On November 1, 2017 Juno Therapeutics, Inc. (NASDAQ: JUNO), a biopharmaceutical company developing innovative cellular immunotherapies for the treatment of cancer, reported financial results and business highlights for the third quarter 2017 (Press release, Juno, NOV 1, 2017, View Source [SID1234521435]).

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“We are pleased with the potential best-in-class profile for JCAR017, and we look forward to presenting an updated dataset at the upcoming ASH (Free ASH Whitepaper) conference,” said Hans Bishop, Juno’s President and Chief Executive Officer. “The clinical data continue to support our belief that a defined cell product can improve patient outcomes. Our broad clinical development programs and ongoing infrastructure and manufacturing investments remain a key part of our strategy to deliver on the potential of CAR T cell therapies for cancer patients across a broad array of diseases.”

Third Quarter 2017 and Recent Corporate Highlights

Clinical Update:

Juno and its collaborators will present 15 abstracts at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH) (Free ASH Whitepaper) and seven abstracts at the upcoming Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meetings.
Presentations at ASH (Free ASH Whitepaper) will include data from the ongoing Phase I TRANSCEND study in patients with relapsed or refractory (r/r) aggressive B-cell NHL who were treated with fludarabine/cyclophosphamide lymphodepletion and JCAR017. New data will be available at multiple presentations, including an oral presentation on Monday, December 11 that will include information on safety and responses. JCAR017 is a defined composition CD19-directed CAR T cell product candidate using a 4-1BB costimulatory domain. Juno believes JCAR017’s clinical profile could enable outpatient administration. The primary TRANSCEND abstract included the following data:

The core group (N=49) includes patients that represent the population that Juno is studying in the ongoing pivotal cohort. The core group includes patients with DLBCL (NOS and transformed from follicular lymphoma) that are ECOG Performance Status 0-1. Topline data from the abstract for both dose levels for the core group as of a data cutoff date of July 7, 2017 included:

Dose level 2 (DL2 = 100 million cells), the dose in our pivotal cohort, showed a 3 month overall response rate (ORR) of 80% (12/15) and a 3 month complete response (CR) rate of 73% (11/15) in the core group. Data support a dose response relationship. Dose level 1 (DL1 = 50 million cells) showed a 3 month ORR of 52% (11/21) and a 3 month CR rate of 33% (7/21).

Across both doses in the core group, the best overall response was 84% (41/49) and the best overall CR rate was 61% (30/49).

There was no increase in cytokine release syndrome (CRS) and neurotoxicity (NT) rates associated with the higher dose or between the full and core groups. Across doses in the full group, 1% (1/69) experienced severe CRS and 14% (10/69) experienced severe NT. 30% (21/69) had any grade CRS and 20% (14/69) had any grade NT. 64% (44/69) had no CRS or NT.

The most common treatment-emergent adverse events other than CRS and NT that occurred at ≥25% in the full group included neutropenia (41%), fatigue (30%), thrombocytopenia (30%), and anemia (26%).
Ongoing enrollment for the pivotal cohort of the TRANSCEND trial at DL2 with BLA filing expected to be completed in the second half of 2018 and with approval as early as 2018.

Announced the Regenerative Medicine Advanced Therapy (RMAT) designation for investigational drug JCAR017 for the treatment of r/r aggressive large B cell NHL, including DLBCL, not otherwise specified (de novo or transformed from indolent lymphoma), primary mediastinal B Cell lymphoma or Grade 3B follicular lymphoma. Similar to breakthrough designation, the pathway enables companies developing cell and tissue based therapies to have earlier and more frequent interactions with the FDA and includes opportunities for accelerated approval, priority review, rolling submissions, and alternative provisions to fulfill post-approval requirements under accelerated approval.

Initiated the PLATFORM trial, a Phase Ib study initially evaluating JCAR017 in combination with durvalumab in adult r/r aggressive NHL patients, in collaboration with Juno’s partner Celgene Corporation.

