On February 12, 2018 Palatin Technologies, Inc. (NYSE American: PTN), a biopharmaceutical company developing targeted, receptor-specific peptide therapeutics for the treatment of diseases with significant unmet medical need and commercial potential, reported results for its second quarter ended December 31, 2017 (Press release, Palatin Technologies, FEB 12, 2018, View Source [SID1234523915]).

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Recent Highlights
● Bremelanotide – Under development for Hypoactive Sexual Desire Disorder ("HSDD"):
● Entered into a license agreement with Kwangdong Pharmaceutical Co., Ltd. ("Kwangdong") in November 2017 for exclusive rights to develop and commercialize bremelanotide in the Republic of Korea.

● Received $417,500 in December 2017, consisting of an upfront payment of $500,000 less $82,500 which was withheld in accordance with tax withholding requirements in the Republic of Korea.

● Entered into a license agreement with Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. ("Fosun"), a subsidiary of Shanghai Fosun Pharmaceutical (Group) Co., Ltd., in September 2017 for exclusive rights to develop and commercialize bremelanotide in the territories of mainland China, Taiwan, Hong Kong S.A.R. and Macau S.A.R.

● Received $4,500,000 in October 2017, consisting of an upfront payment of $5,000,000 less $500,000 which was withheld in accordance with tax withholding requirements in China.

● Working closely with AMAG Pharmaceuticals, Inc. ("AMAG"), our licensee for North America, on completing the tasks and activities necessary to file a New Drug Application ("NDA") with the Food and Drug Administration ("FDA").

● NDA filing with the FDA by AMAG targeted by March 31, 2018.

● Melanocortin Receptor 1 Agonist ("MC1r") – under development for inflammatory bowel diseases:

● Initiated Subject Dosing in First-in-Human Clinical Study of PL-8177, an MC1r agonist.

● Received FDA Clearance of Investigational New Drug ("IND") Application for PL-8177 For Ulcerative Colitis.

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Second Quarter Fiscal 2018 Financial Results
Palatin reported net income of $3.0 million, or $0.02 per basic and $0.01 per diluted share, for the quarter ended December 31, 2017, compared to a net loss of $(10.0) million, or $(0.06) per basic and diluted share, for the same period in 2016.

The difference in financial results between the three months ended December 31, 2017 and 2016 was primarily attributable to the recognition of $10.6 million in license and contract revenue during the 2017 period pursuant to our license agreement with AMAG, and a reduction of $2.1 million in research and development expenses.

Revenue
For the quarter ended December 31, 2017, 100% of the revenue Palatin recognized was related to our license agreement with AMAG.

There were no revenues recorded in the quarter ended December 31, 2016.

Operating Expenses
Total operating expenses for the quarter ended December 31, 2017 were $7.7 million compared to $9.4 million for the comparable quarter of 2016. The decrease in operating expenses was mainly attributable to the relative development stages of bremelanotide for HSDD as we continue our progress of filing an NDA with the FDA.

Other Income/Expense
Total other expense, net was $0.3 million for the quarter ended December 31, 2017 compared to $0.6 million for the quarter ended December 31, 2016. Total other expense, net for both periods consisted primarily of interest expense related to Palatin’s venture debt.

Income Tax
Pursuant to the license agreements with Fosun and Kwangdong, $500,000 and $82,500, respectively, was withheld in accordance with tax withholding requirements in China and the Republic of Korea, respectively, and will be recorded as an expense during the fiscal year ending June 30, 2018. For the quarter ended December 31, 2017, Palatin recorded $100,880 in income tax expense related to those withholding amounts utilizing an estimated effective annual income tax rate applied to income for the quarter and the remaining balance of $256,365 was included in prepaid expenses and other current assets at December 31, 2017. Any potential credit to be received by Palatin on its United States tax returns is currently offset by Palatin’s valuation allowance. The $100,880 of income tax expense is offset by a $500,000 tax benefit that Palatin recorded in the quarter ended December 31, 2017 related to the release of a valuation allowance against Palatin’s federal alternative minimum tax credit as a result of the Tax Cuts and Jobs Act signed in December 2017. Accordingly, $500,000 is included in other long-term assets at December 31, 2017.

