On January 29, 2018 -Advaxis, Inc. (NASDAQ:ADXS), a late-stage biotechnology company focused on the discovery, development and commercialization of immunotherapy products, reported that three abstracts highlighting the Company’s Listeria-based immunotherapy in combination with antibody-based immunotherapies have been accepted for poster presentation at the upcoming 2018 Keystone Symposia on Cancer Immunotherapies: Combinations (C5) taking place March 23-27, 2018 at the Queen Elizabeth Fairmont in Montreal (Press release, Advaxis, JAN 29, 2018, View Source [SID1234523606]).

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Title:

"Agonism of the CD137 co-stimulatory pathway synergizes with the Listeria monocytogenes-based immunotherapy, axalimogene filolisbac, to promote durable tumor regression in a murine HPV+ tumor model"

Poster Number:

2001
Session:

Poster Session 2
Date:

March 25, 2018
Presenter:

Rachelle E. Kosoff, Ph.D., Senior Scientist, Advaxis, Inc.

Title:

"Combination TIGIT blockade with Listeria-based immunotherapy enhances antigen-specific antitumor immunity"

Poster Number:

3015
Session:

Poster Session 2
Date:

March 25, 2018
Presenter:

Daniel O. Villarreal, Ph.D., Principal Scientist, Advaxis, Inc.

Title:

"Targeting of CCR8 induces antitumor activity as a monotherapy that is further enhanced in combination with a Listeria-based immunotherapy"

Poster Number:

3037
Session:

Poster Session 2
Date:

March 25, 2018
Presenter:

Daniel O. Villarreal, Ph.D., Principal Scientist, Advaxis, Inc.

"We are delighted to present these promising data before an audience of researchers and clinicians dedicated to elucidating the best possible combinations of targeted immunotherapies, to develop better treatments for patients with cancer. The data being presented highlight encouraging preclinical results, which show synergistic effects of a variety of antibody-based immunotherapies with our Lm Technology platform," said Robert G. Petit, Ph.D., Executive Vice President and Chief Scientific Officer of Advaxis.

On January 29, 2018 Horizon Pharma plc (Nasdaq:HZNP) reported that its fourth-quarter and full-year 2017 financial results will be released on Wednesday, Feb. 28, 2018 (Press release, Horizon Pharma, JAN 29, 2018, View Source [SID1234523604]). Following the announcement, Horizon’s management will host a live webcast at 8 a.m. Eastern Time to review the Company’s financial and operating results.

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The live webcast and replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes before the live webcast to ensure adequate time for any software download that may be needed to access the webcast.

On January 29, 2018 Ipsen (Euronext: IPN; ADR: IPSEY) reported that Sotirios
G. Stergiopoulos, MD, has been appointed as Chief Medical Officer (Press release, Ipsen, JAN 29, 2018, View Source [SID1234523600]).

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Dr Stergiopoulos joined Ipsen in January 2017 as Senior Vice President, Head of Global Medical Affairs (GMA) and will retain this
position in addition to the role as the new Chief Medical Officer within the company. Dr Stergiopoulos reports to Alexandre Lebeaut, MD, Executive Vice-President R&D and Chief Scientific Officer.

Dr Lebeaut commented, "Sotirios has extensive experience in directing global medical affairs
strategies and a strong expertise in oncology drug development that includes chemotherapy,
immunology drugs and targeted agents across various tumor indications. We are delighted to appoint
him to an expanded role through which he will make the voice of the patient heard at the highest levels
of the organization and represent the company externally as its primary medical representative."

Prior to joining Ipsen, Sotirios was Vice President, Head of Global Medical Affairs Oncology at Baxalta (now Shire, Cambridge, MA), Executive Medical Director Oncology US Medical Affairs at Celgene Corporation (Summit, NJ), Senior Global Brand Medical Director Oncology at Novartis Pharmaceuticals (East Hanover, NJ) and Director Medical Affairs Oncology at Bayer Healthcare
(Montville, NJ).

Dr. Stergiopoulos added, "I am delighted to take on this leading role at Ipsen. This is an exciting
time for Ipsen with significant growth and evolution. Together with our experienced leadership team,
I look forward to helping our company continue to bring innovative new medicines to our patients."

Dr. Stergiopoulos is a physician executive with significant experience in the Pharmaceutical/Biotech industry, especially in Oncology. He has held appointments as an Attending Physician and trainee in institutions such as Albert Einstein College of Medicine, Harvard Medical School and the National Institutes of Health. He holds a Masters in Biotechnology Enterprise and Entrepreneurship (MBEE) from The Johns Hopkins University and a Medical Degree from Poznan University of Medical
Sciences (Poland). Sotirios is a Fellow of the American College of Physicians, the New York Academy of Medicine as well as the Royal Society of Medicine (UK). He is also a Member of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) and of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).
In October 2017 Dr. Stergiopoulos was appointed President of the Board of Governors for the Accreditation Council for Medical Affairs (ACMA); a body whose mission is to establish, certify, and maintain the competencies of qualified medical and scientific professionals who have a focus in Medical Affairs within the pharmaceutical & biotechnology industries.

On January 29, 2018 Santhera Pharmaceuticals (SIX: SANN) reported preliminary, unaudited key financial figures for 2017. The Company reports an increase of 21% year-on-year in net revenues to CHF 22.9 million (2016: CHF 19.0 million) from sales of its lead product Raxone for the treatment of Leber’s hereditary optic neuropathy (LHON) (Press release, Santhera Pharmaceuticals, JAN 29, 2018, View Source [SID1234523599]). Freely available liquid funds by year-end amounted to CHF 58.2 million (December 31, 2016: CHF 49.8 million). Following the recent negative opinion by the Committee for Medicinal Products for Human Use (CHMP) on its Marketing Authorization Application (MAA) for Raxone in Duchenne muscular dystrophy (DMD), the Company will work with regulators and clinical experts to prepare a refiling as soon as possible. Other development projects progressed according to plan.

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Financial and Commercial Highlights

In 2017, Santhera reported net revenues from product sales of Raxone for LHON of CHF 22.9 million which corresponds to a growth of 21% year-on-year (2016: CHF 19.0 million). The roll-out of Raxone in the approved indication is progressing as planned and the product is currently sold in 20 European countries.
By end of 2017, full reimbursement for Raxone in LHON was achieved for 8 European countries. In an additional 12 European countries, Raxone availability is currently governed by special reimbursement schemes.
Commercial operations in the regional country clusters in Europe were expanded to support marketing of Raxone for LHON. In February, US operations were established in the Boston metropolitan area. The US team is currently focused on expanding relationships with patient advocacy groups and clinicians, supporting ongoing studies in the US, assembling a NDA filing for Raxone in DMD and preparing for market entry.
In February 2017, Santhera successfully placed CHF 60 million senior unsecured convertible bonds due 2022. These funds are being primarily used for the commercialization of Raxone in the currently approved indication LHON, for investment into ongoing and further clinical trials with Raxone in DMD to facilitate regulatory filings, to advance the pipeline and for other corporate and business development purposes.
As of December 31, 2017, freely available liquid funds (cash and cash equivalents and short-term financial assets) amounted to CHF 58.2 million (December 31, 2016: CHF 49.8 million). In addition, the Company reported CHF 7.5 million of restricted cash designated for the interest payments related to the convertible bonds during the first three years.
The Company had 6,288,555 shares outstanding as of December 31, 2017.
"We are pleased about Santhera’s strong commercial progress in 2017. At the same time, we are disappointed about the regulatory decision concerning the approval of Raxone in DMD," commented Thomas Meier, PhD, CEO of Santhera. "Our priorities for 2018 are clear: in the interest of patients and convinced of the treatment benefits of Raxone in DMD, we will work with clinical experts, patient advocacy groups and regulators to prepare for a refiling to enable treatment of patients with abnormal respiratory function and not taking glucocorticoids. In parallel, we will push ahead with the commercialization of Raxone in LHON and the advancement of our development pipeline."

