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Merrimack Announces Inclusion of ONIVYDE® (irinotecan liposome injection) as a Category 1 Treatment Option in the 2016 NCCN Guidelines for Pancreatic Adenocarcinoma

On March 24, 2016 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) reported that the National Comprehensive Cancer Network (NCCN) has included ONIVYDE (irinotecan liposome injection) in combination with fluorouracil (5-FU) and leucovorin in its 2016 Clinical Practice Guidelines in Oncology for pancreatic adenocarcinoma (Press release, Merrimack, MAR 24, 2016, View Source [SID:1234509915]).

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The new guidelines recognize the ONIVYDE regimen as a category 1 second-line therapy for patients with metastatic adenocarcinoma of the pancreas who have previously been treated with gemcitabine-based therapy. A category 1 classification represents the highest level of evidence and uniform NCCN consensus that the intervention is appropriate. The new guidelines are published on www.nccn.org .

"The addition of the ONIVYDE regimen to the 2016 NCCN guidelines further validates the importance of this treatment option for patients battling metastatic pancreatic cancer," said Edward J. Stewart, Head of Commercial at Merrimack. "ONIVYDE is the only FDA approved therapy available to patients whose disease has progressed after gemcitabine-based therapy and, more importantly, it addresses a critical unmet need in a patient population with very limited options. We believe these guidelines will further support the adoption of the ONIVYDE regimen as a standard-of-care in metastatic pancreatic cancer."

Pancreatic cancer is a rare and deadly disease. Each year approximately 53,000 patients are diagnosed with pancreatic cancer in the United States with only 7% surviving five years or longer1. The NCCN’s recommendation was based on a review by a multidisciplinary panel of experts from NCCN member institutions and supported by data from the NAPOLI-1 study, published in The Lancet in 2015, and the U.S. Food and Drug Administration (FDA) approval of the ONIVYDE regimen. NAPOLI-1 was a randomized, open label Phase 3 study in patients with metastatic adenocarcinoma of the pancreas who received prior gemcitabine-based therapy, and was the largest Phase 3 study in this setting to date. Patients were enrolled at 76 sites in North America, South America, Europe, Asia and Oceania.

NCCN is an alliance of 26 world-class cancer centers dedicated to the development of treatment guidelines for most cancers and to research that will ultimately improve the quality of patient care and outcomes. The NCCN guidelines are widely recognized as the standard of clinical practice in oncology and provide evidence-based treatment recommendations to assist key stakeholders, including physicians, patients and payers, in directing cancer patient care.

About ONIVYDE [pronounced \ ‘on – īh – vide \]

ONIVYDE (irinotecan liposome injection), also known as MM-398 or "nal-IRI," is a novel encapsulation of irinotecan in a liposomal formulation. The activated form of irinotecan is SN-38, which functions by inhibiting topoisomerase I (an essential enzyme involved in DNA transcription and replication) and promoting cell death. ONIVYDE was recently approved by the U.S. Food and Drug Administration in combination with fluorouracil and leucovorin for the treatment of patients with metastatic adenocarcinoma of the pancreas after disease progression following gemcitabine-based therapy. For full prescribing information, including Boxed WARNING, please visit www.ONIVYDE.com.

20-F – Annual and transition report of foreign private issuers [Sections 13 or 15(d)]

(Filing, Annual, Rosetta Genomics, 2015, MAR 23, 2016, View Source [SID:1234509916])

Arbor Pharmaceuticals to Acquire XenoPort

On May 23, 2016 Arbor Pharmaceuticals, LLC (Arbor) and XenoPort, Inc. (XenoPort) (NASDAQ:XNPT) announced today that they have signed a definitive agreement under which Arbor will acquire XenoPort for $7.03 per share in cash, or a total equity value of approximately $467 million (Press release, Arbor Pharmaceuticals, MAR 23, 2016, View Source [SID1234523639]). The purchase price per share represents a 60 percent premium to the closing price of XenoPort shares on May 20, 2016.

"We are pleased to be adding HORIZANT and the XenoPort pipeline to the growing portfolio of Arbor products," said Ed Schutter, President and Chief Executive Officer of Arbor. "We believe that XenoPort’s lead product HORIZANT offers patients and physicians a valuable treatment option for moderate-to-severe primary restless legs syndrome and postherpetic neuralgia. The XenoPort sales team has done an excellent job of growing HORIZANT, and we look forward to supporting them to continue this significant momentum."