Cash Position
Palatin’s cash, and cash equivalents were $35.0 million as of December 31, 2017, compared to cash, cash equivalents, accounts receivable and investments of $55.6 million at June 30, 2017. Current liabilities were $14.1 million, net of deferred revenue of $9.5 million, as of December 31, 2017, compared to $19.9 million, net of deferred revenue of $35.1 million, as of June 30, 2017.

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Palatin believes that existing capital resources will be sufficient to fund our planned operations through at least the next 12 months.

CONFERENCE CALL / WEBCAST
Palatin will host a conference call and webcast on February 12, 2018 at 11:00 a.m. Eastern Time to discuss the results of operations in greater detail and provide an update on corporate developments. Individuals interested in listening to the conference call live can dial 1-800-289-0449 (domestic) or 1-323-794-2093 (international), conference ID 6111978. The webcast and replay can be accessed by logging on to the "Investor/Webcasts" section of Palatin’s website at View Source A telephone and webcast replay will be available approximately one hour after the completion of the call. To access the telephone replay, dial 1-888-203-1112 (domestic) or 1-719-457-0820 (international), passcode 6111978. The webcast and telephone replay will be available through February 19, 2018.

On February 12, 2018 Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported the issuance of two U.S. patents – US 9,855,297 and US 9,890,393 – for the invention of certain uses of RNA-guided endonucleases, such as Cas9 or Cpf1, for the genetic engineering of T-cells (Press release, Cellectis, FEB 12, 2018, View Source [SID1234523922]). The patents came into force on January 2nd, 2018 and February 13th, 2018, respectively.

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Both patents claim methods by which T-cells are gene edited using transient expression of CRISPR/Cas9 components. These inventions are based on the early work initiated by inventors at Cellectis when the CRISPR technology first came to light.

These therapeutic-focused patents follow the grant by the European Patent Office of patent No. EP3004337 for similar inventions and previous intellectual property that Cellectis has obtained over the last two decades for major gene editing technologies including meganucleases, TALEN, MegaTAL and CRISPR.

"Cellectis is a pioneering gene editing company that has always been keen to be at the forefront of all gene editing technologies," said Dr. André Choulika, Cellectis Chairman & CEO. "We have been the first to explore the potential of CRISPR in its early days in various applications, including therapeutics and plants. These early findings ultimately led to the grant of this set of new patents. As such, these patents only reinforce Cellectis’ leadership position in the gene editing industry."

Convinced of their strong value for the future development of engineered CAR T-cells, Cellectis will make these patents available for licensing to companies that are willing to use CRISPR technologies in T-cells. The technical knowledge in these patents could, for example, help users engineer allogeneic CAR T-cells while suppressing genes involved in checkpoint inhibitions, such as PD-1, engineer drug resistance, or remove MHC (Major Histocompatibility Complex) related genes. The technology could also be used to insert a DNA CAR construct by gene targeting a specific locus in the genome of T-cells.

The inventors of these patents are Dr. André Choulika, Chairman & CEO of Cellectis and one of the pioneers in the development of nuclease-based genome editing technologies; Dr. Philippe Duchateau, Cellectis Chief Scientific Officer and seasoned gene editing expert; and Dr. Laurent Poirot, Cellectis Head of Early Discovery and expert of gene functions in immune cells.

Claims 1 and 2 of US 9,855,297:

1. A method of preparing genetically modified primary T-cells for immunotherapy comprising the steps of: (a) transfecting mRNA encoding an RNA-guided endonuclease into the primary T-cells, wherein the RNA-guided endonuclease is expressed from the transfected m RNA; (b) introducing a DNA vector that encodes a specific guide RNA, wherein the specific guide RNA directs the RNA-guided endonuclease to at least one targeted locus in the T-cell genome into the primary T-cells; (c) cleaving at least one targeted locus in the T-cell genome with the RNA-guided endonuclease; (d) generating a genetic modification at the site of the cleavage; and (e) expanding the resulting genetically modified T-cells.

