On February 14, 2018 Oncobiologics, Inc. (NASDAQ:ONS) today reported financial results and business highlights for its first fiscal quarter ended December 31, 2017 (Press release, Oncobiologics, FEB 14, 2018, View Source;p=RssLanding&cat=news&id=2332432 [SID1234523977]). Oncobiologics had a net loss attributable to common stockholders of $17.7 million for the three months ended December 31, 2017 and total cash of $ 13.8 million at December 31, 2017. On an adjusted basis, Oncobiologics had a net loss attributable to common stockholders for the three months ended December 31, 2017 of $5.0 million.

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Oncobiologics’ Chairman and Chief Executive Officer Dr. Pankaj Mohan commented, "With the closing of the GMS Tenshi strategic investment in October 2017, in 2018 we plan to execute on a newly developed strategy to organically generate funding for our biosimilar development programs, in addition to our ongoing efforts to secure additional development partners. The first step in this strategy is to leverage the capacity and capabilities of our BioSymphony Platform to accelerate and maximize commercial revenues from our core expertise in drug development and manufacturing. As we roll out this new contract development and manufacturing (CDMO) business, we initially plan to assist our clients with the development and manufacturing of their drug product candidates for clinical trials."

"In 2017, Oncobiologics also completed the process of out-licensing rights to ONS-3010 and ONS-1045 biosimilar development programs for emerging markets to GMS Tenshi. In each of these smaller, ex-U.S. markets, we identified potential synergies between our partner’s strategy to enter the biologics marketplace and access to our biosimilar development platform. For many of these emerging market opportunities, our partners may be able to take advantage of differing regulatory requirements that could allow more rapid regulatory approval of these product candidates and commercial sales."

Dr. Mohan continued, "Going forward, we will continue to focus on the development of our biosimilar pipeline and look for partners for our most advanced programs, ONS-3010 and ONS-1045, to move those candidates into Phase 3 clinical trials to support FDA and EMA approvals. Additionally, we are excited to confirm two programs we are preparing for clinical development ONS-4010, a biosimilar of Prolia/Xgeva, and ONS-3040, a biosimilar for Stelara. We have also begun work on ONS-5010, an innovative drug product candidate that will not use the biosimilar regulatory pathway. Our intent is to seek and receive feedback from U.S. regulatory authorities and proceed into Phase 1 clinical trials for ONS-5010 in 2018."

"As we execute this updated strategy with the support of our new partner and investor, GMS Tenshi, we believe that the company is well positioned to begin generating revenue from our new CDMO business in 2019, which we expect to cover the basic operating costs of running our business and allow us to use funds generated from partnerships and other transactions for investment directly in our development pipeline," concluded Dr. Mohan.

First Quarter Highlights

Stockholders approved the strategic investment by GMS Tenshi Holdings Pte. Limited, from which the Company received the remaining $21.7 million of gross proceeds from the sale of Series A Convertible Preferred Stock;
The Company initiated efforts to launch a new CDMO business to support ongoing biosimilar drug development efforts;
Started work in emerging markets with development partners to expedite regulatory approvals and position the Company for potential revenue generation;
Continued discussions with potential development partners to initiate Phase 3 programs for ONS-3010 and ONS-1045;
Confirmed next biosimilar pipeline candidates for clinical development, ONS-4010 and ONS-3040;
Identified ONS-5010, an innovative drug product candidate to be developed outside of the biosimilar regulatory pathway.
2018 – Anticipated Milestones

• Q2 2018

Enter into first CDMO contract;
• Q3/Q4 2018

Initiate clinical development program for ONS-3010 and/or ONS-1045 by partners in emerging markets;
Initiate ONS-5010 Phase 1 clinical development program;
Announce licensing/co-development partnership announced for major market.
Long-term Milestones

• 2019

CDMO business cash flow positive by end of 2019;
Initiate Phase 3 trial for ONS-1045 in major markets with development partner;
• 2020

First revenue from emerging market partnerships;
Initiate Phase 3 trial for ONS-3010 in major markets with development partner;
Initiate ONS-3040 and ONS-4010 clinical development programs;
• 2021

