On October 26, 2017 4SC AG (4SC, FSE Prime Standard: VSC) reported an interim communication on the nine months ended 30 September 2017 that presents all material developments with a focus on Q3 2017 and provides the Company’s current outlook (Press release, 4SC, OCT 26, 2017, View Source [SID1234521182]). The full communication is available for download on 4SC’s website.
Jason Loveridge, Ph.D., CEO of 4SC, commented:

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"With the conclusion of a successful capital increase generating gross proceeds of ca. EUR 41 million we are now well positioned to reach our stated goals over the next few years: to create significant value for our shareholders by executing on accelerated routes to market for our core cancer products resminostat, 4SC-202 and 4SC-208.

We will continue to enroll patients in our pivotal RESMAIN study of resminostat in cutaneous T-cell lymphoma (CTCL) and are looking forward to the initiation of a Phase II study of resminostat in patients with biliary tract cancer by our Japanese development partner Yakult Honsha Co., Ltd. (Yakult Honsha).

After opening the first clinical center within our SENSITIZE study of 4SC-202 in combination with a checkpoint inhibitor in melanoma patients in Q3 2017, we are anticipating to enroll the first patient in Q4 2017. Furthermore, we are expecting the investigator-initiated Phase II EMERGE study of 4SC-202 in combination with another checkpoint inhibitor in patients with microsatellite-stable gastrointestinal tumors to be initiated in Q1 2018.

Formal preclinical testing of 4SC-208 in order to initiate Phase I clinical evaluation is ongoing according to plan.

Finally, we aim to continue to enhance the value of 4SC and to add to our funds through signing deals with the right industry partners to pursue further development of our non-core assets as we did with the Kv1.3 inhibitors which we recently licensed to Maruho Co., Ltd. (Maruho)."

Key highlights of Q3 2017 and beyond

Ca. EUR 41 million secured from successful capital increase; proceeds forecast to be sufficient to finance 4SC’s accelerated development strategy into 2020
Patient enrollment and opening of study centers well on track for the ongoing pivotal RESMAIN study, which examines the potential of resminostat as maintenance therapy in patients with advanced CTCL
Preclinical data presented on resminostat’s potential to significantly alleviate itching in CTCL patients – one of the major disease burdens
Promising results of a Phase I study of resminostat in combination with S-1 chemotherapy in Japanese patients with biliary tract or pancreatic cancer presented by Yakult Honsha, 4SC’s development partner for resminostat in Japan
Phase Ib/II study SENSITIZE of 4SC-202 in combination with the anti-PD-1 checkpoint inhibitor pembrolizumab (Keytruda(R), Merck) in patients with advanced-stage melanoma initiated with the opening of the first study centers
US composition of matter patent secured for structurally related compounds including 4SC-208; preclinical testing ongoing
Preclinical inhibitors of the Kv1.3 ion channel out-licensed to Maruho in return for up to EUR 208 million in upfront, development and commercial milestones plus single-digit royalties
Business outlook

Continue patient enrollment in the pivotal RESMAIN study of resminostat in CTCL
Yakult Honsha to initiate Phase II study of resminostat in Japanese patients with biliary tract cancer
Enroll patients in the Phase Ib/II study SENSITIZE of 4SC-202 in melanoma
Initiate Phase II investigator-initiated study EMERGE of 4SC-202 in patients with microsatellite-stable gastrointestinal tumors
Continue preclinical testing of 4SC-208 to initiate a Phase I clinical study immediately thereafter
Pursue further licensing deals for non-core assets and continue evaluating potential partnering opportunities with pharmaceutical and biotech companies to progress the clinical development of 4SC’s core pipeline assets
Development of cash balance in Q3 2017 and financial forecast

As of 30 September 2017, 4SC holds cash balance/funds of EUR 43,353 thousand as compared to EUR 4,638 thousand as of 30 June 2017. The increase results from a successful cash capital increase in July with gross proceeds of ca. EUR 41 million. The monthly use of cash from operations was within the range forecasted for 2017 amounting to EUR 739 thousand on average in the first nine months of 2017 (9M 2016 EUR 857 thousand). The decrease in 2017 was mainly driven by the upfront payment from the licensing agreement with Maruho offset by an increase in expenses for the preparation of the Phase Ib/II clinical study SENSITIZE of 4SC-202. The Management Board of 4SC confirms that the proceeds of the capital raise will finance 4SC’s stated goals into 2020.