2. The method of claim 1, wherein the RNA-guided endonuclease is Cas9.

Claim 1 of US 9,890,393:

1. A method of preparing T-cells for immunotherapy comprising the step of:

(a) genetically modifying primary T-cells by introduction and/or expression into the cells of at least:

– a RNA-guided endonuclease; and

– a specific guide RNA that directs said endonuclease to at least one targeted locus in the T-cell genome,

wherein said RNA-guided endonuclease is expressed from transfected mRNA;

wherein said RNA-guided endonuclease comprises the amino acid sequence set forth in SEQ ID NO:1 or SEQ ID NO:2; and

(b) expanding the resulting cells.

On February 12, 2018 Supernus Pharmaceuticals, Inc. (NASDAQ:SUPN), a specialty pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported that the Company expects to report the financial results for the fourth quarter and full year of 2017 after 5:00 PM ET on Tuesday, February 27, 2018 (Press release, Supernus, FEB 12, 2018, View Source [SID1234523918]).

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Jack Khattar, President and Chief Executive Officer, and Greg Patrick, Chief Financial Officer, will host a conference call to present the fourth quarter and full year 2017 business results on Wednesday, February 28, 2018 at 9:00 AM ET. Following the management presentation, the call will be open for questions.

A live webcast will be available at www.supernus.com.

Please refer to the information below for conference call dial-in information. Callers should dial in approximately 10 minutes prior to the start of the call.

Conference dial-in: (877) 288-1043
International dial-in: (970) 315-0267
Conference ID: 9385269
Conference Call Name: Supernus Pharmaceuticals Fourth Quarter and Full Year 2017 Earnings Conference Call

Following the live call, a replay will be available on the Company’s website under the ‘Investors’ section. The webcast will be available on the Company’s website for 60 days following the live call.

On February 12, 2018 ERYTECH Pharma (Euronext: ERYP- Nasdaq: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported the selection of Triple Negative Breast Cancer as the next target indication for broadening the scope of eryaspase (GRASPA) development in solid tumors (Press release, ERYtech Pharma, FEB 12, 2018, View Source;p=RssLanding&cat=news&id=2332012 [SID1234523923]).

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L-asparaginase is a cornerstone treatment in acute lymphoblastic leukemia (ALL), especially for pediatric patients, but the excessive toxicity of conventional L-asparaginase formulations has limited its use in other indications.

The positive Phase 2b result of eryaspase (L-asparaginase encapsulated in red blood cells) in metastatic pancreatic cancer, reported in 2017, represents, to our knowledge, the first-ever evidence of clinical benefit of an asparaginase-based product in a solid tumor indication. In this 141-patient randomized Phase 2b study, eryaspase in combination with chemotherapy demonstrated a 40% reduction in risk of death rate (HR=0.60; p=0.009) compared to chemotherapy alone.

Following these very encouraging results, ERYTECH conducted a comprehensive evaluation to determine other potential solid-tumor indications for developing eryaspase. Metastatic Triple-Negative Breast Cancer (TNBC) has now been selected as the next indication to expand the potential use of eryaspase in solid tumors. TNBC is an aggressive and metabolically active form of breast cancer with high rates of symptomatic metastases. A recent publication in Nature1 supports the hypothesis that restricting availability of asparagine can reduce metastatic progression of cancer cells in breast cancer. One of the most striking observations from our Phase 2b trial in pancreatic cancer was the 40% reduction of new lesions in the eryaspase arm compared to the control arm.

"TNBC is a heterogenous subgroup of breast cancer associated with poor patient outcome and high risk of recurrence compared to other breast cancer subtypes. Aside from BRCA1/2 mutation status, treatment options for TNBC remain limited," commented Iman El-Hariry, MD, PhD, Chief Medical Officer of ERYTECH. "There is growing evidence of altered metabolism in TNBC. The evaluation of eryaspase in metastatic TNBC provides a promising new therapeutic approach, which capitalizes on reprogramming of the metabolic pathways in this disease."

Gil Beyen, Chairman and CEO of ERYTECH, added, "The selection of TNBC as the second solid tumor indication for evaluating eryaspase anti-tumor activity brings hope for improving the health of these women. The safety profile of eryaspase provides additional rationale for combination with currently existing therapies to increase treatment options in TNBC."

