On August 6, 2018 Harpoon Therapeutics, an immuno-oncology company pioneering a new class of T cell engaging therapeutics, reported that the first patient has been treated with HPN424 in a Phase 1 clinical study of metastatic castration-resistant prostate cancer (mCRPC) patients (Press release, Harpoon Therapeutics, AUG 6, 2018, View Source [SID1234528457]). HPN424 is the first of multiple compounds in development that are based on the company’s TriTAC (Tri-specific T cell Activating Construct) platform and designed to penetrate solid tumors, have extended serum half-life, and recruit patients’ own T cells to destroy malignant tumor cells.

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 Phase 1 trial is a multicenter, multinational, open-label, ascending dose study designed to evaluate the safety, tolerability and pharmacokinetics of HPN424 in approximately 40 patients with metastatic prostate cancer.

"We are pleased to initiate Harpoon’s first clinical trial with HPN424 in patients with advanced prostate cancer, an important milestone that marks our transition to a clinical-stage company," said Jerry McMahon, PhD, President and Chief Executive Officer of Harpoon Therapeutics. "HPN424 is the first TriTAC compound to enter clinical testing and represents a novel class of T cell therapeutics aiming to achieve superior efficacy in penetrating and killing solid tumors. Data is expected in 2019, and should provide the safety assessment, pharmacokinetics and pharmacodynamics to determine the optimal dose and regimen for our additional planned trials in prostate cancer, the third leading cause of cancer deaths for men in the U.S."

In the first phase of the study, HPN424 will be administered once weekly to patients via intravenous (IV) infusion with dose escalation until a therapeutic dose level has been reached. Following dose escalation, Harpoon will further evaluate the safety and efficacy of HPN424 in additional cohorts of patients at the recommended Phase 2 dose established in the first phase of the study. For more information about the trial, please visit clinicaltrials.gov, identifier number NCT03577028.

"Immunology represents a new frontier for the treatment of prostate cancer," said Charles G. Drake, MD, PhD, Director of Genitourinary Oncology at New York Presbyterian/Columbia University Cancer Center, Co-director of Columbia’s Cancer Immunotherapy Program and Scientific Advisory Board member at Harpoon. "Each year in the U.S., approximately 26,000 men die from prostate cancer following treatment with current available therapies. HPN424, an innovative biologic designed to activate T cells in the tumor, is highly potent in killing prostate cancer cells in preclinical models and could be a promising option for these men with advanced disease."

"HPN424 is designed as an ‘off-the-shelf’ T cell therapy which can overcome loss of human leukocyte antigen (HLA) expression – a frequent mechanism by which cancer cells escape T cell recognition," said Che-Leung Law, PhD, Vice President of Translational Medicine. "Pharmacological insights gleaned from the study should help inform development of both HPN424 and our pipeline of additional products – targeting mesothelin (MSLN), B-cell maturation antigen (BCMA) and DLL3 – in the near future."

About HPN424

HPN424 is a 50-kD single polypeptide that contains three binding domains — for human PSMA, human serum albumin and human CD3. In preclinical studies, HPN424 demonstrated single-digit picomolar potency for PSMA-dependent T cell killing in a panel of human prostate cancer cell lines, which translated to in vivo efficacy with efficacious doses in the low μg/kg range. HPN424 was well tolerated in a multi-dose safety study in non-human primates and showed a serum half-life of about 80 hours supporting once-weekly administration. AACR (Free AACR Whitepaper)_2018_Poster_HPN424

On August 6, 2018 Kura Oncology, Inc., (Nasdaq: KURA) a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported second quarter 2018 financial results and provided a corporate update (Press release, Kura Oncology, AUG 6, 2018, View Source [SID1234528626]).

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!

"I am very pleased with the continued progress we made over the past quarter, with a particular focus on operational execution," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We have enhanced our leadership team, improved our ability to screen and identify patients with HRAS mutations, positioned our pipeline to create additional opportunities and strengthened our balance sheet through a successful public offering. We believe the proceeds from this offering along with our existing funds give us the resources to advance our pipeline through a series of potential milestones, including data from a registration-directed trial of our lead drug candidate tipifarnib in HRAS mutant HNSCC."

