On June 25, 2018 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported that it has completed submission of a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for ELZONRISTM (tagraxofusp; SL-401), a potential treatment for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a CD123+ malignancy of unmet medical need for which the agent was awarded Breakthrough Therapy Designation (BTD) (Press release, Stemline Therapeutics, JUN 25, 2018, View Source [SID1234532234]).

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Survival probability in first-line BPDCN patients who received ELZONRIS (12mcg/kg/day) in Stages 1, 2 and 3

Ivan Bergstein, M.D., Stemline’s CEO, commented, "The completion of our rolling BLA submission is a major milestone for Stemline and the overall BPDCN patient community. We want to recognize the hard work of our dedicated investigators as well as the entire Stemline team in completing this submission. We also want to especially thank all of the patients and their families who participated in the clinical development program. We are committed to bringing this promising agent to BPDCN patients as rapidly as possible."

BPDCN Efficacy – Stages 1, 2, and 3, ELZONRIS (12mcg/kg/day) (n=42)
The trial of investigational agent, ELZONRIS, in patients with BPDCN was comprised of 3 stages, with Stage 3 serving as the pivotal cohort for confirmation of efficacy. To ensure ongoing access to ELZONRIS, patients with BPDCN are being enrolled in an additional cohort, Stage 4. Stage 3 met its primary endpoint with a CR+CRc (complete response + clinical complete response) rate of 54% (95% CI: 25.1, 80.8). A summary of efficacy results is shown below.

Summary table: Efficacy of ELZONRIS (12mcg/kg/day) in patients with BPDCN (Stages 1, 2 and 3 [n=42])

Line of Therapy First-line Relapsed / Refractory
n 29 13
ORR, n (%) 26 (90%) 9 (69%)
CR+CRc+CRi, n (%) 21 (72%) 5 (38%)
CR 12 1
CRc 7 3
CRi 2 1
PR, n (%) 5 (17%) 4 (31%)
Bridged to SCT, n (%) 13 (45%) 1 (8%)
Abbreviations: ORR=overall response rate; CR=complete response; CRc=clinical CR (CR with minimal residual skin abnormality); CRi=CR with incomplete hematologic recovery; PR=partial response; SCT=stem cell transplant.

Overall Safety
148 patients received ELZONRIS (12mcg/kg/day) across all Stemline-sponsored trials, including in BPDCN, myeloproliferative neoplasms, and acute myeloid leukemia. A summary of safety results is shown below.

Summary table: Safety and tolerability of ELZONRIS (12mcg/kg/day) in all Stemline-sponsored clinical trials (n=148)

Most Common Adverse Events (AEs) (>15% treatment-related AEs, TRAEs)
Preferred Term All Grades, n (%) TRAEs, n (%)
TRAEs All AEs Gr 1-2 Gr 3 Gr 4 Gr 5
ALT increased 65 (43.9%) 80 (54.1%) 31 (20.9%) 34 (23.0%) 0 (0.0%) 0 (0.0%)
AST increased 65 (43.9%) 74 (50.0%) 30 (20.3%) 31 (20.9%) 4 (2.7%) 0 (0.0%)
Hypoalbuminaemia 65 (43.9%) 73 (49.3%) 64 (43.2%) 1 (0.7%) 0 (0.0%) 0 (0.0%)
Thrombocytopenia 39 (26.4%) 48 (32.4%) 7 (4.7%) 8 (5.4%) 24 (16.2%) 0 (0.0%)
Nausea 38 (25.7%) 70 (47.3%) 37 (25.0%) 1 (0.7%) 0 (0.0%) 0 (0.0%)
Pyrexia 33 (22.3%) 60 (40.5%) 33 (22.3%) 0 (0.0%) 0 (0.0%) 0 (0.0%)
Fatigue 30 (20.3%) 67 (45.3%) 26 (17.6%) 4 (2.7%) 0 (0.0%) 0 (0.0%)
Weight increased 28 (18.9%) 42 (28.4%) 28 (18.9%) 0 (0.0%) 0 (0.0%) 0 (0.0%)
Chills 26 (17.6%) 40 (27.0%) 25 (16.9%) 1 (0.7%) 0 (0.0%) 0 (0.0%)
Capillary leak syndrome (CLS)a 25 (16.9%) 25 (16.9%) 16 (10.8%) 5 (3.4%) 3 (2.0%) 1 (0.7%)
Hypotension 23 (15.5%) 36 (24.3%) 17 (11.5%) 5 (3.4%) 1 (0.7%) 0 (0.0%)
Oedema peripheral 22 (14.9%) 57 (38.5%) 21 (14.2%) 1 (0.7%) 0 (0.0%) 0 (0.0%)
a0.7% (1/148) for all trials (12mcg/kg/day) and 1.6% (3/182) for all trials (all doses) were grade 5. A myocardial infarction, grade 5, was also reported in a patient who experienced a grade 4 CLS.

