Our Science

Istari Oncology’s second technology platform consists of a series of ADCs for a variety of oncology targets (Company Web Page, Istari Oncology, NOV 15, 2017, View Source [SID1234522093]). These select, highly specific, monoclonal antibodies are presently conjugated with PE-38 toxin to maximize the killing effect.
We believe our platform overcomes some of the challenges associated with other ADCs because it is based on genetic linkage, which provides more stability than chemical linkers, making it is easier to manufacture. The FDA granted our ADC conjugate, D2C7-IT, Orphan Drug designation in 2016.
Beyond the various PE-38 toxins, our technology could be linked with other therapeutic agents for different targets. Toxins used in construction of immunotoxins are typically derived from bacteria, fungi, and plants. These agents and linkers potentially provide another platform for additional pipeline therapeutics. Possible tumor targets include solid, nonlymphoid tumors.

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Abeona Reports Third Quarter 2017 Financial Results and Recent Business Highlights

On November 15, 2017 Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, reported financial results for the third quarter and recent business highlights (Press release, Abeona Therapeutics, NOV 15, 2017, View Source;p=RssLanding&cat=news&id=2317120 [SID1234522085]). The Company will provide investors an update on recent and ongoing business activities and an overview of its 3Q17 financials on Monday, November 20th, at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-269-7756 (toll free domestic) or 201-689-7817 (international).

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"The third quarter was marked with achievements across multiple clinical programs, including initiating enrollments at our global clinical sites for ABO-102 for MPS IIIA and reporting additional data that underscored the durability and clinical benefit of the gene therapy. Our Epidermolysis Bullosa program achieved FDA Breakthrough Therapy designation, completed its Phase 1/2 clinical trial and continues to advance as we finalize the clinical protocol before initiating the pivotal Phase 3 trial next year. We were pleased to have recently initiated screening in our MPS IIIB program and look forward to commencing enrollments shortly," stated Timothy J. Miller, Ph.D., President and CEO. "In addition, work in optimizing our AIM vector platform demonstrated exciting progress, including enhanced tissue tropisms compared to naturally occurring AAV capsids."

3rd Quarter Summary Financial Results:

Cash position: Cash and cash equivalents as of September 30, 2017 were $56.5 million, compared to $58.3 million as of June 30, 2017. Net cash used in operating activities in the nine months ended September 30, 2017 was $17.6 million as compared to $9.6 million in the same period in 2016. Cash and cash equivalents includes approximately $5 million from exercised warrants in the third quarter. Subsequent to the end of the third quarter, the Company closed a public offering of common stock with gross proceeds of $92 million. Total cash as of October 31, 2017 was $142.6 million.
Revenues: Revenues were $219 thousand for the third quarter of 2017, compared to $184 thousand in the third quarter of 2016. Revenues consisted of a combination of royalties from marketed products, primarily MuGard, and recognition of deferred revenues related to upfront payments from early license agreements.
Loss per share: Loss per share was $0.13 for the third quarter of 2017, compared to a loss per share of $0.08 in the comparable period in 2016.
Abeona Recent Highlights:

November 9, 2017: Enrolled First Subject at Spain Clinical Site in Ongoing Phase 1/2 Clinical Trial in MPS IIIA
October 19, 2017: Announced Closing of $92 Million Underwritten Public Offering and Full Exercise of Underwriters’ Option to Purchase Additional Shares
October 16, 2017: Announced a Grant of up to $13.85 Million from Leading Sanfilippo Syndrome Foundations for Clinical Development of MPS III Gene Therapies
October 11, 2017: Hosted inaugural R&D day and announced enrollment of First Two Patients in Global Expansion of Phase 1/2 Clinical Trial in MPS IIIA
October 6, 2017: Announced Top-Line One Year Data from ABO-102 MPS IIIA Trial at ARM’s Cell & Gene Meeting on the Mesa
Gene therapy demonstrated durable and significant reduction of underlying disease pathology across multiple clinical measures in Cohort 1 (n=3) compared to a natural history control group (n=8-12)
Systemic biopotency demonstrated time- and dose-dependent reductions of disease causing Heparan Sulfate in the Cerebrospinal fluid (CSF) and liver volumes
Preservation of deep brain architecture observed after intravenous administration
Stabilization of neurocognitive assessment scores at one year post-injection
October 4, 2017: Announced Dedication of Commercial Gene Therapy Manufacturing Facility in Cleveland, Ohio
September 28, 2017: Announced Collaboration with Brammer Bio for Commercial Translation of ABO-102
August 29, 2017: Received FDA Breakthrough Therapy Designation for EB-101 Autologous Cell Therapy in Epidermolysis Bullosa
July 25, 2017: Announced Appointment of Juan Ruiz, M.D., Ph.D. as Chief Medical Officer
July 18, 2017: Received Guidance from FDA to Commence Pivotal Phase 3 for EB-101 Gene Therapy for Patients with Epidermolysis Bullosa