Corporate Update

Updated data from RUN-HN at ESMO (Free ESMO Whitepaper) – An abstract relating to Kura’s ongoing Phase 2 trial of tipifarnib in HRAS mutant HNSCC has been accepted for oral presentation at the upcoming European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress in Munich in October. The update will also include patients from an exploratory cohort in other HRAS mutant squamous cell carcinomas. Approximately 25 clinical sites are now open for enrollment worldwide and RUN-HN is currently enrolling at a rate of approximately two patients per month.

Screening collaboration to support enrollment – The company has signed an agreement with OncoDNA, an oncology-focused healthcare technology company based in Belgium, to support patient enrollment for Kura’s ongoing Phase 2 trial of tipifarnib in patients with HRAS mutant HNSCC. OncoDNA provides physicians internationally with next-generation sequencing for HNSCC oncogenic mutations, including HRAS, for patients who may be eligible to enroll in RUN-HN.

Registration-directed trial anticipated to initiate by year end – Kura has initiated startup activities for its upcoming registration-directed trial of tipifarnib in at least 59 recurrent or metastatic patients with HRAS mutant HNSCC. The company anticipates that the trial will be conducted at approximately 100 clinical sites worldwide and will take approximately two years to enroll. Based on feedback from the U.S. Food and Drug Administration, Kura believes that the single-arm trial, if positive, could support an application for accelerated approval.

HRAS mutant lung squamous cell carcinoma (LSCC) – An investigator-sponsored trial of tipifarnib in HRAS mutant LSCC has been initiated and is now open for enrollment. The proof-of-concept trial is being conducted in collaboration with the Spanish Lung Cancer Group, a cooperative group consisting of more than 150 public and private oncology centers in Spain.

Biomarker-enriched data in hematologic malignances – Kura is prospectively investigating the CXCL12 pathway and bone marrow homing as potential biomarkers of activity for tipifarnib in its three ongoing Phase 2 trials in hematologic malignancies. The PTCL trial was the first of the three to begin and is actively enrolling into two cohorts: 1) Patients with angioimmunoblastic T-cell lymphoma (AITL) and 2) patients with PTCL who have the absence of a single nucleotide variation in the 3’ untranslated region of the CXCL12 gene. The company expects to have biomarker-enriched data from PTCL and potentially other indications by the end of 2018.

Strengthened management team – Kura expanded its leadership team with the additions of Marc Grasso, M.D., and John Farnam. Dr. Grasso will join the company as Chief Financial Officer and Chief Business Officer on August 21, 2018, after 20 years in healthcare investment banking. Mr. Farnam joined Kura in the newly created position of Chief Operating Officer on July 1, 2018, from Celgene Receptos. The company has also added industry veterans Bridget Martell, M.D., and Blake Tomkinson, Ph.D., as Vice Presidents of Clinical Development.

Public offering of common stock – On July 2, 2018, Kura completed a public offering in which the company sold an aggregate of 4,600,000 shares of common stock at a price of $16.75 per share. Net proceeds from the public offering, after deducting underwriting discounts, commissions and offering expenses, were approximately $74.5 million.

Upcoming Milestones

Update from ongoingPhase 2 trial of tipifarnib in HRAS mutant HNSCC at ESMO (Free ESMO Whitepaper) in October 2018

Initiation of registration-directed trial of tipifarnib in HRAS mutant HNSCC by the end of 2018

Dosing of first patient in investigator-sponsored study of tipifarnib in HRAS mutant LSCC

Biomarker-enriched data from Phase 2 trial of tipifarnib in PTCL by the end of 2018

Submission of an investigational new drug application for KO-539, a potent and selective menin-MLL inhibitor, in late 2018 or early 2019

Financial Results

Research and development expenses for the second quarter of 2018 were $11.5 million, compared to $5.7 million for the second quarter of 2017.

