On November 3, 2016 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported operating results for third-quarter 2016 and provided an update on the Company’s marketed product and development programs (Press release, TESARO, NOV 3, 2016, View Source [SID1234516336]).

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"We are very pleased to have submitted regulatory applications for niraparib in both the United States and in Europe for the maintenance treatment of patients with platinum-sensitive, recurrent ovarian cancer who are in response to platinum-based chemotherapy. Pre-launch activities are well underway in support of four potential product launches in 2017, including VARUBI IV and niraparib in the U.S and VARUBI oral and niraparib in Europe," said Lonnie Moulder, CEO of TESARO. "The recent ESMO (Free ESMO Whitepaper) 2016 Congress was very gratifying for TESARO, highlighted by the presentation of the landmark niraparib NOVA trial results during a Presidential Symposium by Dr. Mansoor Raza Mirza. We look forward to advancing our comprehensive ovarian cancer clinical program and expanding niraparib development into other tumor types in 2017."

Recent Business Highlights

The U.S. launch of VARUBI continues, and unit volume increased by 14% for the third quarter compared to the second quarter. For the month of September, VARUBI achieved a 28% market share in the oral NK-1 market in the U.S.
TESARO officially opened its international commercial headquarters in Zug, Switzerland on October 11.
Earlier this week, the rolling submission of a New Drug Application (NDA) for niraparib to the U.S. Food and Drug Administration (FDA) was completed for the maintenance treatment of patients with recurrent, platinum-sensitive ovarian cancer who are in response to platinum-based chemotherapy. The indication proposed in the niraparib NDA provides for the use of niraparib regardless of tumor biomarker status, and it is anticipated that the BRACAnalysis CDx and myChoice HRD tests would be available to physicians as complementary diagnostics.
The Marketing Authorisation Application (MAA) for niraparib has been submitted to and accepted for review by the European Medicines Agency (EMA) for the maintenance treatment of patients with recurrent, platinum-sensitive ovarian cancer who are in response to platinum-based chemotherapy.
The niraparib Phase 3 ENGOT-OV16/NOVA clinical trial results were presented at the Presidential Symposium at the ESMO (Free ESMO Whitepaper) 2016 Congress by Dr. Mansoor Raza Mirza, M.D., Medical Director of the Nordic Society of Gynecologic Oncology (NSGO) and principal investigator. These data were simultaneously published in the New England Journal of Medicine.
Planning is underway to support initiation of an Early Access Program (EAP) for niraparib in the United States and Europe.
Janssen initiated a Phase 2 clinical trial with niraparib in monotherapy in men with metastatic castration resistant prostate cancer (mCRPC) and recently began a Phase 1 safety and pharmacokinetics study of niraparib plus apalutamide (ARN-509).
Enrollment continues in the PRIMA trial for patients with first-line ovarian cancer, the QUADRA trial of niraparib for the treatment of patients with ovarian cancer who have received three or more prior lines of chemotherapy, and in the BRAVO trial for patients with germline BRCA-mutated, metastatic breast cancer.
Enrollment continues in the TOPACIO trial of niraparib plus KEYTRUDA (pembrolizumab) in patients with ovarian cancer or with triple negative breast cancer and in the AVANOVA trial of niraparib plus bevacizumab in patients with ovarian cancer.
TESARO and Zai Lab (Shanghai) Co., Ltd. announced a collaboration to support the development and commercialization of niraparib for patients in China and the potential to advance two immuno-oncology programs outside of China.
The Phase 1 dose escalation study of TSR-022, an anti-TIM-3 antibody candidate, was initiated in July, and the Phase 1 trial of TSR-042, an anti-PD-1 antibody candidate, continues to enroll.
Third Quarter 2016 Financial Results

TESARO reported a net loss of $101.2 million, or ($1.98) per share, for the third quarter of 2016, compared to a net loss of $66.6 million, or ($1.66) per share, for the third quarter of 2015.

