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 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.

Den 26 oktober 2017 rapporterade Xspray Pharma att de har fått godkännande för två sökta patent i USA (Press release, Xspray, OCT 26, 2017, View Source [SID1234523286]). Patentet omfattar komposition avseende produktkandidaterna HyNap-Sora och HyNap-Nilo. Det är Xsprays andra och tredje produktpatent som godkänns på huvudmarknaden i USA på kort tid. Bolaget har tidigare offentliggjort ett patentgodkännande i Japan och USA avseende HyNap-Dasa och har pågående ansökningsärenden för flera motsvarande patent i bland annat Japan och Europa.
"Nu har vi patent beviljade i USA som täcker de tre produkter vi avser att introducera på den amerikanska marknaden efter respektive original-substanspatent har löpt ut (under perioden 2020-2023). Vi har arbetar systematiskt och strategiskt med våra innovationer både för att förverkliga dem till produkter men också genom att säkra värdet med ett fullgott patentskydd. Jag ser det här som en bekräftelse på det arbetet," säger Per Andersson, vd för Xspray Pharma.

Xspray Pharma har erhållit godkännande ("notice of allowance") för två patent i USA avseende produktkandidaterna HyNap-Sora och HyNap-Nilo som är tänkta för behandling av vissa cancerformer. Det är Xsprays andra och tredje produktpatent som godkänns på den viktigaste marknaden, USA. Beskedet kommer i enlighet med bolagets plan att söka och erhålla patent för komposition och metod för samtliga tre produktkandidater under utveckling på de tre viktigaste marknaderna, USA, Europa och Japan.

"Med dessa patent, och de positiva resultaten från vår kliniska studie som vi nyligen offentliggjorde, har vi tagit viktiga steg mot målet att utveckla våra tre första produkter för lansering på den amerikanska marknaden," kommenterar Xsprays vd Per Andersson.

Xspray Pharmas aktier introducerades den 28 september på Nasdaq First North, efter en lyckosamt genomförd nyemission som tillförde bolaget 132 miljoner kronor före emissionskostnader. Planen är nu att använda kapitalet för att utveckla tre produktkandidater och blivande cancerläkemedel baserade på bolagets egenutvecklade teknologi, samt att introducera de första produkterna på den amerikanska marknaden under perioden 2020-2023.

Den 26 oktober 2017 rapporterade Xspray Pharma att de har fått godkännande för två sökta patent i USA (Press release, Xspray, OCT 26, 2017, View Source [SID1234523286]). Patentet omfattar komposition avseende produktkandidaterna HyNap-Sora och HyNap-Nilo. Det är Xsprays andra och tredje produktpatent som godkänns på huvudmarknaden i USA på kort tid. Bolaget har tidigare offentliggjort ett patentgodkännande i Japan och USA avseende HyNap-Dasa och har pågående ansökningsärenden för flera motsvarande patent i bland annat Japan och Europa.
"Nu har vi patent beviljade i USA som täcker de tre produkter vi avser att introducera på den amerikanska marknaden efter respektive original-substanspatent har löpt ut (under perioden 2020-2023). Vi har arbetar systematiskt och strategiskt med våra innovationer både för att förverkliga dem till produkter men också genom att säkra värdet med ett fullgott patentskydd. Jag ser det här som en bekräftelse på det arbetet," säger Per Andersson, vd för Xspray Pharma.

Xspray Pharma har erhållit godkännande ("notice of allowance") för två patent i USA avseende produktkandidaterna HyNap-Sora och HyNap-Nilo som är tänkta för behandling av vissa cancerformer. Det är Xsprays andra och tredje produktpatent som godkänns på den viktigaste marknaden, USA. Beskedet kommer i enlighet med bolagets plan att söka och erhålla patent för komposition och metod för samtliga tre produktkandidater under utveckling på de tre viktigaste marknaderna, USA, Europa och Japan.

"Med dessa patent, och de positiva resultaten från vår kliniska studie som vi nyligen offentliggjorde, har vi tagit viktiga steg mot målet att utveckla våra tre första produkter för lansering på den amerikanska marknaden," kommenterar Xsprays vd Per Andersson.

Xspray Pharmas aktier introducerades den 28 september på Nasdaq First North, efter en lyckosamt genomförd nyemission som tillförde bolaget 132 miljoner kronor före emissionskostnader. Planen är nu att använda kapitalet för att utveckla tre produktkandidater och blivande cancerläkemedel baserade på bolagets egenutvecklade teknologi, samt att introducera de första produkterna på den amerikanska marknaden under perioden 2020-2023.

On October 26, 2017 Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, reported that Dr. Monica Mita of Cedars-Sinai Medical Center and Principal Investigator of the RGX-104-001 study, will present at the 2017 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) (Press release, Rgenix, OCT 26, 2017, View Source [SID1234523090]). The conference is scheduled to take place from Thursday, October 26 to Monday, October 30 in Philadelphia.

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The details of Rgenix’s presentation are as follows:

Event: AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper): Discovery, Biology, and Clinical Applications

Date: October 29, 2017

Time: 12:30 P.M. – 4:00 P.M. EST

Description: Poster B001, "A phase 1 trial of RGX-104, a first-in-class immunotherapy targeting the liver-X nuclear hormone receptor (LXR), in patients with refractory malignancies"

Location: Hall E, Pennsylvania Convention Center, 1101 Arch St, Philadelphia, PA 19107

Dr. Mita will present data from the Phase 1 trial of RGX-104, a first-in-class investigational immunotherapy.

About RGX-104

RGX-104 is a potent small molecule agonist of the Liver X Receptor (LXR). Activation of the LXR-ApoE pathway by RGX-104 stimulates the innate immune response in cancer via depletion of myeloid-derived suppressor cells and activation of dendritic cells, leading to stimulation of T cells and anti-tumor immunity in tumor models. LXR activation also blocks the ability of tumors to recruit blood vessels. These combined effects result in suppression of tumor growth and metastasis in a broad array of pre-clinical models. The LXR-ApoE pathway was originally identified as a cancer target using a novel microRNA-based discovery platform developed by Rgenix’s scientific co-founders at The Rockefeller University.

Rgenix is conducting a Phase 1a/b clinical trial of RGX-104 in patients with advanced solid malignancies and lymphoma—for more information about the clinical trial, please visit: View Source