On August 15, 2019 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported its financial results for the second quarter ended June 30, 2019 and provided an update on recent corporate developments (Press release, Bio-Path Holdings, AUG 15, 2019, View Source [SID1234538775]).

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"Throughout the second quarter we continued to execute on our clinical development plans for our innovative RNAi nanoparticle therapeutics. We are excited to begin dosing patients in the amended cohorts of our Phase 2 study of prexigebersen, which will first evaluate the safety of prexigebersen in combination with decitabine in untreated AML and high risk MDS patients and refractory/relapsed AML and high risk MDS patients, and then evaluate the efficacy of the triple combination of prexigebersen + decitabine + venetoclax in those patient groups," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings.

"We are also looking forward to completing Investigational New Drug (IND) enabling studies of BP1003, a novel liposome-incorporated STAT3 oligodeoxynucleotide inhibitor for the treatment of pancreatic cancer, and to file an IND application for a Phase 1 study of BP1003 for the treatment of pancreatic cancer in 2020. We are particularly excited to launch this program as it will be our first-in-human validation of this cutting-edge therapy in an especially challenging cancer indication that has limited treatment options," added Mr. Nielsen.

Recent Corporate Highlights

Presented Preclinical Data at American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019. In April 2019, Bio-Path presented data from preclinical studies supporting the potential of BP1003, a novel liposome-incorporated STAT3 oligodeoxynucleotide inhibitor, for the treatment of pancreatic cancer, non-small cell lung cancer (NSCLC) and acute myelogenous leukemia (AML). These data were presented in a poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 in Atlanta, GA.
Financial Results for the Second Quarter Ended June 30, 2019

The Company reported a net loss of $2.5 million, or $0.87 per share, for the three months ended June 30, 2019, compared to a net loss of $1.7 million, or $2.96 per share, for the three months ended June 30, 2018.

Research and development expenses for the three months ended June 30, 2019 increased to $1.5 million, compared to $0.8 million for the three months ended June 30, 2018 primarily due to the commencement of activities related to Stage 2 of our Phase 2 clinical trial in AML to include venetoclax combination treatment with prexigebersen and two cohorts of patients.

General and administrative expenses for the three months ended June 30, 2019 increased to $1.0 million, compared to $0.9 million for the three months ended June 30, 2018 primarily due to increased legal fees and insurance costs.

As of June 30, 2019, the Company had cash of $17.1 million, compared to $1.0 million at December 31, 2018. Net cash used in operating activities for the six months ended June 30, 2019 was $4.2 million compared to $3.4 million for the comparable period in 2018. Net cash provided by financing activities for the six months ended June 30, 2019 was $20.3 million.
Conference Call and Webcast Information

Bio-Path Holdings will host a conference call and webcast today at 8:30 a.m. ET to review these second quarter 2019 financial results and to provide a general update on the Company. To access the conference call, please call (844) 815-4963 (domestic) or (210) 229-8838 (international) and refer to conference ID 3792557. A live audio webcast of the call and the archived webcast will be available in the Media section of the Company’s website at www.biopathholdings.com.

On August 15, 2019 Quest Diagnostics (NYSE: DGX), the world’s leading provider of diagnostic information services, reported that its Board of Directors declared a quarterly cash dividend of $0.53 per share, payable on October 21, 2019 to shareholders of record of Quest Diagnostics common stock on October 4, 2019 (Press release, Quest Diagnostics, AUG 15, 2019, View Source [SID1234538773]).

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On August 15, 2019 Alligator Bioscience (Nasdaq Stockholm: ATORX), reported that Janssen Biotech, Inc. (Janssen) will present data from a Phase I clinical study of ADC-1013 (JNJ-7107) at the upcoming ASCO (Free ASCO Whitepaper) Annual meeting held in Chicago on May 31- June 4 (Press release, Alligator Bioscience, AUG 15, 2019, View Source [SID1234538665]). The study results show that side effects were generally mild and transient.

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The primary aim of the study was safety and to establish a recommended dose for Phase II. The study is still ongoing and has enrolled a total of 95 patients. The maximum tolerated dose has not yet been reached. Doses up to 1200 μg/kg i.v without premedication, and up to 2000 μg/kg with premedication have been shown to be safe and tolerable. Early evidence of clinical activity in this study included a partial response (PR) in a patient with renal cell cancer and 10 patients with prolonged stable disease (SD) ≥6 months.

"I am glad to see the promising safety and tolerability profile of ADC-1013 in patients with cancer. This together with the early signs of clinical activity, gives me great confidence in this CD40 drug candidate", said Per Norlén, CEO of Alligator Bioscience.

A poster (#171) with the title "A Phase 1 Study to Assess Safety, Pharmacokinetics (PK) and Pharmacodynamics (PD) of JNJ-64457107, a CD40 Agonistic Monoclonal Antibody, in Patients (pts) with Advanced Solid Tumors" will be showcased at ASCO (Free ASCO Whitepaper) on June 1, 2019 from 8 – 11 a.m. in the session Developmental Immunotherapy and Tumor Immunobiology.

