On November 10, 2017 Nektar Therapeutics (Nasdaq: NKTR) reported that its corporate presentation will be webcast at the upcoming Jefferies 2017 London Healthcare Conference in London on Wednesday, November 15, 2017 at 10:00 a.m. GMT (Press release, Nektar Therapeutics, NOV 10, 2017, View Source [SID1234521926]).

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The presentation will be accessible via a Webcast through a link posted on the Investors, Events Calendar section of the Nektar website: View Source This Webcast will be available for replay until December 11, 2017.

On November 10, 2017 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported that it is presenting two trials in progress posters summarizing study designs for ongoing clinical trials with MAGE-A4 and NY-ESO SPEAR T-cells at the 2017 SITC (Free SITC Whitepaper) annual meeting at the Gaylord National Hotel & Convention Center in National Harbor, Maryland, United States (Press release, Adaptimmune, NOV 10, 2017, View Source;p=RssLanding&cat=news&id=2316203 [SID1234521929]).

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Overview of Study Designs:

MAGE-A4 SPEAR T-cells targeting multiple solid tumors1:
– Open-label, non-randomized pilot study evaluating the safety, tolerability, and antitumor activity of MAGE-A4 SPEAR T-cells in patients with HLA-A*02 and MAGE-A4 positive inoperable locally advanced or metastatic tumor(s)
– This dose escalation study utilizes a modified 3+3 design:
• Group 1: to enroll 3-6 patients; dose of 100 million transduced SPEAR T-cells, 21-day interval for safety review
• Group 2: to enroll 3-6 patients; dose of 1 billion transduced SPEAR T-cells, 7-day interval for safety review2
• Group 3: to enroll 3-6 patients; dose of 1-5 billion transduced SPEAR T-cells, 7-day interval for safety review2
• Study allows for expansion at optimal dose range up to 20 patients across tumors
– Patients must be: ≥ 18 yrs old; HLA-A*02 positive; have MAGE-A4 positive inoperable locally advanced or metastatic tumor(s) at ≥1+ intensity in ≥ 10% of tumor cells MAGE-A4 expression by immunohistochemistry (IHC); have ECOG status 0 or 1; and adequate organ function
– Lymphodepletion regimen: fludarabine (30 mg/m2/day) and cyclophosphamide (600 mg/m2/day) for 3 days
– Efficacy assessed by overall response rate, time to response, duration of response, progression-free survival, and overall survival at weeks 4, 8, and 12, month 6, and then every 3 months until confirmation of disease progression
– The study is open and enrolling

NY‑ESO SPEAR T-cells with or without KEYTRUDA (pembrolizumab) in multiple myeloma:
– Open-label, randomized pilot study evaluating the safety, tolerability, and antitumor activity of NY-ESO SPEAR T-cells with or without KEYTRUDA in patients with multiple myeloma
– Eligible patients will be randomly assigned to a treatment arm: NY-ESO SPEAR T-cells alone (Arm 1) or NY-ESO-1 SPEAR T-cells in combination with KEYTRUDA (Arm 2)
– Target enrollment is 20 patients with 10 in each arm; eligible patients who do not receive the T‑cell infusion may be replaced.
– Patients must be: ≥ 18 yrs old; HLA-A*02:01, *02:05, or *02:06 positive; have histologically confirmed diagnosis of multiple myeloma with either primary refractory or relapsed/refractory disease expressing NY-ESO-1 and/or LAGE-1a; have received prior therapies including IMiD and a proteasome inhibitor as separate lines or a combined line of therapy; have ECOG status 0 or 1; and adequate organ function
– Lymphodepletion regimen: fludarabine (30 mg/m2/day) and cyclophosphamide (600 mg/m2/day) for 3 days, followed by granulocyte-colony stimulating factor
– For patients in Arm 2, KEYTRUDA will be administered every 3 weeks, starting at week 3 following T-cell infusion until week 108
– Target dose of 1 – 8 × 109 transduced SPEAR T-cells
– Efficacy will be assessed by the International Myeloma Working Group (IMWG) Uniform Response Criteria. Overall response rate, time to response, duration of response, progression-free survival, and overall survival will be determined.
– The study is open and enrolling

On November 10, 2017 Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a leading immunotherapy company focused on developing treatments for autoimmune/inflammatory diseases and cancer, reported immuno-oncology preclinical data characterizing the functional activity of molecules Alpine successfully generated from its variant immunoglobulin domain (vIgD) platform (Press release, Alpine Immune Sciences, NOV 10, 2017, View Source [SID1234521920]). Several novel immuno-oncology molecules were functionally active via multiple mechanisms of action, including the demonstration of tumor suppression in an animal model. The findings will be presented on Friday, November 10, in a poster session titled "Immune Modulation, Cytokines, and Antibodies" [#P343] at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 32nd Annual Meeting in National Harbor, MD.

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"Our unique vIgD platform is capable of producing first-in-class immuno-oncology biologics with potentially unique mechanisms of action," said Stanford Peng, M.D., Ph.D., Executive Vice President of Research and Development and Chief Medical Officer of Alpine. "This promising data highlights the versatility of the platform, showing vIgDs may be implemented in multiple therapeutic formats and may be tailored to modulate multiple molecular pathways according to the desired therapeutic application."

