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8-K – Current report

On May 6, 2016 La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, reported first quarter 2016 financial results and highlighted recent corporate progress (Filing, Q1, La Jolla Pharmaceutical, 2016, MAY 11, 2016, View Source [SID:1234512493]).

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Recent Corporate Progress

• La Jolla’s ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial continued to enroll as planned through the first quarter of 2016. The ATHOS 3 trial is La Jolla’s multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501, the Company’s proprietary formulation of angiotensin II, in catecholamine-resistant hypotension (CRH), which was initiated in 2015. The Company initiated the ATHOS 3 trial after reaching an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA), in which the agreed-upon primary efficacy endpoint in ATHOS 3 is an increase in blood pressure. Results of ATHOS 3 are expected by the end of 2016.

• La Jolla reported interim results in January 2016 of its multicenter, open-label, dose-escalation Phase 1 clinical trial of LJPC-401, the Company’s novel formulation of hepcidin, in patients at risk for iron overload due to conditions such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. These results suggested a dose-dependent reduction in serum iron following a single dose of LJPC-401. Complete results from this Phase 1 clinical trial, which was initiated in October 2015, are expected in the second half of 2016.
"The first quarter was a productive start to 2016 for La Jolla, highlighted by the continued enrollment of our ATHOS 3 trial and encouraging interim data from our Phase 1 clinical trial of LJPC-401," said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. "We look forward to continuing the advancement of each of our exciting programs during the rest of 2016, culminating with the results from the ATHOS 3 trial that are expected by the end of the year."
Results of Operations

As of March 31, 2016, La Jolla had $113.1 million in cash and cash equivalents, compared to $126.5 million as of December 31, 2015. The decrease in cash and cash equivalents was primarily due to net cash used for operating activities. Based on current operating plans and projections, La Jolla believes that its current cash and cash equivalents are sufficient to fund operations into 2018.

La Jolla’s net cash used for operating activities for the three months ended March 31, 2016 was $13.0 million, compared to net cash used for operating activities of $5.5 million for the same period in 2015. La Jolla’s net loss for the three months ended March 31, 2016 was $16.5 million, or $0.96 per share, compared to a net loss of $9.0 million, or $0.59 per share, for the same period in 2015. During the three months ended March 31, 2016, La Jolla recognized contract revenue of approximately $0.2 million, which was pursuant to a services agreement initiated in 2015 under which La Jolla provides research and development services to a related party. The net loss includes non-cash, share-based compensation expense of $3.7 million for the three months ended March 31, 2016, compared to $3.4 million for the same period in 2015.

The increases in net cash used for operating activities and net loss in 2016 as compared to 2015 were primarily due to increased development costs associated with the ATHOS 3 trial and the costs associated with the initiation of the Phase 1 clinical trial of LJPC-401 in iron overload. In addition, there were increases in personnel and related costs, which were mainly due to the hiring of additional personnel and increased facility costs to support the increased development activities.

Onconova Therapeutics, Inc. Reports Recent Business Highlights and First Quarter 2016 Financial Results

On May 11, 2016 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported a corporate update and reported financial results for the first quarter ended March 31, 2016 (Press release, Onconova, MAY 11, 2016, View Source [SID:1234512298]).

"We recently reported two important advances in the development of rigosertib for the unmet medical needs of patients with myelodysplastic syndromes (MDS). Foremost is the elucidation of the novel mechanism of action of rigosertib targeting the RAS pathway, recently published in Cell, and the release of detailed clinical data from our ONTIME Phase 3 study in Lancet Oncology. These peer-reviewed papers provide important validation of the mechanistic rationale for rigosertib as a novel treatment option for patients with MDS," said Ramesh Kumar, Ph.D., President and CEO of Onconova. "We are encouraged by the continued progress in the global Phase 3 INSPIRE trial which is now enrolling patients in the U.S. and Europe."

