On January 29, 2016 ImmunoGen, Inc. (Nasdaq: IMGN), a biotechnology company that develops targeted anticancer therapeutics using its proprietary ADC technology, reported financial results for the three-month period ended December 31, 2015 — the second quarter of the Company’s 2016 fiscal year (Filing, 8-K, ImmunoGen, JAN 29, 2016, View Source [SID:1234508907]). ImmunoGen also provided an update on product programs and reiterated its 2016 fiscal year guidance.

"ImmunoGen is off to a strong start for 2016, with multiple clinical trial initiations underway," commented Daniel Junius, President and CEO. "Of particular importance is the opening of our FORWARD I trial assessing mirvetuximab soravtansine as single-agent therapy for pretreated FRα-positive ovarian cancer, which we believe is the fastest path to registration for this promising ADC. We expect several presentations of mirvetuximab soravtansine data in 2016, including mature results from the 40-patient FRα-positive ovarian cancer Phase 1 cohort."

Mr. Junius continued, "Our partners also are making meaningful progress. Of particular note is Bayer’s initiation of a Phase 2 trial designed to support registration of its anetumab ravtansine product candidate. Roche expects data to be reported from its trial assessing Kadcyla in the neoadjuvant setting this year and — if positive — to bring these to regulatory authorities for potential filing in 2016. A third partner compound is on track to advance into registration testing later this year."

Update on Wholly Owned Product Programs

Mirvetuximab soravtansine — First FRα-targeting ADC is a potential new treatment for ovarian cancer and other FRα-positive solid tumors.

· Assessments as single-agent therapy for pretreated FRα-positive ovarian cancer:

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· Updated Phase 1 findings were presented at the AACR (Free AACR Whitepaper)-NCI-EORTC meeting in November for the dataset reported at ASCO (Free ASCO Whitepaper) in May (abstracts #C47 and #5518, respectively). These included that 35% (7/20) of patients with FRα-positive platinum-resistant disease treated had a confirmed objective response, with most (6/7) responders on mirvetuximab soravtansine for 6 months or longer. This compares with ImmunoGen’s target response rate of 30% or more to advance the ADC as monotherapy. Most of the patients and all of the responders had high or medium FRα levels on their tumors.

· Patient enrollment is open for the Company’s FORWARD I Phase 2 trial, which is designed to support an Accelerated Approval pathway for mirvetuximab soravtansine. FORWARD I is being conducted in partnership with the GOG Foundation, Inc. To qualify for enrollment, patients must have ovarian cancer with high or medium FRα expression that was previously treated with 3 or 4 regimens.

· Patient enrollment was completed in 4Q2015 in the 20-patient Phase 1 expansion cohort requiring biopsies. The Company intends to present initial biomarker data from this assessment at a medical meeting in 2Q2016 in addition to reporting mature data from the 40-patient Phase 1 cohort in this disease at the meeting.

· Assessments as combination therapy for FRα-positive ovarian cancer:

· Encouraging preclinical data with a range of combination regimens were presented at the AACR (Free AACR Whitepaper)-NCI-EORTC meeting (abstract C170).

· In December, patient dosing began in the Phase 1b/2 trial, FORWARD II, assessing mirvetuximab soravtansine in combination with approved anticancer agents.

· Assessments for the treatment of other FRα-positive cancers:

· In 4Q2015, patient enrollment was completed in the 20-patient Phase 1 expansion cohort assessing the ADC for FRα-positive relapsed/refractory endometrial cancer. The Company expects to report findings from this assessment in 2H2016.

· Additional cancer types are being evaluated for FRα expression preclinically.

IMGN529 and coltuximab ravtansine — CD37- and CD19-targeting, respectively, ADCs for diffuse large B-cell lymphoma (DLBCL) and potentially other B-cell malignancies.

· Preclinical findings of strong synergy for IMGN529 used in combination with rituximab were reported at ASH (Free ASH Whitepaper) (abstract #1548) in December.

· Patient enrollment in a Phase 2 trial assessing IMGN529 in combination with rituximab is expected to begin early this year. Enrollment in a Phase 2 trial assessing coltuximab ravtansine in a different combination regimen is expected to begin in 2H2016.

IMGN779 — First CD33-targeting ADC utilizing an IGN cancer-killing agent. IGNs are a

new class of DNA-acting agents invented by ImmunoGen.