Submit applications to FDA for ONS-1045 and ONS-3010.
Financial Highlights

For the three months ended December 31, 2017, Oncobiologics reported a net loss attributable to common stockholders of $17.7 million, or $0.71 per share, compared to $19.1 million, or $0.82 per share for the same period in the preceding year. For the three months ended December 31, 2017, net loss attributable to common stockholders includes $1.9 million of non-cash stock-based compensation expense, $0.7 million of depreciation and amortization, a $1.3 million loss from the extinguishment of debt, $0.1 million from a decrease in the fair value of warrant liability, $3.2 million benefit from the sale of state of New Jersey net operating losses, a $15.4 million beneficial conversion charge related to the Company’s Series A convertible preferred stock and a $3.2 million reduction in expenses from the favorable settlement of the termination of a clinical contract. Adjusting for these items, the Company reported an adjusted net loss attributable to common stockholders of $5.0 million, or $0.20 per share, on a non-GAAP basis as appears in the attached non-GAAP reconciliation. Adjusted net loss attributable to common stockholders for the three months ended December 31, 2016 was $15.2 million, or $0.65 per share, on a non-GAAP adjusted basis comparable to the same period in the current fiscal year.

The primary factor for the decrease in adjusted net loss attributable to common stockholders for the three months ended December 31, 2017 as compared to the same period in the prior year was a significant reduction in research and development expenses, which was related to the Company’s decision to postpone the initiation of planned Phase 3 clinical trials for ONS-3010 and ONS-1045 until additional development partners have been secured.

Cash was $13.8 million as of December 31, 2017, compared to $3.2 million as of September 30, 2017.

Non-GAAP Financial Measure – Adjusted Net Loss Attributable to Common Stockholders

Oncobiologics prepares its consolidated financial statements in conformity with accounting principles generally accepted in the United States of America (GAAP) and pursuant to accounting requirements of the Securities and Exchange Commission. In an effort to provide investors with additional information regarding the results and to provide a meaningful period-over-period comparison of Oncobiologics financial performance, Oncobiologics sometimes uses non-GAAP financial measures (NGFM) as defined by the Securities and Exchange Commission. In this press release, Oncobiologics uses the NGFM, "adjusted net loss attributable to common stockholders." Management uses this NGFM because it adjusts for unusual transactions, transactions not related to the Company’s core business or events that are not expected to recur, such as losses from extinguishment of debt, sales of state net operating losses, as well as the settlement of a clinical development contract in connection with the decision to postpone Phase 3 clinical trials of two biosimilar programs, as well as significant non-cash items that impact financial results but not cash flows, such as the recognition of the beneficial conversion feature due to the issuance of Series A Convertible Preferred Stock to GMS Tenshi, stock-based compensation expense, depreciation and amortization expense, and fair value measurements for the Company’s equity and debt securities. Management used this NGFM to evaluate Oncobiologics’ financial performance against internal budgets and targets. Management believes that this NGFM is useful for evaluating Oncobiologics core operating results and facilitating comparison across reporting periods. Oncobiologics believes this NGFM should be considered in addition to, and not in lieu of, GAAP financial measures. Oncobiologics NGFM may be different from the same NGFM used by other companies.

For additional details on Oncobiologics’ financial performance during the quarter, please see the Company’s filings with the Securities and Exchange Commission at: View Source;owner=exclude&action=getcompany

On February 13, 2018 Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, "Astellas") and Universal Cells, Inc. (CEO: Claudia Mitchell, "Universal Cells") reported that Astellas has acquired Universal Cells (Press release, Astellas, FEB 14, 2018, View Source [SID1234649901]). Astellas will gain Universal Cells’ proprietary Universal Donor Cell technology to create cell therapy products that do not require Human Leukocyte Antigen (HLA) matching, potentially overcoming a huge treatment challenge by reducing the risk of rejection.

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In October 2017, the Astellas Institute for Regenerative Medicine (AIRM) and Universal Cells entered into an exclusive license agreement to utilize Universal Donor Cell technology in a single indication. Today’s acquisition enables Astellas to fully utilize this proprietary technology in even more therapeutic areas. The acquisition combines Astellas’ capability of establishing differentiated functional cells from pluripotent stem cells with Universal Cell’s ability to produce pluripotent stem cells that have lower immunological rejection to further enable investigation of innovative cell therapy treatments for various diseases that currently have few or no treatment options.

"We have been very impressed with Universal Cells’ capabilities in cell therapy, including Universal Donor Cell technology, which led us to our initial collaboration and ultimately this acquisition," commented Yoshihiko Hatanaka, President and CEO, Astellas. "This additional capability will further enable Astellas to develop potential innovative cell therapies for numerous diseases with high unmet medical needs."

"We are thrilled to be able to leverage the full potential of our Universal Donor Cell technology by becoming an intrinsic part of Astellas’ effort to fulfill the promises of Regenerative Medicine to treat diseases," said Claudia Mitchell, CEO of Universal Cells. "The acquisition represents the recognition of the immense potential of our unique technology and of the outstanding work done by our team at Universal Cells."

