On September 12, 2019 Palatin Technologies, Inc. (NYSE American: PTN), a specialized biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin and natriuretic peptide receptor systems, whose product candidates are targeted, receptor-specific therapeutics for the treatment of diseases with significant unmet medical need and commercial potential, reported results for its fourth quarter and fiscal year ended June 30, 2019 (Press release, Palatin Technologies, SEP 12, 2019, View Source [SID1234539467]).

"The last year was a landmark one for Palatin. We are proud of the recent FDA approval of Vyleesi and the continued advancement of our pipeline programs," said Carl Spana, Ph.D., President and Chief Executive Officer of Palatin. "The FDA approval is an incredible achievement and milestone, and we are excited that premenopausal women now have a safe and effective, as-needed option available to them for the treatment of acquired, generalized HSDD. Our cash and accounts receivable balances at June 30, 2019 of $102 million is sufficient to cover planned operations through at least calendar year 2021. We remain focused on advancing discussions on Vyleesi collaborations for territories outside the currently licensed territories of North America, China, and Korea, and initiating multiple clinical trials for our pipeline programs over the next several quarters for the treatment of dry eye disease, non-infectious uveitis and ulcerative colitis."

2019 Fiscal Year Highlights and Recent Events

Hypoactive Sexual Desire Disorder / Vyleesi (bremelanotide injection)
The U.S. Food and Drug Administration (FDA) granted marketing approval of AMAG Pharmaceuticals, Inc.’s New Drug Application (NDA) for Vyleesi (bremelanotide injection), a melanocortin receptor agonist developed by Palatin, indicated for the treatment of premenopausal women with acquired, generalized hypoactive sexual desire disorder (HSDD). The FDA’s approval of the NDA on June 21, 2019 triggered a $60 million milestone payment to Palatin under its North American license agreement with AMAG that was received in July. Additionally, Palatin is entitled to receive tiered royalties on net sales ranging from high single-digit to low double-digit percentages, and sales milestones based on escalating annual net sales thresholds, the first of which is $25 million, triggered at annual net sales of $250 million.

Vyleesi is the first as needed treatment for premenopausal women with acquired, generalized HSDD. Vyleesi is currently available through specialty pharmacies, Avella and BioPlus, and AMAG will launch Vyleesi nationally with its full sales force in mid-September.

Anti-Inflammatory / Autoimmune Programs
Melanocortin agonist products are under development for the treatment of inflammatory and autoimmune diseases such as dry eye, uveitis, diabetic retinopathy and inflammatory bowel diseases (ulcerative colitis).

We announced positive results of a micro-dose study of radiolabeled PL8177, a selective melanocortin receptor 1 ("MC1r") peptide agonist, using an oral, delayed-release, polymer formulation. The study met all primary and secondary endpoints. PL8177 has potential application in treatment of ulcerative colitis and other inflammatory bowel diseases. The FDA has granted orphan drug designation for PL8177 for the treatment of non-infectious intermediate, posterior, pan and chronic anterior uveitis. Non-infectious uveitis (NIU) is a group of inflammatory diseases that produces swelling and destroys eye tissue and can result in vision loss. A Phase 2 proof-of-concept clinical study with a systemic formulation in NIU patients is anticipated to commence in the fourth quarter of calendar year 2019. A Phase 2 proof-of-concept clinical study with an oral formulation in ulcerative colitis patients is anticipated to commence in the first quarter of calendar year 2020.

An IND application for PL9643, a melanocortin peptide agonist, and commencement of a Phase 2 clinical study in dry eye disease, are currently anticipated in the first quarter of calendar year 2020.

Natriuretic Peptide Receptor ("NPR") System Program
The Company has designed and is developing potential drug candidates that are selective agonist for one or more different natriuretic peptide receptors, including natriuretic peptide receptor-A ("NPR-A"), natriuretic peptide receptor B ("NPR-B"), and natriuretic peptide receptor C ("NPR-C"). Active collaborations with several institutions are ongoing for PL3994, an NPR-A agonist that has potential utility in the treatment of a number of cardiovascular diseases, including genetic and orphan diseases resulting from a deficiency of endogenous active NPR-A, and PL5028, a dual NPR-A and NPR-C agonist in development for cardiovascular diseases, including reducing cardiac hypertrophy and fibrosis. A Phase 2A clinical trial evaluating PL3994 in heart failure patients with preserved left ventricular ejection fraction will begin enrollment in the latter half of calendar year 2019. This trial is supported by a grant from the American Heart Association.