· Mechanism of action data were reported at ASH (Free ASH Whitepaper) (abstract #1366).

· ImmunoGen is preparing to initiate Phase 1 testing of IMGN779 for the treatment of acute myeloid leukemia in 1H2016.

Update on Partner Programs

Nine companies are advancing ADCs with ImmunoGen technology. Recent highlights include:

· Patient dosing has begun in Bayer’s global Phase 2 clinical trial designed to support registration of its mesothelin-targeting ADC, anetumab ravtansine. This event triggers a milestone payment to ImmunoGen that will be reflected in the Company’s 3QFY2016 financial results.

· Roche expects data from its KRISTINE trial assessing Kadcyla in the neoadjuvant setting for early HER2-positive breast cancer to be reported this year and, if positive, to bring these to regulatory authorities for potential filing in 2016.

· In December, Takeda took its first license for the exclusive right to develop ADCs to an undisclosed target using ImmunoGen technology.

· Also in December, CytomX announced it is advancing a novel anticancer agent targeting CD166 using its ProbodyTM technology and ImmunoGen’s ADC technology under a strategic collaboration established between the companies in early 2014.

Financial Results

For the Company’s quarter ended December 31, 2015 (2QFY2016), ImmunoGen reported a net loss of $(33.2) million, or $(0.38) per basic and diluted share, compared to net income of $13.6 million, or $0.16 per basic and diluted share, for the same quarter last year (2QFY2015).

Revenues for 2QFY2016 were $18.0 million, compared to $48.3 million for 2QFY2015. The current period includes $8.6 million of amortization of upfront fees previously received from Takeda and the prior year period includes $41.4 million of amortization of upfront fees previously received from Novartis and Lilly. The fees are recognized in their respective quarters due to the partner taking one or more licenses in the quarter. License and milestone fees for 2QFY2016 also include a $2 million milestone earned from Sanofi with the advancement of SAR428926 into clinical testing. Revenues in 2QFY2016 include $6.3 million of non-cash royalty revenues and $0.2 million of cash royalty revenues on Roche sales of Kadcyla for the three-months ended September 30, 2015, compared with $4.6 million in cash royalty revenues for the prior year period.

Operating expenses in 2QFY2016 were $46.3 million, compared to $34.5 million in 2QFY2015. Operating expenses in 2QFY2016 include research and development expenses of $38.2 million, compared to $27.6 million in 2QFY2015. This change is primarily due to increased third-party costs related to the advancement of our wholly owned product candidates, increased clinical trial costs, primarily related to our expansion of the mirvetuximab soravtansine development program, and increased

personnel expenses, principally due to recent hiring. Operating expenses include general and administrative expenses of $8.1 million in 2QFY2016, compared to $6.9 million in 2QFY2015. This increase is primarily due to increased personnel expenses and professional services.

ImmunoGen had approximately $212.3 million in cash and cash equivalents as of December 31, 2015, compared with $278.1 million as of June 30, 2015, and had no debt outstanding in either period. Cash used in operations was $63.0 million in the first six months of FY2016, compared with $34.4 million in the same period in FY2015. Capital expenditures were $7.6 million and $2.6 million for the first six months of FY2016 and FY2015, respectively.

Financial Guidance for Fiscal Year 2016

ImmunoGen’s financial guidance remains unchanged from that issued in July 2015. ImmunoGen expects: its revenues to be between $70 million and $80 million; its operating expenses to be between $175 million and $180 million; its net loss to be between $120 million and $125 million; its cash used in operations to be between $100 million and $105 million; and its capital expenditures to be between $13 million and $15 million. Cash and cash equivalents at June 30, 2016 are anticipated to be between $165 million and $170 million.

On January 29, 2016 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations and a primary focus in Hematology and Oncology, reported the Company has initiated the planned registrational trial for SPI-2012 (eflapegrastim), its novel, long-acting G-CSF (Press release, Spectrum Pharmaceuticals, JAN 29, 2016, View Source [SID:1234508908]). This trial will evaluate the safety and efficacy of SPI-2012 as a treatment for chemotherapy-induced neutropenia in patients with breast cancer, and will serve as the basis for the Biologics License Application (BLA) filing.