The development in TNBC complements ERYTECH’s pipeline of programs, which focus on the development of therapies that target amino acid metabolism of tumor cells. Set-up activities of a Phase 2 proof-of-concept clinical study have started and ERYTECH expects to enroll the first patient in Q3 2018.

About Triple-Negative Breast Cancer (TNBC)

Breast cancer is the most commonly diagnosed cancer in women globally with nearly 1.8 million new cases diagnosed annually2. It is estimated that over 600,000 women each year are diagnosed with breast cancer in the United States and Europe in aggregate3. Approximately 10-20% of breast cancers are TNBC, a form of breast cancer that lacks expression of estrogen receptor (ER), progesterone receptor (PR) and does not overexpress HER24. TNBC is associated with a poorer prognosis when compared to other breast cancer subtypes. As commonly utilized hormone therapy and HER2 targeting agents are not treatment options for women with TNBC, there is significant unmet need for novel therapeutic approaches in this subtype of breast cancer.

On February 12, 2018 EUSA Pharma (EUSA), reported that the National Institute for Health and Care Excellence (NICE) has published a Final Appraisal Determination (FAD) recommending tivozanib as a first-line treatment option for advanced renal cell carcinoma (aRCC) in line with its licensed indication (Press release, EUSA Pharma, FEB 12, 2018, View Source [SID1234523911]). Tivozanib was licensed by the European Commission (EC) in August 20171 and is an oral, once-daily, potent selective vascular endothelial growth factor receptor tyrosine kinase inhibitor (VEGFR-TKI). VEGFR-TKIs work by reducing the supply of blood to the tumour – denying it the food and oxygen it would need to grow in size.2

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In arriving at its positive recommendation, NICE considered data from the global, open-label, randomised, multi-centre Phase III trial (TiVO-1)3 which evaluated the efficacy and tolerability of tivozanib compared to a currently available comparator VEGFR-TKI (sorafenib) in the treatment of 517 patients with aRCC. Patients treated with tivozanib experienced superior progression-free survival (PFS) (11.9 vs. 9.1 months in the overall population and 12.7 vs. 9.1 months in treatment naïve patients) versus sorafenib.3 There was also an improved side effect profile with tivozanib, with only 14% (versus 43% with sorafenib) requiring a dose reduction due to adverse events (AEs).3 In addition, fewer people on tivozanib experienced burdensome side effects, such as diarrhoea (23% vs 33%), and hand-foot syndrome (14% vs 54%).3

Dr Lisa Pickering, Consultant Medical Oncologist at St George’s Hospital, London, commented: This is excellent news for patients with aRCC. A key treatment goal is to enable patients to live without their cancer progressing for longer periods of time – we cannot cure their cancer but we can help to preserve normal living for as long as possible. That is why I’m delighted that NICE has recommended tivozanib as a treatment option."

Around 12 people in the UK die every day of kidney cancer, with more than 40% of cases in England diagnosed at a late (advanced) stage. Kidney cancer is expected to be one of the fastest increasing cancers over the next 10 years, as a result of the UK’s ageing population, and with smoking and a rise in obesity also playing a part.4

Nick Turkentine, Chief Executive of Kidney Cancer UK, said: "With over 12,500 new cases of kidney cancer being diagnosed every year this is a great result for people with aRCC and the overall fight with the disease. It is vitally important patients have access to effective, well-tolerated treatments, so they can spend more precious time with their families and loved ones. We applaud EUSA Pharma, NICE and NHS England for making this happen."

Patients with advanced renal cell carcinoma (aRCC) who may benefit from treatment with tivozanib will be able to access it from today as the final guidance is published, and should be able to have immediate access through the CDF.

Lee Morley, EUSA Pharma’s Chief Executive Officer commented "We are very pleased that NICE are recommending the use of Fotivda for the first line treatment of advanced Renal Cell Carcinoma and that Fotivda will be funded immediately in England and Wales, meaning physicians and patients can experience the benefits of Fotivda. Fotivda has the potential to become an important new first-line therapy and the recommendation by NICE is a great achievement for the EUSA team delivering on their mission to improve the lives of those patients suffering from cancer."