Genetic Obesity Program
The Company’s melanocortin receptor 4 ("MC4r") peptide PL8905 and orally-active small molecule PL9610 are currently under investigation for the treatment of rare genetic metabolic and obesity disorders. These programs are under internal evaluation for orphan designation and potential development.

Corporate
Cash and accounts receivable balances at June 30, 2019 of $102 million is sufficient to cover planned operations through at least calendar year 2021. Included in the accounts receivable balance is a $60 million milestone payment due from AMAG for the Vyleesi FDA approval, which was received in July 2019.

Debt and related liabilities decreased from $7.2 million at June 30, 2018 to $0.8 million at June 30, 2019, with a final payment remitted in July 2019.

Fourth Quarter and Fiscal 2019 Financial Results
Palatin reported net income of $52.2 million, or $0.25 per basic and $0.23 per diluted share, for the fourth quarter ended June 30, 2019, compared to net income of $11.8 million, or $0.06 per basic and diluted share, for the same period in 2018.

The difference between the three months ended June 30, 2019 and 2018 was attributable to the recognition of license and contract revenue pursuant to our license agreement with AMAG of $60.3 million for the quarter ended June 30, 2019 compared to $20.6 million in 2018.

For the year ended June 30, 2019, Palatin reported net income of $35.8 million, or $0.17 per basic and $0.16 per diluted share, compared to net income of $24.7 million, or $0.12 per basic and diluted share for the year ended June 30, 2018.

The difference in net income for the years ended June 30, 2019 and 2018, was primarily the result of a $16.6 million decrease in operating expenses to $24.6 million for the year ended June 30, 2019, compared to $41.2 million for the year ended June 30, 2018, partially offset by the recognition of license and contract revenue of $60.3 million for the year ended June 30, 2019, compared to $67.1 million of license and contract revenue for the year ended June 30, 2018.

Revenue
For the quarter and year ended June 30, 2019, Palatin recognized $60.3 million in license and contract revenue related to our license agreement with AMAG.

For the quarter and year ended June 30, 2018, Palatin recognized $20.6 million and $62.1 million, respectively, in license and contract revenue related to our license agreement with AMAG and an additional $5 million in license revenue for the year ended June 30, 2018 related to our license agreement with Fosun.

Operating Expenses
Total operating expenses for the quarter ended June 30, 2019 were $8.1 million, compared to $8.3 million for the comparable quarter of 2018. For the year ended June 30, 2019, Palatin incurred $24.6 million of operating expenses, compared to $41.2 million for the year ended June 30, 2018.

The decrease in operating expenses reflects the completion of the Vyleesi Phase 3 clinical trial program and ancillary studies necessary to file the NDA with the FDA in March 2018.

Other Income/Expense
Total other income, net, for the quarter and year ended June 30, 2019 was approximately $38,000 and $29,000, respectively.

Total other expense, net, for the quarter and year ended June 30, 2018 was $0.2 million and $1.1 million, respectively.

Total other income (expense), net consisted of investment income offset by interest expense related to venture debt.

Income Tax
Palatin recorded no income tax expense for the quarter and year ended June 30, 2019 as a result of the utilization of net operating losses.

Income tax expense was $0.3 and $0.1 million, respectively, for the quarter and year ended June 30, 2018. Income tax expense for the year ended June 30, 2018 related to $0.6 million in tax withholding requirements related to our Fosun and Kwangdong license agreements that was recorded as an expense during the fiscal year ended June 30, 2018 offset by a tax benefit of $0.5 million 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.

Cash Position
Palatin’s cash and cash equivalents were $43.5 million with accounts receivable of $60.3 million at June 30, 2019, compared to cash and cash equivalents and no accounts receivable of $38.0 million at June 30, 2018. Included in the accounts receivable balance at June 30, 2019 is a $60 million milestone payment due from AMAG for the Vyleesi FDA approval, which was received in July 2019. Current liabilities were $4.2 million as of June 30, 2019, compared to $10.8 million at June 30, 2018.
Palatin Drug Discovery Programs
During the conference call and webcast, management will update and discuss next steps in Palatin’s portfolio of drug development programs. These include Palatin’s melanocortin MC1r agonist peptides for treatment of inflammatory indications and natriuretic peptide receptor agonist compounds for treatment of cardiovascular and fibrotic diseases.