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"The initiation of the registration trial for SPI-2012 is a significant milestone in the history of our company," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "Revenues from our current marketed drugs have helped us invest in this exciting technology that opens the door for us to a blockbuster oncology market. In parallel, Spectrum has built a strong commercial infrastructure with specialized expertise in this indication that positions us well to aggressively compete in this market."

"I am excited to be the lead investigator for this important study, and about the potency and safety of SPI-2012 as demonstrated in Phase 2," said Lee S. Schwartzberg, M.D., FACP Professor of Medicine and Division Chief, Hematology Oncology, The University of Tennessee Health Science Center, and Executive Director, UT/West Cancer Center. "The LAPSCOVERY technology confers long-acting properties and increased bone marrow uptake through decreased renal and vascular clearance, as well as Fc-mediated transport of G-CSF. We look forward to a successfully conducted Phase 3 trial of SPI-2012. I believe this novel biologic drug, if approved, would be a very valuable addition to our supportive care armamentarium for cancer patients receiving myelosuppressive cytotoxic chemotherapy."

"SPI-2012 is a third generation agent for the treatment of neutropenia that has shown promising results in Phase 2 trials," said, Jeffrey L. Vacirca, M.D., FACP CEO, Managing Partner & Chief of Clinical Research at North Shore Hematology/Oncology Associates and Vice-President, Community Oncology Alliance. "In the Phase 2 trial, the duration of severe neutropenia was equivalent to pegfilgrastim at the medium dose and superior at the high dose. No new or significant dose-related toxicities have been observed in over 230 patients who have been treated with SPI-2012, and the incidence of adverse events has been similar to pegfilgrastim."

In accordance with the SPA, this registrational, Phase 3 or ADVANCE study (RAnDomized Trial of SPI-2012 Versus Pegfilgrastim in the Management of Chemotherapy Induced Neutropenia in Breast CANCEr Patients Receiving Docetaxel and Cyclophosphamide) is a multicenter, randomized, active controlled trial that will enroll 580 newly diagnosed early-stage breast cancer patients, who will receive adjuvant or neoadjuvant chemotherapy every 21 days. Adjuvant chemotherapy is treatment given after primary surgical therapy to kill any remaining cancer cells and increase the chance of long-term disease-free survival; neoadjuvant chemotherapy is the administration of cytotoxic agents before surgical resection in early-stage breast cancer to shrink the tumor and potentially allow for breast-conserving surgery. SPI-2012 will be administered subcutaneously as a fixed dose equivalent to 3.6 mg of GCSF, which was selected based on the robust pharmacological and pharmacodynamic data from Phase 2. The primary study endpoint is the Duration of Severe Neutropenia (Absolute Neutrophil Counts [ANC] < 0.5×109/L) in Cycle 1 of chemotherapy, based on central laboratory assessment of ANC over the 21 day cycle. Secondary endpoints include the incidence of neutropenic complications, incidence of Febrile Neutropenia, Relative Dose Intensity, and safety.

About Special Protocol Assessments

A Special Protocol Assessment is a written agreement between a Sponsor and the U.S. Food and Drug Administration on the design, execution and analysis for a clinical trial that may form the basis of a new Biologics License Application or BLA. Final marketing approval depends upon the efficacy results, safety profile and an evaluation of the risk/benefit of treatment demonstrated in the Phase 3 clinical program.

About Breast Cancer

According to the American Cancer Society (ACS), breast cancer is the second most common form of cancer in women after skin cancer, and the second highest cause of female cancer deaths after lung cancer. Unfortunately, it is estimated that about 1 in 8 (12%) of women in the US will develop invasive breast cancer during their lifetime. In 2015 in the United States (US), an estimated 231,840 new cases of invasive breast cancer and 60,290 additional cases of in situ breast cancer will be diagnosed, and approximately 40,290 US women are expected to die from breast cancer. In addition, ~2,350 men are also expected to be diagnosed with breast cancer in 2015 with an estimated 440 deaths.