Recent Business Highlights:

Progress in INSPIRE Pivotal Trial of Rigosertib in Higher-risk MDS (HR-MDS)

The global INSPIRE trial is now enrolling patients in the United States and Europe. As of May 3, 2016, 51 sites, including 13 in the U.S., are open and recruiting patients. This pivotal trial was initiated in 4Q2015.

Results from Onconova’s ONTIME trial were published in the top-tier, peer-reviewed journal, Lancet Oncology. The article, entitled, "Rigosertib versus best supportive care for patients with high-risk myelodysplastic syndromes after failure of hypomethylating drugs (ONTIME): a randomised, controlled, phase 3 trial," appeared in the March 8, 2016 online edition of the journal.
Rigosertib Mechanism of Action

The unique RAS-targeted mechanism of action for rigosertib was published in the prestigious peer-reviewed experimental biology journal, Cell. The paper, entitled "A small molecule RAS-mimetic disrupts RAS association with effector proteins to block signaling," appeared in the April 21, 2016 edition of the journal.
Proprietary NCE targeting ARK5 and CDK4/6

A presentation at the 2016 American Association of Cancer Research Annual meeting demonstrated the important differentiating features of ON 123300, Onconova’s novel pre-clinical ARK5 and CDK4/6 targeted agent. Results indicated that the inhibitory activity of ON 123300 on ARK5 and CDK4/6 resulted in the activation of programmed cell death in colorectal cancer cells, while treatment with an approved CDK4/6 inhibitor merely resulted in cytostasis.
Upcoming Events

Enrollment of the first patient in the Phase 3 INSPIRE trial in Japan: 2Q2016

Presentation of updated Phase 2 data from oral rigosertib combination trial in MDS: 2Q2016

End of Phase 2 meeting with FDA to discuss data from oral rigosertib combination trial: 2H2016
First Quarter 2016 Financial Results

Cash, cash equivalents, and marketable securities as of March 31, 2016 totaled $16.8 million, compared to $19.8 million as of December 31, 2015.
Total net revenue was $1.5 million for the first quarter of 2016 compared to $0.1 million for the first quarter of 2015.
Research and development expenses were $5.8 million for the first quarter of 2016 compared to $9.5 million for the first quarter of 2015.
General and administrative expenses were $3.2 million for the first quarter of 2016 compared to $3.0 million for the first quarter of 2015.

About Rigosertib

Rigosertib is a small molecule inhibitor of cellular signaling and acts as a RAS mimetic. These effects of rigosertib appear to be mediated by direct binding of the compound to the RAS-binding domain (RBD) found in many RAS effector proteins, including the Raf kinases and PI3K. The therapeutic focus for rigosertib is myelodysplastic syndromes (MDS), a group of bone marrow disorders characterized by ineffective formation of blood cells that often converts into acute myeloid leukemia (AML). Clinical trials for rigosertib are being conducted at leading institutions in the United States, Europe, and the Asia-Pacific region. Rigosertib is protected by issued patents (earliest expiry in 2026) and has been awarded Orphan Designation for MDS in the United States, Europe and Japan.

About INSPIRE

The INternational Study of Phase III IV RigosErtib, or INSPIRE, is based on guidance received from the U.S. Food and Drug Administration and European Medicines Agency and derives from the findings of the ONTIME Phase 3 trial. INSPIRE is a multi-center, randomized controlled study to assess the efficacy and safety of IV rigosertib in HR-MDS patients who had progressed on, failed to respond to, or relapsed after previous treatment with an HMA within the first nine months of initiation of HMA treatment. This time frame optimizes the opportunity to respond to treatment with an HMA prior to declaring treatment failure, as per NCCN Guidelines. The trial will enroll approximately 225 patients randomized at a 2:1 ratio into two treatment arms: IV rigosertib plus Best Supportive Care versus Physician’s Choice plus Best Supportive Care. The primary endpoint of INSPIRE is overall survival and an interim analysis is anticipated. Full details of the INSPIRE trial, such as inclusion and exclusion criteria, as well as secondary endpoints, can be found on clinicaltrials.gov (NCT02562443).