Astellas will pay up to $102.5 million of upfront and milestones to acquire 100 percent ownership of Universal Cells depending on achievement of certain specified clinical milestones.

Universal Cells has become a wholly-owned subsidiary of Astellas following the close of the acquisition.

The impact of this transaction on Astellas’ financial results for the fiscal year ending

March 31, 2018 will be immaterial.

On February 14, 2018 PDL BioPharma, Inc. (PDL) (NASDAQ: PDLI) reported that John P. McLaughlin, PDL’s chief executive officer, will present at the following two upcoming investor conferences (Press release, PDL BioPharma, FEB 14, 2018, View Source [SID1234523978]):

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2018 RBC Capital Markets Global Healthcare Conference
Wednesday February 21, 2018 at 9:30 a.m. EST
New York City

Cowen and Company 38th Annual Health Care Conference
Tuesday, March 13, 2018 at 12:00 p.m. EDT
Boston, Massachusetts

To access the live and subsequently archived webcast of these presentations, go to the company’s website at View Source and go to "Presentations and Events." Please connect to the website at least 15 minutes prior to the presentations to allow for any software download that may be necessary. The archived webcasts will be available for at least seven days following the presentations.

On February 14, 2018 Oncoceutics Inc. and Calvert Research, LLC reported that the two companies have entered into a second product development and investment partnership agreement, this time to further develop and enable the submission of an investigational new drug (IND) application for ONC206, Oncoceutics’ next generation molecule (Press release, Oncoceutics, FEB 14, 2018, View Source [SID1234558372]).

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As a part of this second agreement, Calvert Research will enlist the capabilities of its CRO affiliate, Calvert Laboratories, Inc. to conduct certain GLP animal safety and pharmacology studies on ONC206 in its GLP certified facilities. In addition, Calvert Research has made a second equity investment in Oncoceutics. This agreement follows the same successful investment-development partnership model established between Oncoceutics and Calvert Research in 2013 that lead to Oncoceutics being able to open an IND application for ONC201, its lead molecule.

ONC206 is a member of a family of drug candidates called "imipridones" that possess the same core structure of ONC201 and that have demonstrated the same favorable drug characteristics, including efficacy in pre-clinical models against cancer with high levels of safety. Given these attractive attributes, Oncoceutics filed a pre-IND application with the FDA describing its plans for a clinical trial with ONC206 and received positive written feedback to the pre-IND submission from the FDA. Based on the FDA written response, Oncoceutics has defined all of the studies required to have an IND accepted by the FDA. These include manufacturing ONC206 in quantity and at quality sufficient to treat people, and the toxicology work necessary to open an investigational new drug application (IND) that will be completed by Calvert Laboratories. Oncoceutics expects to complete all studies by the end of 2018 and to file the IND and begin first-in-human studies for ONC206 in 2019.

Previously, Oncoceutics has announced that the United States Patent and Trademark Office (USPTO) has issued a patent providing the company with composition of matter protection for ONC206.

"We are pleased to enter into a second investment partnership agreement with Calvert Research, a firm that possesses excellent capabilities in the field of GLP-compliant animal safety studies," said Martin Stogniew Ph.D., Chief Development Officer of Oncoceutics. "By bringing the second member of the imipridone family towards a clinical trial, we are continuing our transformation of Oncoceutics from a company with one drug in development into a company developing a portfolio of drugs."

On February 14, 2018 The Janssen Pharmaceutical Companies of Johnson & Johnson reported that the U.S. Food and Drug Administration (FDA) has approved ERLEADA (apalutamide), a next-generation androgen receptor inhibitor,1 for the treatment of patients with non-metastatic castration-resistant prostate cancer (NM-CRPC) (Press release, Johnson & Johnson, FEB 14, 2018, View Source [SID1234523973]). ERLEADA is the first FDA-approved treatment for these patients. Today’s approval follows an FDA Priority Review designation based upon data from the Phase 3 SPARTAN study, which demonstrated a 72 percent reduction in risk of distant metastasis or death, and an increase in median metastasis-free survival (MFS) by more than two years (difference of 24.31 months) in patients with NM-CRPC.

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"The need to delay metastasis is critical to the treatment of prostate cancer. Nearly 90 percent of patients with castration-resistant prostate cancer will eventually develop bone metastases, at which point the prognosis sharply worsens," said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, LLC. "We are excited about what this approval means for patients living with prostate cancer, and that physicians now have an important and much-needed treatment option that has been shown to delay the progression of castration-resistant prostate cancer."