On January 28, 2016 Exelixis, Inc. (NASDAQ:EXEL) reported that the U.S. Food & Drug Administration (FDA) has determined the company’s New Drug Application (NDA) for cabozantinib as a treatment for patients with advanced renal cell carcinoma (RCC) who have received one prior therapy to be sufficiently complete to permit a substantive review (Press release, Exelixis, JAN 28, 2016, View Source;p=RssLanding&cat=news&id=2133086 [SID:1234508892]). The NDA will be considered officially filed 60 days from the date of the completion of the submission, or February 20, 2016. The FDA granted Priority Review to the filing and assigned a Prescription Drug User Fee Act action date of June 22, 2016.

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"With FDA granting Priority Review to our application, Exelixis is one step closer to offering physicians cabozantinib as an important new therapeutic option for their patients with advanced renal cell carcinoma," said Michael M. Morrissey, Ph.D., president and CEO of Exelixis. "While we work closely with the FDA during the review process, Exelixis will continue to execute on our commercial plans, including our commitment to be ready for a potential launch by April 1st of this year."

A Priority Review designation is granted by the FDA for drugs that, if approved, would be significant improvements in the treatment, prevention or diagnosis of a disease. Previously, the FDA granted cabozantinib Breakthrough Therapy designation (August 2015) and Fast Track designation (April 2015) for the compound’s proposed RCC indication.

The NDA is based on the results of METEOR, a phase 3 pivotal trial comparing cabozantinib to everolimus in patients with advanced RCC who experienced disease progression following treatment with a VEGF receptor tyrosine kinase inhibitor. In July 2015, Exelixis announced top-line results demonstrating that the trial had met its primary endpoint of improving progression-free survival; compared with everolimus, cabozantinib was associated with a 42% reduction in the rate of disease progression or death. These data were later presented at the European Cancer Congress in September 2015 and concurrently published in The New England Journal of Medicine.

On January 11, 2016, Exelixis announced the submission of a Marketing Authorization Application (MAA) for cabozantinib as a treatment for patients with advanced RCC who have received one prior therapy to the European Medicines Agency (EMA). The EMA’s Committee for Medicinal Products for Human Use (CHMP) previously granted accelerated assessment to cabozantinib for advanced RCC. As a result, the company’s MAA will be eligible for a 150-day review, versus the standard 210 days (excluding clock stops when information is requested by CHMP).

Cabozantinib is currently marketed in capsule form under the brand name COMETRIQ in the United States for the treatment of progressive, metastatic medullary thyroid cancer (MTC), and in the European Union for the treatment of adult patients with progressive, unresectable locally advanced or metastatic MTC. COMETRIQ is not indicated for patients with RCC. In the METEOR trial, and all other cancer trials currently underway, Exelixis is investigating a tablet formulation of cabozantinib distinct from the COMETRIQ capsule form. The tablet formulation of cabozantinib is the subject of the NDA for advanced RCC.

About Advanced Renal Cell Carcinoma

The American Cancer Society’s 2015 statistics cite kidney cancer as among the top ten most commonly diagnosed forms of cancer among both men and women in the U.S.1 Clear cell RCC is the most common type of kidney cancer in adults.2 If detected in its early stages, the five-year survival rate for RCC is high; however, the five-year survival rate for patients with advanced or late-stage metastatic RCC is under 10 percent, with no identified cure for the disease.3

Until the introduction of targeted therapies into the RCC setting a decade ago, treatments for metastatic RCC had historically been limited to cytokine therapy (e.g., interleukin-2 and interferon). In the second and later-line settings, which encompass approximately 17,000 drug-eligible patients in the U.S. and 37,000 globally,4 two small-molecule therapies and an immune checkpoint inhibitor have been approved for the treatment of patients who have received prior VEGF receptor TKIs. The currently approved small-molecule agents have shown little differentiation in terms of efficacy and have demonstrated only modest progression-free survival benefit in patients refractory to sunitinib, a commonly-used first-line therapy.

The majority of clear cell RCC tumors exhibit down-regulation of von Hippel-Lindau (VHL) protein function, either due to gene inactivation or epigenetic silencing, resulting in a stabilization of the hypoxia-inducible transcription factors (HIFs) and consequent up-regulation of VEGF, MET and AXL.5 The up-regulation of VEGF may contribute to the angiogenic nature of clear cell RCC, and expression of MET or AXL may be associated with tumor cell viability, a more invasive tumor phenotype and reduced overall survival. 6 Up-regulation of MET and AXL in clear cell RCC has also been shown to occur in response to treatment with VEGF receptor TKIs in preclinical models, indicating a potential role for MET and AXL in the development of resistance to these therapies.7

About Cabozantinib

Cabozantinib inhibits the activity of tyrosine kinases including MET, VEGF receptors, AXL and RET. These receptor tyrosine kinases are involved in both normal cellular function and in pathologic processes such as oncogenesis, metastasis, tumor angiogenesis and maintenance of the tumor microenvironment.