Kura Oncology Reports First Quarter 2016 Financial Results

On May 11, 2016 Kura Oncology, Inc., (NASDAQ:KURA) a clinical stage biopharmaceutical company, reported financial results for the first quarter ended March 31, 2016 (Press release, Kura Oncology, MAY 11, 2016, View Source;p=RssLanding&cat=news&id=2167626 [SID:1234512296]).

"Kura has made important progress in advancing its pipeline of precision oncology drug candidates over the past quarter," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We continue to enroll patients in our on-going Phase 2 clinical trials of tipifarnib in patients with HRAS mutant solid tumors and in relapsed or refractory peripheral T-cell lymphoma, and earlier in May 2016, we initiated our Phase 2 trial of tipifarnib in lower risk myelodysplastic syndromes (MDS)."

Dr. Wilson added: "In the second half or 2016, we are also planning to initiate a Phase 2 clinical trial with tipifarnib in patients with chronic myelomonocytic leukemia (CMML), a population for which prognosis is very poor, with a three-year survival rate estimated at less than 30 percent. Objective responses, including complete responses, have been previously observed with tipifarnib in CMML. Moreover, as part of the study, we plan to test a biomarker hypothesis, which may allow us to identify those patients most likely to experience durable responses. We believe our ongoing Phase 2 clinical trials and our additional planned trial in CMML provide us with multiple potential opportunities to position tipifarnib for registration-enabling Phase 3 studies in late 2017 or early 2018."

Upcoming Clinical and Preclinical Milestones for Kura Oncology Programs

IND submission for ERK inhibitor, KO-947, is anticipated in the second quarter of 2016 followed by initiation of a Phase 1 clinical trial, which is anticipated in the second half of 2016
Receipt of topline data from the Phase 2 clinical trial for tipifarnib in HRAS mutant solid tumors is anticipated in the second half of 2016
Initiation of a Phase 2 clinical trial for tipifarnib in patients with CMML is anticipated in the second half of 2016
Nomination of a development candidate for the menin-MLL program is anticipated in the second half of 2016
Financial Results for the First Quarter 2016

Cash, cash equivalents and short-term investments totaled $78.5 million as of March 31, 2016, compared with $85.7 million as of December 31, 2015. Management expects that existing cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2018.
Research and development expenses for the first quarter of 2016 were $4.6 million, compared to $3.6 million for the first quarter of 2015.
General and administrative expenses for the first quarter of 2016 were $2.4 million, compared to $1.1 million for the first quarter of 2015.
Net loss for the first quarter of 2016 was $6.6 million, or $0.36 per share, compared to a net loss of $4.5 million, or $1.41 per share, for the first quarter of 2015.
Subsequent to the first quarter of 2016, Kura Oncology put in place a $20.0 million long-term debt financing agreement, of which $7.5 million has been drawn down. Use of proceeds is for development programs and general corporate purposes.

Heat Biologics Reports First Quarter 2016 Financial Results

On May 11, 2016 Heat Biologics, Inc. ("Heat") (Nasdaq:HTBX), an immuno-oncology company developing novel therapies that activate a patient’s immune system against cancer, reported its financial results for the first quarter ended March 31, 2016 (Press release, Heat Biologics, MAY 11, 2016, View Source [SID:1234512295]).

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"We are focused on achieving near-term milestones to rebuild shareholder value. Our primary goal is to advance HS-410 for the treatment of non-muscle invasive bladder cancer with one-year disease-free survival, immune response, safety and tolerability data expected in the fourth quarter of this year," said Jeff Wolf, Heat’s Founder and CEO. "In addition, we are looking forward to presenting new data at the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting in June around our Phase 1b trial evaluating HS-110 in combination with an anti-PD-1 checkpoint inhibitor for the treatment of non-small cell lung cancer."