Conference Call

4SC will not hold a telephone conference along with today’s Q3 2017 Interim Communication. According to 4SC’s policy, the Company will only hold conference calls when there is significant or material newsflow.

Further information

About resminostat

Resminostat is orally administered and potentially offers a novel approach to treating a wide variety of cancers, both as monotherapy and in combination therapy with other anti-cancer drugs. Resminostat inhibits tumor growth and proliferation, causes tumor regression, and strengthens the body’s immune response to cancer.

Resminostat has been shown to be well tolerated in several clinical trials. Resminostat is currently being investigated in a Phase II pivotal study in cutaneous T-cell lymphoma (CTCL) by 4SC. A Phase II study in biliary tract cancer is planned by 4SC’s development partner Yakult Honsha in Japan. Amongst others, resminostat has previously been investigated in biliary tract or pancreatic cancer and hepatocellular carcinoma (HCC).

About 4SC-202

4SC-202 is an orally administered small molecule with a unique mode of action that was designed to strengthen the body’s own anti-tumor immune response, open the tumor microenvironment and encourage infiltration of immune cells into the tumor.

4SC-202 has been investigated in a Phase I study with 24 mostly heavily pretreated patients with several types of highly advanced hematologic cancers, and was proven to be tolerated. Positive signs of anti-tumor efficacy were observed with one complete remission for 28 months and one partial responder for 8 months.

In addition to its therapeutic potential in cancer monotherapy, 4SC is evaluating 4SC-202’s capacity as a partner in combination therapies, specifically in the immuno-oncology area. In this respect, 4SC initiated a Phase Ib/II study of 4SC-202 in combination with the anti-PD-1 checkpoint inhibitor pembrolizumab in patients with advanced-stage melanoma. A second Phase II study of 4SC-202 in combination with the anti-PD-L1 checkpoint inhibitor avelumab, which will be conducted by an academic partner in gastrointestinal cancers, is expected to start soon.

As soon as results from the aforementioned trials will be available, 4SC plans to advance 4SC-202 into a pivotal study in combination with a checkpoint inhibitor in PD-(L)1 refractory patients with advanced Merkel cell carcinoma (MCC).

About 4SC-208

Data from several preclinical in vivo models has established the efficacy of 4SC-208 in inhibiting the Hedgehog/GLI signaling. Inhibition of this signaling pathway has emerged as a highly effective strategy in obstructing the tumorigenic capacity of cancer stem cells, as well as tumor development, proliferation and survival.

Available inhibitors of Hedgehog signaling target the pathway upstream of the transcription factor GLI, whereas 4SC-208 inhibits at the level of GLI and is thus potentially able to avoid the tumor recurrence and relapse observed in response to currently available inhibitors.

4SC believes that 4SC-208 is a promising drug candidate and expects to complete formal preclinical testing in 2018 and to enter into a Phase I/II clinical study immediately thereafter. Cancer indications that are particularly promising are those where resistance to therapies targeting the Hedgehog/GLI pathway are emerging, such as in basal cell carcinoma.

On October 26, 2017 Acceleron Pharma Inc. (NASDAQ:XLRN) reported it will host a webcast and conference call on Tuesday, November 7, 2017 at 8:00 a.m. EST to discuss its third quarter 2017 financial results and provide an update on recent clinical development and corporate activities(Press release, Acceleron Pharma, OCT 26, 2017, View Source [SID1234521191]).

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The webcast will be accessible under “Events & Presentations” in the Investors/Media page of the company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and refer to the “Acceleron Third Quarter Earnings Call.”

The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

On October 26, 2017 Transgene (Paris:TNG) (Euronext Paris: TNG), a biotech company that designs and develops viral-based immunotherapies, reported that the first patient with recurrent glioblastoma has been treated at La Pitié-Salpêtrière hospital, Greater Paris University Hospitals, AP-HP (Paris), in the first-in-human clinical trial (Oncovirac trial) of TG6002, a novel oncolytic virus (Press release, Transgene, OCT 26, 2017, View Source [SID1234521218]). TG6002 represents the next generation of oncolytic virus (OV), which is administered intravenously and has multiple functions. It has been engineered to combine oncolysis (the breakdown of cancer cells) with the local production of 5-FU chemotherapy agent in the tumor. It is also expected to induce an immune response following the antigen spreading that is caused by the cancer cells’ breakdown.

