On October 29, 2018 Principia Biopharma Inc. (Nasdaq: PRNB), a clinical-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology, reported the appointment of industry veteran Dolca Thomas, M.D. as its Chief Medical Officer (Press release, Principia Biopharma, OCT 29, 2018, View Source [SID1234530295]). Dr. Thomas joins Principia from Roche, where she was Vice President and Global Head of Translational Medicine for Immunology, Inflammation, and Infectious Disease. She brings approximately 15 years of industry and medical experience to Principia. Steve Gourlay, MBBS, FRACP, Ph.D. will remain with the company as a senior medical advisor through mid-year 2019.

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"We are very excited with the addition of Dolca to the Principia team, particularly as we pursue an expanding scope of clinical development activities including initiating our pivotal Phase 3 trial for PRN1008 in patients with pemphigus. Dolca is a seasoned pharma executive with broad and successful experience in executing late stage programs, specifically in immunology," said Martin Babler, Chief Executive Officer of Principia. "I also want to thank Steve for his leadership, dedication and contributions to Principia over the past five years. Steve led the clinical development of three different molecules, including taking PRN1008 from its first-in-human trial through design and preparation for Phase 3. We are pleased that he will continue to support the company as a senior medical advisor through the first half of next year."

"I am impressed with Principia’s approach to BTK inhibition in autoimmune disease and believe that application of the company’s proprietary Tailored Covalency platform may be applicable across a wide range of autoimmune and inflammatory conditions," said Dr. Thomas. "I have been involved in more than a dozen immunology product candidates, and I look forward to contributing to the potential success of Principia’s pipeline assets and moving the company towards late-stage clinical development."

Dr. Thomas brings 15 years of industry and medical experience with strategic and operational responsibility for clinical development, pharmacovigilance, and safety and medical affairs of approximately two dozen pharmaceutical products. Most recently, Dr. Thomas was Vice President and Global Head of Translational Medicine for Immunology, Inflammation, and Infectious Disease at Roche, where she was responsible for advancing multiple product candidates through clinical development. Prior to Roche, Dr. Thomas held roles of increasing responsibility at Pfizer, including Vice President of Clinical Development and Clinical Immunophenotyping, and Vice President and Chief Development Officer of the Biosimilars Research and Development Unit where she was responsible for all stages of development of multiple assets. Dr. Thomas began her industry career at Bristol-Myers Squibb as Director of Global Clinical Development in Immunology, where she was involved in the development and approval of belatacept.

Dr. Thomas has a B.A. in sociology from Cornell University, and received her M.D. degree from Cornell University. Dr. Thomas completed her residency in internal medicine, in addition to her post-doctoral training in nephrology and transplantation, at New York- Presbyterian Hospital, Weill Cornell Medical Center.

Dr. Thomas represented both Bristol-Myers Squibb and Pfizer on the Board of Directors of the Progressive Multifocal Leukoencephalopathy (PML) Consortium, a cross-industry consortium aimed at identifying more effective methods of predicting, preventing, and developing future treatments for PML. Dr. Thomas has many past collaborations with the Juvenile Diabetes Research Foundation (JDRF) where she has been the recipient of multiple JDRF research grants and awards. In addition, she served on Board of Directors for the NYC JDRF chapter.

On October 29, 2018 Novartis reported it was awarded the 2018 Prix Galien USA Award for Best Biotechnology Product for Kymriah (tisagenlecleucel) following its first-in-class approval by the US Food and Drug Administration (FDA) for the treatment of children and young adults with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL) (Press release, Novartis, OCT 29, 2018, View Source [SID1234530441]). Kymriah is a ground-breaking one-time treatment that uses a patient’s own T cells to fight cancer, and the only chimeric antigen receptor T cell (CAR-T) therapy approved for two distinct B-cell malignancies1. The award, which recognizes excellence in scientific innovation that improves the state of human health, was presented at a ceremony in New York City.

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"It is a great honor to receive this prestigious award for Kymriah," said Liz Barrett, CEO, Novartis Oncology. "The impact for patients with aggressive blood cancers who previously had limited treatment options is our greatest reward and drives us to continue pioneering efforts to reimagine the way cancer is treated."