Vincent J. Angotti, Chief Executive Officer of XenoPort, stated, "This transaction provides immediate and substantial value to our stockholders, and we believe that Arbor is well positioned to provide the proper resources for a more expanded commercialization effort of HORIZANT. We evaluated many potential options to maximize the value for stockholders and believe this transaction represents a great outcome for XenoPort stockholders."

Under the terms of the agreement, Arbor will commence a tender offer to purchase all of the outstanding shares of XenoPort for $7.03 per share. Following the closing of the tender offer, the agreement provides for the parties to effect, as promptly as practicable, a merger that would result in all shares not tendered in the tender offer being converted into the right to receive $7.03 per share in cash. The transaction, which has been unanimously approved by both the Arbor Board of Directors and the XenoPort Board of Directors, is expected to close in the third quarter of 2016.

Closing of the tender offer and merger is subject to certain customary conditions, including the tender of more than 50 percent of all outstanding shares of XenoPort. The transaction is also subject to review by the U.S. Government under the Hart-Scott-Rodino (HSR) Antitrust Improvements Act, as amended, and other customary closing conditions.

Centerview Partners is serving as exclusive financial advisor to XenoPort, and Weil, Gotshal & Manges LLP is serving as legal advisor to XenoPort. Deutsche Bank has provided sole committed debt financing to Arbor in support of the transaction. Alston & Bird, LLP and Simpson, Thacher & Bartlett LLP acted as legal advisors to Arbor.

Foundation Medicine and Memorial Sloan Kettering Publish Validation Data for FoundationOne® Heme in the Journal Blood

On March 23, 2016 Foundation Medicine, Inc. (NASDAQ:FMI) and Memorial Sloan Kettering Cancer Center (MSK) reported the publication of new, seminal data validating FoundationOne Heme – the fully informative comprehensive genomic profiling assay for hematologic malignancies developed as part of their collaboration – further supporting its integration into oncology clinical practice (Press release, Foundation Medicine, MAR 23, 2016, View Source [SID:1234509929]). The data, available online as a first edition and soon to be published in an upcoming issue of the journal Blood, demonstrate that FoundationOne Heme has proven highly accurate in detecting the types of genomic alterations known to impact diagnosis, therapy selection and prognosis in hematologic cancers. Importantly, the publication demonstrates the molecular information gleaned from comprehensive genomic profiling can be utilized to accurately match patients with an appropriate therapeutic approach.

"Foundation Medicine has an established track record of developing genomic profiling assays with the highest standards of analytical and clinical validation," said Vincent Miller, M.D., chief medical officer, Foundation Medicine. "Publication of our validation data in this highly regarded, peer-reviewed journal supports the clinical significance of the FoundationOne Heme assay, in particular, for its ability to identify specific therapeutic targets, to help refine underlying diagnosis, and to improve prognostic and risk stratification of hematologic cancers."

"The development of FoundationOne Heme represents a state-of-the-art genomics assay which can be used to profile patients with hematologic malignancies worldwide, which is a critical step in improving outcomes for all patients," stated Ross Levine, M.D., a physician-scientist and the Laurence Joseph Dineen chair in leukemia research at MSK. "Our team’s expertise in hematologic malignancies and in translating genomics to the clinic has allowed us to partner with Foundation Medicine to bring this innovative genomic test to the patients we treat."

Conventional diagnostic assays, including FISH and real-time PCR, are designed to identify a sub-set of genomic alterations, and in some cases, there are no assays that can reliably identify specific rearrangements. FoundationOne Heme, an integrated DNA/RNA platform using targeted hybrid-capture next-generation sequencing, is a proven and effective comprehensive genomic profile developed to detect all types of genomic alterations with therapeutic relevance, including single-nucleotide substitutions, insertions and deletions, copy number alterations and rearrangements, which are not fully evaluated using conventional diagnostic assays.

MSK and Foundation Medicine collaborated to develop FoundationOne Heme, which was commercially launched in 2013. The assay is performed using archived FFPE, blood or bone marrow samples with high accuracy in a clinically relevant timeframe in Foundation Medicine’s laboratory, which is certified by New York State and CLIA and is CAP accredited. FoundationOne Heme simultaneously detects all classes of genomic alterations in the DNA of 405 cancer-related genes and employs RNA sequencing across 265 genes to capture a broad range of gene fusions, a type of alteration that is a common driver of hematologic cancers. It is designed to provide physicians with clinically actionable information to guide treatment options for patients based on the genomic profile of their cancer.