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TG6002: a novel oncolytic virus allowing the targeted production of chemotherapy in the tumor

TG6002 is a next generation oncolytic immunotherapy, which has a double mechanism of action. It has been designed by Transgene to:

1. induce the breakdown of cancer cells (oncolysis) by tumor-selective viral replication. In preclinical experiments, TG6002 was able to induce response in the primary tumor and an immune-mediated regression of distant metastases (immunogenic cell death);

2. allow the local production of chemotherapy (5-FU), a widely used cancer chemotherapy, in the tumor. TG6002 expresses the proprietary Fcu1 gene in the cancer cells it has infected, leading to the local conversion of the 5-FC into 5-FU.

First-in-human trial to deliver first readouts in H2 2018

Oncovirac is an open-label Phase 1/2a trial evaluating the safety and tolerability of multiple-ascending doses of TG6002 administered intravenously in combination with oral 5-FC, a non-cytotoxic pro-drug, flucytosine, that can be converted in 5-FU. The anti-tumor activity of this novel oncolytic virus will also be monitored. The study will enroll patients suffering from recurrent glioblastoma, who have failed standard of care treatment.

Dr. Ahmed Idbaih, M.D., PhD, neuro-oncologist at La Pitié-Salpêtrière Hospital (Paris, France), is the principal investigator of the study. He is involved in several clinical trials dedicated to primary brain tumor patients. He also coordinates “GlioTex”, a research group focused on glioblastoma and experimental therapeutics at ICM (The Institut du Cerveau et de la Moelle épinière – Brain & Spine Institute). AP-HP Paris Greater Hospitals, is the sponsor of Oncovirac, a trial also supported by INCa (French National Cancer Institute). More information on the trial is available on clinicaltrials.gov (NCT03294486). The first readouts of the study are expected in the second half of 2018.

Maud Brandely, M.D., PhD, Chief Medical Officer of Transgene, added: “TG6002 is a very promising new generation of oncolytic virus, which has the potential to be administered intravenously. Based on our compelling preclinical data, we have established that its replication induces immunogenic cell lysis and the local production of chemotherapy. We are excited to see this novel immunotherapy with multiple modes of action enter the clinic and look forward to obtaining results that will allow further development of TG6002 in several solid tumors indications.”

Dr. Ahmed Idbaih, M.D., PhD, neuro-oncologist at La Pitié-Salpêtrière hospital, AP-HP, and principal investigator of the trial, added: “Current treatments of recurrent glioblastoma are insufficient. By combining the immunogenic lysis of cancer cells with the targeted production of chemotherapy in the tumor, TG6002 has the potential to show anti-tumor efficacy and to avoid systemic side effects of chemotherapy. We are very pleased to be conducting this first in human clinical trial evaluating this novel immunotherapy that we believe could improve the overall survival of recurrent glioblastoma patients while preserving their quality of life.”

About TG6002
TG6002 is a next generation oncolytic immunotherapy. It has been designed to induce the breakdown of cancer cells (oncolysis) and allow the local production of chemotherapy (5-FU) in the tumor. TG6002 is a modified Vaccinia virus, with double gene deletion (TK-RR-), and expressing the proprietary Fcu1 gene in the cancer cells it has infected, leading to the local conversion of the non-cytotoxic pro-drug, flucytosine (5-FC), into 5-FU, a widely used cancer chemotherapy. The oncolytic virus TG6002 has shown efficacy and good safety profile in several preclinical models of glioblastoma in vitro (i.e. cell line) and in vivo (i.e. xenografts in Swiss/Nude mice). Transgene believes that TG6002 may represent a new therapeutic option in recurrent glioblastoma patients. TG6002 could also be investigated in other solid tumors.

About Glioblastoma
Glioblastoma is the most common and the most aggressive primary brain cancer in adults. Approximately 70,000 new cases are diagnosed each year in Europe 28 and in the USA (Globocan 2012). Despite very intensive treatments (i.e. maximal safe surgery, radiotherapy, and several lines of cytotoxic chemotherapy), inducing significant adverse events, the prognosis of glioblastoma patients remains poor. More efficient and less toxic therapies are urgently needed to improve survival and quality of life of glioblastoma patients.

