On April 30, 2018 Altum Pharmaceuticals Inc. ("Altum" or "Altum Pharmaceuticals") reported that it has completed the acquisition of Lexi Pharma Inc. ("Lexi Pharma"), a Canadian pharmaceutical company focused on the development of AP-002, a new chemical entity (NCE) small molecule with an active US FDA IND (Investigational New Drug application) (Press release, Altum Pharmaceuticals, APR 30, 2018, View Source [SID1234598056]). AP-002, is a novel molecule, that selectively targets bone resorption while also demonstrating anti-tumor activity. Preclinical studies have shown that AP-002 selectively inhibits osteoclast differentiation and bone resorption with a novel mechanism of action distinct from other anti-bone resorption agents.

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The acquisition provides Altum with an enhanced pipeline by adding potential therapies for bone and oncology disorders, as well as, the addition of a world class management team. Lexi Pharma team members have collectively filed 15 New Drug Applications (NDAs) and have been involved in the development and launch of successfully marketed products including: Byetta, Bydureon, Camptosar, Dacogen, Ellence, Emcyt, Erbitux, Folotyn, Panretin, Symlin, Sutent, Targretin, Vidaza, Velcade, and Zarnestra.

"We are delighted to have the Lexi team join us at Altum to help us realize the goal of addressing unmet needs for women’s health with novel therapeutic approaches. As part of the merger, we are thrilled to have experienced and motivated female C-level executives joining Altum to help implement our novel strategies," said Altum Pharmaceuticals’ Chief Executive Officer, Dr. Ahmad Doroudian. "The merger represents another important step in the execution of our strategy to become a global player in women’s health."

As part of the Altum’s merger with Lexi Pharma:

Ali Ardakani, the Chief Executive Officer of Lexi Pharma, will join Altum’s board.
Dr. Angela Ogden, will join Altum as the new Chief Medical Officer.
Dr. Gina Stetsko will join Altum as the new Chief Development Officer.
Dr. Hooshmand Sheshbaradaran, will join Altum as Chief Operating Officer.
Lexi Pharma’s Chief Medical Officer Dr. Angela Ogden said, "We are excited to become part of Altum Pharmaceuticals. Our combined pipeline allows us to focus on specific therapeutic needs of women, such as, HPV and bone disorders. Our experienced and cohesive development team looks forward to bringing new treatment paradigms to women around the world."

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On April 30, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported the successful results of long-term studies on the type of cells that will be encapsulated using its Cell-in-a-Box technology and then combined with low doses of the cancer prodrug ifosfamide for the treatment of patients with locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC) (Press release, PharmaCyte Biotech, APR 30, 2018, View Source [SID1234525857]).

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These studies, which have taken over three years to complete, are needed to develop the comprehensive dossier of information concerning the genetically engineered human cells that are encapsulated for use in PharmaCyte’s therapy for LAPC. This work is required by the U.S. Food and Drug Administration ("FDA") to be included in PharmaCyte’s Investigational New Drug Application (IND) PharmaCyte is in the process of preparing for submission to the FDA.

The long-term studies were designed to demonstrate that the cells did not alter their properties after long periods in cell culture. The successful nature of these studies ensured that the properties of the product will not change over time and that product manufactured now will be the same as product manufactured in the future.

Kenneth L Waggoner, Chief Executive Officer of PharmaCyte, said, "We are delighted with the results of these long-term studies. They are crucial to demonstrating the long-term functionality and other characteristics of our genetically engineered cells, the ‘Active Pharmaceutical Ingredient (API),’ we plan to encapsulate as part of our therapy for LAPC. We have shown the API continues to function as designed over lengthy periods of time. We now have a large dossier of data to reference in our IND that documents each of the important characteristics of our API."

The characteristics of the cells that were tested included their genetic composition, their metabolic activity and the amount of the active enzyme (cytochrome P450 2B1) produced that converts the inactive prodrug, ifosfamide, to its active cancer-killing form. Importantly, the studies also showed that the cells were stable in the absence of the use of "selection agents," meaning that the cytochrome P450 gene that had been introduced into the cells was stably integrated in the genome of the cells.

On April 30, 2018 Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) reported that it will host a webcast and conference call to discuss the Company’s financial results for the quarter ended March 31, 2018, and provide a general business overview on Wednesday, May 9, 2018, at 4:30 p.m. ET (1:30 p.m. PT) (Press release, Portola Pharmaceuticals, APR 30, 2018, View Source;p=RssLanding&cat=news&id=2345725 [SID1234525859]).

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Conference Call Details
The live conference call on Wednesday, May 9, 2018, at 4:30 p.m. ET, can be accessed by phone by calling (844) 452-6828 from the United States and Canada or 1 (765) 507-2588 internationally and using the passcode 7068059. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call.

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