On December 12, 2018 Five Prime Therapeutics, Inc. (NASDAQ: FPRX), a clinical-stage biotechnology company discovering and developing innovative immuno-oncology protein therapeutics, reported that Aron Knickerbocker, Chief Executive Officer, will present at the 37th Annual J.P. Morgan Healthcare Conference, Wednesday, January 9, 2019 at 2:00 pm PT (Press release, Five Prime Therapeutics, DEC 12, 2018, View Source [SID1234532039]).

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The presentation will be webcast and may be accessed at the "Events & Presentations" section of the Company’s website at View Source Five Prime will maintain an archived replay of the webcast on its website for 30 days after the conference.

On December 12, 2018 Aptose Biosciences Inc. (NASDAQ: APTO, TSX: APS) reported highlights from a Key Opinion Leader (KOL) breakfast featuring a presentation by Brian Druker, M.D., Professor of Medicine at the School of Medicine at Oregon Health & Science University (OHSU), Director of OHSU’s Knight Cancer Institute, and Aptose management team (Press release, Aptose Biosciences, DEC 12, 2018, View Source [SID1234532023]).

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The webcast of the presentation will be archived on Aptose’s website here.

William G. Rice, Ph.D., Chairman, President and Chief Executive Officer, first provided a recap on CG-806, Aptose’s highly potent pan-FLT3/pan-BTK inhibitor, and included the following key highlights:

CG-806 has a well-differentiated mechanism of action, selectively and potently inhibiting kinase clusters that include all forms of FLT3 and BTK, as well as rescue pathways that can allow resistance to other drugs, and avoiding other kinase clusters associated with toxicity
CG-806 demonstrated superior potency on acute myeloid leukemia (AML) patient cells relative to all other FLT3 inhibitors
CG-806 demonstrated superior potency on B-cell patient cells relative to ibrutinib
In preclinical mouse model studies, CG-806 induced rapid and sustained tumor eradication with no observed toxicity
CG-806’s safety profile remains clean: New results, recently presented at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting from 28-day GLP toxicity and toxicokinetic studies, continue to demonstrate a highly favorable safety profile with no adverse findings to date
Dr. Druker discussed the evolution of kinase inhibitors as anticancer drugs, reviewed the current treatment landscape in AML and B-cell cancers, emphasizing the medical needs for these patient populations, and highlighted his experience with CG-806 alone and in combination to potentially address these medical needs. Key highlights:

CG-806 potently killed primary malignant cells from patients with diverse hematologic malignancies
CG-806 demonstrated superior killing potency on cells from AML patients compared to five other FLT3 inhibitors
AML patient cells with the FLT3-ITD mutation, found in approximately 30% of AML patients, were highly sensitive to CG-806
Just presented at ASH (Free ASH Whitepaper), AML patient cells with the IDH-1 mutation were unexpectedly sensitive to CG-806, a new finding that further broadens CG-806’s potential use and potential paths for rapid development
CG-806 data demonstrated broad and superior killing potency compared to ibrutinib, which is the current standard of care for B-cell malignancies, on cells from patients with CLL and other B-cell cancers
Drug combination studies with CG-806 and venetoclax on patient bone marrow cells suggest the combination may become the preferred drug combination for patients with AML, CLL, ALL and other hematologic malignancies
"CG-806 is unlike any molecule we have investigated, and it is more than just a FLT3 and BTK inhibitor," said Dr. Druker. "806 has the unusual ability to suppress key driver and rescue pathways and overcome the resistance that develops with other kinase inhibitors, and it has demonstrated potent activity against actual primary cells from patients with hematologic malignancies. We are hopeful that clinical testing will prove it to be a new treatment for a patient population in need of new options."

Stephen B. Howell, M.D., Professor of Medicine at the University of California, San Diego, and Associate Director for Clinical Research at the Moores UCSD Cancer Center, who serves as Aptose’s Acting Chief Medical Officer, wrapped up the prepared remarks of the session with current development plans for CG-806:

IND-enabling GLP toxicology and safety studies with CG-806 are complete
As a result of its robust safety profile, Aptose plans to file an IND in the first quarter of 2019 to begin clinical testing of CG-806 both in healthy volunteers (HVS) and in patients with B-cell malignancies
After a therapeutic dose is identified from the HVS or B-cell malignancy clinical trials, Aptose intends to expand into acutely ill AML patients and other sensitive subpopulations
About CG-806
CG-806 is a preclinical stage oral, first-in-class pan-FLT3/pan-BTK multi-cluster kinase inhibitor. This small molecule demonstrates potent inhibition of wild type and all mutant forms of FLT3 (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), eliminates acute myeloid leukemia (AML) tumors in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with AML. Likewise, CG-806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser mutant forms of the BTK enzyme, as well as other oncogenic kinase pathways operative in B cell malignancies, suggesting CG-806 may be developed for various B cell malignancy patients (including CLL, MCL, DLBCL and others) that are resistant/refractory/intolerant to covalent BTK inhibitors. It is in development for acute myeloid leukemia (AML) and B cell lymphoma.

Brian J. Druker, M.D.
Brian Druker, M.D., is the director of the Knight Cancer Institute, Associate Dean for oncology of the OHSU School of Medicine, JELD-WEN Chair of Leukemia Research and a Howard Hughes Medical Institute investigator. He revolutionized the treatment of cancer through research that resulted in the development of imatinib, the first drug to target the molecular defect of a cancer while leaving healthy cells unharmed. Marketed under the name Gleevec, his discovery turned a once-fatal cancer, chronic myeloid leukemia, into a manageable condition. This work changed the life expectancy of patients with CML from an average of 3 to 5 years to a 95% five-year survival, and has resulted in a paradigm-shift in cancer treatment from non-specific chemotherapy to highly targeted therapeutic agents. He is a member of the National Academy of Medicine, the National Academy of Sciences and, among numerous awards, is the recipient of the 2009 Lasker-DeBakey Clinical Medical Research Award and most recently, the 2018 Tang Prize in Biopharmaceutical Science.

Dr. Druker currently serves as the Chair of the Scientific Advisory Board for Aptose. He receives compensation from Aptose for this role. He and his team at OHSU, through the BEAT AML collaboration, have directly conducted studies with CG-806 on fresh bone marrow samples from patients with various hematologic malignancies, and data from these studies serve as the cornerstone of presentations made by Aptose Biosciences.

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On December 11, 2018 PharmaMar (MSE:PHM) reported the Australian Regulatory Agency (TGA) has informed STA that Aplidin (plitidepsin) has been approved for the treatment of multiple myeloma in combination with dexamethasone (Press release, PharmaMar, DEC 11, 2018, View Source [SID1234532007]).

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Aplidin will be available to patients who have failed or are resistant to other therapies, after the TGA decision to approve Aplidin before any other country.

The indication has been approved for the treatment of patients that relapse after three lines of treatment, including proteasome inhibitors or immunomodulators. It can also be administered as 3rd line treatment, when the patient has already received two prior lines and is refractory or intolerant to proteasome inhibitors or immunomodulators.

This approval opens the door to many other markets in South America, Mexico, Canada, Asia Pacific, Middle East and North Africa, among others, that will review Aplidin after TGA’s decision, and where PharmaMar has partners for this product.

PharmaMar signed a licensing agreement with Specialised Therapeutics Asia Pte, Ltd (STA), established in Singapore, to market Aplidin in Australia and New Zealand in August 2015, along with a new agreement for 12 other Asian countries in February 2016.

Professor Andrew Spencer, Head of the Malignant Haematology and Stem Cell Transplantation Service at The Alfred Hospital, said: "Aplidin provides a chance for some myeloma patients to extend their lives. We now have another drug to offer patients who have relapsed after being treated with existing therapies. This is important, because once patients become resistant to standard therapies, there have been very limited treatment options."

Professor Jeff Szer, Peter MacCallum Cancer Centre and Royal Melbourne Hospital haematologist, who was the Australian principal investigator on the pivotal Aplidin registration study, said Aplidin had been shown to be effective and well tolerated. He commented: "More Australian myeloma patients were enrolled into the pivotal international trial of Aplidin than anywhere else in the world. These patients in the Phase 3 study known as ADMYRE have now paved the way for others to have access to a new and novel therapy. This really means that some patients with advanced myeloma have the possibility of improved outcomes, when previous therapies have failed."

Carlo Montagner, Chief Executive Officer of Specialised Therapeutics Asia, said Australian regulatory authorities should be commended for ensuring Australian myeloma patients have the first opportunity to access this cutting-edge therapy. He commented: "It is not often that Australian patients are the first in the world to access new medicines. In this case, the TGA is at the forefront, with decision-makers recognizing the great need that exists in multiple myeloma. This disease remains incurable and patients eventually run out of treatment options."