"We have made great progress in the quarter towards becoming a key player in the development of novel breakthrough gene and cell therapies for rare genetic diseases," stated Steven H. Rouhandeh, Executive Chairman. "The recent investment from high-quality investors and leading foundations is another achievement that demonstrates our internal capabilities and commitment to the advancement of our robust pipeline and next generation vector platform, including MPS III gene therapy products. We look forward to further strengthening our efforts with key hires, advancing clinical capabilities, and commercial expansion in the coming quarters."

Invitae Completes Acquisition of CombiMatrix, Becoming a Leader in Family and Reproductive Health Genetic Information Services

On November 15, 2017 Invitae Corporation (NYSE: NVTA), one of the fastest growing genetic information companies, reported it has completed its acquisition of CombiMatrix, which specializes in providing genetic information for prenatal diagnosis, miscarriage analysis and diagnosis of pediatric developmental disorders, establishing Invitae as a new leader in family and reproductive genetic health services (Press release, CombiMatrix, NOV 15, 2017, View Source [SID1234522094]).

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

"With the addition of CombiMatrix to Invitae, we have completed our entry into prenatal and perinatal genetics, currently the second-largest category of genetic testing services. Our integrated offering will build on the expertise and technologies developed by CombiMatrix to offer customers the most comprehensive offering from a single provider in the category," said Sean George, chief executive officer of Invitae. "Invitae’s platform now delivers comprehensive genetic information services that support the use of genetics in mainstream medical care throughout all stages of life."

CombiMatrix leverages cytogenomic and cytogenetic technologies such as single nucleotide polymorphism chromosomal microarray analysis and next generation sequencing, supported by long-standing expertise in technically challenging sample types, to provide in-depth answers for patients and clinicians addressing complex reproductive health questions.

"Access to actionable genetic information is essential for monitoring pregnancies, particularly for women going through IVF or facing recurrent miscarriages," said Robert Nussbaum, MD, chief medical officer of Invitae. "Our integrated platform and world-class expertise can provide genetic information that helps women and their clinicians with some of the most important decisions of their lives today, even as we continue to advance the understanding of the role genetics plays in having healthy pregnancies."

In connection with the closing, Invitae issued approximately $21.2 million in shares of its common stock to former CombiMatrix securityholders, or approximately 2.7 million shares. Together with the approximately 1.7 million shares of Invitae common stock underlying CombiMatrix Series F warrants assumed in the Merger, the transaction has a total enterprise value of approximately $34.9 million.

The acquisition of CombiMatrix complements Invitae’s recent acquisition of another reproductive genetics company, Good Start Genetics, to establish a category-leading menu with the breadth and depth needed to provide comprehensive support for women, their partners and clinicians to use genetic information when considering their reproductive health options, from carrier screening to preimplantation genetic screening and diagnosis to newborn diagnostics.

Transaction Details

At the closing of the Merger, Invitae issued shares of its common stock to (i) CombiMatrix’s common stockholders, at an exchange ratio of 0.8692 of a share of Invitae common stock (the "Merger Exchange Ratio") for each share of CombiMatrix common stock outstanding immediately prior to the Merger, (ii) CombiMatrix’s Series F preferred stockholders, at the Merger Exchange Ratio for each share of CombiMatrix common stock underlying Series F preferred stock outstanding immediately prior to the Merger, (iii) holders of outstanding and unexercised in-the-money CombiMatrix stock options, which were fully accelerated to the extent of any applicable vesting period and converted into the right to receive a number of shares of Invitae common stock adjusted for the Merger Exchange Ratio and reduced by the aggregate exercise price, and (iv) holders of outstanding and unsettled CombiMatrix restricted stock units ("RSUs"), which were fully accelerated to the extent of any applicable vesting period and converted into the right to receive a number of shares of Invitae common stock adjusted for the Merger Exchange Ratio. No fractional shares were issued in connection with the Merger and Invitae will pay cash in lieu of any such fractional shares. The Merger Exchange Ratio was determined through arm’s-length negotiations between Invitae and CombiMatrix.

