On October 4, 2018 Heat Biologics, Inc. (NASDAQ: HTBX), a biopharmaceutical company developing drugs designed to activate a patient’s immune system against cancer, and its Pelican Therapeutics subsidiary ("Pelican") reported updates its novel PTX-35 co-stimulatory antibody (Press release, Heat Biologics, OCT 4, 2018, View Source [SID1234529923]). PTX-35 is designed to harness the body’s natural antigen-specific immune activation mechanisms. 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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

On October 4, 2018 Nimbus Therapeutics, a biotechnology company applying deep computational chemistry expertise throughout drug discovery and development, reported that it has named Jeb Keiper, M.S., M.B.A., President and Chief Executive Officer, effective immediately (Press release, Nimbus Therapeutics, OCT 4, 2018, View Source [SID1234529887]). Mr. Keiper succeeds Donald Nicholson, Ph.D., and will lead the company forward as it progresses multiple programs toward clinical development.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"After over four years of building Nimbus and this exceptional team, the company is now poised to start the next chapter of developing important medicines for patients with autoimmune disease, cancer, and metabolic diseases," said Donald Nicholson, Ph.D. "In its next phase of growth, Nimbus will be led by a CEO with intimate knowledge of the company and a track record of successful leadership. Jeb’s deep business expertise and leadership experience made him an ideal partner to me and a logical successor. I am very much looking forward to starting the next entrepreneurial chapter of my career, and I am confident that Nimbus, the science and our employees are in great hands."

Mr. Keiper joined Nimbus in 2014 and initially served as Nimbus’ Chief Business Officer, and along with Dr. Nicholson, set the strategic direction of the company. In the ensuing four years, Nimbus brought in over $775 million of partnership and financing into the company, including the Series B financing in March 2015, the sale of Nimbus’ clinical NASH program to Gilead in May 2016 for $400 million upfront, the strategic immunology alliance with Celgene announced in October 2017, and a round of private expansion capital in 2018. Mr. Keiper was also appointed Chief Financial Officer in 2017.

Prior to Nimbus, Mr. Keiper was VP of Business Development at GSK Oncology and spent a decade at GlaxoSmithKline in various BD leadership roles. He led the oncology portion of the $16 billion GSK-Novartis transaction announced in April 2014. In 20 years of industry experience, starting as a bench chemist at Pfizer R&D and working in various roles from consulting at McKinsey to business development at TransForm Pharmaceuticals, Mr. Keiper has a track record of leading teams of talented scientists and managers to achieve stretch objectives, including advancing new medicines into clinical development, filing NDAs, and constructing transformative deals and financings.

"Jeb has been a critical component of the success at Nimbus over the last four years, and we are confident that he will lead the company into the future and its continued successful development of the pipeline," said Nimbus’ Chairman and co-founder Bruce Booth, D.Phil. "We thank Don for his passion and leadership in driving the accomplishments Nimbus has achieved to date and have immense respect for him as a scientist and a leader. I look forward to working with him on future innovative opportunities."

"Nimbus has pioneered new ways of discovering and developing important medicines for patients leveraging a globally distributed, virtually-enabled operating model," said Mr. Keiper. "Our success has challenged and reformed decades-old dogma on how to develop new small molecule medicines, and has been reflected in our partnerships with Gilead, Celgene, and Genentech. As we enter the next chapter, our ambition and business strategy remain the same: to continue innovating with computational methodologies for creating and advancing new medicines, and to couple those with cutting-edge discoveries in the mechanistic underpinnings of human disease."

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

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).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.