Overall Survival (OS)
In first-line BPDCN patients who received ELZONRIS (12mcg/kg/day) in Stages 1, 2 and 3, the median OS has not been reached. Median follow up was 13.8 months (range: 0.2-37.4+ months).

About ELZONRISTM (tagraxofusp; SL-401)
ELZONRISTM (tagraxofusp; SL-401) is a novel targeted investigational therapy directed to CD123, a cell surface receptor expressed on a range of malignancies. ELZONRIS has successfully completed a pivotal trial in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an indication for which it was granted Breakthrough Therapy Designation (BTD). A rolling Biologics License Application (BLA) submission has been completed. ELZONRIS is also being evaluated in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), acute myeloid leukemia (AML), and myeloma.

On June 25, 2018 AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, and Calibr, a nonprofit drug discovery division of Scripps Research, reported a collaboration to develop T-cell therapies aimed primarily at cancer, including solid tumors (Press release, AbbVie, JUN 25, 2018, View Source [SID1234527457]).

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This collaboration broadens AbbVie’s oncology research to access advanced precision medicine technology to expand the development of potentially life-changing treatments for patients with cancer.

Chimeric Antigen Receptor T-cell (CAR-T) therapies harness the power of a cancer patient’s own immune system to attack and destroy cancer cells. Despite promising results in hematological malignancies, current CAR-T therapies in development for solid tumors have demonstrated limitations due to rapid activation and expansion of CAR-T cells that can lead to serious adverse events. Calibr’s novel cell therapy program, led by Travis Young, Ph.D., director of protein sciences at Calibr, is designed to enhance safety, versatility and efficacy through a proprietary modular "switchable" CAR-T cell that uses antibody-based switch molecules to control the activation and antigen specificity of CAR-T cells.1 Calibr’s proprietary technology may enable the development of universal CAR-T-based treatments across several types of hematological and solid tumor indications.

"Calibr has assembled a premier scientific team and developed an innovative cell therapy technology that can take us to the next frontier of cancer treatment," says Mohit Trikha, Ph.D., vice president and head of Oncology Early Development at AbbVie. "The combination of AbbVie’s oncology discovery and early development expertise and Calibr’s novel switchable CAR-T therapy platform aims to advance the current standard of care, with the potential rapidly advancing new treatment options for patients."

"We’re delighted to work together with a strong partner like AbbVie to expand the impact of the CAR-T cell field to a broader range of cancers," says Peter Schultz, Ph.D., chief executive officer of Calibr and Scripps Research.

Under the terms of the license agreement, AbbVie will pay Calibr an upfront license fee and gain exclusive access to Calibr’s switchable CAR-T platform for a term of up to four years. The two organizations will work together to develop T-cell therapies directed to solid tumor targets identified by AbbVie. AbbVie also has the option to develop additional cell therapies toward AbbVie-nominated targets and license existing Calibr cell therapy programs under development for hematological and solid cancers, including Calibr’s lead program. Calibr plans to enter this lead candidate into clinical studies for lymphoma in 2019. In addition, the agreement provides AbbVie with an option to acquire an exclusive license to Calibr’s switchable CAR-T platform and programs within the first four years of the collaboration. The companies will share responsibility for preclinical development, with AbbVie responsible for clinical development and commercialization, and Calibr eligible to receive success-based milestone payments and royalties. The transaction is subject to clearance under the Hart-Scott-Rodino Antitrust Improvements Act.

On June 25, 2018 Pfizer reported overall survival (OS) results from the Phase 3 PALOMA-3 trial, which evaluated IBRANCE (palbociclib) in combination with fulvestrant compared to placebo plus fulvestrant in women with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer whose disease has progressed after prior endocrine therapy (Press release, Pfizer, JUN 25, 2018, View Source [SID1234527458]). The results demonstrated a positive trend in the hazard ratio favoring the IBRANCE combination, although this trend did not reach statistical significance. Overall survival is a secondary endpoint of the PALOMA-3 trial and, as such, the trial design was not optimized to detect a statistically significant difference in OS.