Net product revenue for the third quarter of 2016 totaled $2.8 million and included sales of VARUBI from specialty pharmacy customers to patients and from specialty distributors to providers that were made during the third quarter, as well as sales from specialty distributors to providers that occurred in the second quarter of 2016. License, collaboration and other revenue for the third quarter of 2016 totaled $0.9 million and included amortization of milestone payments and shipments of clinical materials under our license agreements with Hengrui and Janssen.

Research and development expenses increased to $60.8 million for the third quarter of 2016, compared to $40.1 million for the third quarter of 2015, driven primarily by higher costs related to the ongoing registration trials of niraparib, manufacturing costs associated with niraparib, and the initiation of clinical studies for our immuno-oncology portfolio, in addition to increased headcount.

Selling, general and administrative expenses increased to $37.7 million for the third quarter of 2016, compared to $22.8 million for the third quarter of 2015, primarily due to higher commercial headcount, including the establishment of a U.S. field sales organization in August 2015, commercial activities in support of the launch of VARUBI, costs associated with the establishment of our international headquarters, and higher professional service fees.

Operating expenses, as described above, include total non-cash, stock-based compensation expense of $12.9 million for the third quarter of 2016, compared to $8.1 million for the third quarter of 2015.

As of September 30, 2016, TESARO had approximately $647 million in cash and cash equivalents, which includes the $409 million in net proceeds from a follow-on offering of 5.3 million shares of common stock that was completed in July 2016. For the quarter ended September 30, 2016, TESARO had approximately 51.2 million shares outstanding on a weighted average basis.

In anticipation of four product launches in 2017, TESARO will continue to invest in pre-launch inventory manufacturing, development of supply chain capabilities and capacity, and expansion of European and targeted U.S. commercial operations, in addition to making milestone payments for regulatory submissions. As a result of these investments, the Company expects its cash and cash equivalents balance to decline by approximately $100 million during the fourth quarter of 2016.

Corporate Objectives

The following is a summary of TESARO’s key objectives:

VARUBI (rolapitant):

Achieve #1 market share position within the oral NK-1 receptor antagonist market by year-end 2016 in the U.S.;
Launch VARUBI IV into the U.S. market in 1H 2017, pending FDA approval;
Establish a European commercial organization; and
Launch VARUBI oral in Europe in 1H 2017, pending EMA approval.
Niraparib:

Continue commercial preparations in support of the launches of niraparib in the U.S. in 1H 2017 and Europe in 2H 2017, pending regulatory approvals;
Report QUADRA data in 2H 2017;
Report Phase 3 BRAVO data in 2H 2017;
Finalize a potential lung cancer registration strategy and initiate development program in 1H 2017; and
Determine the potential registration strategy for niraparib plus an anti-PD-1 antibody in ovarian cancer and triple-negative breast cancer in 2H 2017.
Immuno-Oncology Portfolio:

Identify a dose and schedule for TSR-042 (anti-PD-1 antibody) by the end of 2016;
Select at least one bispecific antibody clinical candidate by the end of 2016;
Identify the first clinical candidate within the MD Anderson collaboration in 1H 2017;
Initiate a Phase 1 study of TSR-033 (anti-LAG-3 antibody) in 1H 2017;
Finalize the TSR-042 registration strategy and initiate a registration program in 1H 2017; and
Initiate a Phase 1 clinical trial of TSR-022 in combination with an anti-PD-1 antibody in mid-2017.

On November 21 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a preclinical and clinical-stage pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center ("MD Anderson"), reported its financial and operating results for the third quarter ended September 30, 2016 (Press release, Moleculin, NOV 3, 2016, View Source [SID1234516729]).