Initiated a clinical trial conducted by the Fred Hutchinson Cancer Research Center to evaluate a CAR T, FCARH143, with a fully-human BCMA binder that preferentially binds membrane-bound BCMA. Juno intends to begin a Phase I trial early next year using this binder in combination with Juno’s cell manufacturing process. This product candidate, JCARH125, recently received orphan drug designation from the FDA for multiple myeloma.

Corporate News:

Closed a follow-on public offering and concurrent private placement in September of 7,773,327 shares of Juno’s common stock at a price of $41.00 per share. This includes the exercise in full by the underwriters of their option to purchase up to an additional 915,000 shares of common stock and a private placement of 758,327 shares of common stock to a subsidiary of Celgene Corporation. Gross proceeds were approximately $318.7 million.
Third Quarter 2017 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities as of September 30, 2017 were $1.06 billion compared to $801.8 million as of June 30, 2017, and $922.3 million as of December 31, 2016.
Cash Used in Operating Activities and Capital Expenditures: For the third quarter of 2017 cash used in operating activities was $40.3 million and cash used for capital expenditures was $13.9 million, compared to cash used in operating activities of $68.1 million and $6.4 million used for capital expenditures for the same period in 2016.
Cash Burn: Cash burn, which is cash used in operating activities and capital expenditures, excluding cash inflows and outflows from upfront payments related to business development activities, was $54.2 million in the third quarter of 2017, of which $59.1 million was operating cash burn and $4.9 million was net cash provided by a tenant improvement allowance offset by capital expenditures. For purposes of comparing the operating cash burn and cash burn for capital expenditures for the third quarter of 2017 to the Company’s financial guidance, a cash inflow of $18.8 million for a tenant improvement allowance was reclassified from operating activities to capital expenditures.

Cash burn in the third quarter of 2016 was $59.5 million, of which $53.1 million was operating cash burn and $6.4 million was cash burn for capital expenditures.
Revenue: Revenue for the three and nine months ended September 30, 2017 was $44.8 million and $85.4 million, compared to $20.8 million and $58.2 million for the three and nine months ended September 30, 2016, respectively. Revenue increased in the three and nine months ended September 30, 2017 compared to the prior year periods due to milestone revenue recognized in the third quarter of 2017 in connection with the Novartis sublicense agreement. Additionally, revenue recognized under our Celgene Collaboration Agreement and Celgene CD19 License increased in the nine months ended September 30, 2017 compared to the prior year period.
R&D Expenses: Research and development expenses for the three and nine months ended September 30, 2017, inclusive of non-cash expenses and computed in accordance with GAAP, were $140.3 million and $324.3 million, compared to $60.9 million and $206.9 million for the three and nine months ended September 30, 2016, respectively. The increases in 2017 compared to 2016 were primarily due to increased costs to manufacture Juno’s product candidates, execute on Juno’s clinical development strategy, expand its overall research and development capabilities, an increase in expense related to its success payment and contingent consideration obligations, expense incurred for the amortization of the intangible asset associated with the AbVitro, Inc. (AbVitro) acquisition, and an increase in non-cash stock-based compensation expense. These increases were offset by a decrease in milestone expense.
Non-GAAP R&D Expenses: Non-GAAP research and development expenses for the three and nine months ended September 30, 2017 were $98.4 million and $250.6 million, and include $10.6 million and $30.3 million of stock-based compensation expense, respectively. Non-GAAP research and development expenses for the three and nine months ended September 30, 2016 were $62.2 million and $214.5 million, and include $7.9 million and $25.8 million of stock-based compensation expense, respectively. Non-GAAP research and development expenses for 2017 exclude the following:
An expense of $37.2 million and $61.8 million for the three and nine months ended September 30, 2017, respectively, associated with the change in the estimated fair value and elapsed service period for Juno’s potential success payment liabilities to Fred Hutchinson Cancer Research Center (FHCRC) and Memorial Sloan Kettering Cancer Center (MSK).
Non-cash stock-based compensation expense of $1.4 million and $3.0 million for the three and nine months ended September 30, 2017, respectively, related to a 2013 restricted stock award to a co-founding director that became a consultant upon his departure from Juno’s board of directors in 2014.
An expense of $2.4 million and $4.8 million for the three and nine months ended September 30, 2017, respectively, associated with amortization of the intangible asset recorded in connection with the AbVitro acquisition.
An expense of $0.8 million and $4.0 million for the three and nine months ended September 30, 2017, respectively, associated with the change in the estimated fair value of the contingent consideration liabilities recorded in connection with the Stage and X-Body acquisitions.
G&A Expenses: General and administrative expenses on a GAAP basis for the three and nine months ended September 30, 2017 were $26.3 million and $70.7 million, respectively, compared to $18.4 million and $51.2 million for the same periods in 2016. The increases in 2017 compared to 2016 were primarily due to an increase in consulting and other expenses to support the growing organization including costs related to commercial readiness, increased personnel expenses primarily related to increased headcount to support the business, an increase in litigation and patent legal costs, and an increase in stock-based non-cash compensation expense. The increases in the nine month period were partially offset by decreased business development expenses. General and administrative expenses include $6.9 million and $19.9 million of non-cash stock-based compensation expense for the three and nine months ended September 30, 2017, compared to $5.4 million and $15.9 million for the three and nine months ended September 30, 2016, respectively.
GAAP Net Loss: Net loss for the three and nine months ended September 30, 2017 was $118.1 million, or $1.12 per share, and $301.1 million, or $2.88 per share, compared to $56.9 million, or $0.56 per share and $192.8 million, or $1.91 per share, for the three and nine months ended September 30, 2016, respectively.
Non-GAAP Net Loss: Non-GAAP net loss, which incorporates the non-GAAP R&D expense, for the three and nine months ended September 30, 2017 was $76.3 million, or $0.73 per share, and $227.4 million, or $2.17 per share, compared to $58.3 million, or $0.57 per share, and $200.4 million, or $1.99 per share for the three and nine months ended September 30, 2016, respectively.
Reconciliations of cash burn to GAAP cash used in operating activities and capital expenditures, non-GAAP net loss to GAAP net loss, and non-GAAP R&D expense to GAAP R&D expense are presented below under “Non-GAAP Financial Measures.”

