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 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 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 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported that two of its cancer immunotherapies demonstrated antigen-specific T-cell stimulation in phase 1 studies. INO-3112 (now called MEDI0457), an investigational T-cell activation immunotherapy that targets cancers caused by human papillomavirus (HPV) types 16 and 18 and licensed to MedImmune, the global Research and Development arm of AstraZeneca, also led to a head and neck cancer patient’s complete response when matched with a PD-1 checkpoint inhibitor (Press release, Inovio, NOV 10, 2017, View Source [SID1234521931]). In addition, INO-1400, Inovio’s investigational cancer immunotherapy targeting hTERT, which is over-expressed in a majority of cancers, generated hTERT-specific IFN-γ secreting T cells, suggesting an ability to break immune tolerance. These results were revealed at poster presentations at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 32nd Annual Meeting starting today at National Harbor, Md.

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Dr. J. Joseph Kim, Inovio’s President and CEO, said, "This study of MEDI0457 shows efficacy signals that Inovio’s activation immunotherapy coupled with checkpoint inhibitor could have meaningful therapeutic impact, given an unusual complete response in one patient. Separately, we are evaluating our hTERT therapy, INO-1400, in nine different solid tumors including breast, lung and pancreatic cancers in a clinical study. Any success we see in our hTERT therapy gives us added confidence in our recently initiated efficacy studies combining PD-1/PD-L1 inhibitors and INO-5401, which includes three of Inovio’s top SynCon cancer antigens – hTERT, WT1, and PSMA, which are over-expressed in multiple tumor types. We look forward to sharing further data on our immunotherapies as studies progress."

In a phase 1 study of MEDI0457 in 22 HPV-positive patients with squamous cell carcinoma of the head and neck, Inovio has previously demonstrated that this cancer immunotherapy generated robust antigen-specific CD8+ killer T cell responses in both tumor tissue and peripheral blood. One patient which initially displayed a slight increase in T cell immune responses developed progressive disease at 11 months into the study and received nivolumab, a PD-1 checkpoint inhibitor. Subsequently, the patient had a sustained complete response after only four doses, and continues on therapy with no evidence of disease, 16 months after initiation of nivolumab.

Detailed immune analyses found that MEDI0457 had activated HPV16-specific CD8+ T cells in the patient and the subsequent treatment with nivolumab helped to unleash the expansion of these killer T cells, which is contributing to the sustained complete response observed in this patient. Medimmune is conducting a separate phase 1/2 trial combining its PD-L1 inhibitor (durvalumab) with MEDI0457 in HPV-associated head and neck cancer patients to evaluate the clinical efficacy of the combination treatment.

Dr. Charu Aggarwal, MD, MPH, medical oncologist and assistant professor of medicine at the hospital of the University of Pennsylvania and the principal investigator of this study, said "This observation suggests that treatment with MEDI0457 prior to PD-1 inhibition can be synergistic, and increase efficacy of checkpoint inhibitors."

In Inovio’s phase 1 dose-escalation study of its synthetic optimized DNA plasmids that target hTERT, the immunotherapies were administered via Inovio’s CELLECTRA delivery device to assess the safety, tolerability, and immune effects of INO-1400 or INO-1401 (two different versions of HTERT constructs), alone or co-administered with a plasmid encoding for IL-12 (INO-9012), in 90 patients with 9 different solid tumors. Interim results presented at the conference show positive safety and tolerability data as well as the generation of hTERT-specific IFN-γ secreting T cells, suggestive of an ability to break immune tolerance.

Dr. Robert Vonderheide, MD, DPhil, Director of the Abramson Cancer Center of the University of Pennsylvania and a principal investigator for the study, said: "If successful, this vaccine platform could represent not only a novel type of immune therapy for cancer patients, but also one day offer an opportunity for immune prevention of cancer."

High levels of human telomerase reverse transcriptase (hTERT) have been reported in many tumor types such as breast, lung, and pancreas. Inovio’s hTERT therapy may prove to be a promising immuno-oncology target for the treatment of these cancers. In 2017, over 530,000 new cases of breast, lung, or pancreatic cancers were reported in the United States and over 240,000 people died from these cancers. Despite available treatments, mortality rates remain unacceptably high in these tumor types. In addition, many existing treatment modalities are associated with significant adverse events.

On November 9, 2017 LIDDS reported that it has entered a research collaboration with the Department of Laboratory Medicine at Karolinska Institute, Sweden (Press release, Lidds, NOV 9, 2017, View Source [SID1234555923]). The collaboration regards fundamental preclinical studies on formulations based on the NanoZolid technology, as well as access to risk laboratories for formulation development.

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LIDDS broadens its R&D activities to develop novel NanoZolid formulations for local tumor therapy by establishing a collaboration with Professor Mustapha Hassan and Dr. Ying Zhau at the Department of Laboratory Medicine (LABMED) at Karolinska Institute in Huddinge.

– LIDDS appreciate very much the collaboration with KI, and I am confident that the ongoing collaborative research will be fruitful and supportive to develop new efficient formulations with the NanoZolid technology. We are also pleased with the preclinical expertise that we have encountered at the Department Laboratory Medicine at Novum, states CEO of LIDDS Monica Wallter.
The collaboration includes fundamental preclinical studies and gives access to risk laboratories for formulation development with hazardous substances. Within the frame of the collaboration, novel NanoZolid formulations will be investigated prior to entering the clinical phase.