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During the third quarter and year to date, key activities included:
Updated the Annamycin clinical strategy to add a Phase I arm to its next Phase II trial that leverages a potential increase in the maximum tolerable drug dose, which could significantly increase the chance for positive outcomes. Despite some likely cost increases, which the Company believes will be offset by the Dermin supply agreement, as well as a likely extension in approval timing by several months, the Company believes that it remains on track to generate useful Phase II data by the second half of 2017;
Secured an agreement with Dermin Sp. Zo. O. to utilize its supply of Annamycin for the Company’s clinical trial, substantially reducing expenditures required of Moleculin for drug product and shortening the time required to produce clinical supplies;
Announced it had received verbal positive guidance from the FDA regarding its planned IND submission indicating that the Company may incorporate by reference the IND established by a prior developer;
Benefitting from additional grant funded research at MD Anderson for WP1066;
Announced promising initial results on the preclinical toxicology work for WP1122. Preliminary escalating single dose toxicity testing in mice was successfully completed. No toxic death was observed and the drug was well tolerated;
Appointed a new CFO, Jonathan P. Foster; and
Completed successful initial public offering and bridge financing.
Planned activities and milestones for the remainder of 2016 include:
Receive pre-IND guidance from FDA for liposomal Annamycin, an anthracycline for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML);
Submit IND for liposomal Annamycin based on FDA guidance;
Strengthen license and IP portfolio; and
Continue development of pipeline assets, including drug and other molecular portfolio.
Walter Klemp, Chairman and CEO of Moleculin stated: "We continue to make progress towards executing on our clinical programs and are pleased with recent developments that allow us to more cost effectively fund our activities and potentially improve target outcomes, while maintaining our milestone to report Phase II data by the second half of 2017. We continue to believe we have sufficient funds to pursue our planned operations through the generation of Phase II data for Annamycin through the end of the third quarter of 2017."
Unaudited Financial Results for the Third Quarter Ended September 30, 2016
Research and development expense was $496,659 and $38,409 for the three months ended September 30, 2016 and 2015, respectively. The increase of approximately $458,000 mainly represents accrued license fees to MD Anderson for approximately $40,000, $37,500 for research performed by HPI, and approximately $228,000 related to MD Anderson sponsored research.
General and administrative expense was $924,041 and $184,344 for the three months ended September 30, 2016 and 2015, respectively. The expense increase of approximately $740,000 was mainly attributable to additional payroll and related expenses of approximately $459,000 related to a full three months of our Chief Financial Officer’s, Chief Operating Officer’s and Chief Executive Officer’s salaries and compensation for our Board of Directors. Also, included in this quarter’s expense was $118,000 related to the severance of the former Chief Financial Officer. The Company also incurred approximately $289,000 of expenses related to investor relations, audit and accounting, and insurance costs.
Interest expense included expense accrued on the Company’s convertible promissory notes issued in 2015 and 2016 bearing interest at the rate of 8% per annum.
The Company’s net loss for the three months ended September 30, 2016 amounted to $1,432,079.
As of September 30, 2016, the Company had $6,183,783 in cash. During the period from January 1, 2016 through May 2, 2016, the Company sold 234,296 common shares for $702,888. On May 31, 2016, the Company completed its initial public offering, pursuant to which it sold 1,540,026 shares of common stock at $6.00 per share for net proceeds of $8,464,183 after deducting underwriting discounts and commissions and direct offering expenses.
Net cash used in operating activities was $2,596,647 for the nine months ended September 30, 2016 and mainly included payments made for payroll, travel, insurance and professional fees to the Company’s consultants, attorneys and accountants for services related to becoming a publicly traded company and related filing fees, along with payments made to MD Anderson for license and maintenance fees. Additionally, prepayments were made for insurance.
Net cash used in investing activities was $109,793 for the nine months ended September 30, 2016 and primarily represents the cash paid to acquire Moleculin, LLC.
Net cash provided by financing activities was $8,862,132 for the nine months ended September 30, 2016. The Company received $8,464,183 net proceeds from its IPO stock issuance, $702,888 from issuance of common shares at $3 per share, and $165,000 from issuance of convertible notes. Net cash used in financing activities included approximately $470,000 for payments of notes payable.
(Tables to follow)
Restatement of the Unaudited Financial Results for the Second Quarter Ended
June 30, 2016
Today, the Company filed its restated unaudited financial statements for the Second Quarter Ended June 30, 2016 with the Security and Exchange Commission ("SEC") on Form 10-Q/A.
The Company identified the following non-cash errors due to an error in the accounting for the business combination of Moleculin, LLC. The impact of the correction of the error was as follows:
1 – The net loss for the three and six months ended June 30, 2016 was overstated by approximately $256,889 due to amortization of an intangible which was recorded in error. Upon correction, the net loss for the period will be $738,727 and $1,070,968, respectively.
2 – A liability in the amount of $750,000 should not have been reflected in the balance sheet as of June 30, 2016. Upon correction for this and item 1 above, the total for Liabilities and Stockholders’ Equity was $18,740,288.
3 – Intangibles were overstated by $750,000 before the amortization mentioned above. Upon correction, total assets was $18,740,288.