2017 Financial Guidance

Juno expects to be in the lower half of 2017 cash burn guidance, which is cash used in operating activities and capital expenditures, excluding cash inflows or outflows from upfront payments related to business development activities, of between $270 million and $300 million.

Conference Call Information

Juno will host a conference call today to review Juno’s financial results for the third quarter 2017 beginning at 1:30 p.m. Pacific Time (PT) / 4:30 p.m. Eastern Time (ET). Analysts and investors can participate in the conference call by dialing (855) 780-7198 for domestic callers and (631) 485-4870 for international callers, using the conference ID# 2899809.

The webcast can be accessed live on the Investor Relations page of Juno’s website, www.JunoTherapeutics.com, and will be available for replay for 30 days following the call.

On November 1, 2017 Novartis reported that it will present new data from across its hematology portfolio at the upcoming 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, Atlanta, December 9-12 (Press release, Novartis, NOV 1, 2017, View Source [SID1234521393]). More than 75 abstracts will be presented, highlighting the robust Novartis development program for serious blood diseases.

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“This is an exceptionally productive time in hematology, and the breadth of our Novartis Oncology data and presence at ASH (Free ASH Whitepaper) underscore our commitment to this space,” said Vas Narasimhan, Global Head Drug Development and Chief Medical Officer, Novartis. “Following the launch of Kymriah, the first FDA-approved CAR-T therapy, we are particularly excited about presenting additional data on this new approach to cancer treatment, as well as a new analysis for crizanlizumab, an investigational treatment for patients with sickle cell disease.”

KymriahTM* (tisagenlecleucel) suspension for intravenous infusion is a CD19-directed genetically modified autologous T cell immunotherapy, indicated for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. Additional results evaluating Kymriah in pediatric ALL and in relapsed/refractory diffuse large B-cell lymphoma (DLBCL) will be presented.

