Immune Design to Report Third Quarter 2017 Financial Results & Provide Corporate Update

On October 27, 2017 Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on cancer, reported that it will report third quarter 2017 financial results and provide a corporate update after the close of U.S. financial markets on Wednesday, November 1, 2017 (Press release, Immune Design, OCT 27, 2017, View Source [SID1234521241]). Immune Design management will host a webcast and conference call at 1:30 p.m. Pacific Time / 4:30 p.m. Eastern Time on November 1, 2017.

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The live call may be accessed by dialing 844-266-9538 for domestic callers and 216-562-0391 for international callers. A live webcast of the call will be available online from the investor relations section of the company website at View Source and will be archived there for 30 days. A telephone replay of the call will be available for five days by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference code: 4793869.

10-Q – Quarterly report [Sections 13 or 15(d)]

La Jolla Pharmaceutical has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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La Jolla Pharmaceutical Company Announces Financial Results for the Three and Nine Months Ended September 30, 2017 and Recent Corporate Progress

On October 26, 2017 La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, reported financial results for the three and nine months ended September 30, 2017 and highlighted recent corporate progress (Press release, La Jolla Pharmaceutical, OCT 26, 2017, View Source [SID1234521288]).

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Recent Corporate Progress


In August 2017, La Jolla announced that the U.S. Food and Drug Administration (FDA) accepted for review the Company’s New Drug Application (NDA) for the investigational drug LJPC‑501, La Jolla’s propriety formulation of synthetic human angiotensin II, for the treatment of hypotension in adult patients with distributive or vasodilatory shock (dangerously low blood pressure with adequate cardiac function) who remain hypotensive despite fluid and vasopressor therapy (catecholamines and/or vasopressin). The review classification for the application is Priority, and the user fee goal date under the Prescription Drug User Fee Act (PDUFA) is February 28, 2018. In its letter to the Company, the FDA stated that it does not currently plan to hold an advisory committee meeting to discuss this application. The NDA for LJPC-501 is based on data from the ATHOS-3 (Angiotensin II for the Treatment of High Output Shock) multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical study of LJPC-501 in patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy, which were published by The New England Journal of Medicine in May 2017.


In September 2017, an analysis from ATHOS-3 entitled, "Baseline angiotensin levels and ACE effects in patients with vasodilatory shock treated with angiotensin II," was presented during the 30th European Society of Intensive Care Medicine Annual Congress. The pre-specified analysis showed that a relatively low angiotensin II state (as measured by the ratio of angiotensin I to angiotensin II) predicted increased mortality in patients with vasodilatory shock, suggesting that a low angiotensin II state is a negative prognostic indicator of outcomes. Furthermore, the analysis showed a statistically significant treatment effect of LJPC-501 compared to placebo on mortality in these patients with a relatively low angiotensin II state (relative risk reduction of 36%; HR=0.64; 95% CI: 0.41-1.00; p=0.047).


In September 2017, La Jolla announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) issued favorable Scientific Advice regarding the EU regulatory pathway for LJPC‑501 for the treatment of hypotension in adult patients with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. Based on this Advice, La Jolla intends to submit a Marketing Authorization Application (MAA) for LJPC-501 in the third quarter of 2018.

"The first nine months of 2017 have been exciting for La Jolla, highlighted by the positive results from ATHOS-3, the publication of these results in The New England Journal of Medicine and the FDA acceptance of our NDA for LJPC-501," said George Tidmarsh, M.D., Ph.D., President and Chief Executive Officer of La Jolla. "We look forward to building on this momentum with the preparation for the potential commercial launch of LJPC-501, if approved by the FDA, and the initiation of our pivotal study of LJPC-401 in beta thalassemia patients suffering from iron overload."

Results of Operations

As of September 30, 2017, the Company had $120.8 million in cash and cash equivalents, compared to $65.7 million of cash and cash equivalents at December 31, 2016. Cash used in operating activities for the nine months ended September 30, 2017 was $60.4 million, compared to $40.1 million for the same period in 2016. Net loss for the three and nine months ended September 30, 2017 was $26.3 million and $76.3 million, or $1.19 per share and $3.65 per share, respectively, compared to a net loss of $21.3 million and $53.3 million, or $1.23 per share and $3.10 per share, respectively, for the same periods in 2016.

BICYCLE THERAPEUTICS TO PRESENT PRECLINICAL DATA ON LEAD MOLECULE BT1718 AT THE AACR-NCI-EORTC INTERNATIONAL CANCER CONFERENCE

On October 26, 2017 Bicycle Therapeutics, a biotechnology company pioneering a new class of therapeutics based on its proprietary bicyclic peptide (Bicycle) product platform, reported that the company will present data describing the mechanism of action of BT1718, Bicycle’s lead molecule, which is being developed to target cancers of high unmet need including triple negative breast cancer and non small cell lung cancer (Press release, Bicycle Therapeutics, OCT 26, 2017, View Source [SID1234521249]). The data will be presented at the 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), held in Philadelphia, Pennsylvania from October 26 – 30, 2017.

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First Patient Treated in a Phase 1/2a Trial (Oncovirac) of Novel Oncolytic Virus TG6002 in Recurrent Glioblastoma

On October 26, 2017 Transgene (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 [SID1234521244]). 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."