On November 2, 2016 Foundation Medicine, Inc. (NASDAQ:FMI) reported financial and operating results for its third quarter ended September 30, 2016 (Press release, Foundation Medicine, NOV 2, 2016, View Source [SID1234516193]). Highlights for the quarter included:

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Achieved third quarter revenue of $29.4 million, 16% year-over-year growth;
Reported 11,627 clinical tests in the third quarter, 45% year-over-year growth;
Grew FoundationCORE, the company’s molecular information knowledgebase, to nearly 100,000 patient cases;
Announced acceptance of FoundationOne for Parallel Review by FDA and CMS. If approved, FoundationOne could be the first FDA-approved comprehensive genomic profiling (CGP) assay to incorporate multiple companion diagnostics to support precision medicine in oncology and would be offered as a covered benefit to Medicare beneficiaries nationwide;
Added new immunotherapy clinical markers, Tumor Mutational Burden (TMB) and Microsatellite Instability (MSI), to FoundationOne and FoundationOne Heme to help guide personalized immunotherapy-based treatment plans;
Published an additional 17 manuscripts in high-quality, peer-reviewed journals and delivered six podium and poster presentations at various medical and scientific meetings providing further evidence for the clinical impact of integrating CGP into routine cancer care;
Expanded patient access to CGP through Palmetto, the Medicare Administrative Contractor in North Carolina, who broadened a Local Coverage Determination, covering CGP for all stage IIIB and IV non-small cell lung cancer (NSCLC) patients at diagnosis by removing a patient’s smoking status as a coverage factor.
Foundation Medicine reported total revenue of $29.4 million in the third quarter of 2016, compared to $25.4 million in the third quarter of 2015. Revenue from biopharmaceutical partners grew 77% to $20.7 million in the third quarter of 2016, compared to $11.7 million in the third quarter of 2015. The increase in revenue demonstrates the company’s leading role in molecular information and the value of this information in informing and accelerating drug development for its oncology focused biopharmaceutical customers.

Revenue from clinical testing in the third quarter of 2016 was $8.7 million, compared to $13.7 million in the third quarter of 2015. The decrease was driven by various factors, the most significant of which was the transition in-network with a large national payer for stage IV NSCLC testing, which resulted in the termination of payments for testing in other indications.

The company reported 11,627 clinical tests in the third quarter of 2016, a 45% increase from the same quarter last year. This reported volume number includes 9,398 FoundationOne tests, 1,325 FoundationOne Heme tests and 904 FoundationACT tests.

"Foundation Medicine delivered another solid quarter highlighted by strong biopharma revenue and record clinical testing volume," said Michael Pellini, M.D., chief executive officer of Foundation Medicine. "We believe our progress on building distinct, yet synergistic clinical and biopharma businesses, developing applications that enable patient access to therapies and clinical trials, aggressively working towards reimbursement coverage and payment for our comprehensive genomic profiling assays and pursuing parallel review with FDA and CMS for FoundationOne will drive continued growth and value creation."

The company’s molecular information knowledgebase, FoundationCORE, grew to nearly 100,000 patient cases at the end of the third quarter. FoundationCORE is a unique asset and critical component of the value that Foundation Medicine delivers to its biopharmaceutical and physician customers. The increasing scale and breadth of this high quality, clinically relevant oncology data set derived from the company’s testing platform continues to enhance clinical practice and help enable improved outcomes for patients.