Data for Kymriah include results from the primary analysis of the JULIET study in adult patients with relapsed or refractory DLBCL, demonstrating sustained complete response rates based on extended follow up, and efficacy and safety findings from additional treated patients compared to a previously presented interim analysis. Additionally, results of a cost-effectiveness analysis of Kymriah for the treatment of relapsed or refractory ALL in the United States will be presented in an oral presentation.

Primary Analysis of JULIET: A Global, Pivotal, Phase 2 Trial of CTL019 in Adult Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma [Abstract #577; Monday, December 11, 7:00 AM EST]
Cost-Effectiveness Analysis of CTL019 for the Treatment of Pediatric and Young Adult Patients with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia in the United States [Abstract #609; Monday, December 11, 7:30 AM EST]
Patient-Reported Quality of Life (QoL) Following CTL019 Infusion in Adult Patients with Relapsed/Refractory (r/r) Diffuse Large B-cell Lymphoma (DLBCL) [Abstract #5215; publication only]
Expert Elicitation of Long-Term Survival for Pediatric Acute Lymphoblastic Leukemia Patients Receiving CTL019 in ELIANA Phase II Study [Abstract #3377; Sunday, December 10, 6:00 PM EST]
Outcomes for chimeric antigen receptor T cell (CAR-T) pipeline therapies in other malignant blood cancers will also be shared at ASH (Free ASH Whitepaper):

Updated Safety and Efficacy of B-cell Maturation Antigen (BCMA)-specific Chimeric Antigen Receptor T Cells (CART-BCMA) for Refractory Multiple Myeloma (MM) [Abstract #505; Sunday, December 10, 4:30 PM EST]
Durable Remissions with Humanized CD19-Targeted Chimeric Antigen Receptor (CAR)-Modified T Cells in Children and Young Adults with Relapsed/Refractory Acute Lymphoblastic Leukemia, Including After Prior CAR Therapy [Abstract #1319; Saturday, December 9, 5:30 PM EST]
Data from a post-hoc sub-group analysis of the Phase II SUSTAIN investigational trial of crizanlizumab for time to first on-treatment sickle cell pain crisis will be featured:

Crizanlizumab 5.0 mg/kg Increased the Time to First On-Treatment Sickle Cell Pain Crisis: A Subgroup Analysis of the Phase II SUSTAIN Study [Abstract #613; Monday, December 11, 10:30 AM EST]
A matched comparison of Molecular Recurrence-free Survival (MRecFS) following treatment discontinuation in chronic myeloid leukemia (CML) patients on Tasigna (nilotinib) in ENESTfreedom versus patients on imatinib in the EURO-SKI trials will be presented in addition to updates from ENESTfreedom and ENESTop on Treatment-free Remission (TFR) outcomes:

Molecular Recurrence-Free Survival (MRecFS) Following Imatinib vs Nilotinib in Patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP): Matched Analysis of Patients in EURO-SKI and ENESTfreedom [Abstract #1601; Saturday, December 9, 5:30 PM EST]
Impact of Treatment Cessation on Overall Disease Outcomes in Patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) Attempting Treatment-Free Remission (TFR): Findings from ENESTfreedom and ENESTop [Abstract #1598; Saturday, December 9, 5:30 PM EST]
Treatment-Free Remission (TFR) Among Patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) Not Initially Eligible for Treatment Discontinuation Due to Unstable Deep Molecular Response (DMR): ENESTfreedom and ENESTop [Abstract #2878; Sunday, December 10, 6:00 PM EST]
Additionally, new insights will be presented from the pivotal, Phase III RATIFY trial of Rydapt (midostaurin) in adults with FLT3+ acute myeloid leukemia (AML):

