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]).

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"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]).

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"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.

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

On August 6, 2018 Alteogen Inc. (KOSDAQ: 196170) reported that it has successfully got an approval for orphan drug designation from the US Food and Drug Administration (FDA) with one of its assets (ALT-P7) for gastric cancer (designation letter 18-6439) last week (Press release, Alteogen, AUG 6, 2018, View Source [SID1234528458]). ALT-P7 is an antibody-drug conjugate (ADC) using a Trastuzumab variant form of antibody, and this approval would guarantee Alteogen the market exclusivity rights for seven years after FDA’s approval of ALT-P7 for gastric cancer treatment.

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The FDA designation of orphan drug is designed to support the development and approval of drugs for the treatment of rare diseases or life-threatening illnesses. Products designated as orphan drugs would be recognized for not only market exclusivity after FDA approval, but also get additional benefits, such as tax benefit of the total costs for clinical trial studies, scientific advice meetings with FDA during the development process, and waiver of marketing application user fees.

ALT-P7, a candidate ADC treatment drug for gastric and breast cancers, is an anti-cancer ADC utilizing the company’s unique "NexMabTM" platform technology, a proprietary next-generation site-specific conjugation methodology developed by the company. The related patents are registered in seven countries so far.

ALT-P7 is currently undergoing a first-in-human phase 1 clinical trial for breast cancer patients in Korea, after the investigational new drug (IND) approval last year from the Ministry of Food and Drug Safety (MFDS) of Korea (NCT03281824). Alteogen is planning to start phase 2 clinical trial for breast cancer patients in 2019. Following the current breast cancer trial, Alteogen will extend the clinical development of ALT-P7 for gastric cancer as well, which already has proven efficacy in pre-clinical in vitro and in vivo studies.

"The orphan drug designation of ALT-P7 by the US FDA will accelerate the advancement of gastric cancer treatment in the US," said Dr. Soon Jae Park, Ph.D., CEO of Alteogen. "We believe that ALT-P7 can provide a breakthrough in the treatment of Her-2 overexpressing gastric cancer, for which there is no effective target treatment yet".

On August 6, 2018 Neon Therapeutics, Inc. (Nasdaq: NTGN), a clinical-stage immuno-oncology company developing neoantigen-targeted therapies, reported financial results and provided a business update for the second quarter of 2018 (Press release, Neon Therapeutics, AUG 6, 2018, View Source [SID1234528483]).

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"The first half of 2018 was transformative for Neon, highlighted by the successful completion of our initial public offering. The capital raised will enable the continued execution of our strategy to develop neoantigen-targeted therapies that have the potential to transform patient lives," said Hugh O’Dowd, President and Chief Executive Officer of Neon. "We are pleased by the continued progress across our entire product portfolio of vaccine and T-cell programs, including completion of enrollment of our first Phase 1b clinical trial for NEO-PV-01. In addition, updated data from this trial will be presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in October 2018."

Second Quarter Business Highlights

Neon achieved several key operational milestones during the second quarter of 2018, including the following:

· In June 2018, Neon completed its initial public offering of common stock, raising $100 million in gross proceeds.

· In May 2018, Neon announced that the first patient was dosed in NT-002, its Phase 1b clinical trial evaluating the company’s personal neoantigen vaccine, NEO-PV-01, in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), along with chemotherapy.

· In April 2018, Neon presented updated data from NT-001, its Phase 1b clinical trial of NEO-PV-01 in the metastatic setting, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

Pipeline Overview and Upcoming Milestones

NEO-PV-01

· NT-001: Phase 1b Clinical Trial of NEO-PV-01 in the Metastatic Setting

· Updated data expected in October 2018 at ESMO (Free ESMO Whitepaper)

· 52-week data expected in the first half of 2019

· NT-002: Phase 1b Clinical Trial of NEO-PV-01 in Metastatic Non-Small Cell Lung Cancer

· 52-week data expected in the second half of 2019

· NT-003: Phase 1b Clinical Trial of NEO-PV-01 in Metastatic Melanoma Combinations

· Trial initiation expected in the second half of 2018

· NT-004: Phase 1b Clinical Trial of NEO-PV-01 in earlier disease setting

· Planning ongoing

NEO-PTC-01

· Phase 1 Clinical Trial in Solid Tumor Setting: Neon intends to submit a European Clinical Trial Application (CTA) for NEO-PTC-01, a personal neoantigen T-cell therapy, in the first half of 2019.

NEO-SV-01

· Phase 1 Clinical Trial in Subset of ER+ Breast Cancer: Following the completion of target validation and preclinical product development work, Neon expects to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the first half of 2019.

Second Quarter 2018 Financial Results

· Cash Position: As of June 30, 2018, cash, cash equivalents and marketable securities were $138.6 million, as compared to cash, cash equivalents and marketable securities of $79.7 million as of December 31, 2017.

· R&D Expenses: R&D expenses were $14.8 million for the quarter ended June 30, 2018, as compared to $7.3 million for the same quarter last year. The increase of $7.5 million was due primarily to increased costs related to the advancement of NEO-PV-01, including increased external manufacturing and other external costs to support our ongoing and planned clinical trials, as well as increased personnel-related costs due to additional headcount to support the growth of our research and development organization.

· G&A Expenses: G&A expenses were $4.3 million for the quarter ended June 30, 2018, compared to $2.4 million for the same quarter last year. The increase of $1.9 million was driven by increased personnel-related costs due to additional headcount, as well as increased consulting and professional fees.

· Net Loss Attributable to Common Stockholders: Net loss attributable to common stockholders was $22.1 million for the quarter ended June 30, 2018, or $7.84 per basic and diluted share, as compared to a net loss attributable to common stockholders of $12.1 million for the same quarter last year, or $7.55 per basic and diluted share.

Financial Guidance: Based on its current operating plan, Neon expects that its cash, cash equivalents and marketable securities as of June 30, 2018, including the proceeds from its initial public offering, will enable it to fund its operating expenses and capital expenditure requirements into at least the first quarter of 2020.