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"While the difference in overall survival narrowly missed the threshold for statistical significance – a high bar for any trial in this patient population – it is similar, in absolute terms, to the improvement in median progression-free survival previously demonstrated in this trial.1 We are encouraged by these results, which build on the compelling clinical benefit delivered by IBRANCE," said Mace Rothenberg, M.D., chief development officer, Oncology, Pfizer Global Product Development. "IBRANCE in combination with endocrine therapy has transformed the treatment landscape for patients with HR+, HER2- metastatic breast cancer."

PALOMA-3 met its primary endpoint of progression-free survival (PFS) at interim analysis and results were published in The New England Journal of Medicine in June 2015; updated PFS data were later presented at the 2016 San Antonio Breast Cancer Symposium. The trial demonstrated a statistically significant and clinically meaningful improvement in PFS for IBRANCE plus fulvestrant compared to placebo plus fulvestrant. PFS is a well-established measure of clinical benefit in metastatic breast cancer trials.2 IBRANCE in combination with fulvestrant has been approved in more than 80 countries around the world based on the PFS demonstrated in PALOMA-3.

"The duration of the survival in hormone receptor-positive metastatic breast cancer patients, and the potential for subsequent therapies to confound overall survival outcomes, make demonstrating statistically significant improvement in overall survival extremely difficult," said Nicholas Turner, M.D., Ph.D., professor of molecular oncology at The Institute of Cancer Research, London, and consultant medical oncologist at The Royal Marsden NHS Foundation Trust, as well as principal investigator of the PALOMA-3 trial. "The results from this overall survival analysis support the strong progression-free survival results from PALOMA-3 and, while not statistically significant, are encouraging for physicians and patients. We look forward to presenting the detailed data at an upcoming medical meeting."

The most common adverse reactions in PALOMA-3 included neutropenia, leukopenia, infections, fatigue and nausea; no new safety signals were identified as part of this final OS analysis.

IBRANCE in combination with endocrine therapy is a standard of care for HR+, HER2- metastatic breast cancer. IBRANCE has been prescribed to more than 120,000 patients globally to date.

The full prescribing information for IBRANCE can be found at www.pfizer.com.

About IBRANCE (palbociclib) 125 mg capsules

IBRANCE is an oral inhibitor of CDKs 4 and 6,3 which are key regulators of the cell cycle that trigger cellular progression.4,5 In the U.S., IBRANCE is indicated for the treatment of HR+, HER2- advanced or metastatic breast cancer in combination with an aromatase inhibitor as initial endocrine based therapy in postmenopausal women, or fulvestrant in women with disease progression following endocrine therapy.

IMPORTANT IBRANCE (palbociclib) SAFETY INFORMATION FROM THE U.S. PRESCRIBING INFORMATION

Neutropenia was the most frequently reported adverse reaction in PALOMA-2 (80%) and PALOMA-3 (83%). In PALOMA-2, Grade 3 (56%) or 4 (10%) decreased neutrophil counts were reported in patients receiving IBRANCE plus letrozole. In PALOMA-3, Grade 3 (55%) or Grade 4 (11%) decreased neutrophil counts were reported in patients receiving IBRANCE plus fulvestrant. Febrile neutropenia has been reported in 1.8% of patients exposed to IBRANCE across PALOMA-2 and PALOMA-3. One death due to neutropenic sepsis was observed in PALOMA-3. Inform patients to promptly report any fever.

Monitor complete blood count prior to starting IBRANCE, at the beginning of each cycle, on Day 15 of first 2 cycles and as clinically indicated. Dose interruption, dose reduction, or delay in starting treatment cycles is recommended for patients who develop Grade 3 or 4 neutropenia.

Based on the mechanism of action, IBRANCE can cause fetal harm. Advise females of reproductive potential to use effective contraception during IBRANCE treatment and for at least 3 weeks after the last dose. IBRANCE may impair fertility in males and has the potential to cause genotoxicity. Advise male patients with female partners of reproductive potential to use effective contraception during IBRANCE treatment and for 3 months after the last dose. Advise females to inform their healthcare provider of a known or suspected pregnancy. Advise women not to breastfeed during IBRANCE treatment and for 3 weeks after the last dose because of the potential for serious adverse reactions in nursing infants.