An Analysis of the Maintenance and Post Completion Effect of Midostaurin Therapy in the International Prospective Randomized Placebo-Controlled, Double-Blind Trial (CALGB 10603/RATIFY [Alliance]) for Newly Diagnosed Acute Myeloid Leukemia (AML) Patients with FLT3 Mutations [Abstract #145; Saturday, December 9, 12:00 PM EST]
The Addition of Midostaurin to Standard Chemotherapy Decreases Cumulative Incidence of Relapse (CIR) in the International Prospective Randomized, Placebo-Controlled, Double-Blind Trial (CALGB 10603/RATIFY [Alliance]) for Newly Diagnosed Acute Myeloid Leukemia (AML) Patients with FLT3 Mutations [Abstract #2580; Sunday, December 10, 6:00 PM EST]
Prognostic Impact of NPM1/FLT3-ITD Genotypes from Randomized Patients with Acute Myeloid Leukemia (AML) Treated Within the International RATIFY Study [Abstract #467; Sunday, December 10, 5:30 PM EST]
Sandoz, a Novartis division, the pioneer and global leader in biosimilars, will present two studies examining the impact of granulocyte colony-stimulating factor (G-CSF) on patient outcomes, cost savings and expanded access for biosimilars including Zarxio (filgrastim-sndz).

Expanded Access to Obinutuzumab from Cost-Savings Generated by Biosimilar Filgrastim (BIOSIM-FIL) in the Prophylaxis of Chemotherapy-Induced (Febrile) Neutropenia: A Simulation Study [Abstract #3380; Sunday, December 10, 6:00 PM EST]
A Systemic Literature Review of Overall Survival and Delivered Dose Intensity in Cancer Patient Receiving Chemotherapy and G-CSF in Randomized Control Trials [Abstract #3424; Sunday, December 10, 6:00 PM EST]
Additional abstracts of note from the meeting are as follows.

Exjade/Jadenu (deferasirox)

Predicting Serum Ferritin Levels in Patients with Iron Overload Treated with the Film-Coated Tablet of Deferasirox During the ECLIPSE Study [Abstract #3508; Monday, December 11, 6:00 PM EST]
Jakavi (ruxolitinib)**

Primary Analysis of JUMP, a Phase 3b, Expanded-Access Study Evaluating the Safety and Efficacy of Ruxolitinib in Patients with Myelofibrosis (N = 2233) [Abstract #4204; Monday, December 11, 6:00 PM EST]
Results from the 208-Week (4-Year) Follow-Up of Response Trial, a Phase 3 Study Comparing Ruxolitinib (Rux) with Best Available Therapy (BAT) for the Treatment of Polycythemia Vera (PV) [Abstract #322; Sunday, December 10, 7:30 AM EST]
Role of Symptom Burden in Disability Leave Among Patients with Myeloproliferative Neoplasms (MPNs): Findings from the Living with MPN Patient Survey [Abstract #1637; Saturday, December 9, 5:30 PM EST]
Revolade/Promacta (eltrombopag)***

Occurrence and Management of Cataracts in Patients with Chronic Immune Thrombocytopenia (cITP) During Long-Term Treatment with Eltrombopag (EPAG): Results from the EXTEND Study [Abstract #1053; Saturday, December 9, 5:30 PM EST]
Eltrombopag (EPAG) Treatment Improved Platelet Counts in Patients with Persistent or Chronic Immune Thrombocytopenia During a 2-Year, Phase IV, Open-Label Study [Abstract #3628; Monday, December 11, 6:00 PM EST]
A Retrospective Chart Review to Assess Burden of Illness Among Patients with Severe Aplastic Anemia with Insufficient Response to Immunosuppressive Therapy [Abstract #678; Monday, December 11, 10:30 AM EST]
Product Information
Approved indications for products vary by country and not all indications are available in every country. The product safety and efficacy profiles have not yet been established outside the approved indications. Because of the uncertainty of clinical trials, there is no guarantee that compounds will become commercially available with additional indications.

For full prescribing information, including approved indications and important safety information about marketed products, please visit View Source (link is external).

Crizanlizumab, CART-BCMA and CTL119 are investigational compounds. Efficacy and safety have not been established. There is no guarantee these compounds will become commercially available.