Cabozantinib, marketed under the brand name COMETRIQ, is currently approved by the U.S. Food and Drug Administration for the treatment of progressive, metastatic medullary thyroid cancer (MTC).

The European Commission granted COMETRIQ conditional approval for the treatment of adult patients with progressive, unresectable locally advanced or metastatic MTC. Similar to another drug approved in this setting, the approved indication states that for patients in whom Rearranged during Transfection (RET) mutation status is not known or is negative, a possible lower benefit should be taken into account before individual treatment decisions.

Important Safety Information, including Boxed WARNINGS

WARNING: PERFORATIONS AND FISTULAS, and HEMORRHAGE

Serious and sometimes fatal gastrointestinal perforations and fistulas occur in COMETRIQ-treated patients.

Severe and sometimes fatal hemorrhage occurs in COMETRIQ-treated patients.

COMETRIQ treatment results in an increase in thrombotic events, such as heart attacks.

Wound complications have been reported with COMETRIQ.

COMETRIQ treatment results in an increase in hypertension.

Osteonecrosis of the jaw has been observed in COMETRIQ-treated patients.

Palmar-Plantar Erythrodysesthesia Syndrome (PPES) occurs in patients treated with COMETRIQ.

The kidneys can be adversely affected by COMETRIQ. Proteinuria and nephrotic syndrome have been reported in patients receiving COMETRIQ.

Reversible Posterior Leukoencephalopathy Syndrome has been observed with COMETRIQ.

Avoid administration of COMETRIQ with agents that are strong CYP3A4 inducers or inhibitors.

COMETRIQ is not recommended for use in patients with moderate or severe hepatic impairment.

COMETRIQ can cause fetal harm when administered to a pregnant woman.

Adverse Reactions – The most commonly reported adverse drug reactions (≥25%) are diarrhea, stomatitis, palmar-plantar erythrodysesthesia syndrome (PPES), decreased weight, decreased appetite, nausea, fatigue, oral pain, hair color changes, dysgeusia, hypertension, abdominal pain, and constipation. The most common laboratory abnormalities (≥25%) are increased AST, increased ALT, lymphopenia, increased alkaline phosphatase, hypocalcemia, neutropenia, thrombocytopenia, hypophosphatemia, and hyperbilirubinemia.

Please see full U.S. prescribing information, including Boxed WARNINGS, at www.COMETRIQ.com/downloads/Cometriq_Full_Prescribing_Information.pdf

Please refer to the full European Summary of Product Characteristics for full European Union prescribing information, including contraindication, special warnings and precautions for use at www.sobi.com once posted.

On January 28, 2016 ArQule, Inc. (NASDAQ:ARQL) reported a pipeline update for its AKT inhibitors, including ARQ 092 and ARQ 751, both orally available, selective pan-AKT inhibitors (Press release, ArQule, JAN 28, 2016, View Source [SID:1234508895]).

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ARQ 092 – Lead AKT Inhibitor

The phase 1 trial for ARQ 092 in Proteus syndrome, which was opened for enrollment in November 2015, has completed dosing of its first cohort. The first cohort, consisting of three patients, is being observed for safety. The phase 1 trial is being conducted by our collaborators at the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH). Proteus syndrome is a rare disease that is characterized by overgrowth of the skeleton, skin, adipose tissue and central nervous system. ARQ 092 was recently granted orphan drug designation by the Food and Drug Administration (FDA) in this indication which impacts less than one in a million people worldwide.

Recently, a manuscript was published by the NHGRI of the NIH in Scientific Reports (available on-line at View Source) discussing pre-clinical research with ARQ 092 in Proteus syndrome. This pre-clinical study tested the efficacy of ARQ 092 in suppressing AKT signaling in cells and tissues from patients with Proteus syndrome. Reduced phosphorylation of AKT and downstream targets of AKT in a concentration-dependent manner were observed in as little as two hours without reduction in cell viability.