Preclinical Study Design and Results

Alpine scientists used the vIgD directed evolution platform to engineer a number of vIgDs with unique binding profiles to proteins relevant to the immune synapse, including PD-1, PD-L1, CTLA-4, TIGIT, CD155, CD28, and/or ICOS. The poster describes the vIgD domains in multiple therapeutic formats, including tumor-localized Fc fusion proteins, multi-checkpoint inhibitors, and vIgDs fused with tumor-specific monoclonal antibodies (V-mAbs). Various in vitro and in vivo tests characterized the functional activity of these potentially novel therapeutics. Data include:

Tri-specific vIgDs for treating cancer with a single domain capable of interacting with three different B7 family members. Depending on formatting, tri-specific vIgDs are potentially capable of agonizing CD28, blocking PD-L1, blocking CTLA-4, and/or depleting tumor cells and/or regulatory T cells. Initial formats investigated in an animal model of cancer demonstrated activity with tumor growth suppression.
A dual ICOS/CD28 costimulatory vIgD fused with the HER2-targeting monoclonal antibody trastuzumab to provide immune stimulation in the tumor microenvironment. These V-mAbs demonstrated in vitro proof of principle for immune cell stimulation and proliferation in response to HER2-positive tumor cells.
Multiple vIgD Fc fusions capable of targeting TIGIT and PD-1 while sparing CD226. These multi-checkpoint inhibitory molecules blocked checkpoint activity and improved IFN-γ production by "exhausted" T cells.
"The SITC (Free SITC Whitepaper) data suggest the versatile vIgD platform has the potential to contribute to the next generation of immuno-oncology therapeutics. Based on these and other encouraging preclinical data, we are continuing to identify and develop appropriate candidates from our vIgD platform for clinical trials for both oncology and inflammation," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine.

On November 10, 2017 BioSpecifics Technologies Corp. (NASDAQ: BSTC), a biopharmaceutical company that originated and continues to develop collagenase based-therapies with a first in class collagenase-based product marketed as XIAFLEX in the U.S. and Xiapex in Europe, reported its financial results for the third quarter ended September 30, 2017 and provided a corporate update (Press release, BioSpecifics Technologies, NOV 10, 2017, View Source [SID1234521921]).

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"BioSpecifics continues to make important progress as we remain focused on further developing XIAFLEX for the treatment of serious medical conditions. We are excited about the potential of our ongoing Phase 1 trial of XIAFLEX for the treatment of uterine fibroids and look forward to reporting data in 2018," said Thomas L. Wegman, President of BioSpecifics. "Additionally, we look forward to our partner Endo initiating Phase 3 trials of XIAFLEX for the treatment of cellulite in the coming months."

Third Quarter 2017 Financial Results

BioSpecifics reported net income of $2.7 million for the third quarter ended September 30, 2017, or $0.38 per basic share and $0.37 per share on a fully diluted basis, compared to net income of $3.1 million, or $0.43 per basic share and $0.42 per share on a fully diluted basis, for the same period in 2016.

Total revenue for the third quarter ended September 30, 2017 was $6.5 million, compared to $6.9 million for the same period in 2016. Royalty revenue increased by $0.4 million for the 2017 quarter as compared to the 2016 quarter primarily due to increased sales of XIAFLEX for the treatment of Peyronie’s disease and Dupuytren’s contracture, whereas total revenue was lower due to the exercise of an opt-in right by Endo for the human lipoma indication of $750,000 in the 2016 period.

Research and development expenses for the third quarter ended September 30, 2017 were $0.4 million compared to $0.3 million for the same period in 2016.

General and administrative expenses for the third quarter ended September 30, 2017 were $2.2 million compared to $1.8 million for the same period in 2016.

Provision for income taxes for the third quarter ended September 30, 2017 were $1.5 million, compared to $1.8 million for the same period in 2016.

As of September 30, 2017, BioSpecifics had cash and cash equivalents and investments of $61.3 million, compared to $52.8 million as of December 31, 2016.

XIAFLEX Commercial Highlights

On November 9, 2017, Endo reported commercial highlights for XIAFLEX for the third quarter of 2017 (Endo’s third quarter 2017 financials are reported in BioSpecifics’ fourth quarter 2017 financials). For the third quarter of 2017, total revenues were $52.5 million compared to $47.7 million in the third quarter of 2016, an 10 percent growth. Endo continues to expect high-single to low-double digit revenue growth for sales of XIAFLEX in 2017.

Endo announced a partnership with Tim Herron, a four-time PGA Tour winner, and Damon Adamany, MD, of the CORE Institute, launched Facts on Hand, an unbranded campaign to raise awareness of Dupuytren’s Contracture, a progressive, potentially disfiguring hand condition. Endo also recently launched several direct-to-consumer initiatives intended to increase patient awareness of XIAFLEX as a possible treatment option for Dupuytren’s Contracture and Peyronie’s Disease.

