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 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).

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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."

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.