In addition, at the closing of the Merger, (a) all outstanding and unexercised out-of-the money CombiMatrix stock options were cancelled and terminated without the right to receive any consideration, (b) all CombiMatrix Series D Warrants and Series F Warrants outstanding and unexercised immediately prior to the closing of the Merger were assumed by Invitae and converted into warrants to purchase the number of shares of Invitae common stock determined by multiplying the number of shares of CombiMatrix common stock subject to such warrants by the Merger Exchange Ratio, and with the exercise price adjusted by dividing the per share exercise price of the CombiMatrix common stock subject to such warrants by the Merger Exchange Ratio, and (c) certain entitlements under CombiMatrix’s executive compensation transaction bonus plan (the "Transaction Bonus Plan") were paid in shares of Invitae common stock or RSUs to be settled in shares of Invitae common stock. All outstanding and unexercised CombiMatrix Series A, Series B, Series C, Series E, and PIPE warrants were repurchased by CombiMatrix prior to closing pursuant to that certain CombiMatrix Common Stock Purchase Warrants Repurchase Agreement dated July 11, 2016.

Invitae’s previously announced offer to exchange each outstanding Series F warrant (the "CombiMatrix Series F warrants") to acquire one share of common stock of CombiMatrix for 0.3056 of a share of Invitae common stock (the "Exchange Offer") expired at 12:00 midnight (one minute after 11:59 p.m.), New York City time, on November 13, 2017. Because the minimum tender condition of 90% was not achieved in the Exchange Offer, Invitae did not accept any of the CombiMatrix Series F warrants that were tendered in the Exchange Offer prior to its expiration. Accordingly, any CombiMatrix Series F warrants that were tendered will be promptly returned to the holder by the exchange agent.

Invitae issued an aggregate of 2,726,324 shares of its common stock and 214,976 RSUs in connection with the Merger (including shares and RSUs issued pursuant to the Transaction Bonus Plan). Immediately after the Merger, (i) there were approximately 52.9 million shares of Invitae common outstanding, (ii) the former CombiMatrix securityholders and executives owned approximately 8.6% of the fully-diluted common stock of the combined company, and (iii) Invitae securityholders, whose shares of Invitae capital stock remain outstanding after the Merger, owned approximately 91.4% of the fully-diluted common stock of the combined company.

Upon completion of the Merger, CombiMatrix became a wholly owned subsidiary of Invitae. As a result, the CombiMatrix common stock and Series F warrants will cease trading on the Nasdaq Capital Market and will be delisted.

About Invitae’s Family and Reproductive Health Genetic Services

Invitae’s reproductive genetics products, which include CombiPGS, CombiPGD and CombiSNP from CombiMatrix and Good Start Genetics’ GeneVu, EmbryVu and VeriYou, provide affordable and accessible genetic information to help people have healthy families. Good Start Genetics became part of Invitae in August 2017.

Mateon Provides Corporate Update and Reports Third Quarter 2017 Financial Results

On November 14, 2017 Mateon Therapeutics, Inc. (OTCQX:MATN), a biopharmaceutical company developing investigational drugs for the treatment of orphan oncology indications, reported a corporate update and reported financial results for the three months ended September 30, 2017 (Press release, Mateon Therapeutics, NOV 14, 2017, View Source [SID1234522046]).

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Recent Corporate Highlights

Announced phase 1b data for OXi4503 in Study OX1222 for the treatment of relapsed/refractory acute myeloid leukemia/myelodysplastic syndromes, which show complete remissions and evidence of a dose response;
Elevated OXi4503 to lead program, representing the primary focus of Mateon’s drug development efforts; and
Terminated FOCUS Study in platinum-resistant ovarian cancer, terminated clinical development of CA4P, and restructured company down to six employees to reduce expenditures.
"OXi4503 destroys the protective environment that bone marrow tumors provide to AML stem cells while also simultaneously attacking the tumor cells themselves. Thus, if ultimately approved, our lead compound would be a completely new way to treat AML and should offer many advantages over other drugs currently on the market or in development for this indication," stated William D. Schwieterman, M.D., President and Chief Executive Officer of Mateon. "With patent protection in AML to 2033, we believe that OXi4503 represents an outstanding business development or financing proposition, and are working to secure an arrangement that will allow us to accrue clinical data in higher-dose cohorts in Study OX1222."

Financial Results for the Third Quarter of 2017

For the three months ended September 30, 2017, Mateon reported a net loss of $3.5 million, compared to a net loss of $3.2 million for the three months ended September 30, 2016. Research and development expenses increased to $2.8 million for the three months ended September 30, 2017, compared to $2.1 million for the three months ended September 30, 2016, primarily due to higher clinical costs associated with the recently terminated FOCUS study. General and administrative expenses decreased to $0.7 million for the three months ended September 30, 2017, compared to $1.2 million for the three months ended September 30, 2016.

