On November 13, 2018 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company" or "Sophiris"), a biopharmaceutical company studying topsalysin (PRX302), a first-in-class, pore-forming protein, in late-stage clinical trials for the treatment of patients with urological diseases, reported financial results for the third quarter 2018 and recent corporate highlights (Press release, Sophiris Bio, NOV 13, 2018, View Source [SID1234531295]).

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"During the third quarter, we reported top-line six-month biopsy data from two additional patients following a single administration of topsalysin in our ongoing Phase 2b clinical trial in low to intermediate risk localized prostate cancer," said Randall E. Woods, president and CEO of Sophiris. "Both patients were considered partial responders, bringing the total number of patients with a partial response to 15 out of the 37. We continue to believe that the data obtained to date from the single administration of topsalysin supports advancing topsalysin into a single Phase 3 trial for the treatment of localized prostate cancer. We remain on track to report data by the end of next month from patients who received a second administration of topsalysin as part of our Phase 2b trial. Finally, we are preparing for Phase 3 guidance meetings with regulatory agencies. We expect these meetings to take place in the first half of 2019, after which we will provide an update on our Phase 3 trial design."

Third Quarter Corporate Highlights:

Updates from Phase 2b trial in localized prostate cancer. In the third quarter, Sophiris reported top-line six-month follow-up biopsy data from the final two patients enrolled in the trial with pre-identified, clinically-significant localized prostate cancer that were treated with a single administration of topsalysin.

Based on the six-month follow-up biopsy results, 27% of patients (10/37) demonstrated a clinical response. Of the 10 clinical responders in the Phase 2b trial, six patients experienced a complete ablation with no histological evidence of the targeted tumor remaining. In addition, 41% of patients (15/37) were considered partial responders, meaning that while an effect was seen, some clinically-significant lesion remained as identified by targeted biopsy. 68% of patients (25/37) demonstrated a partial or clinical response to the single administration of topsalysin.

The Phase 2b study was designed to include an option for qualified patients to receive a second administration of topsalysin six-months after the first administration, provided: (1) the patient did not have any clinically-significant adverse events to either topsalysin or the dosing procedure and (2) some response to the first administration was observed following a targeted biopsy. The objective of re-administering topsalysin is to assess the safety of giving a second administration of topsalysin and to determine if additional clinical benefit is observed six-months after the second administration.

The Company expects to have six-month follow-up safety and biopsy data from patients who received a second administration of topsalysin in its ongoing Phase 2b trial in localized prostate cancer patients next month.

Preparations for Phase 3 trial in localized prostate cancer. The Company believes that the data generated in the single-administration portion of the Phase 2b prostate cancer study supports the advancement of the program into a single Phase 3 pivotal trial. Currently, the Company is in the process of preparing information for regulatory guidance meetings with the U.S. Food and Drug Administration and the European Medicines Agency which are expected to take place in the first half of 2019, after which the Company will provide an update on the Phase 3 trial design. The Company will evaluate whether future clinical development will include an option to administer a second dose of topsalysin once the Company receives more information from the 10 patients who have received a second dose.

Interest only period extended under loan and security agreement. In September the Company announced that it had met the requirements within its existing loan and security agreement with Silicon Valley Bank to extend the interest only period to March 31, 2019. The Company will begin making interest and principal payments starting on April 1, 2019 and ending on the final payment date of September 1, 2021.

Financial Results:

At September 30, 2018, the Company had cash, cash equivalents and securities available-for-sale of $14.5 million and working capital of $11.7 million. The Company expects that its existing cash and cash equivalents will be sufficient to fund its operations through June 2019, assuming no new clinical trials are initiated. The Company will require significant additional funding to advance topsalysin in clinical development. As of September 30, 2018, the outstanding principal balance of the Company’s term loan was $7 million on which the Company is currently making monthly interest only payments.

For the three months ended September 30, 2018

The Company reported a net loss of $2.9 million or ($0.10) per share for the three months ended September 30, 2018, compared to net loss of $2.7 million or ($0.09) per share for the three months ended September 30, 2017.