Conference Call / Webcast
Palatin will host a conference call and audio webcast on September 12, 2019 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 1-888-254-3590 (U.S./Canada) or 1-323-994-2093 (international), conference ID 1394071. The audio 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 (U.S./Canada) or 1-719-457-0820 (international), passcode 1394071. The webcast and telephone replay will be available through September 19, 2019.

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On September 12, 2019 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a clinical stage biotechnology company focused on the development of novel therapeutics for the treatment of rare diseases, reported it will present clinical data on its lead product candidate, mavorixafor (X4P-001), in combination with Inlyta (axitinib) at the upcoming European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Congress, taking place September 27 – October 1 in Barcelona, Spain (Press release, X4 Pharmaceuticals, SEP 12, 2019, View Source [SID1234539483]). The presentation will detail final safety and efficacy results from the Company’s Phase 2a portion of an open-label Phase 1/2 clinical trial of mavorixafor in combination with axitinib in patients with advanced clear cell renal cell carcinoma (ccRCC).

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Details of the presentation are as follows:

Abstract #2521: Safety and Efficacy of the Oral CXCR4 Inhibitor X4P-001 + Axitinib in Advanced Renal Cell Carcinoma Patients: An Analysis of Subgroup Responses by Prior Treatment
Date and Time: Monday, September 30, 2019; 8:45 – 9:45 AM CEST
Session Type: Poster Discussion Session, Discussion 2 – Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)
Presentation #: 1186PD
Location: Malaga Auditorium, Hall 5

Details of the investor conference call and webcast are as follows:

Time and Date: Monday, September 30 at 8:00 AM EDT / 2:00 PM CEST
US Toll-Free Dial-In Number: (866) 721-7655
International Dial-In Number: (409) 216-0009 / Spain 0934923253
Conference ID: 4787329
Webcast: A live audio webcast of the conference call may be accessed in the "Investors" section of the Company’s website at the following link.

About Mavorixafor

X4 Pharmaceuticals’ lead product candidate, mavorixafor (X4P-001), is a potential first-in-class, once-daily, oral inhibitor of CXCR4, currently in Phase 3 development for the treatment of WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. Mavorixafor has demonstrated proof of concept in WHIM syndrome in a Phase 2 trial, including clinically meaningful increases in neutrophil and lymphocyte biomarker counts, as well as a trend of reduction in infection rates and wart burden, and a favorable safety profile. Mavorixafor was designated orphan drug status by the U.S. Food and Drug Administration in 2018 and by the European Commission in 2019 for the treatment of WHIM syndrome, and is also in development for the treatment of Severe Congenital Neutropenia (SCN), Waldenström’s macroglobulinemia (WM), and clear cell renal cell carcinoma (ccRCC).

On September 12, 2019 NOXXON Pharma N.V. (Paris:ALNOX) (Euronext Growth Paris: ALNOX), a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), reported the start of recruitment of newly diagnosed brain cancer patients in a Phase 1/2 clinical trial combining the CXCL12 inhibitor NOX-A12 with radiotherapy (Press release, NOXXON, SEP 12, 2019, View Source [SID1234539484]). NOX-A12 is thought to inhibit the influx of "repair cells" from the bone marrow to the tumor, an unwanted consequence of radiotherapy that leads to a replacement of the destroyed blood vessels within the tumor which ultimately results in a recurrence of the disease.

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The safety and first efficacy data obtained in this study will support the definition of a Recommended Phase 2 Dose (RP2D) for this new treatment approach to guide further clinical developments. The non-invasive assessment of the changes in tumor vascularization by MRI is expected to confirm the predicted mechanism mode of action for the inhibition of CXCL12 in combination with radiotherapy.

"NOX-A12 combined with radiotherapy is a novel and promising approach with the potential to effectively treat brain cancer patients for which there are currently no optimal therapies. The demand from the clinical community to test this combination has been very strong and we are pleased to initiate this trial. We expect data from the first cohort of patients to be available in mid-2020," said Jarl-Ulf Jungnelius, Chief Medical Officer of NOXXON.

