cancer (TNBC), as well as other types of cancer.
Trovagene has three ongoing clinical trials of onvansertib: A Phase 2 trial of onvansertib in combination with Zytiga (abiraterone acetate)/prednisone in patients with mCRPC who are showing signs of early progressive disease (rise in PSA but minimally symptomatic or

asymptomatic) while currently receiving Zytiga (NCT03414034); a Phase 1b/2 Study of onvansertib in combination with FOLFIRI and Avastin for second-line treatment in patients with mCRC with a KRAS mutation (NCT03829410); and a Phase 1b/2 clinical trial of onvansertib in combination with low-dose cytarabine or decitabine in patients with relapsed or refractory AML (NCT03303339). Onvansertib has been granted orphan drug designation by the FDA in the U.S. and by the EC in the European Union for the treatment of patients with AML.
Trovagene licensed onvansertib (also known as NMS-1286937 and PCM-075) from Nerviano Medical Sciences (NMS), the largest oncology-focused research and development company in Italy, and a leader in protein kinase drug development. NMS has an excellent track record of licensing innovative drugs to pharma/biotech companies, including Array (recently acquired by Pfizer), Ignyta (acquired by Roche) and Genentech.

On November 14, 2019 Alector, Inc. (Nasdaq: ALEC), a clinical stage biotechnology company pioneering immuno-neurology, reported that Sabah Oney, Ph.D., chief business officer of Alector, will present at the Jefferies 2019 London Healthcare Conference on Thursday, Nov. 21, at 8:00 a.m. local time (GMT) in London (Press release, Alector, NOV 14, 2019, View Source [SID1234551281]).

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To access the live webcast of the presentation, please visit the "Events & Presentations" page within the Investors section of the Alector website at View Source A replay will be available on the Alector website for 90 days following the conference.

On November 14, 2019 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines, reported third quarter 2019 financial results and provided an overview of operational progress (Press release, Rubius Therapeutics, NOV 14, 2019, View Source [SID1234551280]).

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"Over the last quarter, our team has successfully manufactured RTX-134 for our Phase 1b trial for the treatment of patients with phenylketonuria. Our clinical sites are actively recruiting patients, and we plan to announce when we have treated the first patient. In order for us to share follow up data from the first patients treated in the study, we expect to report initial clinical results during the first quarter of 2020," said Pablo J. Cagnoni, M.D., chief executive officer of Rubius Therapeutics. "We continue to make good progress with our entire pipeline, having presented preclinical oncology data showcasing the versatility and potential power of the RED PLATFORM. We remain on track to file our first oncology Investigational New Drug application for RTX-240 by early 2020."

Recent Highlights and Upcoming Milestones

·Over the third quarter, Rubius Therapeutics successfully manufactured RTX-134 for our Phase 1b trial for the treatment patients with phenylketonuria (PKU).

·Rubius has clinical sites actively recruiting patients and plans to announce when the first patient has been treated.

· In order for Rubius to share follow up data from the first patients treated in the study, the Company expects to report initial clinical results during the first quarter of 2020, including:

·Preliminary safety;

· Longevity of RTX-134 in circulation; and

· Proof-of-mechanism as measured by production of trans-cinnamic acid, a metabolite of phenylalanine when degraded by phenylalanine ammonia lyase, or PAL.

·On November 8, 2019, Rubius presented preclinical data at the SITC (Free SITC Whitepaper) Annual Meeting, supporting its lead artificial antigen presenting cell program, RTX-321, for the treatment of HPV 16-positive tumors.

· RTX-321 is an allogeneic artificial antigen presenting cell engineered to express, on the cell surface, an HPV peptide antigen, 4-1BBL and IL-12 to mimic human T cell-APC interactions. RTX-321 is currently in IND-enabling studies.

·Proof-of-concept data generated in a B16-F10 melanoma mouse model demonstrated successful engineering of artificial antigen presenting cells against a melanoma tumor-associated antigen, gp100, that significantly expanded antigen-specific T cells and eliminated lung metastases with limited, reversible toxicity.

·On October 29, 2019, Rubius Therapeutics was issued U.S. Patent No. 10,456,421 covering Red Cell Therapeutic (RCT) compositions comprising 4-1BBL and their use for the treatment of cancer. 4-1BBL is expressed on the cell surface of each of the Company’s lead oncology product candidates: RTX-240, RTX-224 and RTX-321.

·Today, in total, Rubius Therapeutics has nine issued U.S. patents, including five composition of matter patents, 32 patent families and more than 150 pending patent applications worldwide.