Research and development expenses

Research and development expenses were $1.8 million for the three months ended September 30, 2018, compared to $1.6 million for the three months ended September 30, 2017. The increase in research and development costs is primarily attributable to increases in the costs associated with manufacturing activities for topsalysin offset in part by a decrease in clinical costs associated with its Phase 2b clinical trial of topsalysin for the treatment of localized prostate cancer.

General and administrative expenses

General and administrative expenses were $1.2 million for the three months ended September 30, 2018, compared to $1.7 million for the three months ended September 30, 2017. The decrease in general and administrative expense is primarily due to decreases in marketing research activities and to a lesser extent a decrease in personnel related expenses.

Gain on revaluation of the warrant liability

Gain on revaluation of the warrant liability was $0.2 million for the three months ended September 30, 2018, compared to a gain of $0.7 million for the three months ended September 30, 2017. As these warrants may require the Company to pay the warrant holder cash under certain provisions of the warrant, the Company accounts for these warrants as a liability, and the Company is required to calculate the fair value of these warrants each reporting date. The non-cash gain reported for the three months ended September 30, 2018, is associated with a decrease in the fair value of the Company’s warrant liability from June 30, 2018, to September 30, 2018, which is calculated using a Black-Scholes pricing model. Certain inputs utilized in the Company’s Black-Scholes fair value calculation may fluctuate in future periods based upon factors which are outside of the Company’s control. A significant change in one or more of these inputs used in the calculation of the fair value may cause a significant change to the fair value of the Company’s warrant liability, which could also result in a material non-cash gain or loss being reported in the Company’s consolidated statement of operations and comprehensive loss.

For the nine months ended September 30, 2018

The Company reported a net loss of $12.3 million or ($0.41) per share for the nine months ended September 30, 2018 compared to a net loss of $4.7 million or ($0.15) per share for the nine months ended September 30, 2017.

Research and development expenses

Research and development expenses were $8.7 million for the nine months ended September 30, 2018 compared to $4.2 million for the nine months ended September 30, 2017. The increase in research and development costs is primarily attributable to increases in the costs associated with manufacturing activities for topsalysin.

General and administrative expenses

General and administrative expenses were $3.5 million for the nine months ended September 30, 2018 compared to $4.4 million for the nine months ended September 30, 2017. The decrease in general and administrative expense is primarily due to decreases in non-cash stock-based compensation expense and marketing research activities and to lesser extent a decrease in its personnel related costs.

Gain on revaluation of the warrant liability

Gain on revaluation of the warrant liability was $0.1 million for the nine months ended September 30, 2018 as compared to a gain of $3.9 million for the nine months ended September 30, 2017. The non-cash gain reported for the nine months ended September 30, 2018, is associated with a decrease in the fair value of the Company’s warrant liability from December 31, 2017, to September 30, 2018.

On November 12, 2018 Coherus BioSciences, Inc. (Nasdaq: CHRS), reported that senior management will be presenting at the 27th Annual Credit Suisse Healthcare Conference being held in Scottsdale, Arizona on Thursday, November 15 at 9:45 am MST (Press release, Coherus Biosciences, NOV 12, 2018, View Source [SID1234531698]).

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The audio portion of the presentation will be available on the investors page of the Coherus BioSciences website at View Source

On November 12, 2018 Ziopharm Oncology, Inc. (Nasdaq: ZIOP), reported that it has entered into definitive securities purchase agreements for the sale of its common stock and warrants to purchase common stock in a private placement that is expected to result in gross proceeds to the Company of approximately $50 million, before deducting placement agent and other offering expenses (Press release, Ziopharm, NOV 12, 2018, View Source [SID1234531224]). The private placement is being led by existing stockholder, MSD Partners, L.P. Other participants include Miller Value Partners, White Rock Capital Management and Level One Partners, LLC.