The historic approval of Kymriah was a result of collaboration with researchers at University of Pennsylvania (Penn) who demonstrated the first successful use of gene transfer therapy to use the body’s immune cells to fight cancer. Novartis further researched this emerging class of drug, initiating the first global CAR-T trials and in August 2017, became the first company to gain a regulatory approval of a CAR-T cell therapy. This year, Kymriah has also been approved in the United States for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL); and for the treatment of pediatric and young adult patients with r/r ALL and adults with r/r DLBCL in the European Union, Canada and Switzerland. Of note, Kymriah is not approved for the treatment of patients with primary central nervous system lymphoma1.

Both B-cell ALL and DLBCL are aggressive malignancies, and for patients who relapse or don’t respond to therapy, there are limited treatment options2,3.

Novartis Prix Galien Awards
Considered "the pharmaceutical industry’s Nobel Prize," the Prix Galien recognizes excellence in scientific innovation that improves the state of human health, and acknowledges the technical, scientific and clinical research skills necessary to develop such innovative medicines. As a true testament to the company’s commitment to reimagine medicine, since 1970, Novartis has received more than 40 national Prix Galien awards in 15 countries for innovative therapies including Gleevec (imatinib mesylate), Kymriah (tisagenlecleucel), Parlodel (bromocriptine mesylate), Rimactane (rifampin), Sandimmune (cyclosporine), Sandostatin (octreotide acetate), Simulect (basiliximab) and Visudyne (verteporfin)4.

About Kymriah (tisagenlecleucel)
Kymriah is an innovative immunocellular therapy, manufactured individually for each patient by reprogramming the patient’s own immune system cells. Kymriah is the only approved CAR-T cell therapy manufactured using the 4-1BB costimulatory domain, which is critical for full activation of the therapy, enhancement of cellular expansion and durable persistence of the cancer-fighting cells. In 2012, Novartis and Penn entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including Kymriah, for the investigational treatment of cancers.

Kymriah (tisagenlecleucel) US Important Safety information
Kymriah may cause side effects that are severe or life-threatening, such as Cytokine Release Syndrome (CRS) or Neurological Toxicities. Patients with CRS may experience symptoms including difficulty breathing, fever (100.4◦F/38◦C or higher), chills/shaking chills, severe nausea, vomiting and diarrhea, severe muscle or joint pain, very low blood pressure, or dizziness/lightheadedness. Patients may be admitted to the hospital for CRS and treated with other medications.

Patients with neurological toxicities may experience symptoms such as altered or decreased consciousness, headaches, delirium, confusion, agitation, anxiety, seizures, difficulty speaking and understanding, or loss of balance. Patients should be advised to call their healthcare provider or get emergency help right away if they experience any of these signs and symptoms of CRS or neurological toxicities.

Because of the risk of CRS and neurological toxicities, Kymriah is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called Kymriah REMS.

Serious allergic reactions, including anaphylaxis, may occur after Kymriah infusion.

Kymriah can increase the risk of life-threatening infections that may lead to death. Patients should be advised to tell their healthcare provider right away if they develop fever, chills, or any signs or symptoms of an infection.

Patients may experience prolonged low blood cell counts (cytopenia), where one or more types of blood cells (red blood cells, white blood cells, or platelets) are decreased. The patient’s healthcare provider will do blood tests to check all of their blood cell counts after treatment with Kymriah. Patients should be advised to tell their healthcare provider right away if they get a fever, are feeling tired, or have bruising or bleeding.

Patients may experience hypogammaglobulinemia, a condition in which the level of immunoglobulins (antibodies) in the blood is low and the risk of infection is increased. It is expected that patients may develop hypogammaglobulinemia with Kymriah, and may need to receive immunoglobulin replacement for an indefinite amount of time following treatment with Kymriah. Patients should tell their healthcare provider about their treatment with Kymriah before receiving a live virus vaccine.

After treatment with Kymriah, patients will be monitored lifelong by their healthcare provider, as they may develop secondary cancers or recurrence of their cancer.

Patients should not drive, operate heavy machinery, or do other dangerous activities for eight weeks after receiving Kymriah because the treatment can cause temporary memory and coordination problems, including sleepiness, confusion, weakness, dizziness, and seizures.