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; general economic and industry conditions, including the effects of the persistently weak economic and financial environment in many countries; safety, quality or manufacturing issues, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

On November 1, 2017 Diplomat Pharmacy, Inc. (NYSE: DPLO) reported that it has been named to the limited pharmacy panel to dispense CALQUENCE (acalabrutinib) (Press release, Diplomat Speciality Pharmacy, NOV 1, 2017, View Source [SID1234521424]).

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CALQUENCE, recently granted accelerated approval by the Food and Drug Administration, is indicated to treat mantle cell lymphoma (MCL)—a type of non-Hodgkin lymphoma—in adult patients who have received at least one prior therapy.

The average age at MCL diagnosis is the mid-60s, according to the Leukemia & Lymphoma Society.

“CALQUENCE is a needed alternative to treat a life-threatening blood cancer for patients who might not be responding to therapy or whose cancer has relapsed,” said Joel Saban, Diplomat president.

To learn more about Diplomat’s oncology program, visit diplomat.is/oncology.

CALQUENCE was developed by Acerta Pharma, LLC, a member of the AstraZeneca Group. For full prescribing information, click here.

Agios Pharmaceuticals has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Agios Pharmaceuticals, 2017, NOV 1, 2017, View Source [SID1234521409]).

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On November 1, 2017 Pain Therapeutics, Inc. (Nasdaq:PTIE) reported financial results for the third quarter ended September 30, 2017 (Press release, Pain Therapeutics, NOV 1, 2017, View Source [SID1234521394]). Net loss for the third quarter of 2017 was $2.6 million, or $0.40 per share, respectively, compared to a net loss for the same period in 2016 of $3.5 million, or $0.54 per share. Net cash used during the third quarter was $2.2 million. Cash and investments were $11.9 million as of September 30, 2017, with no debt. The Company still expects net cash usage in the calendar year 2017 may be approximately $10 million. Following the resubmission of the REMOXY NDA in Q1 2018, the Company believes net cash usage in 2018 will decrease significantly compared to 2017.

“The White House recently declared the opioid epidemic a public health emergency,” said Remi Barbier, President & CEO. “We fully support this policy position, and have been a voice in support of such a policy for many years. Nearly 15 years ago, Pain Therapeutics pioneered abuse-deterrent technology for opioid drugs specifically to provide policy makers, regulators, physicians, pharmacists and patients an additional tool to help combat the opioid epidemic. In partnership with all constituents, we look forward to doing our part to address the issues of overdose and death from extended-release opioid drugs.”

Operating Highlights for Q3 2017

In September, the National Institutes of Health (NIH) awarded us a $1.8 million research grant to develop a blood-based diagnostic to detect Alzheimer’s disease.
In September, The National Institute on Drug Abuse (NIDA) awarded us a $2.2 million research grant to further develop FENROCK, an abuse-deterrent transdermal patch that contains the prescription drug fentanyl.
In October, we announced a successful Phase I clinical study for PTI-125, our drug candidate for the treatment of Alzheimer’s disease. As previously announced, our scientists plan to present full results of this study at the 10th Annual International Conference on Clinical Trials on Alzheimer’s Disease, in Boston, MA, on November 1-4th.
In October, we announced the FDA had agreed to a pre-NDA guidance meeting on November 14th to discuss the upcoming resubmission of an NDA for REMOXY ER. We will provide details of this FDA meeting after receipt of final meeting minutes.
Recently, we substantially completed a previously announced human nasal study with REMOXY ER. We plan to announce top-line results of this study by yearend 2017.
Financial Highlights for Q3 2017