ArQule continues to enroll patients harboring AKT1 or PI3K mutations in the company sponsored phase 1b expansion cohort of its oncology trial. Cohorts for lymphoma and endometrial are fully enrolled. Thus far the company has observed five partial responses in the phase 1b portion of the trial, three of which occurred in patients whose tumors have AKT1 or PI3K mutations and two of which occurred in patients where the mutational status is unknown.

ARQ 751 – Next Generation AKT Inhibitor

ArQule received FDA approval of its investigational new drug (IND) application for ARQ 751, a next generation AKT inhibitor, in oncology. As published in PLOS ONE, (available on-line at View Source), ARQ 751 has demonstrated signal abrogation and efficacy in pre-clinical in vitro and in vivo models harboring AKT1 and PI3K mutations. The company expects to commence a phase 1 trial in oncology during the first half of 2016 targeting AKT1 and PI3K mutations.

"We are pleased with the two recent publications on our AKT program, and we look forward to further advancing our AKT pipeline in 2016 with ARQ 092 and our next generation AKT inhibitor, ARQ 751," said Brian Schwartz, Chief Medical Officer and Head of Research and Development at ArQule. "The understanding accumulated by us and our collaborators on our AKT program in oncology and rare diseases positions us for leadership in this class. We plan to explore opportunities to advance our trials in oncology as well as in rare over-growth indications beyond Proteus syndrome."

"2015 was an exciting year for ArQule as we were able to bring forward clinical results from our proprietary pipeline which now includes two orphan drug designations from the FDA," said Paolo Pucci, Chief Executive Officer at ArQule. "Looking ahead into 2016, we expect the interim analysis for the tivantinib phase 3 METIV-HCC trial to occur early in the second quarter, and we also expect to be able to make decisions on next steps in clinical development for ARQ 092 in oncology and Proteus syndrome, as well as for ARQ 087 in intrahepatic cholangiocarcinoma (iCCA)."

About Proteus Syndrome

According to the patient advocacy and support group, the Proteus syndrome Foundation (View Source), the condition was named for Proteus, the Greek god who could transform his shape. Patients experience changes in the shapes of certain body structures over time, including abnormal, often asymmetric, massive growth (overgrowth) of the skeleton, skin, adipose tissue and central nervous system out of proportion to the rest of the body, which may appear normal. Although patients may have minimal or no manifestations at birth, the disease develops and becomes apparent in early childhood (6-18 months) and rapidly progresses with intense growth in the first ten years of life. It is primarily a childhood-onset disease but there are very few living affected adults.

Proteus syndrome is a rare condition with an incidence of less than 1 in 1 million people worldwide. Only a few hundred individuals have been reported in the medical literature. At this time, there are more than 120 documented cases worldwide, but because not all cases are documented, it is not known how many individuals have this syndrome. The incidence of Proteus syndrome classifies it as a rare disorder, defined by the National Organization of Rare Diseases (NORD) as any disease affecting fewer than 200,000 Americans.

About the AKT Pathway, ARQ 092 and ARQ 751

ARQ 092 and ARQ 751 are orally bioavailable, selective small molecule inhibitors of the AKT kinases. The AKT pathway when abnormally activated is implicated in multiple oncogenic processes such as cell proliferation and apoptosis. This pathway has emerged as a target of potential therapeutic relevance for compounds that inhibit its activity, which has been linked to a variety of cancers as well as to select non-oncology indications.

ARQ 092, the lead compound in ArQule’s AKT program, has completed phase 1a clinical testing and has advanced into phase 1b expansion testing in cohorts of patients with endometrial cancer, lymphoma and tumors harboring either AKT or PI3K mutations. A number of next-generation compounds in the Company’s AKT program are in early to late stages of pre-clinical development. The company plans to initiate a phase 1 clinical trial in the first half of 2016 for ARQ 751, a next generation AKT inhibitor.

On January 28, 2016 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company" or "Sophiris"), a biopharmaceutical company developing PRX302 (topsalysin) for the treatment of urological diseases, reported the biopsy data at 6 months for the first seven patients to complete the Phase 2a proof-of-concept study in localized prostate cancer (Press release, Sophiris Bio, JAN 28, 2016, View Source [SID:1234508896]).