Total operating expenses for the third quarter of 2016 were approximately $44.9 million compared with $35.6 million for the third quarter of 2015. Net loss was approximately $31.3 million in the third quarter of 2016, or a $0.90 loss per share. At September 30, 2016, the company held approximately $167.9 million in cash, cash equivalents and marketable securities.

Recent Enterprise Highlights

Announced a collaboration with Sarah Cannon Research Institute (SCRI) in which SCRI will utilize Foundation Medicine’s full suite of assays to accelerate patient enrollment into clinical trials and facilitate data sharing across its network in the U.S.
Selected as the molecular information provider of choice by The Leukemia & Lymphoma Society for its landmark Beat AML Master Trial, a prospective study aimed at delivering a precision medicine approach to treat newly diagnosed Acute Myeloid Leukemia patients.
Announced the election of life sciences leader, Michael R. Dougherty, to its Board of Directors and the Board’s Audit Committee. Mr. Dougherty joins as an independent director and fills an existing vacancy on the Board.
2016 Outlook

Foundation Medicine’s business and financial outlook for 2016 is the following:

The company continues to expect 2016 revenue will be in the range of $110 to $120 million.
The company is increasing the bottom-end of its clinical volume guidance range and now expects to deliver between 40,000 and 41,000 FoundationOne and FoundationOne Heme clinical tests in 2016.
The company continues to expect operating expenses will be in the range of $175 and $185 million.

On November 2, 2016 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported financial results for the third quarter ended September 30, 2016 (Press release, NanoString Technologies, NOV 2, 2016, View Source [SID1234516304]).

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Third Quarter Financial Highlights

Total revenue of $23.9 million, 53% year-over-year growth
Total product and service revenue of $19.2 million, 38% year-over-year growth
Consumables revenue of $11.5 million, including $1.1 million of Prosigna IVD kits, 27% year-over-year growth
Instrument revenue of $6.9 million, 62% year-over-year growth
Collaboration revenue of $4.8 million
"We continued to execute well during the third quarter, generating strong growth across our business while advancing our product pipeline and partnerships," said president and chief executive officer Brad Gray. "A highlight of the quarter was the robust demand for our nCounter SPRINT Profiler, which helped drive 62% year-on-year growth in instrument revenue and validated that SPRINT’s ability to reach new customers is accelerating instrument placement."

Recent Business Highlights

Grew installed base to approximately 450 nCounter Analysis Systems at September 30, 2016
Launched new nCounter Vantage 3D Solid Tumor Panels for proteins and single nucleotide variations to enable simultaneous analysis of DNA mutations, messenger RNA, fusion genes, and proteins on a single platform
Presented data demonstrating the potential workflow advantages of Hyb & Seq sequencing chemistry, requiring less than 60 minutes of sample processing to enable initiation of a sequencing run
Appointed Kirk Malloy, Ph.D., seasoned life sciences executive, to the company’s board of directors
Third Quarter Financial Results

Revenue for the three months ended September 30, 2016 increased by 53% to $23.9 million, as compared to $15.7 million for the third quarter of 2015. Instrument revenue was $6.9 million, up 62% versus the prior year period, with nCounter SPRINT systems representing approximately half of systems sold. Consumables revenue, excluding Prosigna, was $10.3 million for the third quarter of 2016, 23% higher than in the comparable 2015 quarter. Prosigna IVD kit revenue was $1.1 million for the quarter, an increase of 73% over the third quarter of 2015. Collaboration revenue totaled $4.8 million, compared to $1.8 million for the third quarter of 2015. Gross margin on product and service revenue was 58% for the third quarter of 2016, up from 55% for the prior year period.

Research and development expense increased by 50% to $8.7 million for the third quarter of 2016 versus $5.8 million for the third quarter of 2015, reflecting increased costs associated with biopharma collaborations announced earlier this year and new products and technologies under development for the life science research market. Selling, general and administrative expense increased by 30% to $15.6 million for the third quarter of 2016 compared to $12.0 million for the prior year period.

Net loss for the three months ended September 30, 2016 increased to $10.1 million, or a loss of $0.51 per share, compared with $9.5 million, or a loss of $0.49 per share, for the third quarter of 2015.