The most common adverse reactions (≥10%) of any grade reported in PALOMA-2 for IBRANCE plus letrozole vs placebo plus letrozole were neutropenia (80% vs 6%), infections (60% vs 42%), leukopenia (39% vs 2%), fatigue (37% vs 28%), nausea (35% vs 26%), alopecia (33% vs 16%), stomatitis (30% vs 14%), diarrhea (26% vs 19%), anemia (24% vs 9%), rash (18% vs 12%), asthenia (17% vs 12%), thrombocytopenia (16% vs 1%), vomiting (16% vs 17%), decreased appetite (15% vs 9%), dry skin (12% vs 6%), pyrexia (12% vs 9%), and dysgeusia (10% vs 5%).

The most frequently reported Grade ≥3 adverse reactions (≥5%) in PALOMA-2 for IBRANCE plus letrozole vs placebo plus letrozole were neutropenia (66% vs 2%), leukopenia (25% vs 0%), infections (7% vs 3%), and anemia (5% vs 2%).

Lab abnormalities of any grade occurring in PALOMA-2 for IBRANCE plus letrozole vs placebo plus letrozole were decreased WBC (97% vs 25%), decreased neutrophils (95% vs 20%), anemia (78% vs 42%), decreased platelets (63% vs 14%), increased aspartate aminotransferase (52% vs 34%), and increased alanine aminotransferase (43% vs 30%).

The most common adverse reactions (≥10%) of any grade reported in PALOMA-3 for IBRANCE plus fulvestrant vs placebo plus fulvestrant were neutropenia (83% vs 4%), leukopenia (53% vs 5%), infections (47% vs 31%), fatigue (41% vs 29%), nausea (34% vs 28%), anemia (30% vs 13%), stomatitis (28% vs 13%), diarrhea (24% vs 19%), thrombocytopenia (23% vs 0%), vomiting (19% vs 15%), alopecia (18% vs 6%), rash (17% vs 6%), decreased appetite (16% vs 8%), and pyrexia (13% vs 5%).

The most frequently reported Grade ≥3 adverse reactions (≥5%) in PALOMA-3 for IBRANCE plus fulvestrant vs placebo plus fulvestrant were neutropenia (66% vs 1%) and leukopenia (31% vs 2%).

Lab abnormalities of any grade occurring in PALOMA-3 for IBRANCE plus fulvestrant vs placebo plus fulvestrant were decreased WBC (99% vs 26%), decreased neutrophils (96% vs 14%), anemia (78% vs 40%), decreased platelets (62% vs 10%), increased aspartate aminotransferase (43% vs 48%), and increased alanine aminotransferase (36% vs 34%).

Avoid concurrent use of strong CYP3A inhibitors. If patients must be administered a strong CYP3A inhibitor, reduce the IBRANCE dose to 75 mg. If the strong inhibitor is discontinued, increase the IBRANCE dose (after 3-5 half-lives of the inhibitor) to the dose used prior to the initiation of the strong CYP3A inhibitor. Grapefruit or grapefruit juice may increase plasma concentrations of IBRANCE and should be avoided. Avoid concomitant use of strong CYP3A inducers. The dose of sensitive CYP3A substrates with a narrow therapeutic index may need to be reduced as IBRANCE may increase their exposure.

For patients with severe hepatic impairment (Child-Pugh class C), the recommended dose of IBRANCE is 75 mg. The pharmacokinetics of IBRANCE have not been studied in patients requiring hemodialysis.

On June 25, 2018 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, reported the company will hold a conference call on Friday, June 29th at 8:30 a.m. EDT/5:30 a.m. PDT to discuss the ROLONTIS Phase 3 ADVANCE study data being presented at Multinational Association of Supportive Care in Cancer (MASCC) 2018 annual meeting (Press release, Spectrum Pharmaceuticals, JUN 25, 2018, http://investor.sppirx.com/news-releases/news-release-details/spectrum-pharmaceuticals-host-conference-call-highlighting [SID1234527459]). The call will feature a presentation from ADVANCE study lead investigator, 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.