José María Fernández Sousa-Faro, President of PharmaMar, said: "This approval for an incurable disease, corroborates the work that the PharmaMar team has done over the years with Aplidin. Patients and the medical community will now have a new therapeutic alternative with a new mechanism of action, that is different from the products currently in use."

Luis Mora, Managing Director of PharmaMar´s Oncology Business Unit, added: "The approval of Aplidin is a very important step forward for the company. This increases PharmaMar’s presence with a second drug on the Australian market and, together with our partners, we are initiating procedures for other markets, such as South America, Mexico, Canada, Asia and Israel."
In Europe, as already announced, the decision of the European Medicines Agency (EMA) is being appealed to the Luxembourg Court.

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On December 11, 2018 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2018 fourth quarter and year ended September 30, 2018 (Press release, Arrowhead Research Corporation, DEC 11, 2018, View Source [SID1234532026]). The company is hosting a conference call at 4:30 p.m. EST to discuss results.

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 6744427.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 6744427.

Selected Fiscal 2018 and Recent Events

Hosted an Analyst R&D Day in September 2017 to highlight the following:

The Targeted RNAi Molecule platform, or TRiMTM, which utilizes ligand-mediated delivery and is designed to enable tissue-specific targeting, while being structurally simple. The TRiMTM platform offers several potential competitive advantages including:

A more sophisticated RNAi trigger selection and screening process that identifies potent sequences rapidly in locations that RNAi competitors may miss

Multiple routes of administration including subcutaneous, intravenous, and inhaled

Faster time to clinical candidates

Optimal pharmacologic activity and long duration-of-effect

Potentially wide safety margins

Simplified manufacturing at reduced cost

And, the promise of taking RNAi to tissues beyond the liver

Presented new clinical data at HEP DART 2017 and EASL 2018 demonstrating up to 5.3 Log10 reduction in HBV s-antigen and a Sustained Host Response in 50% of hepatitis B patients following first generation RNAi therapy, ARC-520, in the 2001 open label extension study

One patient serocleared all viral markers, including HBsAg

Began a Phase 1 study of ARO-AAT, Arrowhead’s second generation subcutaneously administered RNAi therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency

Began a Phase 1/2 study of ARO-HBV, a third-generation subcutaneously administered RNAi therapeutic candidate being developed as a potential treatment for patients with chronic hepatitis B virus infection

Announced that Amgen had administered the first dose of AMG 890, formerly ARO-LPA, in a Phase 1 clinical study, which earned Arrowhead a $10 million milestone payment

Presented clinical data on ARO-AAT at the Alpha-1 National Education Conference and at the AASLD Liver Meeting 2018 demonstrating:

Three monthly doses of 300 mg ARO-AAT led to reductions in serum alpha-1 antitrypsin to below the level of quantitation in 100% of subjects

Reductions were sustained for greater than 14 weeks indicating that quarterly or less frequent dosing appears feasible

Single and multiple doses of ARO-AAT appeared to be well-tolerated at all doses tested

Presented clinical data on ARO-HBV at the World Gastroenterologists Summit and at the AASLD Liver Meeting 2018 demonstrating:

Mean HBsAg reduction of -1.9 Log10 (-98.7%) with a range of -1.3 Log10 (-95.0%) to -3.8 Log10 (-99.98%)

ARO-HBV appeared to be well-tolerated at monthly doses up to 400 mg

Signed a license agreement with Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceuticals Companies of Johnson & Johnson, for ARO-HBV and a collaboration agreement for up to three RNAi therapeutic candidates that use our proprietary TRiMTM platform against new targets to be selected by Janssen

The total potential deal value is approximately $3.7 billion plus royalties on commercial sales

Received $175 million as an upfront payment and received $75 million in the form of an equity investment by Johnson & Johnson Innovation – JJDC, Inc., at a price of $23.00 per share of Arrowhead common stock

Hosted an R&D Day in October 2018 to discuss in more detail our emerging pipeline of RNAi therapeutics that leverage the TRiMTM platform

Filed for regulatory clearance to begin a Phase 1 study of ARO-ANG3, an RNAi-based investigational medicine targeting angiopoietin like protein 3 (ANGPTL3) being developed for the treatment of dyslipidemias and metabolic diseases