On October 26, 2017 argenx (Euronext Brussels & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported financial results and provided a business update for the third quarter ended September 30, 2017 (Press release, argenx, OCT 26, 2017, View Source [SID1234521183]).

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"We made strong progress during the third quarter launching the Phase 2 study of ARGX-113 in pemphigus vulgaris (PV) and delivering on our promise to expand the ARGX-113 program into new, carefully selected indications. With data readouts expected in 2018 from the Phase 2 studies in myasthenia gravis (MG), immune thrombocytopenia (ITP) and now PV, we will evaluate ARGX-113 in indications where disease progression is driven by pathogenic autoantibodies of the Immunoglobulin G (IgG) type," commented Tim Van Hauwermeiren, CEO of argenx. "We are also progressing well with our lead oncology candidate, ARGX-110, and expect interim data from both our Phase 1/2 trial in acute myeloid leukemia (AML) and Phase 2 trial in cutaneous T-cell lymphoma (CTCL) by the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (December, 2017). Between our wholly-owned pipeline and our programs partnered with AbbVie, Leo Pharma and through our Innovative Access Program, we believe we are very well-positioned for a catalyst-rich 2018."

THIRD QUARTER 2017 AND RECENT HIGHLIGHTS

Products in clinical development:

ARGX-113

Completed enrollment for Phase 2 clinical trial of MG.

Announced orphan drug designation by U.S. Food and Drug Administration for ARGX-113 for the treatment of MG.

Reached 50% enrollment in Phase 2 proof-of-concept clinical trial of ARGX-113 in ITP.

Launched Phase 2 proof-of-concept clinical trial of ARGX-113 for the treatment of PV.

ARGX-111

Met safety endpoints in the Phase 1 clinical trial in treatment-refractory patients with advanced cancers whose tumors overexpress the MET protein. Complete data set from ARGX-111 Phase 1b clinical trial presented at Best of ASCO (Free ASCO Whitepaper) Asia 2017 (Singapore).

Collaborations:

ARGX-116

< >nnounced publication of new preclinical data in ‘Nature Medicine’ on ARGX-116 inhibiting ApoC3, a metabolic target correlated with blood lipid levels that provide further rationale for the development of ARGX-116 for the treatment of dyslipidemia.
Established argenx US, Inc., a U.S. subsidiary located in Boston.

UPCOMING CLINICAL MILESTONES

ARGX-113

< >line data from Phase 2 clinical trials in MG expected in 1Q 2018 at the latest and ITP expected in 2H 2018. Interim data from Phase 2 clinical trial in PV expected in 2H 2018.
Initiation of Phase 1 clinical trial of subcutaneous dosing in healthy volunteers expected in Q4 2017.

ARGX-110

Interim data from Phase 1/2 clinical trial in AML and Phase 2 clinical trial in CTCL each expected by ASH (Free ASH Whitepaper) (December 11, 2017).

FINANCIAL HIGHLIGHTS (as of September 30, 2017) (compared to financial highlights as of September 30, 2016)

Operating income of €30.5 million (September 30, 2016: €12.5 million).

Total comprehensive loss of €16.5 million (September 30, 2016: €12.6 million).

Cash position of €161.7 million (cash, cash-equivalents and current financial assets) allowing argenx to pursue development of its pipeline as planned.

On October 26, 2017 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company developing new gene and cell-based immunotherapies for cancer, reported that it will host a conference call and webcast slide presentation on Monday, November 6, 2017 at 4:30 p.m. ET to provide a corporate update and discuss financial results for the third quarter ended September 30, 2017 (Press release, Ziopharm, OCT 26, 2017, View Source [SID1234521204]).

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The call can be accessed by dialing 1-844-309-0618 (U.S. and Canada) or 1-661-378-9465 (international). The passcode for the conference call is 8769629. To access the slides and live webcast or the subsequent archived recording, visit the “Investors & Media” section of the ZIOPHARM website at www.ziopharm.com. The webcast will be recorded and available for replay on the Company’s website for two weeks.