The trial will be conducted at three hospitals in Germany. Up to three escalating doses of NOX-A12 will be administered in combination with standard radiotherapy to newly diagnosed patients with brain tumors who would not benefit from the current standard of care of chemoradiotherapy and whose tumors cannot be fully resected by surgery. The main objective of the study is to assess the safety and tolerability of this combination. Secondary endpoints include activity of the therapy, assessed through the monitoring of tumor vascularization by MRI scans, progression-free survival, overall survival and rates of response.

CEO of NOXXON, Aram Mangasarian, will host a webinar on September 23, 2019 at 03.00 p.m. CEST, and will be joined by Dr. Frank Giordano, Vice Chairman of Radiation Oncology Department at University Medical Centre Mannheim for the presentation and Q&A session. To join the webinar, please send an email to [email protected].

On September 12, 2019 Celgene Corporation (NASDAQ: CELG) reported top-line results from the international phase 3, randomized, double-blind, placebo-controlled study, QUAZAR AML-001 (Press release, Celgene, SEP 12, 2019, View Source [SID1234539485]). The study evaluated the efficacy and safety of investigational therapy CC-486 as maintenance therapy in patients with newly diagnosed acute myeloid leukemia (AML) who achieved first complete response (CR) or complete response with incomplete blood count recovery (CRi) with induction chemotherapy (with or without consolidation). The study demonstrated that maintenance treatment with CC-486 resulted in a highly statistically significant and clinically meaningful improvement in overall survival compared to placebo. The key secondary endpoint of relapse-free survival (RFS) also showed a statistically significant improvement.

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CC-486 was well-tolerated and there were no unexpected safety events in QUAZAR AML-001. This phase 3 study enrolled 472 patients, randomized 1:1 to receive either oral CC-486 300mg or placebo once daily for 14 days of a 28-day cycle plus best supportive care until disease relapse.

"AML remains a deadly blood cancer where most patients are not curable and less than 30% of patients survive five years1," said Jay Backstrom, M.D., M.P.H., Chief Medical Officer for Celgene. "The CC-486 QUAZAR AML-001 study is the first phase 3 trial to demonstrate that the addition of maintenance therapy has the potential to extend overall survival in a broad population of patients with newly diagnosed AML who have achieved remission with induction chemotherapy."

Data from QUAZAR AML-001 will be submitted to a future medical meeting. Celgene also plans regulatory submissions for CC-486 beginning in the first half of 2020.

CC-486 is an investigational compound and not approved for any use in any geography.

About QUAZAR AML-001

Phase 3, randomized, double-blind, placebo-controlled study of CC-486 as AML maintenance therapy in patients who achieved first CR or complete response with incomplete blood count recovery (CRi) with induction chemotherapy (with or without consolidation) The primary endpoint of the study was overall survival. Key secondary endpoints included relapse-free survival (RFS), safety and tolerability, healthcare resource utilization and patient-reported outcomes per the FACIT-Fatigue Scale and EQ-5D questionnaire. The study enrolled 472 patients, randomized 1:1 to receive either oral CC-486 300mg or placebo once daily for 14 days of a 28-day cycle plus best supportive care until disease progression.

About CC-486

CC-486 is a cytidine nucleoside analogue and incorporates into DNA and RNA. The main mechanism of action is thought to cause DNA hypomethylation and direct cytotoxicity on abnormal hematopoietic cells in the bone marrow. Hypomethylation may restore normal function to genes that are critical for differentiation and proliferation. The antineoplastic effect of CC-486 is hypothesized to cause death of rapidly dividing cells, including cancer cells that are no longer responsive to normal growth control mechanism.

About AML

Acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal blast cells that are supposed to grow into different types of blood cells. It may present in multiple subtypes based on the maturity of the cancer cells at diagnosis.2 There will be an estimated 21,450 new cases of AML in the United States this year, accounting for 1.2% of all cancer cases, with an estimated 10,920 deaths resulting from the disease. There are an estimated 61,048 people living with AML in the United States.3

On September 12, 2019 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported positive data from the Phase III IMpower110 study evaluating Tecentriq (atezolizumab) as a first-line (initial) monotherapy compared with cisplatin or carboplatin and pemetrexed or gemcitabine (chemotherapy) in advanced non-squamous and squamous non-small cell lung cancer (NSCLC) without ALK or EGFR mutations (Wild-Type or WT) (Press release, Hoffmann-La Roche, SEP 12, 2019, View Source [SID1234539486]). The study met its primary endpoint in an interim analysis showing that Tecentriq monotherapy demonstrated a statistically significant overall survival (OS) benefit in people with high PD-L1 expression (TC3/IC3-WT), compared with chemotherapy alone. Safety for Tecentriq appeared to be consistent with its known safety profile and no new safety signals were identified. The study will continue to final analysis for patients with lower levels of PD-L1 expression.