General and administrative expenses for the second quarter of 2018 were $3.8 million, compared to $2.3 million for the second quarter of 2017.

Net loss for the second quarter of 2018 was $14.7 million, or $0.45 per share, compared to $7.8 million, or $0.40 per share, for the second quarter of 2017.

Cash, cash equivalents and short-term investments totaled $125.9 million as of June 30, 2018, compared with $93.1 million as of December 31, 2017.

As adjusted for the $74.5 million in net proceeds resulting from the company’s public offering of common stock that closed on July 2, 2018, Kura had, on a pro forma basis, $200.4 million in cash, cash equivalents and short-term investments at June 30, 2018.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund its current operations through 2021.

Conference Call and Webcast

Kura’s management will host a webcast and conference call today at 4:30 p.m. ET / 1:30 p.m. PT today, August 6, 2018, to discuss the financial results for the second quarter of 2018 and provide a corporate update. The live call may be accessed by dialing (877) 516-3514 for domestic callers and (281) 973-6129 for international callers

and entering the conference code: 1588245. A live webcast of the call will be available from the Investors and Media section of the company website at www.kuraoncology.com, and will be archived there for 30 days.

On August 6, 2018 Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, reported financial results for the second quarter ended June 30, 2018, and provided an update on recent achievements and upcoming events (Press release, Editas Medicine, AUG 6, 2018, View Source;p=RssLanding&cat=news&id=2362273 [SID1234528651]).

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!

"During the second quarter, we continued to drive towards our first IND and to advance our broader pipeline of transformative CRISPR medicines," said Katrine Bosley, President and Chief Executive Officer of Editas Medicine. "Our lead candidate, EDIT-101 to treat the genetic disease LCA10, is poised to be the first in vivo CRISPR medicine in human trials with an anticipated IND filing in October. Our broader pipeline of ocular and engineered cell medicines is advancing as well."

Recent Achievements and Outlook

Allergan Pharmaceuticals International Limited (Allergan) exercises option to develop and commercialize EDIT-101 globally and Editas exercises option to co-develop and equally share profits and losses in the United States. Editas and Allergan announced today that Allergan has exercised its option for EDIT-101 and Allergan has paid an option exercise fee of $15 million, which will be recorded in the third quarter. In addition, Editas is eligible to receive a $25 million milestone payment from Allergan upon clearance of an IND application for EDIT-101.

EDIT-101 advancing towards clinical trials with NIH filing submitted in July and IND filing anticipated in October 2018. Editas submitted the requisite data package for human gene transfer clinical protocol registration to the United States National Institutes of Health (NIH) for potential review by the Recombinant DNA Advisory Committee. Editas plans to file an IND application for EDIT-101 with the United States Food and Drug Administration in October 2018. In addition, the Company presented new pre-clinical data on EDIT-101 at the American Society of Gene & Cell Therapy 21st Annual Meeting (ASGCT Meeting) demonstrating that EDIT-101 was well tolerated in a study of non-human primates (NHPs). Therapeutically relevant levels of editing were achieved in NHPs regardless of pre-existing or induced immunity to Staphylococcus aureus Cas9.

Broader ocular pipeline moving forward. Editas is pursuing product candidates for Usher Syndrome type 2A (USH2A) and recurrent ocular Herpes Simplex Virus type 1 (HSV-1). At the ASGCT (Free ASGCT Whitepaper) Meeting, Editas and collaborators from Massachusetts Eye and Ear presented in vitro data demonstrating that deletion of exon 13 in the human USH2A gene using CRISPR/Cas9 can restore cilia formation, providing the basis for a potential medicine. Editas also presented pre-clinical in vivo proof-of-concept data in a rabbit model for its recurrent ocular HSV-1 program at the Association for Research in Vision and Ophthalmology 2018 Annual Meeting.