·On October 27, 2019, Rubius Therapeutics presented preclinical data at the AACR (Free AACR Whitepaper)-NCI-EORTC, demonstrating the ability to engineer RCTs to create loadable artificial antigen presenting cells for personal neoantigens.

RTX-aAPCs with peptide-loaded MHC constructs functionally engaged TCRs and achieved robust expansion of primary cytomegalovirus (CMV)-specific T cells in healthy donor PBMCs with prior exposure to CMV.

Rubius Therapeutics’ loadable aAPC system has the potential to generate aAPCs containing multiple neoantigens in a single therapeutic.

·The Company continued to strengthen its leadership team by appointing Maiken Keson-Brookes as chief legal officer and corporate secretary.

Third Quarter Financial Results

Net loss for the third quarter of 2019 was $47.0 million or $0.59 per common share, compared to $26.4 million or $0.42 per common share in the third quarter of 2018.

In the third quarter of 2019, Rubius invested $33.5 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $14.4 million in the third quarter of 2018. This year-over-year increase was driven primarily by $8.6 million in incremental R&D program spending in preparation for the Company’s Phase 1b clinical trial for RTX-134 and towards preclinical activities for Rubius’ lead oncology programs, including RTX-240. In addition, $7.8 million in incremental R&D spending was driven by increased R&D headcount, a move into larger facilities and purchasing lab supplies to support Rubius’ goal of delivering four to five INDs across 2019 and 2020. Contract research costs increased by $1.9 million and R&D stock-based compensation also increased by $0.8 million.

G&A expenses were $15.0 million during the second quarter of 2019, as compared to $13.2 million for the third quarter of 2018. The higher costs were primarily driven by a $2.1 million increase in personnel and facility costs due to increased headcount in the general and administrative function, as well as increases in professional fees and infrastructure costs to support the Company’s growth.

Nine Month Financial Results

Net loss for the first nine months of 2019 was $119.0 million or $1.52 per common share, compared to $62.0 million or $2.33 per common share in the first nine months of 2018.

In the nine months ended September 30, 2019, Rubius invested $81.9 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $35.2 million in the first nine months of 2018. This year-over-year increase was largely due to an additional $22.4 million in R&D personnel, external research and facilities and lab supplies to support the Company’s pipeline expansion in 2019 and 2020 and $19.8 million in R&D program spending, including preparing for the RTX-134 Phase 1b clinical trial and preclinical activities for Rubius’ lead oncology programs, including RTX-240. R&D stock-based compensation also increased by $4.5 million.

G&A expenses were $42.3 million during the first nine months of 2019, as compared to $27.3 million for the same period in 2018. The higher costs were primarily driven by a $7.6 million increase in stock-based compensation and a $7.4 million increase in personnel costs and facility costs due to increased headcount in our general and administrative function, as well as increases in professional fees and infrastructure costs to support the Company’s growth.

Cash Position

As of September 30, 2019, cash, cash equivalents and investments were $324.7 million as compared to $404.1 million as of December 31, 2018, providing Rubius with a cash runway into 2021. During the year, the Company used $81.5 million of cash to fund operations and $27.4 million to fund capital expenditures, including work related to the buildout of Rubius’ manufacturing facility. In addition, during the year, the Company drew down a second tranche of $25.0 million from its $75.0 million loan agreement with Solar Capital in June 2019, which leaves a third tranche of $25.0 million that can be drawn through June 2020, subject to the satisfaction of certain financial covenants.

On November 14, 2019 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing and providing access to innovative therapies for patients with liver and other chronic diseases with high unmet medical need, reported that management will participate in two investor conferences including the Stifel 2019 Healthcare Conference taking place November 19-20, 2019 in New York City and the Evercore ISI 2nd Annual HEALTHCONx Conference in Boston, MA – December 3-5, 2019 (Press release, CymaBay Therapeutics, NOV 14, 2019, View Source [SID1234551279]).

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Stifel 2019 Healthcare Conference
Date: Tuesday, November 19
Time: 8:00am Eastern Time
Webcast: View Source

Evercore ISI 2nd Annual HEALTHCONx Conference
Date: Tuesday, December 3
Time: 11:45am Eastern Time
Webcast: View Source

On November 14, 2019 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported that members of the management team will participate in the following upcoming investor conferences (Press release, Pieris Pharmaceuticals, NOV 14, 2019, View Source [SID1234551278]):

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Jefferies 2019 London Healthcare Conference

Wednesday, November 20, 2019 in London. The Company will conduct one-on-one meetings with institutional investors at this conference.

Evercore ISI HealthCONx Conference 2019

Tuesday, December 3, 2019 at 10:35 AM EST in Boston. A webcast of the Company’s presentation will be available at this link.