At September 30, 2017, Mateon had cash and short-term investments of $1.9 million.

"We very rapidly closed out the FOCUS Study in October. Following the other cost reductions implemented in late September, we now project that our existing cash, when combined with expected refunds from certain vendors, should sustain our OXi4503-focused business development and financing efforts into approximately February 2018," concluded Dr. Schwieterman.

Aptose Reports Results for the Third Quarter Ended September 30, 2017

On November 14, 2017 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, reported financial results for the three months ended September 30, 2017 and reported on corporate developments. Unless specified otherwise, all amounts are in Canadian dollars (Press release, Aptose Biosciences, NOV 14, 2017, View Source;p=RssLanding&cat=news&id=2316911 [SID1234522060]).

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The net loss for the quarter ended September 30, 2017 was $3.3 million ($0.14 per share) compared with $4.0 million ($0.31 per share) in the quarter ended September 30, 2016. Total cash and cash equivalents and investments as of September 30, 2017 were $13.6 million (or $10.9 million US dollars) which, based on current operations, provide the Company with sufficient resources to fund research and development and operations into Q4 2018.

"During the third quarter, we made important progress with both of our novel small molecule compounds for the treatment of certain hematologic malignancies," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "We’ve completed manufacture of high quality drug substance and explored multiple formulations of CG’806, our oral first-in-class pan-FLT3/pan-BTK inhibitor. We triggered PK and dose range finding studies with CG’806 in order to prepare the agent for advancement into the clinic for patients with acute myeloid leukemia and certain B-cell malignancies. We also generated renewed excitement about the potential for APTO-253, a clinical-stage compound that effectively inhibits expression of the c-Myc oncogene, and we believe we have effectively addressed the formulation and manufacturing setbacks that led to a clinical hold for APTO-253. Our goal is to return APTO-253 to the clinic for the treatment of patients with AML or MDS."

Corporate Highlights
Successful completion of APTO-253 formal root cause studies – Aptose successfully completed Formal Root Cause Studies for the manufacturing setback related to the clinical batch supply that failed stability testing and established a Corrective and Prevention Action (CAPA) plan.

Manufacture of APTO-253 clinical supply has begun – The Company has initiated the process to manufacture a cGMP clinical supply that will be required for a potential return of APTO-253 to the clinic. Upon manufacture of the new clinical supply, Aptose plans to perform stability, sterility, mock infusion, animal bridging and blood compatibility studies. Following completion of those studies, we would plan to submit findings to the FDA to seek release of the clinical hold and allow return of APTO-253 to the open Phase 1b trial in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

CG’806 manufacturing and preclinical testing progress – Aptose produced a highly purified batch of drug substance (API) to support pharmacokinetic (PK), formulation evaluation and dose range finding studies of CG’806. The PK and formulation evaluation studies have been completed, and the dose range finding preclinical studies are expected to begin before or shortly after year-end. Separately, the Company has initiated the process to manufacture multi-kilogram batches of GLP-grade API for use in the formal GLP/IND-enabling animal toxicity studies.

Intellectual property protection for CG’806 – During the third quarter, Aptose continued to strengthen its patent portfolio. In September, Aptose and partner CrystalGenomics announced that the United States Patent and Trademark Office issued U.S. Patent No. 9,758,508 which claims numerous compounds, including the CG’806 compound, pharmaceutical compositions comprising the CG’806 compound, and methods of treating various diseases. The patent is expected to provide protection until the end of 2033. In August, Aptose received notice allowing U.S. Patent Application No. 14/655,954. The allowed ‘954 application claims numerous compounds, including the CG’806 compound, pharmaceutical compositions comprising the CG’806 compound, and methods of treating various diseases caused by abnormal or uncontrolled activation of protein kinase in a mammal by administering a compound, including the CG’806 compound.

Strengthened financial position – The Company recently announced that it entered into a Common Shares Purchase Agreement with Aspire Capital Fund, LLC ("Aspire Capital") to sell up to US$15.5 million of common shares to Aspire Capital. Under the terms of the agreement, Aspire Capital made an initial investment of US$500,000 to purchase APTO common shares at US $1.40 per share on October 31, 2017. In addition, Aspire Capital has committed to purchase up to an additional US$15.0 million of APTO common shares, at Aptose’s request from time to time during a 30 month period beginning on the effective date of a registration statement related to the transaction, and at prices based on the market price at the time of each sale. Under terms of the agreement, the Company also issued 321,429 common shares to Aspire Capital as consideration for Aspire Capital entering into the Purchase Agreement.