Recent Developments & First Quarter 2016 Corporate Highlights

In May, Heat had an abstract accepted for poster presentation at the ASCO (Free ASCO Whitepaper) Annual Meeting being held on June 3-7, 2016 in Chicago, IL. The poster is entitled "Broadening response rates to PD-1 therapy in advanced lung adenocarcinoma: Viagenpumatucel-L (HS-110) in combination with nivolumab in the ongoing DURGA trial" (Abstract #TPS9102). Abstracts will be made available on the ASCO (Free ASCO Whitepaper) website at www.asco.org in line with the conference’s embargo policy.

In April, Heat presented three posters at the AACR (Free AACR Whitepaper) Annual Meeting. In the poster entitled "Phase I/II Study of Patients with Non-Muscle Invasive Bladder Cancer (NMIBC) Treated with Vesigenurtacel-L (HS-410) with or without BCG," Heat reported that no additional recurrences had been reported to-date, with all patients now at least 18 months out from enrollment. In another poster, Heat reported initial preclinical results from its collaboration with OncoSec Medical Incorporated in which researchers concluded that combining Heat’s ComPACT vaccine with OncoSec’s intratumoral DNA electroporation delivery platform stimulated an expansion of neoantigen-specific CD8+ T cells, leading to a regression in both treated and untreated cancer lesions in two mouse studies. In the third poster, Heat reported positive preclinical data on its next generation ComPACT platform technology, which combines a T cell priming vaccine and a T cell co-stimulator in a single product.

In April, Heat implemented cost-saving measures and a focused corporate strategy to achieve data readout, with its current cash on-hand, anticipated in the fourth quarter of 2016 for its lead Phase 2 program evaluating HS-410 for the treatment of NMIBC. These cost-saving measures included a workforce reduction of approximately 22 percent of the company’s headcount.

In April, Heat appointed John Prendergast, Ph.D., to its Board of Directors.

In March, Heat closed a public offering of approximately $6.8 million in gross proceeds, which will primarily be used to complete its Phase 2 clinical trial evaluating HS-410 for the treatment of NMIBC. Remaining funds will be used to advance the current eight patients enrolled in our Phase 1b trial evaluating HS-110 in combination with nivolumab for the treatment of non-small cell lung cancer (NSCLC) through the reporting of topline data, as well as for licensing or acquisition of assets complementary to our existing programs and working capital and general corporate purposes.

In March, Heat presented additional preclinical data from its ComPACT platform technology at the Keystone Symposia on Cancer Vaccines. Data presented demonstrated that ComPACT secreting OX40L generated the most potent immune response among other ComPACT co-stimulator variations including TL1A, 4-1BBL and ICOSL, as well as compared to systemic delivery of OX40 agonist antibody and vaccine alone.

In February, Heat announced that it will no longer enroll new patients in its Phase 2 monotherapy trial arm evaluating HS-410 alone for the treatment of NMIBC following the resolution of the standard of care BCG shortage and discussions with the U.S. FDA. Heat anticipates reporting topline 6-month data from the 16 enrolled patients in the fourth quarter of 2016, contemporaneous with reporting data from Heat’s BCG combination cohorts.

In February, Heat announced that the U.S. FDA lifted the partial clinical hold on its HS-410 Phase 2 clinical trial and patient enrollment was resumed after less than one week; clinical timelines were materially unchanged. The partial clinical hold came after Heat concluded that the cell line on which HS-410 is based had been previously misidentified and immediately notified FDA of this conclusion. The FDA placed Heat’s HS-410 Phase 2 clinical trial on partial clinical hold while they reviewed updated documentation. The partial clinical hold did not relate to concerns regarding the safety or efficacy of HS-410.

In January, Heat reported three-month interim data from the unblinded, monotherapy arm in its Phase 2 trial evaluating HS-410 for the treatment of NMIBC. Images of the bladder taken from several patients treated with HS-410 alone showed changes that resemble T cell-rich structures that Heat has observed in biopsy samples, indicating that systemic administration with HS-410 leads to a localized immune response within the bladder that cannot be attributed to standard of care.