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"We are encouraged that Tecentriq monotherapy has shown a significant survival benefit over chemotherapy as an initial treatment in people with squamous or non-squamous non-small cell lung cancer with high PD-L1 expression," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "These findings reinforce the potential of Tecentriq to play an important role in the treatment of multiple forms of lung cancer, and we look forward to discussing these data with health authorities."

Roche will now submit these data to global health authorities, including the FDA and EMA, and will discuss how best to bring this option to patients as quickly as possible. These data will be presented at an upcoming medical congress.

Currently, Roche has nine Phase III lung cancer studies underway evaluating Tecentriq as a monotherapy or in combination with other medicines across different types of lung cancer. Roche has an extensive development programme for Tecentriq, including multiple ongoing and planned Phase III studies, across lung, genitourinary, skin, breast, gastrointestinal, gynaecological and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

About the IMpower110 study
IMpower110 is a Phase III, randomised, open-label study to evaluate the efficacy and safety of Tecentriq monotherapy compared with cisplatin or carboplatin and pemetrexed or gemcitabine (chemotherapy) in programmed death-ligand 1 (PD-L1)-selected, chemotherapy-naive participants with advanced non-squamous or squamous NSCLC without ALK or EGFR mutations (Wild-Type or WT).

A total of 572 people (555 WT) were enrolled and were randomised 1:1 to receive:

Tecentriq monotherapy, until loss of clinical benefit (as assessed by the investigator) or
Cisplatin or carboplatin (per investigator discretion) combined with either pemetrexed (non-squamous) or gemcitabine (squamous), followed by maintenance therapy with pemetrexed alone (non-squamous) or best supportive care (squamous) until disease progression, unacceptable toxicity or death.
The primary efficacy endpoint is OS by PD-L1 subgroup (TC3/IC3-WT; TC2/3/ IC2/3-WT; and TC1,2,3/IC1,2,3-WT), as determined by the SP142 assay test. Key secondary endpoints include investigator-assessed progression-free survival (PFS), objective response rate (ORR) and duration of response (DoR).

About NSCLC
Lung cancer is the leading cause of cancer death globally.1 Each year 1.76 million people die as a result of the disease; this translates into more than 4,800 deaths worldwide every day.1 Lung cancer can be broadly divided into two major types: NSCLC and small cell lung cancer. NSCLC is the most prevalent type, accounting for around 85% of all cases.2 NSCLC comprises non-squamous and squamous-cell lung cancer, the squamous form of which is characterised by flat cells covering the airway surface when viewed under a microscope.2

About Tecentriq (atezolizumab)
Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1, which is expressed on tumour cells and tumour-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the activation of T cells. Tecentriq is a cancer immunotherapy (CIT) that has the potential to be used as a foundational combination partner with other immunotherapies, targeted medicines and various chemotherapies across a broad range of cancers. The development of Tecentriq and its clinical programme is based on our greater understanding of how the immune system interacts with tumours and how harnessing a person’s immune system combats cancer more effectively.

Tecentriq is approved in the US, EU and countries around the world, either alone or in combination with targeted therapies and/or chemotherapies in various forms of non-small cell and small cell lung cancer, certain types of metastatic urothelial cancer, and in PD-L1-positive metastatic triple-negative breast cancer.

About Roche in cancer immunotherapy
For more than 50 years, Roche has been developing medicines with the goal to redefine treatment in oncology. Today, we’re investing more than ever in our effort to bring innovative treatment options that help a person’s own immune system fight cancer.

By applying our seminal research in immune tumour profiling within the framework of the Roche-devised cancer immunity cycle, we are accelerating and expanding the transformative benefits with Tecentriq to a greater number of people living with cancer. Our cancer immunotherapy development programme takes a comprehensive approach in pursuing the goal of restoring cancer immunity to improve outcomes for patients.

To learn more about the Roche approach to cancer immunotherapy please follow this link: View Source