Designing novel medicines for Sickle Cell Disease and Beta-Thalassemia. Editas reported data at the ASGCT (Free ASGCT Whitepaper) Meeting demonstrating that lead molecules targeting the beta-globin locus drove the upregulation of fetal hemoglobin in human mobilized peripheral blood stem cells. This was achieved by editing a novel genomic site that has potential to result in a best-in-class medicine. Editas expects to present additional data on this program in the second half of 2018.

Improving efficacy of engineered T cell medicines to treat cancer with CRISPR-based gene editing. In May, Editas expanded its collaboration with Juno Therapeutics, Inc., a Celgene company (Celgene), to develop and commercialize engineered T cell medicines for cancer. The recently expanded collaboration now encompasses four programs, including checkpoint inhibitors, tumor microenvironment, T cell receptor locus editing, and an undisclosed program.

Strong balance sheet to advance Company through multiple value inflection points. The Company held cash, cash equivalents, and marketable securities of $344.1 million as of June 30, 2018, providing at least 24 months of funding for operating expenses and capital expenditures without any assumption of future cash received from milestones or additional financings.

Upcoming Events

Editas will participate in the following investor conferences:

Citi 13th Annual Biotech Conference, Gene Editing Panel, September 5, 1:15 p.m. ET, Boston;
Morgan Stanley 16th Annual Global Healthcare Conference, Fireside Chat, September 12, 4:50 p.m. ET, New York City;
Jefferies Gene Therapy Summit, September 27, New York City; and
Chardan 2nd Annual Genetic Medicines Conference, October 9, New York City.
Editas will also participate in the following scientific and medical conferences:

26th Annual Congress of the European Society of Gene & Cell Therapy, October 16-19, Lausanne.

Second Quarter 2018 Financial Results

Cash, cash equivalents, and marketable securities at June 30, 2018, were $344.1 million, compared to $329.1 million at December 31, 2017.

For the second quarter ended June 30, 2018, net loss attributable to common stockholders was $38.7 million, or $0.82 per share, compared to $26.4 million, or $0.65 per share, for the same period in 2017.

Collaboration and other research and development revenues were $7.4 million for the quarter ended June 30, 2018, compared to $3.1 million for the same period in 2017. The $4.3 million increase was primarily attributable to $3.9 million in revenue recognized pursuant to a license agreement with Beam Therapeutics Inc. and a $2.8 million increase in revenue recognized pursuant to our collaboration agreement with Celgene, partially offset by a $2.4 million decrease in revenue recognized pursuant to our strategic alliance with Allergan.
Research and development expenses were $32.7 million for the quarter ended June 30, 2018, compared to $17.3 million for the same period in 2017. The $15.4 million increase was primarily attributable to $9.6 million in increased sublicensing and success payment expenses resulting from $12.5 million in research funding payments related to our sponsored research agreement with the Broad Institute which were partially offset by a decrease in sublicensing fees, $3.1 million in increased process and platform development expenses, $1.4 million in increased employee related expenses, $0.9 million in increased stock-based compensation expenses, and $0.4 million in increased facility-related expenses.
General and administrative expenses were $14.3 million for the quarter ended June 30, 2018, compared to $11.9 million for the same period in 2017. The $2.4 million increase was attributable to $1.1 million in increased stock-based compensation expenses, $0.7 million in increased employee related expenses, and $0.7 million in increased professional service expenses, partially offset by $0.2 million in decreased intellectual property and patent related fees.

Conference Call

The Editas management team will host a conference call and webcast today, August 6, 2018, at 5:00pm ET. To access the call, please dial 844-348-3801 (domestic) or 213-358-0955 (international) and provide the passcode 4379216. A live webcast of the call will be available on the Investors & Media section of the Editas Medicine website at www.editasmedicine.com and a replay will be available approximately two hours after its completion.

