On November 2, 2016 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the third quarter ending September 30, 2016 (Press release, PTC Therapeutics, NOV 2, 2016, View Source [SID1234516313]).

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"For the past 18 years, we have focused significant effort towards developing Translarna for those affected by Duchenne," stated Stuart W. Peltz, Ph.D. Chief Executive Officer, PTC Therapeutics, Inc. "Over 400 Duchenne patients have participated in our clinical trials dating back to 2005 and the vast majority continue to remain on Translarna including approximately 130 boys in the United States. With this high compliance rate, we believe these actions speak to the benefit Translarna is providing to patients in the U.S. and around the world. In the E.U., regulatory discussions continue and we anticipate an opinion from the CHMP before the end of the year. With respect to the U.S., we will continue to escalate our appeal so that we may have the opportunity to have the Translarna NDA submission reviewed by the FDA. Our goal remains to deliver this novel therapy to nonsense mutation Duchenne patients globally."

Key Third Quarter and Other Corporate Highlights:

Translarna net sales of $22M in the third quarter represents 125% year-over-year growth. Commercial access to Translarna continued to expand during the quarter with a significant number of new patients coming from England. While pricing and reimbursement discussions with a number of European countries are ongoing, PTC has now successfully concluded pricing and reimbursement negotiations in both Italy and Romania. PTC anticipates growth from both additional penetration into existing markets as well as expanding access in new geographies across Central and Eastern Europe, the Middle East and Latin America.
EMA regulatory discussions regarding Translarna’s marketing authorization are ongoing. PTC continues to interact with the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) in connection with the company’s ongoing request for annual renewal of its marketing authorization for Translarna. Following the company’s CHMP meeting in October, the committee requested additional information regarding the risk-benefit profile of Translarna, its efficacy and the design of a potential trial that would provide comprehensive clinical data. While we have provided information in response to the CHMP’s requests and expect to continue to engage in further interactions, recent dialogue has introduced a higher degree of uncertainty as to the outcome. PTC anticipates that an opinion regarding its marketing authorization renewal request will be adopted by the CHMP before the end of 2016. The current marketing authorization remains valid while the EMA assessment is ongoing and until a decision is made by the European Commission.
Appeal process of FDA’s Refuse to File regarding Translarna for DMD continues. As PTC previously stated, the RTF appeal could be an iterative process requiring multiple rounds before a conclusion. Having had its appeal denied, the company intends to continue the appeal to higher levels of the FDA. PTC continues to believe that the FDA can only accomplish a proper assessment of the data and analyses based on the results of the company’s extensive clinical research and experience in more than 400 boys in the context of a full and fair review. This would include an advisory committee meeting that allows DMD clinical experts and representatives of the patient community to express their views on Translarna for the treatment of nmDMD. PTC intends to provide an update following a final conclusion of the appeal process or, alternatively, following a determination by the company to pursue an alternate regulatory strategy for advancing the potential approval of Translarna for the treatment of nmDMD in the U.S.
ACT CF Phase 3 clinical trial results expected late first quarter 2017. PTC’s confirmatory Phase 3 ACT CF clinical trial, a 48-week placebo-controlled trial designed to evaluate the efficacy of Translarna in patients six years of age or older with nonsense mutation cystic fibrosis, is ongoing. Following feedback from the FDA and the company’s CHMP rapporteurs during the third quarter, PTC modified the protocol for ACT CF. In-line with clinical trials for other approved CF therapies, the primary endpoint of lung function as measured by FEV1 was updated from relative change to absolute change in percent predicted FEV1. Pulmonary exacerbations will be an important secondary endpoint.
SMA program advancing with Phase 2 SUNFISH trial initiated in Type 2/3 patients. PTC’s joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation initiated a Phase 2 study in pediatric and adult Type 2/3 SMA patients. SUNFISH, is a two-part study investigating the safety, tolerability and efficacy of RG7916, an oral small molecule survival motor neuron 2 (SMN2) splicing modifier. The first part of the study will evaluate safety and tolerability through escalating doses of RG7916. After dose selection, the study will transition into the pivotal second part evaluating the efficacy of RG7916. Initiation of the pivotal second part of the study is expected to begin in 2017 and will trigger a $20 million milestone payment to PTC from Roche. A similarly designed two-part study to evaluate RG7916 in Type 1 SMA patients is expected to begin in the coming months.
PTC596 cancer stem cell program to advance in clinical development in 2017.
Phase 1 safety data from PTC’s clinical oncology program were recently presented at the European Society for Medical Oncology Congress. The ongoing Phase 1 dose-escalation study is evaluating the safety, tolerability and pharmacokinetics of PTC596 in patients with advanced solid tumors as a monotherapy. Preliminary results demonstrate that PTC596 is generally well tolerated at doses associated with exposures that achieved or exceeded efficacious target plasma concentrations in preclinical studies. Escalating doses are being evaluated for safety, pharmacodynamics, and to determine a target dose for subsequent studies. PTC596 is a novel, oral investigational drug that reduces the levels of BMI1, a protein that is required for cancer stem cell survival. Based on its proposed mechanism of action, PTC596 is expected to be most efficacious when used as part of combination therapy. Continued clinical development of PTC596 is being planned for 2017.
Third Quarter Financial Highlights:

Translarna net product sales were $22.0 million for the third quarter of 2016, representing a 125% increase versus $9.8 million in the third quarter of 2015. Net sales increased in conjunction with continued expansion of commercial access to Translarna for nonsense mutation DMD boys outside of the U.S.
Total revenues for the third quarter of 2016 were $23.0 million, an increase of $13.2 million or 135% compared to $9.8 million in the same period of 2015. The change in total revenue was primarily a result of the expanded commercial launch of Translarna.
GAAP R&D expenses were relatively flat at $31.4 million for the third quarter of 2016 compared to $30.6 million for the same period in 2015. Non-GAAP R&D expenses were $27.1 million for the third quarter of 2016, excluding $4.3 million in non-cash, stock-based compensation expense, compared to $26.8 million for the same period in 2015, excluding $3.8 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $23.7 million for the third quarter of 2016 compared to $21.4 million for the same period in 2015. Non-GAAP SG&A expenses were $19.0 million for the third quarter of 2016, excluding $4.6 million in non-cash, stock-based compensation expense, compared to $17.1 million for the same period in 2015, excluding $4.2 million in non-cash, stock-based compensation expense. The increase in SG&A expense for the third quarter 2016 as compared to the prior year period primarily resulted from additional costs associated with commercial activities in support of Translarna across Europe and other regions.
Net interest expense for the third quarter of 2016 was $2.1 million compared to net interest expense of $0.9 million in the same period in 2015. The increase in interest expense is primarily a result of the $150 million convertible debt offering completed during mid-third quarter 2015. The debt was recorded on PTC’s balance sheet at a discount, which will be amortized over the life of the bond.
Net loss for the third quarter of 2016 was $35.2 million, a decrease of $8.1 million compared to a net loss of $43.2 million for the same period in 2015.
Cash, cash equivalents, and marketable securities totaled approximately $248.3 million at September 30, 2016 compared to approximately $338.9 million at December 31, 2015.
Shares issued and outstanding as of September 30, 2016 were 34.3 million, which includes 0.2 million shares of unvested restricted stock.
2016 Guidance:

PTC expects to achieve total ex-US nmDMD Translarna net sales in the middle of our guidance of $65 to $85 million for 2016.
Operating expenses for the full year 2016 are now anticipated to be between $180 million and $190 million, excluding expected non-cash stock-based compensation expense of approximately $35 million, for total operating expenses of approximately $215 million to $225 million. These expenses are in support of our ongoing clinical trials for Translarna in nmDMD and nmCF, commercial launch activities for Translarna outside of the U.S., and the continued research and clinical development of other product pipeline candidates.
PTC expects to end 2016 with cash and cash equivalents of approximately $220 million.

On November 2, 2016 Neurocrine Biosciences, Inc. (NASDAQ:NBIX) reported its financial results for the quarter ended September 30, 2016 (Press release, Neurocrine Biosciences, NOV 2, 2016, View Source;p=RssLanding&cat=news&id=2218719 [SID1234516306]). For the third quarter of 2016, the Company reported a net loss of $36.9 million, or $0.43 loss per share, compared to a net loss of $34.4 million, or $0.40 loss per share, for the same period in 2015. For the nine months ended September 30, 2016, the Company reported a net loss of $96.4 million, or $1.11 loss per share, as compared to net loss of $59.6 million, or $0.71 loss per share, for the first nine months of last year.

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The Company’s balance sheet at September 30, 2016 reflected total assets of $399.2 million, including cash, cash equivalents, investments and receivables of $385.3 million.