Some of the most common side effects of Kymriah are difficulty breathing, fever (100.4°F/38°C or higher), chills/shaking chills, confusion, severe nausea, vomiting and diarrhea, severe muscle or joint pain, very low blood pressure, dizziness/lightheadedness, and headache. However, these are not all of the possible side effects of Kymriah. Patients should talk to their healthcare provider for medical advice about side effects.

Prior to a female patient starting treatment with Kymriah, their healthcare provider may do a pregnancy test. There is no information available for Kymriah use in pregnant or breast-feeding women. Therefore, Kymriah is not recommended for women who are pregnant or breast feeding. Patients should talk to their healthcare provider about birth control and pregnancy.

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

After receiving Kymriah, patients should be advised that some commercial HIV tests may cause a false-positive test result. Patients should also be advised not to donate blood, organs, or tissues and cells for transplantation after receiving Kymriah.

On October 29, 2018 Aura Biosciences, a leader in the development of novel targeted therapies in ocular oncology, reported that updated clinical data from its Phase 1b/2 clinical trial evaluating the safety and efficacy of light-activated AU-011, the Company’s lead product candidate for the primary treatment of choroidal melanoma, was highlighted in an oral presentation at the American Academy of Ophthalmology (AAO) 2018 Annual Meeting being held October 27-30, 2018, at McCormick Place in Chicago (Press release, Aura Biosciences, OCT 29, 2018, View Source [SID1234530245]).

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Brian P. Marr, M.D. , Director, Division of Ophthalmic Oncology, New York-Presbyterian/Columbia University Medical Center, and principal investigator of the study, gave the oral presentation titled, "One-Year Results of a Phase 1b/2 Open-Label Clinical Trial of AU-011 for the Treatment of Primary Choroidal Melanoma." This open-label, multicenter trial is designed to investigate single and multiple ascending doses of light-activated AU-011 in approximately 36 adult subjects with clinically diagnosed primary choroidal melanoma.

The data presented at the meeting show that multiple administrations of light-activated AU-011 are well-tolerated with no related serious adverse events, severe adverse events or dose-limiting toxicities observed. Drug related adverse events were all expected and included anterior chamber inflammation, posterior chamber inflammation and increase in intraocular pressure, but all were manageable with standard-of-care treatments and resolved without clinical sequelae. Notably, the posterior inflammation appears to originate within and/or around the tumor which is consistent with AU-011’s mechanism of action of acute tumor necrosis.

Treatment with light-activated AU-011 achieved preservation of best corrected visual acuity (BCVA) with a mean change of -1.06 letters at 6 months and a mean change of -0.75 letters at 12 months. BCVA was preserved even in high risk patients with tumors close to the fovea or the optic disk, a factor that typically correlates with a higher risk of irreversible severe vision loss following radioactive treatments. Importantly, all patients (100%) achieved stable disease at the prespecified preliminary efficacy endpoint at 3 months. Biological activity has been confirmed with long term tumor control in those patients with documented growth before treatment, reduction in tumor thickness and localized inflammation around the tumor. An expansion cohort of the study is currently underway. The Company plans to initiate a pivotal Phase 3 clinical program following the Phase 1b/2 study.

"The currently available treatments for choroidal melanoma come with the risk of severe vision loss, especially for patients with melanomas that are located close to the fovea or optic disk," commented Dr. Marr. "These 12-month data demonstrate that light-activated AU-011 continues to be well-tolerated, including with multiple administrations, has evidence of tumor control and preservation of visual acuity. We look forward to executing on the expansion phase of the study and generating more clinical data for this innovative targeted therapy."

"To date, light-activated AU-011 has shown a compelling degree of tolerability and vision preservation, especially given the alternative radioactive treatment options for choroidal melanoma," said Cadmus Rich, M.D., Chief Medical Officer of Aura. "We believe that a minimally invasive, non-radiation-based treatment option that enables early intervention while preserving vision has the potential to transform the therapeutic landscape for this difficult to treat, often deadly form of melanoma. We are actively preparing the Phase 3 study designs and look forward to initiating this pivotal program."