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Conference Call Details

Friday, June 29, 2018 @ 8:30 a.m. Eastern/5:30 a.m. Pacific

Domestic: (877) 837-3910, Conference ID# 6798817

International: (973) 796-5077, Conference ID# 6798817

The conference call will also be webcast live. To access the webcast and additional documents related to the call, please visit the Investor Relations page of the Spectrum Pharmaceutical website at View Source

For interested individuals unable to join the call, a webcast replay will be available online from June 29, 2018, @ 11:30 a.m. ET/8:30 a.m. PT through July 6, 2018 until 11:30 a.m. ET/8:30 a.m. PT.

Domestic Replay Dial-In #: (855) 859-2056, Conference ID# 6798817

International Replay Dial-In #: (404) 537-3406, Conference ID# 6798817

On June 25, 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 top-line interim safety and biopsy data following a single administration of topsalysin from its ongoing open-label, Phase 2b clinical trial (Press release, Sophiris Bio, JUN 25, 2018, View Source [SID1234527461]). A single administration of topsalysin continues to demonstrate an ability to ablate targeted prostate cancer cells with 10 of 35 patients (29%) demonstrating a clinical response of which 6 patients had a complete ablation with no detectable cancer on targeted biopsy of the treated area. Separately, Sophiris was recently notified that a patient death occurred on the same day as their second administration. The company is currently investigating the cause and as a precaution no additional patients will receive a second administration of topsalysin.

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"We are extremely saddened by the death of a patient after receiving a second administration of topsalysin," said Randall E. Woods, president and CEO of Sophiris. "Understanding the cause of the patient’s death is our first priority and essential to determining the potential for re-administration of topsalysin in future clinical trials."

To date, over 450 patients have received a single administration of topsalysin at various doses. The drug continues to appear to be well-tolerated in patients who received a single administration, with no new safety signals reported. In addition, biopsy data from the Phase 2b trial demonstrated that 29% (10/35) of patients sustained a clinical response at six-month follow-up – defined as no detectable tumor following targeted biopsy of the treated lesion or a reduction in the tumor to clinically insignificant.

"We are very encouraged by the safety and biopsy results from a single administration of topsalysin in the Phase 2b study. Biopsy results improved from what we saw in the Phase 2a proof of concept trial and safety and tolerability remains in-line with what we have seen historically," stated Mr. Woods. "We believe that the safety and biopsy data from the first administration of topsalysin supports moving forward into potential registration studies. We will continue to evaluate whether future clinical development will include an option to administer a second dose as we receive more information about the patient death and additional information from the 10 patients who received a second dose. We will be able to evaluate this towards the end of this year."

Top-Line Interim Safety Results from a Single Administration of Topsalysin:

The primary objective of this trial is to evaluate the safety and tolerability of a single, and if applicable, a second administration of topsalysin, when used to focally ablate a histologically-proven, clinically-significant lesion in patients with localized prostate cancer.

To date, a single administration of topsalysin continues to appear safe and well tolerated by patients. No hypersensitivity reactions or other serious systemic reactions to study medication were observed after a single administration. Adverse events considered related to topsalysin and occurring in more than one patient were: dysuria (n=3 patients), urinary retention (n=3 patients), nocturia (n=2 patients), micturition urgency (n=2 patients) and strangury (n=2 patients). All adverse events were considered mild and typically resolved within the same day. One event of micturition urgency was considered severe and resolved the same day and one event of urinary retention was considered moderate and the event was considered resolved after the patient underwent a transuretheral resection of the prostate.

In May 2018, an independent data monitoring committee (IDMC) met to review the safety data from all 38 patients administered a single dose of topsalysin as well the safety data available on the first seven patients who received a second administration of topsalysin. At that time, the IDMC unanimously recommended the clinical trial continue without changes to the protocol.

Top-Line Interim Biopsy Results From a Single Administration of Topsalysin:

A secondary objective of the study is to evaluate the efficacy of a single administration of topsalysin and, if applicable, a second administration of topsalysin to selectively target and focally ablate a pre-identified lesion.

In the Phase 2b clinical trial, 38 patients with pre-identified, clinically-significant low-to-intermediate risk localized prostate cancer received a single administration of topsalysin. Six months after administration, patients received a follow-up targeted biopsy of the treated lesion. At the time of this release, targeted follow-up biopsies have been undertaken and evaluated from 35 of 38 patients treated with a single dose of topsalysin. Two of the remaining patients are expected to receive follow-up biopsies in the coming weeks.