"Our NDA for INGREZZATM in tardive dyskinesia was recently accepted by the FDA for Priority Review and we look forward to continuing our work with the FDA to potentially bring this important treatment option to patients and physicians," said Kevin Gorman, President and Chief Executive Officer of Neurocrine Biosciences. "In addition, our partner AbbVie presented Phase III data for elagolix in endometriosis at the 72nd American Society for Reproductive Medicine Scientific Congress & Expo. The results from the Phase III Violet Petal and Solstice clinical trials demonstrate that elagolix has the potential to be an important treatment for women suffering from endometriosis."

Research and development expenses were $20.9 million during the third quarter of 2016 compared to $24.4 million for the same period in 2015. Quarterly external development expense decreased by approximately $2.5 million from 2015 to 2016 primarily due to the completion of certain INGREZZA (valbenazine) manufacturing activities and the conclusion of the Kinect 3 clinical trial in 2016. Additionally, share-based compensation expense for research and development was $3.6 million lower during the third quarter of 2016 compared to the third quarter of 2015. The decrease in share-based compensation expense is primarily due to certain performance-based restricted stock units which met the criteria for expensing during the third quarter of 2015.

For the nine months ended September 30, 2016, research and development expenses were $71.7 million, compared to $59.7 million for the same period last year. This increase was primarily due to expenses related to the Company’s compilation and submission of the New Drug Application (NDA) for INGREZZA in tardive dyskinesia. Additionally, external clinical development expenses related to INGREZZA, which is being evaluated in both tardive dyskinesia and Tourette syndrome, accounted for $4.0 million of the increase in year-to-date expenses.

General and administrative expenses increased from $11.5 million for the third quarter of 2015 to $17.5 million for the third quarter of 2016. For the nine months ended September 30, 2016, general and administrative expenses were $44.4 million, compared to $23.5 million for the first nine months of 2015. The overall increase in general and administrative expense is primarily due to pre-commercialization activities for INGREZZA. General and administrative share-based compensation expense was $4.0 million lower during the third quarter of 2016 compared to the third quarter of 2015. The decrease in quarterly share-based compensation expense is primarily due to certain performance-based restricted stock units which met the criteria for expensing during the third quarter of 2015. Other personnel related costs increased by $3.8 million quarter over quarter, and by $7.6 million for the first nine months of 2015 compared to the first nine months of 2016 primarily due to the expansion of sales and marketing and medical affairs personnel. Additionally, professional costs related to market research and other pre-commercial activities increased by $5.4 million quarter over quarter and by $11.9 million for the first nine months of 2016 compared to the same period in 2015.

Updated 2016 Financial Guidance

The Company expects to end 2016 with approximately $340 million in cash, investments and receivables. The previous financial guidance was to end 2016 with approximately $320 million in cash, investments and receivables. Total expenses for 2016 are expected to be approximately $160 to $170 million. The previous financial guidance for 2016 expenses ranged from $185 to $195 million. The lower than anticipated cost of preclinical and clinical development as well as lower headcount contributed to this reduction in estimated 2016 expenses.

Pipeline Highlights

INGREZZA (valbenazine) Update

The NDA for INGREZZA for the treatment of tardive dyskinesia was submitted to the U.S. Food and Drug Administration (FDA) during the third quarter of 2016. The NDA was accepted for Priority Review by the FDA and received a Prescription Drug User Fee Act (PDUFA) target action date of April 11, 2017.

During the third quarter of 2016, the Company completed the Kinect 3 study, a Phase III trial that included moderate to severe tardive dyskinesia in patients with underlying schizophrenia, schizoaffective disorder, bipolar or major depressive disorder who underwent six weeks of placebo controlled assessment. Subsequent to the initial six weeks of treatment, subjects were eligible to continue in the Kinect 3 study for up to 42 weeks of additional INGREZZA treatment. The Company had previously announced positive efficacy results from the six-week placebo-controlled portion of the Kinect 3 study during the fourth quarter of 2015.

The Company is currently conducting a separate one-year open-label safety study of INGREZZA, Kinect 4. This study is fully enrolled and expected to complete its one year of dosing in early 2017.

The Company is also supporting an INGREZZA roll-over study for those patients who complete the one year of dosing in either the Kinect 3 or Kinect 4 studies. This roll-over study is designed to permit open-label access to INGREZZA for up to an additional 72 weeks of treatment.

INGREZZA is also being investigated in Tourette syndrome through two ongoing placebo-controlled Phase II Tourette syndrome studies in adults and pediatrics, the T-Forward study and T-Force GREEN study, respectively.