About Choroidal Melanoma

Choroidal melanoma is a rare and aggressive type of eye cancer. Choroidal melanoma is the most common primary ocular tumor and develops in the uveal tract of the eye. No targeted therapies are available at present, and current radiotherapy treatments can be associated with severe visual loss and other long-term sequelae such as dry eye, glaucoma, cataracts and radiation retinopathy. The most common current treatment is plaque radiotherapy, which involves surgical placement of a radiation device on the exterior of the eye over the tumor. The alternative is enucleation, or total surgical removal of the eye. Choroidal melanoma metastasizes to the liver in about 40-50 percent of cases in the long term (source: OMF), and only 15 percent of patients whose melanoma has metastasized survive beyond five years after diagnosis (source: ACS).

About Light-Activated AU-011

AU-011 is a first-in-class targeted therapy in development for the primary treatment of choroidal melanoma. The therapy consists of proprietary viral-like particle bioconjugates (VPB) that are activated with an ophthalmic laser. The VPBs bind selectively to unique receptors on cancer cells in the eye and are derived from technology originally pioneered by Dr. John Schiller of the Center for Cancer Research at the National Cancer Institute (NCI), recipient of the 2017 Lasker-DeBakey Award. Upon activation with an ophthalmic laser, the drug rapidly and specifically disrupts the cell membrane of tumor cells while sparing key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. AU-011 can be delivered using equipment commonly found in an ophthalmologist’s office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians. AU-011 for the treatment of choroidal melanoma has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration and is currently in clinical development.

On October 29, 2018 Protalix BioTherapeutics, Inc. (NYSE American:PLX, TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx, reported that it will report third quarter 2018 financial results and provide a corporate update on Wednesday, November 7, 2018 at 8:30 am ET (Press release, Protalix, OCT 29, 2018, View Source;p=RssLanding&cat=news&id=2373839 [SID1234530296]).

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To participate in the conference call, please dial the following numbers prior to the start of the call: United States: +1-844-358-6760; International: +1-478-219-0004. Conference ID number 8567317.

The conference call will also be broadcast live and available for replay for two weeks on the Company’s website, www.protalix.com, in the Events Calendar of the Investors section. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.

On October 29, 2018 Neuralstem, Inc. (Nasdaq:CUR), a biopharmaceutical company focused on developing novel treatments for nervous system diseases, reported that it has entered into definitive agreements with institutional investors for the purchase of 3,000,000 shares of its common stock, at a purchase price per share of $0.70, in a registered direct offering (Press release, Neuralstem, OCT 29, 2018, View Source [SID1234530381]). Additionally, Neuralstem has also agreed to issue to the investors unregistered warrants to purchase up to 3,000,000 shares of its common stock. The closing of the offering is expected to take place on or about October 29, 2018, subject to the satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The warrants have an exercise price of $0.75 per share of common stock, will be exercisable commencing six months following the issuance date and will expire five and one-half years from the issuance date.

The gross proceeds to Neuralstem, before deducting placement agent fees and other offering expenses, are expected to be $2.1 million. Neuralstem intends to use the net proceeds from this offering to further its clinical and preclinical programs, and for general working capital.

The shares of common stock (but not the warrants or the shares of common stock underlying the warrants) are being offered by Neuralstem pursuant to a "shelf" registration statement on Form S-3 that was filed and declared effective by the Securities and Exchange Commission ("SEC") on June 23, 2017 and the base prospectus contained therein (File No. 333-218608). The offering of the shares of common stock will be made only by means of a prospectus supplement and accompanying base prospectus that form a part of the registration statement.

A final prospectus supplement and accompanying base prospectus relating to the shares of common stock being offered will be filed with the SEC. Copies of the final prospectus supplement and accompanying base prospectus may be obtained, when available, on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, New York 10022, by phone at 646-975-6996 or e-mail at [email protected].

The warrants described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act"), and Regulation D promulgated thereunder and, along with the shares of common stock underlying the warrants, have not been registered under the Act, or applicable state securities laws. Accordingly, the warrants and underlying shares of common stock may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws.

This press release shall not constitute an offer to sell, or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction