On April 30, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported eight oral and poster presentations at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2018 Annual Meeting being held May 16 -19, 2018 in Chicago, IL (Press release, Rocket Pharmaceuticals, APR 30, 2018, View Source;p=RssLanding&cat=news&id=2345662 [SID1234526550]).

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"Rocket has a robust pipeline of five innovative gene therapy programs across lentiviral vector (LVV) and adeno-associated viral vector (AAV) platforms, and we are pleased that our programs to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) are featured at ASGCT (Free ASGCT Whitepaper)," said Gaurav Shah, Chief Executive Officer and President of Rocket. "Our most advanced program to treat FA is in a Phase 1/2 trial for which updated data will be presented during the Presidential Symposium. In children with FA, FANCA gene mutations enable chromosomal abnormalities that frequently lead to bone marrow failure, acute myeloid leukemia and death, with relatively toxic bone marrow transplant regimens as a principal therapy. We are hopeful that our FANCA-focused LVV gene therapy has the potential to enable broadly-applicable prevention of bone marrow failure, leading to safer and transformative outcomes."

Jonathan Schwartz, M.D., Chief Medical Officer of Rocket, added, "Our second most advanced program is our LVV gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I), which is expected to enter the clinic early next year. We are focused on the most severe form of LAD-I in which approximately 61-75% of children do not survive past the age of 2 due to life threatening infections. Based on our research, a modest correction of the defective gene encoding for the CD18 receptor can enable survival to adulthood. Both FA and LAD-I are examples of Rocket’s focus on exploring definitive therapies for patients with clearly defined monogenic diseases by targeting the underlying genetic deficit; these are disorders where a modest number of gene-corrected stem cells can make a meaningful difference for patients otherwise facing very limited treatment options. "

Oral Presentations:

Title: Engraftment and Phenotypic Correction of Hematopoietic Stem Cells in Non-Conditioned Fanconi Anemia Patients Treated with Ex Vivo Gene Therapy
Session: 330 Presidential Symposium & Presentation of the Top Abstracts
Presenter: Juan Bueren, Ph.D. Head of the Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) / CIBER-Rare Diseases / IIS-Fundación Jiménez Díaz, and principal investigator of the RP-L102 trial.
Date: Friday, May 18, 2018
Session Time: 1:15 p.m. – 3:15 p.m. Central Time
Presentation Time: 2:45 p.m. – 3:00 p.m. Central Time
Location: International Ballroom North & South

Title: Immunotoxin-Based Conditioning Facilitates Autologous Hematopoietic Stem Cell Engraftment and Multi-Lineage Development in a Fanconi Anemia Mouse Model
Session: 115 Hematopoietic Cell Therapies
Presenter: Meera Srikanthan, M.D., Seattle Children’s Hospital; Fred Hutchinson Cancer Research Center
Date: Wednesday, May 16, 2018
Session Time: 10:30 a.m. – 12:00 p.m. Central Time
Presentation Time: 10:45 a.m. – 11:00 a.m. Central Time
Location: Salon A-5

Title: Gene Editing in Fanconi Anemia Hematopoietic Stem and Progenitor Cells
Session: 302 Advances in Genome Editing in HSCs – Organized by the Hematologic and Immunologic Gene and Cell Therapy Committee
Presenter: Paula Rio, Ph.D., Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT)
Date: Friday, May 18, 2018
Session Time: 8:00 a.m. -10:00 a.m. Central Time
Presentation Time: 9:00 a.m. – 9:30 a.m. Central Time
Location: International Ballroom North

Poster Presentations:

Title: Improvements in the Transduction Conditions of Human Hematopoietic Progenitors with the CPcoRPKW-17 Therapeutic Lentiviral Vector to be Used in a Pyruvate Kinase Deficiency Gene Therapy Clinical Trial
Session: Hematologic & Immunologic Diseases I
Date: Wednesday, May 16, 2018
Time: 5:30 p.m. – 7:30 p.m. Central Time
Location: Stevens Salon C, D

Title: Leukocyte Adhesion Deficiency I: A Closer Step to a Gene Therapy Clinical Trial
Session: Hematologic & Immunologic Diseases I
Date: Wednesday, May 16, 2018
Time: 5:30 p.m. – 7:30 p.m. Central Time
Location: Stevens Salon C, D

Title: Pairs of Guide RNAs Mediate Precise Deletions on the PKLR Gene via Non Homologous End Joining Generating a Human Hematopoietic Progenitor Model of Pyruvate Kinase Deficiency
Session: Hematologic & Immunologic Diseases II
Date: Thursday, May 17, 2018
Time: 5:15 p.m. – 7:15 p.m. Central Time
Location: Stevens Salon C, D

Title: Leukocyte Adhesion Deficiency-I: A Comprehensive Review of Published Cases
Session: Hematologic & Immunologic Diseases III
Date: Friday, May 18, 2018
Time: 5:45 p.m. – 7:45 p.m. Central Time
Location: Stevens Salon C, D

Title: Towards the Gene Therapy Clinical Trial for Pyrivate Kinase Deficiency
Session: Hematologic & Immunologic Diseases III
Date: Friday, May 18, 2018
Time: 5:45 p.m. – 7:45 p.m. Central Time
Location: Stevens Salon C, D

On April 27, 2018 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the company will webcast presentations from its Investor Day, including first quarter financial results, on Friday, May 4, 2018 at 8:00 a.m. ET in New York (Press release, Agios Pharmaceuticals, APR 27, 2018, View Source [SID1234525778]).

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Presentations will be given by members of Agios’ leadership team and external speakers including:

David Schenkein, M.D., Chief Executive Officer
Scott Biller, Ph.D., Chief Scientific Officer
Chris Bowden, M.D., Chief Medical Officer
Andrew Hirsch, Chief Financial Officer and Head of Corporate Development
Steve Hoerter, Chief Commercial Officer
Darrin Miles, Vice President, IDH Program Management
Susan Pandya, M.D., Senior Medical Director, Clinical Development
Kevin Marks, Ph.D., Senior Director, Head of Cancer Biology
Maeve Lowery, M.B., B.Ch., B.A.O, Trinity College Dublin
A live webcast of the presentations can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The presentations are scheduled to begin at 8:00 a.m. ET and conclude at 12:00 p.m. ET. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

On April 27, 2018 IntelGenx Technologies Corp. (TSX VENTURE:IGX) (OTCQX:IGXT) (the "Company" or "IntelGenx") reported that its President and Chief Executive Officer, Dr. Horst Zerbe, is scheduled to present at the 2018 Bloom Burton & Co. Healthcare Investor Conference on Thursday, May 3 at 3:00 p.m. EasternTime at the Sheraton Centre Hotel Toronto (Press release, IntelGenx, APR 27, 2018, View Source;Co-Healthcare-Investor-Conference/default.aspx [SID1234525779]).

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Dr. Zerbe will provide an overview of IntelGenx’ business during the presentation and, along with the Company’s Executive Vice-President and Chief Financial Officer, Andre Godin, will be available to participate in one-on-one meetings with investors who are registered to attend the conference.

The presentation will be webcast live and archived for 90 days on the Company’s website, at www.intelgenx.com, under "Investors"

On April 27, 2018 The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency reported that it has adopted a positive opinion, recommending a change to the terms of the Marketing Authorisation for Tagrisso (osimertinib) to include the 1st-line treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with activating epidermal growth factor receptor (EGFR) mutations (Press release, AstraZeneca, APR 27, 2018, View Source [SID1234525785]). The recommendation is based on results from the Phase III FLAURA trial, which were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress and published in the New England Journal of Medicine.

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Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, said: "This positive recommendation acknowledges Tagrisso’s potential as a new 1st-line standard of care for patients with EGFR-mutated NSCLC in Europe. It reflects the strength of the FLAURA data that show Tagrisso delivered a statistically-significant and clinically-meaningful improvement in progression-free survival over the EGFR-TKI comparator arm across all pre-specified patient subgroups, including those with or without central nervous system metastases."

Safety data for Tagrisso were in line with those observed in prior clinical trials. Tagrisso was well tolerated, with fewer Grade 3 or higher adverse events (AEs) than with standard EGFR-TKIs (34% vs. 45%). In all patients, the most common adverse reactions were rash (58% [1.1% Grade ≥3] for Tagrisso vs. 78% [6.9% Grade ≥3] for the comparator arm), diarrhoea (58% [2.2% Grade ≥3] for Tagrisso vs. 57% [2.5% Grade ≥3] for the comparator arm) and dry skin (36% [<1% Grade ≥3] for Tagrisso vs. 36% [1.1% Grade ≥3] for the comparator arm).

The positive opinion from the CHMP will now be reviewed by the European Commission, which has the authority to approve medicines for the 28 EU member countries plus Iceland, Norway and Liechtenstein. Earlier this month, Tagrisso was approved in the US for the 1st-line treatment of patients with metastatic NSCLC whose tumours have EGFR mutations (exon 19 deletions or exon 21 L858R mutations). In addition to the EU, Tagrisso is under regulatory review in Japan for use in the 1st-line treatment setting with a decision anticipated in the second half of 2018. Other global health authority reviews and submissions are also ongoing.

Notes to Editors
About NSCLC

Lung cancer is the leading cause of cancer death among both men and women, accounting for about one-fifth of all cancer deaths, more than breast, prostate and colorectal cancers combined. Approximately 10-15% of patients in the US and Europe, and 30-40% of patients in Asia have EGFR-mutated (EGFRm) NSCLC. These patients are particularly sensitive to treatment with EGFR-TKIs, which block the cell-signalling pathways that drive the growth of tumour cells. Tumours almost always develop resistance to EGFR-TKI treatment, however, leading to disease progression. Approximately half of patients develop resistance to approved EGFR-TKIs such as gefitinib, erlotinib and afatinib due to the EGFR T790M resistance mutation. There is also a need for medicines with improved CNS efficacy, since approximately 25% of patients with EGFRm NSCLC have brain metastases at diagnosis, increasing to approximately 40% within two years of diagnosis.

About Tagrisso

Tagrisso (osimertinib) is a third-generation, irreversible EGFR-TKI designed to inhibit both EGFR-sensitising and EGFR T790M-resistance mutations, with clinical activity against CNS metastases. Tagrisso 40mg and 80mg once-daily oral tablets have been approved in the US and Brazil for 1st-line EGFRm advanced NSCLC, and in more than 75 countries, including the US, EU, Japan and China for patients with EGFR T790M mutation-positive advanced NSCLC. Tagrisso is also being tested in the adjuvant setting and in combination with other treatments.

About the FLAURA trial

The FLAURA trial assessed the efficacy and safety of Tagrisso 80mg once daily vs. standard-of-care EGFR-TKIs (either erlotinib [150mg orally, once daily] or gefitinib [250mg orally, once daily]) in previously-untreated patients with locally-advanced or metastatic EGFRm NSCLC. The trial was double-blinded and randomised, with 556 patients across 29 countries.

About AstraZeneca in Lung Cancer

AstraZeneca is committed to developing medicines to help every patient with lung cancer. We have three approved medicines and a growing pipeline that targets genetic changes in tumour cells and boosts the power of the immune response against cancer. Our unrelenting pursuit of science aims to deliver more breakthrough therapies with the goal of extending and improving the lives of patients across all stages of disease and lines of therapy.

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance Oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

On April 27, 2018 Sanofi reported financial results for the first quarter ended March 31, 2018 (Press release, Sanofi, APR 27, 2018, View Source [SID1234525827]).

First-quarter 2018 reflected strong Specialty Care sales offset by U.S. Lantus and sevelamer exclusivity losses

Net sales were €7,898 million, down 8.7% on a reported basis, down 0.4%(3) at CER and down 1.1% at CS/CER(4).
Sanofi Genzyme sales grew strongly, up 16.2%(5), driven by contribution from the new immunology franchise.
Vaccines sales (-0.9%) reflected strong performance in EU, offset by expected Pentaxim supply constraint in China.
CHC sales grew 2.0% supported by double-digit growth in Emerging Markets(6).
Diabetes and Cardiovascular GBU sales down 15.7%; global Diabetes franchise sales declined 10.0%.
Emerging Markets sales(6) increased 8.3%, driven by double-digit growth in China and Latin America.
2018 guidance confirmed

First-quarter 2018 business EPS(1) increased 1.4% at CER to €1.28.
First-quarter 2018 IFRS EPS was €0.81 (-82.0%) due to a gain on disposal of the Animal Health business in 2017.
Sanofi continues to expect 2018 Business EPS to grow between 2% and 5% at CER(7) barring unforeseen major adverse events. Applying the average April 2018 exchange rates, the currency impact on 2018 Business EPS is estimated to be around -7%.
Announcement of a €1.5bn share buyback program(8) expected to be completed in mid-2019

Sanofi strengthens leadership in Specialty Care through the addition of a Rare Blood Disorder franchise

Sanofi completed the acquisition of Bioverativ and consolidated its financial results from March 9.
First patient dosed with fitusiran, a novel RNAi therapeutic for hemophilia, in phase 3 ATLAS program.
Ablynx acquisition(9) will add caplacizumab for aTTP(10) (submitted in EU) and innovative Nanobody platform.
Sustaining innovation in R&D

Praluent significantly reduced the risk of cardiovascular events in high risk patients in the ODYSSEY OUTCOMES study and was associated with a lower death rate.
Dupixent supplemental BLA filed in the U.S., Japan and EU for moderate-to-severe asthma in adults and adolescents.
Cemiplimab filed in EU for metastatic cutaneous squamous cell carcinoma.
Sotagliflozin submitted in the U.S. and EU for type 1 diabetes.