The T-Forward study is a randomized, double-blind, placebo-controlled, multi-dose, parallel group study that has completed enrollment of 124 adults with moderate to severe Tourette’s. The adult Tourette patients are receiving once-daily dosing of INGREZZA or placebo during the eight-week treatment period to assess the safety, tolerability and efficacy of INGREZZA. The primary endpoint of this study is a change from baseline of placebo vs. active scores utilizing the Yale Global Tic Severity Scale at the end of Week 8. Top-line data from this study is expected in January 2017.

The T-Force GREEN study is a randomized, double-blind, placebo-controlled, multi-dose, parallel group study of up to 90 children and adolescents. Pediatric Tourette subjects receive once-daily dosing of INGREZZA or placebo during a six-week treatment period to assess the safety, tolerability and efficacy of INGREZZA. The primary endpoint of this study is the change from baseline of the Yale Global Tic Severity Scale between placebo and active treatment groups at the end of Week 6. Top-line data from this study is expected in early 2017.

Additionally, the Company has also launched an open-label, fixed-dose study of INGREZZA in up to 180 subjects with Tourette syndrome. This study is designed to enroll up to 90 children and adolescents and up to 90 adults who have completed either of the two placebo-controlled Tourette clinical trials: T-Force GREEN or T-Forward. This Phase II study will assess the long-term safety and tolerability of INGREZZA in children and adults with Tourette’s.

Elagolix Update

In October, AbbVie presented multiple scientific abstracts at the 72nd American Society for Reproductive Medicine Scientific Congress & Expo in Salt Lake City. The posters and oral presentations highlighted positive primary and secondary efficacy endpoint data from the Phase III studies of elagolix in premenopausal women who suffer from endometriosis as well as research on the economic burden of endometriosis and endometriosis-related surgery in women in the United States:

Elagolix, An Oral Gonadotropin-Releasing Hormone (GnRH) Antagonist, For The Management Of Endometriosis-Associated Pain: Safety And Efficacy Results From Two Double-Blind, Randomized, Placebo-Controlled Studies; Taylor H, et al.
Use Of Elagolix For The Management Of Endometriosis-Associated Pain: Secondary Efficacy Results From Two Randomized, Placebo-Controlled Studies; Surrey, et al.
The Effect of Elagolix On Bone Mineral Density: Safety Results From Two Randomized, Placebo-Controlled Studies In Women With Endometriosis-Associated Pain; Archer et al.
Incremental Costs of Healthcare and Work Loss Attributed to Endometriosis in a Cohort of Commercially Insured Women; Soliman et al.
Incidence of Comorbidities Among Women with Endometriosis: A Retrospective Matched Cohort Study; Soliman et al.
The Effect Of Elagolix On The Endometrium: Safety Results From Two Randomized, Placebo-Controlled Studies In Women With Endometriosis-Associated Pain; Diamond et al.
The Impact of Elagolix on Quality of Life in Women with Endometriosis-Associated Pain: Results From Two Randomized, Placebo-Controlled Studies Using the Endometriosis Health Profile Questionnaire; Taylor H et al.
Direct and Indirect Costs Associated with Endometriosis-Related Surgery Among Employed Women in the US; Soliman et al.
During the first quarter of 2016, AbbVie announced positive top-line results from the second of two Phase III clinical trials, the Solstice Study, a multinational study designed to evaluate the efficacy and safety of elagolix in 815 premenopausal women with endometriosis. The top-line results from this trial were consistent with those of the initial Phase III clinical trial, the Violet Petal Study, where after six months of treatment, both doses of elagolix (150 mg once-daily and 200 mg twice-daily) met the study’s co-primary endpoints of reducing scores of non-menstrual pelvic pain and menstrual pain (or dysmenorrhea) associated with endometriosis at month three, as well as month six, as measured by the Daily Assessment of Endometriosis Pain scale. The observed safety profile of elagolix in the Solstice Study was consistent with observations from prior studies. Among the most common adverse events (AEs) were hot flush, headache and nausea. While most AEs were similar across treatment groups some, such as hot flush and bone mineral density loss, were dose-dependent. AbbVie is targeting a 2017 NDA filing with the FDA for elagolix in endometriosis.