First Quarter 2016 Financial Highlights

Research and development expenses decreased to approximately $500,000 in the first quarter of 2016 compared to approximately $504,000 in the first quarter of 2015, a decrease of approximately $4,000. The decrease is attributable to reductions in patent, license and other professional fees offset by compensation costs associated with new hires, as well as supplies and facilities as we bring more capabilities in-house.

Clinical and regulatory expenses increased to approximately $3.2 million in the first quarter of 2016 compared to approximately $2.2 million in the first quarter of 2015, an increase of approximately $1.0 million. The increase is attributable to clinical trial execution expenses, personnel costs and expenses related to the production of our clinical trial material.

General and administrative expenses decreased to approximately $1.0 million in the first quarter of 2016 compared to approximately $1.3 million in the first quarter of 2015, a decrease of approximately $0.3 million. The decrease is attributable to non-cash stock compensation expense for non-employees associated with the company’s reduced shared price, as well as reduced professional services and third party expenses.

Net loss for the first quarter of 2016 was $4.7 million compared to a net loss of $4.0 million for the first quarter of 2015.

Cash, cash equivalents and short-term investments totaled approximately $11.8 million at March 31, 2016 compared to $11.6 million at December 31, 2015. This includes the $6.1 million in net proceeds raised during our March 2016 public offering.

Cellectis Reports First Quarter 2016 Financial Results

On May 11, 2016 Cellectis S.A. (Alternext: ALCLS – Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene edited CAR T-cells (UCART), reported its results for the three-month period ended March 31, 2016 (Press release, Cellectis, MAY 11, 2016, View Source [SID:1234512287]).

"We are excited to monitor the results presented by Great Ormond Street Hospital – University College of London describing clinical application of allogeneic, off-the-shelf CAR T-cells in young ALL patients with high medical need who exhausted all other treatment options. We are looking forward to seeing more data updates from our partners and bringing our CAR T-cell programs into the clinic, starting with UCART123 for AML patients" said André Choulika, CEO, Cellectis.

Recent Corporate Highlights
• New agreement with CELLforCURE for the cGMP manufacturing of clinical batches of UCART123, Cellectis’ lead product candidate, and for the implementation of cGMP manufacturing processes designed and developed by Cellectis.

• Supply and license agreement with Takara Bio Inc. for recombinant human fibronectin fragment RetroNectin to support Cellectis’ manufacturing processes and production capabilities.

• Publication in Scientific Reports, part of Nature Publishing Group, describing the design and development of a new CAR architecture with an integrated switch-on system that allows control over CAR T-cell functions.

• Research collaboration and license agreement with MabQuest SA for the development of a new class of anti PD-1 monoclonal antibodies.

• Cellectis gave a presentation at the Cowen and Company 36th Annual Health Care Conference on March 9, 2016 in Boston, MA.

• Scientific presentations at AACR (Free AACR Whitepaper), New Orleans:

Allogeneic TCRα/CS1 double knockout T-cell bearing an anti-CS1 chimeric antigen receptor: an improved immunotherapy approach for the treatment of Multiple Myeloma, presented by Roman Galetto, Cellectis.
Improved safety by a non-lethal switch to control CAR activity at the T-cell surface membrane, presented by Laurent Poirot, Cellectis.
• Appointment of Dr. Loan Hoang-Sayag to the role of Chief Medical Officer. Dr. Hoang-Sayag joined Cellectis from Quintiles Transnational, where she was most recently Senior Director of Medical Science.

• Calyxt, Cellectis’ plant science subsidiary, has purchased a 10-acre parcel in the St. Paul suburb of Roseville, MN, to build a new greenhouse and company headquarter.

Financial Results
As previously announced, commencing with this report of first quarter results Cellectis will now publish quarter-over-quarter comparative figures.