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Pursuant to the terms of the securities purchase agreement, at the closing of the private placement, Ziopharm will issue and sell 18,939,394 shares of common stock and warrants to purchase up to 18,939,394 additional shares of common stock at a per unit purchase price of $2.64. The warrants will become exercisable on the date that is six months following the date of issuance, have a per share exercise price of $3.01 and will expire five years from the date of issuance. The private placement is expected to close on or about November 13, 2018, subject to the satisfaction of customary closing conditions. Additional details regarding the private placement will be included in a Form 8-K filed by Ziopharm with the Securities and Exchange Commission (the "SEC").

Neither the shares of Ziopharm common stock nor the warrants to be issued in the private placement have been registered under the Securities Act of 1933, as amended (the "Securities Act"). Accordingly, these securities may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act. Ziopharm has agreed to file a registration statement with the SEC to register the resale of Ziopharm common stock to be issued in the private placement as well as the Ziopharm common stock issuable upon exercise of the warrants.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

On November 12, 2018 Cellectar Biosciences, Inc. (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted an exemption to the Import Alert placed on the Centre for Probe Development and Commercialization (CPDC), the sole supplier of the CLR 131 (Press release, Cellectar Biosciences, NOV 12, 2018, View Source [SID1234531227]). The exemption for CLR 131 is effective immediately for all hematology studies and, in response, Cellectar is preparing to dose patients in the second fractionated dose cohort of the Phase 1 relapsed refractory (R/R) multiple myeloma study and the Phase 2 study for R/R hematologic malignancies. The company awaits authorization from the FDA for any future shipments in connection with its Phase 1 study of pediatric patients with neuroblastoma, sarcomas, lymphomas (including Hodgkin’s lymphoma) and malignant brain tumors.

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"We thank the FDA for their diligence and for providing this exemption for CLR 131 hematology studies. Our ability to advance our clinical trials and achieve stated business objectives remains our top priority," said James Caruso, president and CEO of Cellectar Biosciences. "I also want to recognize our team for their outstanding execution in support of a rapid resolution."

In its efforts to obtain an exemption for CLR 131 from the Import Alert in hematology and pediatrics, Cellectar has collaborated with the various divisions within the FDA that oversee the company’s investigational new drug applications evaluating CLR 131 in multiple indications. Cellectar executed a series of actions requested by the FDA to obtain an exemption to the Import Alert for its hematology programs. Similarly, the company continues to work with the appropriate division of the FDA to secure an exemption for the pediatric program.

As background, on August 10, 2018, Cellectar announced that CPDC was informed of an FDA Import Alert that prohibited CPDC from supplying CLR 131. While the Import Alert disrupted CLR 131 supply, the basis of the Import Alert was not related to CLR 131 specifically, or to CPDC’s production facility associated with CLR 131. The company actively supported CPDC’s efforts to have the Import Alert lifted as quickly as possible. The FDA subsequently initiated direct talks with Cellectar concerning a possible exemption for CLR 131 from the Import Alert. Those discussions and subsequent actions resulted in the exemption Cellectar is announcing today.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131 is in a Phase 2 clinical study in relapsed/refractory multiple myeloma (R/R MM) and a range of B-cell malignancies, and a Phase 1b clinical study in patients with R/R MM exploring fractionated dosing. The objective of the multicenter, open-label, Phase 1b dose-escalation study is the characterization of safety and tolerability of CLR 131 in patients with R/R MM. Patients in Cohorts 1-4 received single doses of CLR 131 ranging from 12.5 mCi/m2 to 31.25 mCi/m2 as well as a fractionated dose of 15.625 mCi/m2 given twice over seven days in Cohort 5. All study doses and regimens have been deemed safe and well tolerated by an independent Data Monitoring Committee. The company plans to initiate a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma as well as a second Phase 1 study in combination with external beam radiation for head and neck cancer.