Key Study Findings

Established analytic accuracy of detecting substitutions, insertions and deletions (indels) and copy number alterations (CNAs) by comparing the performance of the new assay with Foundation Medicine’s DNA-only assay that has previously undergone comprehensive validation across a large number of clinical samples. Compared to FoundationOne, FoundationOne Heme contains an additional 90 genes relevant to hematologic malignancies.

Samples that were previously profiled with a validated test in which 169 alterations were identified in 55 genes common to both assays. The concordance between the two sets of results was 99.4%.

Blinded comparisons were performed with CLIA-certified diagnostic assays, including Sequenom, RT-PCR, FISH and PCR fragment analysis, for 76 clinical specimens previously tested for 214 clinical relevant alterations in 11 genes that are known and routinely tested in clinical practice in AML, ALL and MDS.

Overall concordance was 99% (211/214).

In addition to the concordance analysis, genomic profiling of the 76 test samples identified 126 additional somatic alterations which are not covered by available hot spot assays in the given disease type, including clinically relevant genomic alterations in KRAS, TET2, EZH2, and DNMT3A.

In independent low frequency variants ( < 10% mutant allele frequency), 20 of 21 variants were confirmed from AmpliSeq assay and another hotspot clinical assay.

Combined DNA and RNA sequencing approach accurately detects a wide variety of genomic rearrangements and gene fusions with immediate clinical value in hematologic malignancies.

Sensitivity for fusion detection at 20% or greater tumor fraction was 100% (161/161) and 98% (84/86) at 10% tumor fraction.
Clinical experiences from 3,696 hematologic malignancies are summarized, with a high fraction of clinically relevant genomic alteration detected.

At least one driver alteration was identified in 95% tumor specimens, and 77% cases harbored at least one alteration linked to a commercially available targeted therapy or one that is in clinical development. In addition, 61% of cases harbored at least one alteration with known prognostic relevance in that tumor type.

Genomic rearrangements were detected from 37% of clinical hematologic malignancies; known and novel fusions in kinase drug targets are highlighted.

In 16 cases of high-risk, BCR-ABL-negative B-ALL malignancies, known and novel clinical relevant genomic alterations were detected by FoundationOne Heme, and gene fusions involving JAK2, CRLF2 and EPOR were detected in 9 of 16 cases.

Sophiris Bio Reports Fourth Quarter and Full Year 2015 Financial Results and Key Business Highlights

On March 23, 2016 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company" or "Sophiris"), a biopharmaceutical company developing topsalysin (PRX302) for the treatment of urological diseases, reported financial results for the three months and year ended December 31, 2015 (Press release, Sophiris Bio, MAR 23, 2016, View Source [SID:1234509857]).

"Compelling data have been announced from both topsalysin programs over the past few months. Topsalysin demonstrated promising therapeutic activity in four of the initial seven patients with localized prostate cancer – including one with a complete tumor ablation. This encouraging data follows the announcement in November of 2015 that topsalysin met the primary endpoint in a Phase 3 study in BPH," stated Randall Woods, president and CEO of Sophiris Bio. "We believe that these robust and consistent BPH results will help us secure the capital needed to advance the BPH program into the final clinical study required for marketing approval, in a non-dilutive manner such as through a development and/or commercial partner."

Liquidity:

At December 31, 2015, we had cash, cash equivalents and securities available-for-sale of $8.4 million and net working capital of $5.6 million. We expect that our cash, cash equivalents and securities available-for-sale as of December 31, 2015 will be sufficient to fund our operations through September 2016 assuming that we do not initiate any additional clinical development of topsalysin beyond our on-going Phase 2a proof of concept study in localized prostate cancer. We will need to obtain additional capital to fund a second Phase 3 clinical trial of topsalysin for the treatment of the symptoms of BPH and for any future clinical development of topsalysin for the treatment of localized prostate cancer beyond our ongoing Phase 2a proof of concept clinical trial and to fund our ongoing operations. As of December 31, 2015, the outstanding principal balance of our term loan was $5.3 million on which we make principal and interest payments monthly.