The licensing agreement with Janssen encompasses milestone payments up to a potential total value of USD 695 million. Alligator is also eligible to receive tiered royalties on worldwide net sales upon successful launch and commercialization of ADC-1013.

For further information, please contact:
Cecilia Hofvander, Director IR & Communications
Phone +46 46 540 82 06
E-mail: [email protected]

This information is such information as Alligator Bioscience AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. This information was submitted for publication, through the agency of the contact person set out above, at 11:00 p.m. CEST (5:00 p.m. ET) on May 15, 2019.

About ADC-1013 (JNJ-7107)
ADC-1013 is a drug candidate intended for immunotherapy of different types of cancer. Results from a previous clinical Phase I first-in-human study performed by Alligator (International Journal of Cancer, View Source) showed that ADC-1013 was generally well tolerated. Preclinical data have previously shown that the ADC-1013 antibody effectively activates T cells, mediated through binding to the co-stimulatory receptor CD40 on dendritic cells. The increased T cell activation enables the immune system to attack the cancer.

On August 14, 2019 Cleveland BioLabs, Inc. (NASDAQ:CBLI) reported financial results and development progress for the second quarter ended June 30, 2019 (Press release, Cleveland BioLabs, AUG 14, 2019, View Source [SID1234538834]).

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Cleveland BioLabs reported a net loss of $(0.6) million, excluding minority interests, for the second quarter of 2019, or $(0.05) per share, compared to a net loss, excluding minority interests, of $(0.9) million, or $(0.08) per share, for the same period in 2018. The decrease in net loss was primarily due to reduced research and development expenses related to the oncology applications of the entolimod family of compounds, a decrease in General and Administrative costs, and a decrease in the non-cash adjustment to our warrant liabilities, partially offset by reduced revenues.

As of June 30, 2019, the Company had $2.6 million in cash, cash equivalents and short-term investments, which, based on the Company’s current operational plan, is expected to fund operations into June 2020.

Yakov Kogan, Ph.D., MBA, Chief Executive Officer, stated, "The pursuit of regulatory approval and commercialization for entolimod as a medical radiation countermeasure remains our main priority and focus."

Further Financial Results

Revenue for the second quarter of 2019 decreased to $0.28 million compared to $0.39 million for the second quarter of 2018. The net decrease was primarily attributable to decreased revenue from our Joint Warfighter Medical Research Program ("JWMRP") contract from the Department of Defense ("DoD") for the continued development of the entolimod as a medical radiation countermeasure and by decreased revenue from our service contract with Incuron.

Research and development costs for the second quarter of 2019 decreased to $0.60 million compared to $0.91 million for the second quarter of 2018. The reduction in research and development costs is due to a $0.36 million decrease in expenses related to the oncology applications of the entolimod family of compounds, partially offset by a $0.05 million increase in spending for biodefense applications of entolimod.

General and administrative costs for the second quarter of 2019 decreased to $0.45 million compared to $0.55 million for the second quarter of 2018. This decrease was primarily attributable to a $0.03 million decrease in CBLI’s legal and professional fees, a $0.03 million decrease in travel expense, and a $0.03 million decrease in personnel and subcontractor expenses relating to outsourced assistance.

On August 14, 2019 Scholar Rock Holding Corporation (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the quarter ended June 30, 2019 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, AUG 14, 2019, View Source [SID1234538815]).

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"Throughout the first half of 2019, we made significant progress both clinically and financially as we advanced SRK-015 into a Phase 2 trial in SMA, SRK-181 towards the initiation of a Phase 1 trial in cancer immunotherapy in mid-2020, as well as successfully completed a follow-on offering to ensure that we are well-capitalized to support our portfolio of product candidates and overall company growth," said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. "We look forward to building upon this momentum as we continue to progress our broad pipeline, with a number of important milestones at the end of this year and into 2020."

Company Highlights and Upcoming Milestones

SRK-015 Program:

Positive Final Results from the Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers Presented at the Cure SMA Annual Conference. In June 2019, Scholar Rock presented positive detailed results from the Phase 1 trial of SRK-015, which consisted of data from both the single-ascending and multiple-ascending dose portions of the trial. SRK-015 was observed to be well-tolerated with no dose-limiting toxicities identified in up to the highest evaluated dose of 30 mg/kg. The pharmacologic profile showed robust engagement of latent myostatin, which was saturated and sustained up to at least Day 140 after three doses of SRK-015 given once every two weeks at 20 mg/kg or 30 mg/kg. In contrast, no meaningful change was observed in the latent myostatin biomarker concentrations in subjects who received placebo.

Preliminary Pharmacokinetic (PK) and Pharmacodynamic (PD) Data from TOPAZ Phase 2 Trial Expected by End of 2019. Patient enrollment is underway in the TOPAZ Phase 2 proof-of-concept trial to assess the safety and efficacy of SRK-015 in patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA). Approximately 55 patients are anticipated to be enrolled in the U.S., Canada, and Europe across three distinct and parallel cohorts and treated with SRK-015, either as a monotherapy or in conjunction with an approved survival motor neuron (SMN) upregulator therapy.