ERLEADA received FDA approval based on the Phase 3 data from the SPARTAN clinical trial, which assessed the efficacy and safety of ERLEADA versus placebo in patients with NM-CRPC who had a rapidly rising PSA while receiving continuous androgen deprivation therapy.2 The study was recently presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU) on Thursday, February 8, 2018 in San Francisco and published in The New England Journal of Medicine.2,3

"The SPARTAN trial results demonstrated impressive clinical benefits in patients with non-metastatic castration-resistant prostate cancer," said Matthew Smith, M.D., Ph.D., Director of the Genitourinary Malignancies Program at the Massachusetts General Hospital Cancer Center, Professor of Medicine at Harvard Medical School, and co-principal investigator of the SPARTAN study. "As an oncologist and clinical investigator, I know how devastating it can be for patients and their families to hear that the cancer has spread. With this approval, doctors now have the chance to offer hope for delaying metastases in patients with castration-resistant prostate cancer."

"As the impact of prostate cancer continues to grow, we are reminded every day of the critical need for therapeutic options that offer patients with prostate cancer more time with their loved ones," Mark Scholz, M.D., Executive Director of the Prostate Cancer Research Institute. "Today’s approval is significant, as it means that patients with non-metastatic castration-resistant prostate cancer now have a treatment option that offers renewed hope."

SPARTAN, a Phase 3, randomized, double-blind, placebo-controlled, multi-center study, enrolled 1,207 patients with non-metastatic castration-resistant prostate cancer.4 Patients were randomized 2:1 to receive either ERLEADA orally at a dose of 240 mg once daily (n=806), or placebo once daily (n=401).4 All patients in the SPARTAN trial received a concomitant gonadotropin-releasing hormone (GnRH) analog or had a bilateral orchiectomy.4

ERLEADA decreased the risk of distant metastasis or death by 72 percent compared to placebo (HR = 0.28; 95% CI, 0.23-0.35; P<0.0001).4 The median MFS was 40.51 months for ERLEADA compared to 16.20 months for placebo, prolonging MFS by more than two years (difference of 24.31 months).4 MFS benefit was consistently seen across patient subgroups including prostate specific antigen doubling time (PSADT) (≤6 months or >6 months), use of a prior bone-sparing agent (yes or no), and locoregional disease (N0 or N1).4

The major efficacy outcome was supported by statistically significant improvements for the following secondary endpoints: time to metastasis (TTM), progression-free survival (PFS) and time to symptomatic progression.4 The median TTM was 40.51 months for ERLEADA compared to 16.59 months for placebo (HR=0.27; 95% CI, 0.22-0.34; P<0.0001) and the median PFS was 40.51 months compared to 14.72 months for placebo (HR=0.29; 95% CI, 0.24-0.36; P<0.0001).4 Overall survival data were not mature at the time of final MFS analysis (24% of the required number of events).4

Warnings and Precautions include seizure, falls and fractures.4 In the SPARTAN trial, the most common adverse reactions (≥10%) were fatigue, hypertension, rash, diarrhea, nausea, weight decreased, arthralgia, fall, hot flush, decreased appetite, fracture, and peripheral edema.4

About Non-Metastatic Castration-Resistant Prostate Cancer
Non-metastatic castration-resistant prostate cancer (NM-CRPC) refers to a disease state when the cancer no longer responds to medical or surgical treatments that lower testosterone, but has not yet been discovered in other parts of the body using a total body bone scan or CT scan.5 Features include: lack of detectable metastatic disease;5 rapidly rising prostate-specific antigen while on androgen deprivation therapy (ADT) and serum testosterone level below 50 ng/dL.6,7 Ninety percent of patients with CRPC will eventually develop bone metastases, which can lead to pain, fractures and spinal cord compression.8 The relative 5-year survival rate for patients with distant stage prostate cancer is 30 percent.9 It is critical to delay the onset of metastasis in patients with NM-CRPC.

About ERLEADA
ERLEADA (apalutamide) is an androgen receptor (AR) inhibitor indicated for the treatment of patients with non-metastatic castration-resistant prostate cancer.4

ERLEADA is an AR inhibitor that binds directly to the ligand-binding domain of the AR. ERLEADA inhibits AR nuclear translocation, inhibits DNA binding, and impedes AR-mediated transcription.4 A major metabolite, N-desmethyl apalutamide, is a less potent inhibitor of AR, and exhibited one-third the activity of ERLEADA in an in vitro transcriptional reporter assay.4 ERLEADA administration caused decreased tumor cell proliferation and increased apoptosis leading to decreased tumor volume in mouse xenograft models of prostate cancer.4

Full prescribing information will be available soon at www.ERLEADA.com.