In early 2016, AbbVie announced the initiation of the Phase III uterine fibroids program consisting of two replicate randomized, parallel, double-blind, placebo-controlled clinical trials evaluating elagolix alone or in combination with add-back therapy in women with heavy uterine bleeding associated with uterine fibroids. The studies are expected to enroll approximately 400 subjects each for an initial six-month placebo-controlled dosing period. At the end of the six-months of placebo-controlled evaluation, subjects are eligible to enter an additional six-month safety extension study. The primary efficacy endpoint of the study is an assessment of the change in menstrual blood loss utilizing the alkaline hematin method comparing baseline to month six. Additional secondary efficacy endpoints will be evaluated including assessing the change in fibroid volume and hemoglobin. Bone mineral density will be assessed via DXA scan at baseline, the conclusion of dosing and six months post-dosing. The Company expects the initial top line efficacy data from the uterine fibroid Phase III program in 2017.

Essential Tremor Program (NBI-640756) Update

The Company has successfully completed an initial Phase I single site, randomized, double-blind, placebo-controlled, sequential dose-escalation, pharmacokinetic study assessing the safety and tolerability of a single dose of NBI-640756 in up to 32 healthy volunteers.

Based on the results of this initial study, the Company initiated a second Phase I, single site, randomized, double-blind, placebo-controlled, multiple-dose, sequential dose-escalation study to evaluate the safety, tolerability and pharmacokinetics of NBI-640756 in up to 30 healthy volunteers over a week of continuous dosing. The study is being conducted in multiple sequential cohorts of ten subjects per cohort; data from this second Phase I study is expected later in 2016. The data from this study, in conjunction with the single dose Phase I study and preclinical studies, will be evaluated and utilized in the design of the anticipated Phase II program for NBI-640756 in subjects with essential tremor.

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 Immunomedics, Inc. (Nasdaq:IMMU) reported financial results for the first quarter ended September 30, 2016. The Company also highlighted recent key developments and planned activities for its clinical pipeline (Filing, Q3, Immunomedics, 2016, NOV 2, 2016, View Source [SID1234516285]).

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First Quarter Fiscal 2017 Results

Total revenues for the first quarter ended September 30, 2016, were $0.7 million, the same amount reported for the quarter ended September 30, 2015.

Total costs and expenses for the quarter ended September 30, 2016 were $15.7 million, compared to $14.8 million for the same quarter in fiscal 2016, an increase of $0.9 million, or approximately 6%. The increase was due primarily to a $4.9 million increase in research and development expenses related to manufacturing and Phase 2 clinical trials of the antibody-drug conjugates, including sacituzumab govitecan (IMMU-132), which was offset partially by a $3.0 million decrease in research and development expense from the early termination of the Phase 3 PANCRIT-1 clinical trial during the third quarter of fiscal 2016, and a $1.1 million decrease from adjustments for deferred unearned executive bonuses.

Interest expense related to the 4.75% Convertible Senior Notes due 2020 was $1.4 million for both quarters ended September 30, 2016 and September 30, 2015, including amortization of $0.2 million debt issuance costs in each quarter.

Net loss attributable to stockholders was $16.2 million, or $0.17 per basic and diluted share, for the first quarter of fiscal year 2017, compared with net loss attributable to stockholders of $15.4 million, or $0.16 per basic and diluted share, for the same quarter in fiscal 2016, an increase of $0.8 million, or approximately 5%. The increase was due primarily to increased research and development expenses, as described above.

Cash, cash equivalents, and marketable securities were $33.0 million as of September 30, 2016. On October 12, 2016, the Company sold 10 million shares of its common stock and warrants to purchase up to 10 million shares of common stock for net proceeds of approximately $28.5 million.

"The recent offering of stock and warrants has strengthened our balance sheet, which we believe should facilitate uninterrupted clinical and manufacturing activities for IMMU-132, while simultaneously continuing our out-licensing efforts," commented Michael R. Garone, Vice President Finance and Chief Financial Officer. "We are committed to pursuing these strategic goals to completion for the benefit of cancer patients; particularly those with metastatic triple-negative breast cancer, for whom there are no targeted therapies currently available." Mr. Garone added.

The Company’s key clinical developments and future planned activities:

Sacituzumab Govitecan (IMMU-132)

Updated Phase 2 clinical trial results of IMMU-132 in patients with metastatic triple-negative breast (TNBC) have been submitted for publication

Manufacturing of clinical materials for the Phase 3 confirmatory trial in TNBC is progressing according to plan. Large-scale batches have been produced by our Contract Manufacturing Organizations, and are moving through quality control and comparability testing requirements

Patient enrollment in the Phase 2 clinical trial in TNBC is nearing completion with the 100 assessable patients for planned accelerated approval application

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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