On August 6, 2018 Protagonist Therapeutics, Inc. (Nasdaq:PTGX), a biopharmaceutical company leveraging its proprietary technology platform to discover and develop novel peptide-based drugs, reported that it has signed a securities purchase agreement with investors including BVF Partners L.P. and their affiliates for the sale of 2,750,000 shares of common stock for gross proceeds of $22 million (Press release, Protagonist, AUG 6, 2018, View Source;p=RssLanding&cat=news&id=2362104 [SID1234528667]). The investors also received five-year warrants to purchase 1,375,000 shares of common stock at $10.00 per share and 1,375,000 shares of common stock at $15.00 per share. The transaction is expected to close on Aug. 8, 2018.

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!

Proceeds from the financing will be used to advance development of drug candidate PTG-100

On August 6, 2018 DotBio, a new company focused on the development of novel immuno-oncology drugs based on humanized domain antibodies, reported the company has officially launched and has successfully raised US$2.3 million in seed financing led by the HeungKong Group via Futec Biomedical Investments Limited (Press release, DotBio, AUG 6, 2018, View Source [SID1234646736]). DotBio is an independent biotechnology company that has spun out from Singapore’s Nanyang Technological University (NTU).

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!

DotBio aims to develop a broad pipeline of drug candidates to address the pressing need for new oncology treatments based on its proprietary DotBody technology. DotBodies are domain therapeutic antibodies which are multi-specific, humanized and highly-stable.

As a result of their small size, domain antibodies benefit from superior tumor penetration and can be used as building blocks for multi-specific antibodies. DotBodies are optimized by a unique proprietary technology that improves antibody stability, reduces aggregation and lowers the risk of immunogenicity – increasing the probability of their success in clinical trials. The higher stability and small size of DotBodies make them highly modular, allowing rapid optimization of pharmacokinetics, multi-valency and multi-specificity.

DotBio was founded by Professor Pär Nordlund, a world leading structural biologist at NTU and Karolinska Institute, who has pioneered strategies to define cancer drug mechanisms, and Dr. Ignacio Asial, who designed and conceptualized the DotBody technology based on his expertise in protein and antibody engineering at NTU’s School of Biological Sciences. Dr. Asial will lead DotBio as Chief Executive Officer and Dr. Kelly Hew will serve as Chief Operating Officer. Professor Nordlund will be scientific advisor to the company.

As part of the spin-out agreement with NTU, DotBio will acquire the rights to the domain antibody technology through NTUitive – the university’s innovation and enterprise company – as well as certain assets developed under a previous collaboration agreement between ASLAN Pharmaceuticals (ASLAN) and NTU. NTU and ASLAN will take minority equity stakes in DotBio. Kingsley Leung, representing HeungKong Group, and Carl Firth, Chief Executive Officer of ASLAN, will join the Board of DotBio as non-executive directors.

The funds raised will enable DotBio to generate a number of therapeutic candidates and complete validation studies. In addition to agreed collaborations with NTU and Karolinska Institute, DotBio is establishing partnerships in industry and academia to advance DotBodies in clinical development. DotBio’s current internal pipeline is focused on multi-specific immuno-oncology drugs targeting different checkpoint blockades, positive immune signals and tumor specific processes with several candidates planned to enter preclinical studies during 2018. The broad applicability of the DotBody technology will enable DotBio to consider other therapeutic areas on a case-by-case basis.

Dr Ignacio Asial, Chief Executive Officer, DotBio, commented: "This is an exciting time for us to launch DotBio, the potential of domain antibody technology to change the way we treat cancer is clear. Our focus is on applying the world-leading protein science expertise of our team to revolutionize multi-specific, CAR-T and ADC therapies, positioning DotBio as a leader in next-generation immuno-oncology drugs."

Professor Pär Nordlund, Co-Founder of DotBio, added: "Multi-specific domain antibodies offer a more refined means to activate the antitumor immune response and to minimize adverse effects as compared to standard antibody-based combination therapies. It is our belief that DotBio´s powerful domain antibody technology uniquely positions the company to become a leader in next-generation multi-specific cancer therapies. The DotBody technology can also be applied to many other therapeutic areas and we look forward to opportunities to collaborate with industry partners and academia to realize the enormous potential of our technology."