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A review of the biopsy data from the first seven men 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.

"This new trial is very exciting — we have promising data showing that topsalysin can ablate cancer cells and we look forward to reviewing the results from the remaining 11 patients as they complete the study. We could be on the cusp of a new class of therapeutics for the focal treatment of localized prostate cancer," stated Professor Mark Emberton, Dean, Faculty of Medical Sciences, University College London and Honorary Consultant Urologist University College London Hospital NHS Foundation Trust.

Dr. Hashim Ahmed, Principal Investigator for the study, Division of Surgery and Interventional Sciences, University College London, said, "Topsalysin could offer a tissue-sparing cancer treatment that carries little in the way of side effects. This treatment has the potential to help men avoid radical treatments such as radiation therapy or complete removal of the prostate."

This one-time administration of topsalysin directly into a pre-identified clinically significant tumor appears to be well tolerated with no serious adverse events and no new safety signals being reported. This is consistent with safety observed in the 365 patients that have been treated with topsalysin in the Company’s BPH program to date.

"The biological activity that we have observed further validates the mechanism of action of topsalysin. We are gaining valuable experience on how we might best optimize both the delivery and dose of topsalysin based on lesion size, and the remaining patients to complete the study will help in that assessment," said Dr. Ahmed.

The ongoing Phase 2a proof of concept study is a single-center, open-label study at University College London, which is well known for the focal treatment of prostate cancer in the UK. In this study, previously obtained multiparametric magnetic resonance images (mpMRIs) of each patient’s prostate tumor lesions are mapped to real-time three-dimensional transrectal ultrasound. These images are used to guide the injection of topsalysin to treat a single, histologically-proven, clinically significant prostate cancer lesion. The primary objective of the study is safety and tolerability, and the key efficacy variable is the change in the treated lesion on targeted biopsy after 6 months. The study is designed to assess whether topsalysin has the potential to provide patients with clinically significant, localized, low to intermediate risk prostate cancer a tissue-sparing cancer treatment that carries little in the way of side effects. A total of 18 patients were enrolled and treated in this study. Sophiris expects to have final data on all patients by the end of the second quarter of 2016.

Webcast scheduled for today at 11:00 a.m. Pacific Time
The Sophiris management team will host a conference call and webcast today, January 28, at 11:00 a.m. Pacific Time to review the topsalysin prostate cancer data as well as the previously announced results of the topsalysin Phase 3 study in BPH. Dr. Hashim Ahmed, University College London and an investigator in the prostate cancer study, and Dr. Marc Gittelman, Director of South Florida Medical Research and investigator in the Phase 3 BPH study, will also participate in the call.

A live audio webcast will be accessible on the "Investor Relations" page of the Sophiris corporate website at www.SophirisBio.com. A replay will be available at the same location for 60 days.

About Prostate Cancer
Prostate cancer is the second most common form of cancer in men in the US with an estimated 220,800 new cases in 2015. Approximately 80 percent of patients in the US are diagnosed with localized disease. Research has shown that patients with early, localized disease have a low likelihood of the cancer spreading beyond the confines of the prostate; however, many men with clinically significant localized disease do choose to undergo radical treatment. Radical therapies include surgery to remove the entire prostate and/or radiation. Potential toxicities from radical treatments can be significant and permanent and include erectile dysfunction, urinary incontinence, and rectal toxicity.

Topsalysin for the Targeted Treatment of Localized Prostate Cancer
Topsalysin (PRX302) has the potential to provide a focal targeted therapy for the ablation of localized prostate cancer while potentially avoiding many of the complications and side effects associated with whole gland radical treatments. The increasing use of multi-parametric magnetic resonance imaging (mpMRI) and advances in mapping previously obtained mpMRI images with real-time three-dimensional ultrasound images enables physicians to more accurately locate tumors within the prostate when taking biopsies. This increases the accuracy with which men with clinically significant lesions are identified. It also enables the injection of an ablative agent, such as topsalysin, directly into previously identified clinically significant tumors located within the prostate. Topsalysin, an inactivated pore-forming protein, was engineered to be activated only by enzymatically-active PSA, which is present only in prostate tissue. The targeted focal treatment of prostate cancer is in line with current treatments for solid tumors such as breast and liver, where the goal is to remove the tumor and preserve as much of the organ as possible