On November 12, 2018 AstraZeneca and Merck & Co., Inc., Kenilworth, N.J., US (Merck: known as MSD outside the US and Canada) reported that the US Food and Drug Administration (FDA) has accepted a supplemental New Drug Application (sNDA) for Priority Review for the use of Lynparza (olaparib) tablets as a maintenance treatment in patients with newly-diagnosed, BRCA-mutated (BRCAm) advanced ovarian cancer who were in complete or partial response following 1st-line standard platinum-based chemotherapy (Press release, AstraZeneca, NOV 12, 2018, View Source [SID1234531207]). A Prescription Drug User Fee Act (PDUFA) date is set for the first quarter of 2019.

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This is the first US regulatory submission acceptance for a poly ADP-ribose polymerase (PARP) inhibitor in the 1st-line maintenance setting for advanced ovarian cancer, and if approved will be the fourth indication for Lynparza in the US.

This submission was based on positive results from the pivotal Phase III SOLO-1 trial. The trial showed a statistically-significant and clinically-meaningful improvement in progression-free survival (PFS) for Lynparza compared to placebo, reducing the risk of disease progression or death by 70% in patients with newly-diagnosed, BRCAm advanced ovarian cancer who were in complete or partial response to platinum-based chemotherapy (HR 0.30 [95% CI 0.23-0.41], p<0.001). Of those receiving Lynparza, 60% remained progression-free at 36 months compared to 27% of women in the placebo arm. These data were recently presented for the first time at the ESMO (Free ESMO Whitepaper) 2018 Congress (European Society for Medical Oncology) and published online in the New England Journal of Medicine.

Lynparza is currently approved in over 60 countries for the treatment of platinum-sensitive relapsed ovarian cancer regardless of BRCA status. It is also approved in several countries, including the US and Japan, for germline BRCAm HER2-negative metastatic breast cancer – regulatory reviews are underway in the EU, Japan and other markets.

About SOLO-1

SOLO-1 is a Phase III randomised, double-blinded, placebo-controlled, multicentre trial to evaluate the efficacy and safety of Lynparza tablets (300mg twice daily) as maintenance monotherapy compared with placebo, in newly-diagnosed patients with BRCAm advanced ovarian cancer following platinum-based chemotherapy. The trial randomised 391 patients with a deleterious or suspected deleterious BRCA1 or BRCA2 mutation who were in clinical complete or partial response following platinum-based chemotherapy. Patients were randomised (2:1) to receive Lynparza or placebo for up to two years or until disease progression (at the investigator’s discretion). The primary endpoint was PFS and key secondary endpoints included time to second disease progression or death, time to first subsequent treatment and overall survival.

About Lynparza

Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to potentially exploit DNA damage response (DDR) pathway deficiencies, such as BRCA mutations, to preferentially kill cancer cells. Specifically, in vitro studies have shown that Lynparza-induced cytotoxicity may involve inhibition of PARP-enzymatic activity and increased formation of PARP-DNA complexes, resulting in DNA damage and cancer cell death. Lynparza is being tested in a range of DDR-deficient tumour types.

Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, is approved for advanced ovarian cancer and metastatic breast cancer and has been used in over 20,000 patients worldwide. Lynparza has the broadest and most advanced clinical trial development programme of any PARP inhibitor and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza is the foundation of AstraZeneca’s industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.

About ovarian cancer

Ovarian cancer is a leading cause of cancer death in women worldwide, with a five-year survival rate of 19%.[i] In 2018, there were over 295,000 new cases diagnosed and around 185,000 deaths.[ii] For newly-diagnosed advanced ovarian cancer, the primary aim of treatment is to delay progression of the disease for as long as possible and maintain the patient’s quality of life with the intent of achieving complete remission or cure.[iii],[iv],[v],[vi]

About BRCA mutations

BRCA1 and BRCA2 are human genes that produce proteins responsible for repairing damaged DNA and play an important role in maintaining the genetic stability of cells. When either of these genes is mutated, or altered, such that its protein product either is not made or does not function correctly, DNA damage may not be repaired properly, and cells become unstable. As a result, cells are more likely to develop additional genetic alterations that can lead to cancer.