On October 4, 2018 ArQule, Inc. (Nasdaq: ARQL) reported that it will be presenting clinical data on the company’s lead AKT inhibitor, miransertib (ARQ 092), in three poster presentations at the American Society of Human Genetics (ASHG) 2018 Annual Meeting to be held from October 16 to 20, 2018 in San Diego, CA (Press release, ArQule, OCT 4, 2018, View Source [SID1234529813]).

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The data to be presented is from the Phase 1/2 company-sponsored trial in PROS and the single patient use program with select physicians.

Presentation Details

Title:

Personalized medicine in rare diseases and cancer: A case report of a lasting response in a young teenage patient with Proteus syndrome and secondary ovarian cancer

Program #:

1251

Session:

Complex Traits and Polygenic Disorders
Date:

Wednesday, October 17, 2018

Time:

2:00-3:00 PM PT
Location:

Exhibit Hall

Title:

An open-label, phase 1/2 study of miransertib (ARQ 092), an oral pan-AKT inhibitor, in patients (pts) with PIK3CA-related Overgrowth Spectrum (PROS): Preliminary results

Program #:

2538

Session:

Complex Traits and Polygenic Disorders
Date:

Wednesday, October 17, 2018
Time:

3:00-4:00 PM PT
Location:

Exhibit Hall

Title:

Severe PI3Kinase overgrowth syndrome treated with the AKT inhibitor miransertib

Program #:

1288

Session:

Mendelian Phenotypes
Date:

Thursday, October 18, 2018
Time:

3:00-4:00 PM PT
Location:

Exhibit Hall
About Miransertib
Miransertib (ARQ 092) is an orally available, selective, pan-AKT (protein kinase B) inhibitor that potently inhibits AKT1, 2 and 3 isoforms. Dysregulation of AKT has been implicated in a variety of rare overgrowth diseases and cancers; however, there are currently no approved inhibitors of AKT. AKT inhibitors, either as single agent or combination therapy, show significant promise in molecularly defined patient populations. Miransertib is currently in a Phase 1/2 company-sponsored study for PIK3CA-Related Overgrowth Spectrum (PROS), a Phase 1 study for ultra-rare Proteus syndrome conducted by the National Institutes of Health (NIH/NHGRI), and a Phase 1b study in combination with the hormonal therapy, anastrozole, in patients with advanced endometrial cancer with AKT and PI3K mutations. Miransertib has been granted Rare Pediatric Disease Designation and Fast Track Designation by the U.S. Food and Drug Administration (FDA), as well as Orphan Designation by the FDA and European Medicines Agency in the rare overgrowth disease, Proteus syndrome.

About PROS
PROS is a term used to refer to a spectrum of rare diseases identified by somatic mutations in the PIK3CA gene, that result in excess growth in certain areas of the body. While the individual diseases that fall within the overgrowth spectrum have similar symptoms, each disease is defined by unique clinical characteristics. The implementation of genetic sequencing has led to the identification of the underlying genetic mutations that drive these overgrowth disorders, allowing for the development of medicines that target the specific causes of disease.

About Proteus Syndrome
Proteus syndrome is an ultra-rare condition characterized by the aberrant overgrowth of multiple tissues of the body. Patients with Proteus syndrome experience changes in the shapes of certain body structures over time, including abnormal, often asymmetric, massive growth (overgrowth) of the skeleton, skin, adipose tissue and central nervous system out of proportion to the rest of the body. Although patients may have minimal or no manifestations at birth, the disease develops and becomes apparent in early childhood (6-18 months) and rapidly progresses with intense growth in the first 10 years of life. The worldwide incidence is believed to be approximately one in a million. There are currently no approved medicinal treatments for Proteus syndrome, leaving patients with minimal treatment options to manage the disease and a mortality of 25% by age 22.

A Phase I, open-label, dose finding study (NCT02641002) of CC-90002 in subjects with acute myeloid leukemia and high-risk myelodsplastic syndrome has been terminated due to that preliminary monotherapy data in relapsed/refractory AML and high-risk MDS did not offer a sufficiently encouraging profile for further dose escalation/expansion (Clinical trial, ClinicalTrials.gov, OCT 4, 2018, View Source;B=13&C=merged#StudyPageTop [SID1234531854]).

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However another Phase I, open-label, dose finding study (NCT02367196) of CC-90002 in subjects with advanced solid and hematologic cancers is still active and confirmed by Celgene October 16, 2018 (Clinicaltrials.gov, OCT 16, 2018, View Source).

On October 4, 2018 Celyad (Euronext Brussels and Paris, and NASDAQ: CYAD), a clinical-stage biopharmaceutical company focused on the development of specialized CAR-T cell-based therapies, reported an exclusive agreement with Horizon Discovery Group plc (LSE: HZD), for the use of its shRNA technology to generate Celyad’s second non-gene-edited allogeneic platform (Press release, Celyad, OCT 4, 2018, View Source [SID1234530340]).