XIAFLEX Pipeline Updates and Anticipated Upcoming Milestones

BioSpecifics manages the development of XIAFLEX for the treatment of uterine fibroids. The Phase 1 clinical trial is currently enrolling. BioSpecifics has the right to initiate the development of any new potential indication not licensed by Endo. Endo’s licensed indications include Dupuytren’s Contracture and Peyronie’s Disease, both approved and marketed; in addition to cellulite, adhesive capsulitis, human and canine lipoma, lateral hip fat and plantar fibromatosis.

The Phase 1 clinical trial of XIAFLEX for the treatment of uterine fibroids is ongoing and BioSpecifics plans to announce results in 2018. The study, being conducted at the Department of Gynecology & Obstetrics at Johns Hopkins University, is designed to enroll 15 subjects administered XIAFLEX prior to hysterectomy. The primary endpoint of the study will assess the safety and tolerability of a single injection of XIAFLEX directly into the uterine fibroid under transvaginal ultrasound guidance. The secondary endpoints will assess symptoms of pain and bleeding, quality of life throughout the study as well as size, collagen content and rate of apoptosis of XIAFLEX treated fibroids.
Endo plans to initiate Phase 3 clinical trials of XIAFLEX for the treatment of cellulite in the coming months following discussions with the U.S. Food and Drug Administration.
Endo continues its commercial review of additional indications.

On November 10, 2017 Carna Biosciences reported that it has revised its consolidated results forecast for the fiscal year ending December 31, 2017 (Press release, Carna Biosciences, NOV 10, 2017, View Source [SID1234526522])

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(January 1, 2017 to December 31, 2017) announced on February 10, 2017.

1. Revised consolidated results forecast

Reason of the revision
Carna Biosciences has revised its full year consolidated sales forecast to 701 million yen. Sales forecast for the
Drug Discovery Support business was revised to 701 million yen from 1,000 million yen and sales forecast for
the Drug Discovery and Development business was revised to zero from 440 million yen.
At the Drug Discovery and Development business, we decided to revise our forecast because we concluded that
it would be difficult to receive the expected milestone payment of 440 million yen within this fiscal year for
SRA141 that Carna out-licensed to Sierra Oncology, Inc. Sierra is currently conducting preclinical research for
SRA141 as initially planned, aiming to expand its pipeline targeting DNA Damage Response (DDR). However,
given its near-term focus on broadening the development program for its lead asset SRA737, Sierra rescheduled
the development timeline of SRA141, and the IND submission has been postponed until next year. We decided
to revise our forecast, examining the current development status of SRA141 and other revenue from the Drug
Discovery and Development business.
At the Drug Discovery Support business, in order to achieve sales target of 1,000 million yen, we focused on the
marketing activity to win large scale contract for DGK assay kits from major pharmaceutical companies and
biotech companies mainly in North America. As a result of our efforts, such as providing DGK proteins and
smaller scale assay kits, we expect to receive medium scale orders within this fiscal year. However, we currently
expect that it would be difficult to achieve 220 million sales target for DGK assay kits and decided to revise our
sales forecast for North America to 238 million yen, down 48.9 % compared to the previous forecast and up
19.4% compared to the previous fiscal year. In Japan, sales from profiling services provided to Ono
Pharmaceutical are expected to fall short of the initial plan. As a result, we decided to revise sales forecast for
Japan to 366 million yen, down 14.1% compared to the previous forecast and down 12.5% compared to the
previous fiscal year. We also revised sales forecast for Europe to 70 million yen, down 16.8% compared to the
previous forecast and down 2.0% compared to the previous fiscal year and revised sales forecast for other region
to 26 million yen, up 18.5% compared to the previous forecast and up 19.6% compared to the previous fiscal year.
– 2 –
For operating income, we initially expected 39 million yen at consolidated level, 443 million yen from the Drug
Discovery Support business and loss of 403 million yen from the Drug Discovery and Development business.
However, after careful consideration, we decided to revise our consolidated operating profit forecast to operating
loss of 727 million yen. By segment, we now expect operating profit of 173 million yen for the Drug Discovery
Support business, down 270 million yen from the previous forecast, mainly due to a decline in gross profit caused
by the lower than expected sales. For the Drug Discovery and Development business, we now expect operating
loss of 901 million yen mainly due to a lack of milestone payment from Sierra.
As a result, we now expect ordinary loss of 738 million yen after considering non-operating income and expenses
and net loss attributable to owners of parent of 766 million yen.
We expect to receive milestone payment from Sierra in FY2018 since preclinical research of SRA141 is advancing
as expected. For DGK assay kits in North America, we plan to strengthen our marketing activity in an effort to
expand the expected medium scale contracts to large scale contract.
We plan to reduce unnecessary expenses thoroughly. At the same time, we continue to advance preclinical trials
and lead optimization of our compounds. We also aim to initiate in-house clinical trials and to out-license our
pipeline in order to maximize our corporate value.
* The forward-looking statements in this document have been prepared based on information available at the
time of the issuance of this release as well as on the certain assumptions considered by the management to be
reasonable. Actual results may differ from this forecast due to variety of factors.