Cellectis’ consolidated financial statements have been prepared in accordance with International Financial Reporting Standards, or IFRS, as issued by the International Accounting Standards Board ("GAAP").

First Quarter 2016 Financial Results

Cash: As of March 31, 2016 Cellectis had €276.5 million in total cash, cash equivalents and current financial assets compared to €314.2 million as of December 31, 2015. This notably reflects (i) the initiation of industrial GMP UCART123 production, (ii) increased expenses in GMP materials (iii) payment of €7.2 million of Value Added Taxes related to the proceeds received in the fourth quarter of 2015 from Servier and (iv) Calyxt’s acquisition of a 10-acre parcel for €5.2 million. The change was also attributable to the unrealized translation effect of exchange rate fluctuations on our U.S. dollar cash and cash equivalent accounts.

Revenues and Other Income: During the three months ended March 31, 2015 and 2016, we recorded €9.2 million and €9.5 million, respectively, in revenues and other income.

Total Operating Expenses and Other Operating Income: Total operating expenses and other operating income for the first quarter of 2016 were €29.9 million, compared to €12.8 million for the first quarter of 2015. The non-cash stock-based compensation expenses included in these amounts were €13.4 million and €0.8 million, respectively.

R&D Expenses: For the three months ended March 31, 2015 and 2016, research and development expenses increased by €11.4 million from €7.4 million in 2015 to €18.9 million in 2016. Personnel expenses increased by €7.2 million from €4.7 million in 2015 to €11.9 million in 2016, notably due to a €1.0 million increase in wages and salaries, and a €7.2 million increase in non-cash stock based compensation expense, partly offset by a €1.0 million decrease in social charges on stock option and free shares grants. Purchases and external expenses increased by €4.2 million from €2.4 million in 2015 to €6.6 million in 2016, due to increased expenses related to innovation and platform development, including payments to third parties participating in product development, purchases of biological raw materials and expenses associated with the use of laboratories and other facilities.

SG&A Expenses: During the three months ended March 31, 2015 and 2016, we recorded €5.4 million and €10.5 million, respectively, of selling, general and administrative expenses. The increase of €5.2 million primarily reflects (i) an increase of €4.5 million in personnel expenses from €3.7 million to €8.3 million, attributable, among other things, to an increase of €5.4 million of non-cash stock-based compensation expense, partly offset by a decrease of €1.1 million of social charges on stock options and free share grants, and (ii) an increase of €0.8 million in purchases and external expenses.

Financial gain (loss): The financial gain was €9.9 million for the first quarter of 2015 compared with financial loss of €9.1 million for the first quarter of 2016, which does not reflect actions undertaken to mitigate the impact of currency exchange rate fluctuations that were adopted at the end of the first quarter of 2016. The change in financial result was primarily attributable to the effect of exchange rate fluctuations on our U.S. dollar cash and cash equivalent accounts.

Net Loss Attributable to Shareholders of Cellectis: During the three months ended March 31, 2015 and 2016, we recorded a net income of €6.3 million (or €0.20 per share on a basic basis and €0.19 per share on a diluted basis) and a loss of €29.5 million (or €0.84 per share on both a basic and diluted basis), respectively. Adjusted net loss attributable to shareholders of Cellectis for the first quarter of 2016 was €16.1 million (€0.46 per share on both a basic and a diluted basis) compared to adjusted net income attributable to shareholders of Cellectis of €7.0 million (€0.22 per share on both a basic and a diluted basis), for the first quarter of 2015. Adjusted net income (loss) attributable to shareholders of Cellectis for the first quarter of 2016 and 2015 excludes a non-cash stock-based compensation expense of €13.4 million and €0.8 million, respectively. Please see "Note Regarding Use of Non-GAAP Financial Measures" for a reconciliation of GAAP net income to adjusted net income.

Financial Guidance: Cellectis expects that its cash, cash equivalents and Current financial assets of €276.5 million as of March 31, 2016 will be sufficient to fund its current operations through 2018.