Important Safety Information4

CONTRAINDICATIONS

Pregnancy – ERLEADA can cause fetal harm and potential loss of pregnancy.

WARNINGS AND PRECAUTIONS

Falls and Fractures – In the SPARTAN study, falls and fractures occurred in 16% and 12% of patients treated with ERLEADA compared to 9% and 7% treated with placebo respectively. Falls were not associated with loss of consciousness or seizure. Evaluate patients for fracture and fall risk. Monitor and manage patients at risk for fractures according to established treatment guidelines and consider use of bone targeted agents.

Seizure – In a randomized study (SPARTAN), two patients (0.2%) treated with ERLEADA experienced a seizure. Permanently discontinue ERLEADA in patients who develop a seizure during treatment. It is unknown whether anti-epileptic medications will prevent seizures with ERLEADA. Advise patients of the risk of developing a seizure while receiving ERLEADA and of engaging in any activity where sudden loss of consciousness could cause harm to themselves or others.

ADVERSE REACTIONS

Adverse Reactions – The most common adverse reactions (≥10%) were fatigue, hypertension, rash, diarrhea, nausea, weight decreased, arthralgia, fall, hot flush, decreased appetite, fracture, and peripheral edema.

Laboratory Abnormalities

Hematology: anemia ERLEADA 70% (Grade 3-4 0.4%) placebo 64% (Grade 3-4 0.5%) leukopenia ERLEADA 47% (Grade 3-4 0.3%) for, placebo 29% (Grades 3-4 0%), lymphopenia ERLEADA 41% (Grade 3-4 2%), placebo 21% (Grade 3-4 2%);
Chemistry – hypercholesterolemia ERLEADA 76%(Grade3-4 0.1%), placebo 46% (Grade 3-4 0%); hypertriglycemia ERLEADA 70%(Grade 3-4 2%) Placebo 59% (0.8%); hypertriglyceridemia ERLEADA 67%(Grade 3-4 2%) placebo 49%(Grade 3-4 0.8%); Hyperkalemia ERLEADA 32%(Grade 3-4 2%) Placebo 22%(Grade 3-4 0.5%)
Rash – Rash was most commonly described as macular or maculo-papular. Adverse reactions were 24% with ERLEADA versus 6% with placebo. Grade 3 rashes (defined as covering > 30% body surface area [BSA]) were reported with ERLEADA treatment (5%) versus placebo (0.3%).

The onset of rash occurred at a median of 82 days. Rash resolved in 81% of patients within a median of 60 days (range: 2 to 709 days) from onset of rash. Four (4%) of patients treated with ERLEADA received systemic corticosteroids. Rash recurred in approximately half of patients who were re-challenged with ERLEADA.

Hypothyroidism – Hypothyroidism was reported for 8% of patients treated with ERLEADA and 2% of patients treated with placebo based on assessments of thyroid-stimulating hormone (TSH) every 4 months. Elevated TSH occurred in 25% of patients treated with ERLEADA and 7% of patients treated with placebo. The median onset was Day 113. There were no Grade 3 or 4 adverse reactions. Thyroid replacement therapy, when clinically indicated, should be initiated or dose-adjusted.

Effect of Other Drugs on ERLEADA – Co-administration of a strong CYP2C8 or CYP3A4 inhibitor is predicted to increase the steady-state exposure of the active moieties. No initial dose adjustment is necessary however, reduce the ERLEADA dose based on tolerability [see Dosage and Administration (2.2)].

DRUG INTERACTIONS

Effect of ERLEADA on Other Drugs – ERLEADA is a strong inducer of CYP3A4 and CYP2C19, and a weak inducer of CYP2C9 in humans. Concomitant use of ERLEADA with medications that are primarily metabolized by CYP3A4, CYP2C19, or CYP2C9 can result in lower exposure to these medications. Substitution for these medications is recommended when possible or evaluate for loss of activity if medication is continued. Concomitant administration of ERLEADA with medications that are substrates of UDP-glucuronosyl transferase (UGT) can result in decreased exposure. Use caution if substrates of UGT must be co-administered with ERLEADA and evaluate for loss of activity.

P-gp, BCRP or OATP1B1 substrates – Apalutamide is a weak inducer of P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), and organic anion transporting polypeptide 1B1 (OATP1B1) clinically. Concomitant use of ERLEADA with medications that are substrates of P-gp, BCRP, or OATP1B1 can result in lower exposure of these medications. Use caution if substrates of P-gp, BCRP or OATP1B1 must be co-administered with ERLEADA and evaluate for loss of activity if medication is continued.