At September 30, 2017, cash and investments were $11.9 million, compared to $14.1 million at June 30, 2017. The Company has no debt.
Net cash used during the three months ended September 30, 2017 was $2.2 million.
Research and development expenses for the three months ended September 30, 2017 decreased to $1.6 million, respectively, from $2.7 million for the same period in 2016, primarily due to decreases in REMOXY related expenses and the receipt of research grant funding from the National Institutes of Health for FENROCK and PTI-125, recorded as a reduction in research and development expenses activities. Research and development expenses included non-cash stock-related compensation of $0.3 million in both three months ended September 30, 2017 and 2016.
General and administrative expenses increased slightly to $1.0 million in the three months ended September 30, 2017 from $0.9 million for the same period in 2016. General and administrative expenses included non-cash stock-related compensation of $0.4 million in the three months ended September 30, 2017 compared to $0.5 million for same period in 2016.
About REMOXY ER (extended-release oxycodone capsules CII)
REMOXY ER is a proprietary, abuse-deterrent, extended-release oral formulation of oxycodone. The proposed indication for this drug candidate is for “the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate.” We developed REMOXY to make oxycodone difficult to abuse yet provide 12 hours of steady pain relief when used appropriately by patients. In particular, REMOXY’s thick, sticky, high-viscosity gel-cap formulation may deter unapproved routes of drug administration, such as injection, snorting or smoking.

About Opioid Abuse
Opioid drugs such as oxycodone are an important treatment option for patients with severe chronic pain. However, oxycodone abuse and diversion remains a serious, persistent problem. Drug overdose deaths exceeded 64,000 in 2016, according to the CDC. For over a decade, Pain Therapeutics has pioneered Abuse-Deterrent Formulations (ADFs) to help in the fight against prescription drug abuse. ADFs attempt to raise the bar on prescription drug abuse by making it difficult, longer or aversive to tamper with long-acting opioid formulations, recognizing that no drug can be made abuse-proof.

About Pain Therapeutics, Inc.
We develop proprietary drugs that offer significant improvements to patients and physicians. Our expertise consists of developing new drugs and guiding these through various regulatory and development pathways in preparation for their eventual commercialization. We generally focus our drug development efforts around disorders of the nervous system. The FDA has not yet established the safety or efficacy of our drug candidates.

Our pipeline of drug assets includes:

REMOXY ER — (extended-release oxycodone capsules) Proprietary abuse-deterrence, twice-daily oxycodone targeted at severe chronic pain. NDA resubmission planned for Q1 2018.

PTI-125 Rx — Proprietary small molecule drug targeted at the treatment of Alzheimer’s disease. Phase I clinical-stage program, substantially funded by a research grant award from the NIH.

PTI-125 Dx — Blood-based diagnostic to detect Alzheimer’s disease. Early-stage program, substantially funded by a research grant award from the NIH.

FENROCK — (transdermal fentanyl patch system) Proprietary abuse-deterrent skin patch to treat severe pain. Early-stage program, substantially funded by a research grant award from National Institute on Drug Abuse.

NOTE: REMOXY ER and FENROCK are trademarks of Pain Therapeutics, Inc.

Important Note Regarding Forward-Looking Statements: This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Pain Therapeutics disclaims any intent or obligation to update these forward-looking statements, and claims the protection of the Safe Harbor for forward-looking statements contained in the Act. Examples of such statements include, but are not limited to, statements regarding our projected cash usage in CY2017 and CY2018, statements regarding potential discussions with the FDA and the abuse-deterrent properties and potential benefits of REMOXY ER. Such statements are based on management’s current expectations but actual results may vary materially due to various factors, many of which are beyond the control of management. Drug development involves substantial risks and uncertainties, including but not limited to those risks and uncertainties relating to successfully completing the activities required to address the issues raised by the FDA in the September 2016 Complete Response Letter for REMOXY ER and the time required to do so, including the time required to reach resolution with the FDA on the scope of the appropriate actions to be undertaken and the possibility that the FDA may raise additional issues in the future that were not raised in the past. In addition, the development of abuse-deterrent drug products is a young and still emerging area of drug development, with regulatory guidance that may be inconsistent, unclear or still in development. Such statements are based on management’s current expectations, but actual results may differ materially due to various factors. For further information regarding these and other risks related to our business, investors should consult our filings with the U.S. Securities and Exchange Commission.