Business Highlights:

Topsalysin BPH Program:

On November 10, 2015, the Company announced final positive results from its Phase 3 "PLUS-1" study of topsalysin (PRX302) as a treatment for lower urinary tract symptoms of benign prostatic hyperplasia (BPH, enlarged prostate)
Topsalysin met the primary endpoint of the study, demonstrating a statistically significant improvement in International Prostate Symptom Score (IPSS) total score from baseline over 12 months compared to the vehicle-only control group, (p = 0.043).
The clinical relevance of the overall improvement in IPSS was assessed by an additional efficacy endpoint, the patient self-assessment of the impact of treatment on their quality of life. Patients reported a sustained improvement in their quality of life from Weeks 18 through 52, which was statistically significantly superior to patients treated with vehicle for every post-baseline visit beginning at week 18 (reaching p = 0.004).
Topsalysin continues to have an attractive safety profile, with no evidence of any treatment related sexual or cardiovascular side effects.
Topsalysin Localized Prostate Cancer Program:

As of December 31, 2015, a total of 18 patients with clinically significant, localized low to intermediate risk prostate cancer (prostate cancer that has not yet metastasized) had been enrolled in the ongoing Phase 2a proof of concept study.
On January 28, 2016, the Company announced that a review of the 6-month biopsy data from the first seven patients to complete the study showed that four patients experienced a response to treatment: One patient experienced complete ablation of the tumor where no evidence of the treated tumor remained on a targeted biopsy at 6 months; three patients experienced either a reduction in the maximum cancer core length or a reduction in Gleason pattern; three patients had no response to treatment.
The Company held a conference call to review clinical data with principal investigators from the BPH program and the prostate cancer program, which can be accessed at View Source
The Company expects to have final data on all patients by the end of second quarter of 2016.
Woods added, "Practicing urologists view topsalysin as a compelling alternative for their BPH patients who have discontinued their oral medications and are unwilling to take the leap to the more invasive surgical procedures. Given the convenience of this four minute administration of topsalysin in the urologist’s office, 12 months of benefit and a benign safety profile, we believe topsalysin has the potential to obtain a significant position within the BPH treatment paradigm."

Financial Results:

For the fourth quarter ended December 31, 2015

The Company reported a net loss of $2.5 million ($0.15 per share) for the three months ended December 31, 2015 compared to a net loss of $5.3 million ($0.31 per share) for the three months ended December 31, 2014.

Research and development expenses

Research and development expenses were $1.7 million for the three months ended December 31, 2015 compared to $4.1 million for the three months ended December 31, 2014. The decrease in research and development costs is attributable to decreases in the costs associated with the Company’s completed Phase 3 PLUS-1 clinical trial of topsalysin, costs associated with the manufacturing activities for topsalysin and personnel related costs. These decreases are partially offset by an increase in costs associated with the Phase 2a proof of concept clinical trial for localized low to intermediate risk prostate cancer which enrolled its first patient in the second quarter of 2015.

General and administrative expenses

General and administrative expenses were $0.7 million for the three months ended December 31, 2015 compared to $1.0 million for the three months ended December 31, 2014. The decrease is primarily due to a decrease in non-cash stock-based compensation expense, consulting, and personnel related costs.

For the year ended December 31, 2015

The Company reported a net loss of $14.2 million ($0.84 per share) for the year ended December 31, 2015 compared to a net loss of $30.7 million ($1.85 per share) for the year ended December 31, 2014.

Research and development expenses

Research and development expenses were $9.9 million for the year ended December 31, 2015 compared to $24.7 million for the year ended December 31, 2014. The decrease in research and development costs is attributable to decreases in the costs associated with the Company’s Phase 3 PLUS-1 clinical trial of topsalysin, costs associated with the manufacturing activities for topsalysin and personnel related costs. These decreases are partially offset by an increase in costs associated with the Phase 2a proof of concept clinical trial for localized low to intermediate risk prostate cancer.

General and administrative expenses

General and administrative expenses were $3.6 million for the year ended December 31, 2015 compared to $5.3 million for the year ended December 31, 2014. The decrease is primarily due to a decrease in non-cash stock based compensation expense and, to a lesser extent, decreases in legal, travel, professional, consulting, and personnel related costs.