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Celyad recently announced its first-in-class non-gene edited allogeneic CAR-T candidate, CYAD-101 a non-gene-edited allogeneic NKG2D-based CAR using the TIM (TCR Inhibiting Molecule). As a result of the agreement with Horizon Discovery, Celyad now also has access to a novel shRNA-based platform.

Data from preclinical studies demonstrating the versatility of the shRNA platform in the allogeneic setting, will be presented at the upcoming 2018 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in Washington, D.C., November 7th – 11th. These promising preclinical data will pave the way for the next steps in the development of Celyad’s differentiated non-gene edited allogeneic approach to CAR-T cell therapy.

"We are excited to have the opportunity to leverage Horizon’s shRNA platform to further advance our pioneering approach to non-gene edited allogeneic CAR-T cells", said Dr. Christian Homsy, CEO of Celyad. "Celyad is committed to rapidly advancing its allogeneic program based on highly promising preclinical data which will be presented at SITC (Free SITC Whitepaper). These data provide proof of concept for our shRNA based non-gene-edited allogeneic approach. In addition to very promising preclinical data, our allogeneic approach is also strengthened by Celyad’s strong patent estate in the U.S., which broadly covers the use of allogeneic CAR-T using cells that are TCR inhibited or suppressed by any means."

Jon Moore, CSO of Horizon Discovery added: "The high performance shRNA technology licensed by Celyad is the same as that deployed in our range of SMARTvector products and is designed to deliver efficient target knock down with high specificity. Horizon’s collaboration with Celyad is designed to let Celyad find a highly effective solution for its needs. Horizon sees its shRNA technology as a serious rival to gene editing approaches for delivering enhanced performance to therapeutic cell products. We see enormous promise in cell therapies and are committed to develop and supply innovative technologies that allow our partners to bring transformative cell therapies to the clinic and fulfil unmet clinical needs."

On October 4, 2018 Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) reported that the Company will report its financial results for the third quarter ended September 30, 2018 before the US financial markets open on October 24, 2018 (Press release, Alexion, OCT 4, 2018, View Source [SID1234529852]). Following the release of the financial results, Alexion management will conduct a conference call and audio webcast at 8:00 a.m. Eastern Time (ET).

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To participate in this conference call, dial 866-762-3111 (USA) or 210-874-7712 (International), conference ID 5947065 shortly before 8:00 a.m. ET. The audio webcast can be accessed on the Investor page of View Source and an archived version will be available for a limited time following the presentation.

On October 4, 2018 Heat Biologics, Inc. ("Heat") (NASDAQ: HTBX), a biopharmaceutical company developing drugs designed to activate a patient’s immune system against cancer, and its Pelican Therapeutics subsidiary ("Pelican") reported on its novel PTX-35 co-stimulatory antibody. PTX-35 is designed to harness the body’s natural antigen-specific immune activation mechanisms (Press release, Heat Biologics, OCT 4, 2018, View Source [SID1234529871]). When combined with immunotherapies, including checkpoint inhibitors as well as Heat’s ImPACT and ComPACT technologies, PTX-35 has been shown to enhance antigen-specific T-cell activation to eliminate tumor cells in pre-clinical models.

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Recent PTX-35 highlights:

Completed cell line development and creation of validated master cell bank for cGMP manufacturing
Established CMC path for the production of GMP clinical material and non-clinical preliminary pharmacology / non-GLP toxicology studies
Preliminary non-GLP pharmacology demonstrates positive results, including efficient binding and activation on cells expressing the TNFRSF25 receptor, as well as increased expansion of T-cells in-vivo
2-week IND enabling dose range finding toxicology studies in primates receiving two doses show no signs or signals of clinical toxicity across wide dose range
Ongoing pre-IND discussions with FDA; expect to submit IND in Q1 2019
Rahul Jasuja, Ph.D., CEO of Pelican, commented, "We are progressing rapidly with our pre-clinical activities and expect to submit an IND for PTX-35 in the first quarter of 2019. We are strongly encouraged by the preliminary pre-clinical efficacy and safety data which shows no signs of toxicity across a wide range of doses."

Dr. Jasuja continued, "We have been efficient in our use of funds, which has allowed us to come in under budget, further extending our runway for this program. Given our operating efficiency thus far, we expect to receive the next tranche of grant funding once we fully utilize the funds that the Cancer Prevention Research Institute of Texas ("CPRIT") has previously provided. As we progress, our plan is to advance a broad clinical development program that could include combination therapy with Heat’s ImPACT and ComPACT therapies, as well as other costimulatory agonists, checkpoint inhibitors and immune modifiers to address the unmet need for patients who do not respond well to current cancer therapies."

The Company further reported that PTX-35 was featured in Nature’s Biopharma Dealmakers September 2018 edition, which is available at: View Source

To-date, Pelican has received $8.3 million in grants from CPRIT. The CPRIT award supports pre-clinical development, manufacturing and clinical development through a comprehensive 70-patient Phase 1 clinical trial for PTX-35. The Company expects to meet the qualifications to receive the third tranche of its $15.2 million CPRIT grant award, totaling $6.9 million, later this year.