Based on the six-month follow-up biopsy results, 29% of patients (10/35) demonstrated a clinical response, defined in this study as no detectable tumor on targeted biopsy of the treated lesion or a sufficient reduction to deem the lesion clinically-insignificant (cancer lesion of Gleason Score 6 (3+3) and a Maximum Cancer Core Length (MCCL) of less than 6 millimeters). This compares favorably to 17% of patients (3/18) moving to clinically-insignificant disease in the previously completed Phase 2a localized prostate cancer study. Of the 10 clinical responders in the Phase 2b trial, six experienced a complete ablation with no histological evidence of the tumor remaining.

Additionally, the Phase 2b single administration follow-up biopsy data show that:

37% of patients (13/35) experienced a partial response, defined as a reduction in MCCL and/or Gleason pattern, but the targeted lesion was still deemed clinically-significant.
34% (12/35) of patients did not respond to treatment defined as no change in the targeted lesion or an increase in MCCL and/or Gleason pattern
"The initial biopsy results released today, following a single intraprostatic administration of topsalysin are highly encouraging and definitely improve upon the Proof of Concept study results with a greater proportion of patients experiencing successful treatment of their treated lesion," stated Dr. Hashim Ahmed, the study’s chief investigator and chair of urology & consultant urological surgeon, Imperial College Healthcare NHS Trust & professor urology Imperial College. "Importantly, we have shown that the targeted intraprostatic injection of a single administration of topsalysin continues to appear safe and well tolerated. Furthermore, targeted focal therapy with topsalysin in this patient population is transferable to other clinicians with at least half of the clinical sites observing patients with no detectable lesion on re-biopsy of the targeted tumor and all eight sites observing patients in which lesions were at least partially ablated."

"Advances in the imaging of the prostate – by virtue of MRI – and the precise risk-stratification that this now permits has opened up new therapeutic opportunities for men with low-intermediate risk prostate cancer," stated Professor Mark Emberton, Dean University College London Faculty of Medical Sciences. "Men truly welcome the opportunity to undergo a targeted treatment of their prostate cancer (often in an ambulatory setting) without exposure to the commonly occurring side-effects of urinary incontinence, sexual dysfunction and rectal symptoms that have tended to accompany the more traditional approaches of surgery and radiotherapy. Topsalysin is beginning to show the spectrum of attributes we would want to see in a prostate cancer treatment of the future."

Administration of a Second Topsalysin Dose:

The Phase 2b prostate cancer study represents the first trial designed to allow qualified patients to receive a second administration of topsalysin six months after initial treatment. To be eligible to receive a second administration, patients could not have experienced a clinically-significant adverse event attributable to either topsalysin or the dosing procedure. Additionally, patients must have demonstrated evidence of a response to treatment with topsalysin, either through a reduction in lesion size, Gleason pattern, or MCCL. The objective of re-administering topsalysin is to determine if additional clinical benefit is observed.

Eleven patients elected to receive a second dose of topsalysin. The patients will continue to be monitored per the trial’s protocol and data are expected to be available in the fourth quarter of 2018.

Webcast scheduled for today at 11:00 a.m. Eastern Time

The Sophiris management team will host a conference call and webcast today, June 25, at 11:00 a.m. Eastern Time to review the topsalysin prostate cancer data. Dr. Hashim Ahmed, Chair of Urology, Imperial College of London & Imperial College Healthcare NHS Trust and investigator for the Phase 2b clinical trial 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.

About Localized Prostate Cancer

Prostate cancer is the second most common form of cancer in men in the US with an estimated 161,000 new cases in 2017. 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 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.

About Topsalysin

Topsalysin (PRX302), an innovative, "First-in-Class" transmembrane pore-forming protein, was genetically modified to be activated only by enzymatically-active PSA, which is produced in large quantities within the prostate of men with prostate cancer. The targeted focal treatment of prostate cancer is in line with current treatment trends for solid tumors such as breast and liver, where the goal is to remove the tumor and preserve as much of the organ and organ function as possible.

Topsalysin has the potential to provide a targeted focal therapy for the ablation of localized prostate cancer lesions while potentially avoiding many of the complications and side effects associated with whole gland radical treatments. The increasing use of multiparametric magnetic resonance imaging (mpMRI) and advances in software to co-register previously obtained mpMRI images with real-time three-dimensional ultrasound images enables urologists 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.