Outlook for 2016

The company’s financial outlook for 2016 is unchanged, and includes:

Total revenue in the range of $89 million to $93 million
Gross margin on product and service revenues in the range of 54% to 55%
Operating expenses in the range of $94 million to $99 million
Operating loss in the range of $37 million to $40 million
Net loss per share in the range of $2.15 to $2.30
Cash from collaborations in 2016 in the range of $40 million to $45 million

On November 2, 2016 Bio-Path Holdings, Inc., (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported the enrollment and dosing of the first patient in the efficacy portion of its Phase 2 clinical study of BP1001, a liposomal Grb2 antisense for the treatment of acute myeloid leukemia (AML) (Filing, 8-K, Bio-Path Holdings, NOV 2, 2016, View Source [SID1234516251]). The objective of the Phase 2 study is to further assess the efficacy and safety of BP1001, Bio-Path’s lead development candidate.

The Phase 2 clinical trial is a multicenter study of BP1001 in combination with low dose cytarabine (LDAC) in patients with previously untreated AML who are not otherwise eligible for standard or high-intensity chemotherapy regimens or who have elected a low-intensity regimen.

The trial is a single arm, open label, two-stage design to assess the safety profile, pharmacokinetics, pharmacodynamics, and efficacy of 60 mg/m2 of BP1001 in combination with LDAC compared to historical response rates documented for LDAC alone. Evaluable patients will receive an initial dose intravenous (IV) infusion of BP1001 over 60 minutes and every three days thereafter, as eight doses per 28-day cycle of 60 mg/m2 BP1001, and will be administered LDAC as a subcutaneous (SQ) injection, twice daily for 20 consecutive doses per 28-day cycle.

The primary endpoint of the study is the number of patients who achieve Complete Remission (CR), including CR with incomplete hematologic recovery (CRi) and CR with incomplete platelet recovery (CRip). Secondary endpoints assessing the safety and efficacy of BP1001 include overall survival, time to response, duration of response, and adverse events as evaluated by physical examination findings, vital signs and clinical laboratory tests.

The full trial design includes approximately 54 evaluable patients with an interim analysis performed after 19 patients. In the event the interim results exceed the primary endpoint in a number of patients that meets or exceeds statistically determined thresholds, the Company may seek to convert the trial into a registration trial for accelerated approval.

Among the sites registered to conduct the study are Weill Medical College of Cornell University, Baylor Scott & White Health, The University of Kansas and The University of Texas MD Anderson Cancer Center.

"This is an exciting milestone for Bio-Path as it will be the first study to confirm the efficacy of BP1001 as a treatment for AML and to validate our DNAbilizeTM platform," said Peter H. Nielsen, Chief Executive Officer of Bio-Path Holdings. "We are particularly pleased with the Phase 2 trial design, which has a built-in interim analysis that offers a pathway to an accelerated approval should the efficacy results for the first 19 evaluable patients demonstrate the high response rate seen in the safety segment of our Phase 2 trial. We look forward to the completion of this study and expect its results to replicate these very promising early data," added Mr. Nielsen.

Patients in the safety segment of the trial treated with 60 mg/m2 and 90 mg/m2 of BP1001 twice a week over a four-week period, in combination with a standard regimen of frontline low-dose cytarabine (LDAC), showed BP1001 to be safe and well tolerated, with signs of significant anti-leukemia activity. Of the six evaluable patients included in both cohorts of the safety segment, three achieved complete remissions, while two others achieved partial remission. There were no attributable adverse events reported.

As previously reported, BP1001’s pharmacokinetics at a dose of 60 mg/m2 had a 30-hour half-life, significantly better than the half-life with a dose of 90 mg/m2. The final analysis of these data, along with the demonstrated reductions in bone marrow blasts, suggested that 60 mg/m2 is the appropriate dose for use in the Phase 2 trial. Administratively, this required Bio-Path to reformat documents for the Phase 2 trial with the 60 mg/m2 dose and resubmit for approvals with the U.S. Food and Drug Administration (FDA) and site Institutional Review Boards, requiring additional time prior to starting the Phase 2 trial.

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