Scholar Rock is on track to report preliminary PK/PD data for a subset of patients in the Phase 2 TOPAZ trial by the end of 2019, which will provide initial observations on target engagement of latent myostatin in patients with Type 2 and Type 3 SMA. These preliminary PK/PD results will be followed by interim safety and efficacy results for a subset of patients with six months of treatment exposure expected in the first half of 2020 and top-line data for the full 12-month treatment period expected beginning in the fourth quarter of 2020.
Details of Scholar Rock-Developed Biomarker Assay that Quantifies Serum Latent Myostatin Published in Peer-Reviewed Journal. In July 2019, Scholar Rock published details of its immunoassay used to measure serum latent myostatin in peer-reviewed journal Society of Laboratory Automation and Screening Discovery. The publication, "A Sensitive and Selective Immunoassay for the Quantitation of Serum Latent Myostatin After In-Vivo Administration of SRK-015, a Selective Inhibitor of Myostatin Activation,"(1) highlights the ability of the immunoassay to quantify serum latent myostatin without cross-reactivity to promyostatin, mature myostatin, or closely related members of the TGFβ superfamily and the establishment of the robustness, reproducibility, and stability of the immunoassay.

Through use of this biomarker assay, robust and sustained target engagement of latent myostatin has been observed in both preclinical studies and the Phase 1 clinical trial in healthy volunteers, establishing proof-of-mechanism of Scholar Rock’s therapeutic approach of targeting the latent form of growth factors.
Identification of Second Indication for SRK-015 Planned for 2020. Scholar Rock continues to evaluate multiple potential opportunities for which SRK-015 could offer clinical benefit and is assessing additional potential clinical settings in which the selective inhibition of the activation of myostatin may offer therapeutic benefit.
SRK-181 Program:

Initiation of the Phase 1 Clinical Trial of SRK-181 in Patients with Solid Tumors Anticipated in Mid-2020. In March 2019, Scholar Rock selected SRK-181, a highly specific inhibitor of TGFβ1 activation, as the first product candidate in its TGFβ1 cancer immunotherapy program based on the strength of preclinical data and human translational insights. Scholar Rock intends to develop SRK-181 for the treatment of tumors resistant to checkpoint blockade therapies (CBTs), such as anti-PD(L)1 antibodies, and believes there is significant opportunity to expand the number of patients who can benefit from CBTs.

Scholar Rock plans to initiate a Phase 1 trial in patients with primary resistance to approved checkpoint blockade therapies in mid-2020 with initial clinical data anticipated by the end of 2021.
RGMc Program:

Nomination of a Product Candidate from the RGMc Program Planned in First Half of 2020. For its third product candidate, Scholar Rock is evaluating a number of highly specific inhibitors of repulsive guidance molecule C (RGMc) and plans to nominate an antibody in the first half of 2020. RGMc is a co-receptor of bone morphogenetic protein 6 (BMP6), a member of the TGFβ superfamily that plays an important role in iron metabolism. RGMc’s known function is localized to hepatocytes and the identification of RGMc selective-antibodies may offer the potential for liver-specific modulation of BMP6 signaling to address iron-restricted anemias.
Corporate Update:

Public Offering of Common Stock Completed in June/July 2019. Scholar Rock successfully completed a public offering of 3,450,000 shares of common stock, inclusive of the full exercise of the over-allotment option by the underwriters, raising gross proceeds of approximately $51.8 million.
Second Quarter 2019 Financial Results

For the quarter ended June 30, 2019, net loss was $12.5 million or $0.48 per share compared to a net loss of $14.7 million or $1.39 per share for the quarter ended June 30, 2018.

Research and development expense was $13.7 million for the quarter ended June 30, 2019 compared to $11.4 million for the quarter ended June 30, 2018. The increase year-over-year reflects preclinical and manufacturing costs for SRK-181 and higher personnel-related costs, partially offset by a decrease year-over-year in manufacturing costs associated with SRK-015.

General and administrative expense was $4.7 million for the quarter ended June 30, 2019 compared to $3.5 million for the quarter ended June 30, 2018. The increase year-over-year was primarily attributable to increased headcount, stock compensation, and higher operational fees associated with operating as a public company.
As of June 30, 2019, Scholar Rock had cash, cash equivalents, and marketable securities of $185.1 million, compared to $175.6 million as of December 31, 2018. The cash balance as of June 30, 2019 is inclusive of the approximately $42.2 million in net proceeds from the public offering of 3,000,000 shares of common stock and exclusive of $6.3 million in net proceeds from the underwriter’s purchase of an additional 450,000 shares of common stock that was settled in July 2019.

Cote, S. M., Jackson, J., Pirruccello-Straub, M., Carven, G. J., & Wawersik, S. (2019). A Sensitive and Selective Immunoassay for the Quantitation of Serum Latent Myostatin after In Vivo Administration of SRK-015, a Selective Inhibitor of Myostatin Activation. SLAS DISCOVERY: Advancing Life Sciences R&D.