Pipeline and Regulatory Matters and Outlook

On January 26, 2018, Santhera announced that the European Medicines Agency’s CHMP had maintained its negative opinion on the Type II extension application for Raxone (idebenone) in DMD following a re-examination procedure. The CHMP concluded that an approval for Raxone in DMD applied as Type II variation of the existing marketing authorization cannot be granted at the present time based on the currently existing evidence base. Santhera remains fully committed to addressing this unmet need and is convinced of the treatment benefits of Raxone in DMD. The Company intends to strengthen the clinical data package for Raxone in preparation of a refiling of a Marketing Authorization Application (MAA) in Europe. In light of the CHMP’s opinion, Santhera has withdrawn the corresponding regulatory application in Switzerland, with the intention to refile at a later stage.
In June 2017, as the first drug for DMD, Raxone was granted a positive scientific opinion by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) through the Early Access to Medicines Scheme (EAMS). This allows patients with DMD and respiratory function decline, who are not taking glucocorticoids and meet the criteria defined under this scheme, to gain access to Raxone already prior to marketing approval. The Company will seek consultations with MHRA concerning the continuation of the program.
Santhera’s randomized, double-blind, placebo-controlled Phase III trial (SIDEROS) designed to assess the efficacy of Raxone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid therapy is enrolling patients. The trial is currently recruiting patients in 56 centers in Europe and the US. The study duration is 18 months and completion of the trial is expected in H2 2020. If successful, this study will provide data that support use of Raxone in all DMD patients experiencing respiratory decline irrespective of their glucocorticoid use.
In December 2017, Santhera announced the launch of an educational disease awareness campaign for the DMD community in the U.S. The "Take a Breath DMD" campaign, TakeABreathDMD.com, underscores the importance of respiratory care and also helps people living with DMD and their families receive information to help manage respiratory complications, including information about breathing, coughing and pulmonary care.
The Phase I/II trial (IPPoMS) evaluating the safety and effectiveness of using Raxone to treat primary progressive multiple sclerosis (PPMS) has been completed. Top line study results of the trial which was carried out in collaboration with the U.S. National Institute of Neurological Disorders and Stroke (NINDS) are expected to be announced in Q1 2018.
The Phase I trial (CALLISTO) evaluating the safety and tolerability of omigapil in pediatric and adolescent patients with congenital muscular dystrophy (CMD) was also conducted in collaboration with NINDS. The study has been completed and the announcement of top line results is planned for early Q2 2018. Omigapil has a Fast Track Designation and a grant from the FDA’s Office of Orphan Products Development.
Guidance

For 2017, Santhera anticipates a net result of CHF -50 to -55 million.
For 2018, the Company expects net sales of Raxone for the currently approved indication LHON to reach CHF 28 to 30 million.

On January 26, 2018 The U.S. Food and Drug Administration reported the approval of Lutathera (lutetium Lu 177 dotatate) for the treatment of a type of cancer that affects the pancreas or gastrointestinal tract called gastroenteropancreatic neuroendocrine tumors (GEP-NETs) (Press release, US FDA, JAN 26, 2018, View Source [SID1234523587]). This is the first time a radioactive drug, or radiopharmaceutical, has been approved for the treatment of GEP-NETs. Lutathera is indicated for adult patients with somatostatin receptor-positive GEP-NETs.

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"GEP-NETs are a rare group of cancers with limited treatment options after initial therapy fails to keep the cancer from growing," said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "This approval provides another treatment choice for patients with these rare cancers. It also demonstrates how the FDA may consider data from therapies that are used in an expanded access program to support approval for a new treatment."

GEP-NETs can be present in the pancreas and in different parts of the gastrointestinal tract such as the stomach, intestines, colon and rectum. It is estimated that approximately one out of 27,000 people are diagnosed with GEP-NETs per year.

Lutathera is a radioactive drug that works by binding to a part of a cell called a somatostatin receptor, which may be present on certain tumors. After binding to the receptor, the drug enters the cell allowing radiation to cause damage to the tumor cells.

The approval of Lutathera was supported by two studies. The first was a randomized clinical trial in 229 patients with a certain type of advanced somatostatin receptor-positive GEP-NET. Patients in the trial either received Lutathera in combination with the drug octreotide or octreotide alone. The study measured the length of time the tumors did not grow after treatment (progression-free survival). Progression-free survival was longer for patients taking Lutathera with octreotide compared to patients who received octreotide alone. This means the risk of tumor growth or patient death was lower for patients who received Lutathera with octreotide compared to that of patients who received only octreotide.

The second study was based on data from 1,214 patients with somatostatin receptor-positive tumors, including GEP-NETS, who received Lutathera at a single site in the Netherlands. Complete or partial tumor shrinkage was reported in 16 percent of a subset of 360 patients with GEP-NETs who were evaluated for response by the FDA. Patients initially enrolled in the study received Lutathera as part of an expanded access program. Expanded access is a way for patients with serious or immediately life-threatening diseases or conditions who lack therapeutic alternatives to gain access to investigational drugs for treatment use.

Common side effects of Lutathera include low levels of white blood cells (lymphopenia), high levels of enzymes in certain organs (increased GGT, AST and/or ALT), vomiting, nausea, high levels of blood sugar (hyperglycemia) and low levels of potassium in the blood (hypokalemia).

Serious side effects of Lutathera include low levels of blood cells (myelosuppression), development of certain blood or bone marrow cancers (secondary myelodysplastic syndrome and leukemia), kidney damage (renal toxicity), liver damage (hepatotoxicity), abnormal levels of hormones in the body (neuroendocrine hormonal crises) and infertility. Lutathera can cause harm to a developing fetus; women should be advised of the potential risk to the fetus and to use effective contraception. Patients taking Lutathera are exposed to radiation. Exposure of other patients, medical personnel, and household members should be limited in accordance with radiation safety practices.

Lutathera was granted Priority Review, under which the FDA’s goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition. Lutathera also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.