On October 18, 2018 Aclaris Therapeutics, Inc. (Nasdaq:ACRS), a dermatologist-led biopharmaceutical company focused on identifying, developing, and commercializing innovative therapies to address significant unmet patient needs in aesthetic and medical dermatology and immunology, reported the pricing of its underwritten public offering of 8,645,000 shares of its common stock at a price to the public of $10.75 per share (Press release, Aclaris Therapeutics, OCT 18, 2018, View Source [SID1234530333]). In addition, Aclaris has granted to the underwriters a 30-day option to purchase up to 1,296,750 additional shares of common stock at the public offering price, less the underwriting discount. The gross proceeds from the offering to Aclaris are expected to be approximately $92.9 million, before deducting underwriting discounts and commissions and offering expenses, but excluding any exercise of the underwriters’ option. The offering is expected to close on or about October 22, 2018, subject to customary closing conditions.

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Leerink Partners and Evercore ISI are acting as joint book-running managers for the offering. Cantor is acting as lead manager for the offering. Guggenheim Securities is acting as co-manager for the offering.

A shelf registration statement relating to this offering was filed with the Securities and Exchange Commission (SEC) on November 2, 2016 and declared effective by the SEC on November 14, 2016. The offering is being made only by means of a written prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering has been filed with the SEC and is available on the SEC’s website at www.sec.gov. A final prospectus supplement and accompanying prospectus will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus may also be obtained by contacting Leerink Partners LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, telephone: (800) 808-7525, ext. 6132, or by email at [email protected]; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 36th Floor, New York, New York 10055, by telephone at 888-474-0200, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy the securities being offered, nor shall there be any sale of the securities being offered in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On October 18, 2018 Bexion Pharmaceuticals, Inc., a clini cal stage biotechnology company focused on developing innovative cures for cancer, reported that Dr. Ray Takigiku, Founder and CEO of the Company presented recently at the 15 th Annual BioNetwork Partnering Summit, held in Laguna Nigel, CA from October 10 – 12, 2018 (Press release, Bexion, OCT 18, 2018, View Source [SID1234530294]). Chosen by the National Cancer Institute as part of its SBIR Investor Initiatives program, Bexion was among a select group of companies th at the N CI sponsored to present at this annual biotech industry financial partnering forum.

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Over 200 se nior decision makers in pharma and biotech deal making attend ed the conference to pursue relationships for partnerships and investment opportunities.

Dr. Takigiku presented the Bexion story to the audience and participated in numerous one to one meetings with pharma organizations.

About NCI SBIR Investor Initiatives Program

Through the Investor Initiatives Program, the National Cancer Institute (NCI) Small Business Innovation Research (SBIR) Development Center connects NCI SBIR funded companies wi th potential investors and strategic partners to continue the research and commercialization efforts initially funded by NCI. Companies must apply and are selected by a panel of independent experts including over 50 Venture Capital organizations and potent ial strategic partners.

On October 18, 2018 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported its new data from three of its programs, including the company’s first data on complex edits, at the 26th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), in Lausanne, Switzerland (Press release, Intellia Therapeutics, OCT 18, 2018, View Source [SID1234530291]).

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"We are extremely pleased to present an outstanding compilation of data today reflecting progress in our preclinical genome editing programs," said Intellia President and Chief Executive Officer John Leonard, M.D. "We showed that we can efficiently introduce complex edits in mice by inserting genes to express proteins that are deficient in some genetic diseases. By using our LNP delivery system in combination with AAV to deliver template DNA, we are opening the door for the development of therapies for a wide range of genetic diseases that require stable gene insertion and expression. In parallel, we are driving forward our ex vivo programs and other in vivo programs. Our researchers are gaining further insights into our ATTR program through our ongoing NHP studies, as well as working with our collaborators at Ospedale San Raffaele (OSR) to make excellent progress in our quest to advance the next generation of engineered cell therapy."

CRISPR-mediated, Targeted Gene Insertion Data

In a collaboration between Intellia and Regeneron Pharmaceuticals, Inc., researchers combined Intellia’s modular lipid nanoparticle (LNP) delivery system of CRISPR/Cas9 with a modular adeno-associated viral (AAV) insertion template to achieve supratherapeutic levels (levels higher than those required in a clinical setting) of gene expression in mice. Using Factor 9 (F9) as a model gene, the team demonstrated the first robust, efficient CRISPR-mediated targeted insertion into the liver. F9 is a gene that encodes Factor IX (FIX), a blood-clotting protein that is often missing or defective in hemophilia B patients.

Using Intellia’s proprietary bi-directional template, researchers detected hybrid mAlb-hF9 transcripts in >50 percent of hepatocytes following a single dose. Circulating human FIX protein levels of >30,000 ng/mL were achieved, which are predicted to correspond to levels 40-300 times higher than those capable of preventing bleeding episodes in hemophilia B patients, when using a wildtype or hyperfunctional version of F9 (sources: George, et al, NEJM, 2017; Simioni et al, NEJM, 2009). Researchers were able to vary FIX levels by modulating either the LNP or the AAV dose, and expression levels remained stable and ongoing in all cases throughout 12 weeks of observation.

This approach was repeated with Intellia’s wholly owned preclinical in vivo program in alpha-1 antitrypsin deficiency (AATD), another genetic disease of the liver associated with a mutation in the SERPINA1 gene that causes liver and lung dysfunction. Researchers used the LNP-AAV delivery combination of CRISPR/Cas9 components to insert donor template DNA encoding the SERPINA1 gene for AATD. The insertion resulted in blood protein levels in mice that corresponded to a range of SERPINA1 systemic levels required for normal lung function in humans.

Today’s presentation, titled "Supra-therapeutic levels of transgene expression achieved in vivo by CRISPR/Cas9 mediated targeted gene insertion," was made by Jonathan Finn, Ph.D., executive director, platform biology, Intellia. This presentation will be accessible through the Events and Presentations page of the Investor Relations section of Intellia’s website at www.intelliatx.com.

New Non-Human Primate Data from Intellia’s ATTR Program

Intellia also presented new data from non-human primate (NHP) studies in its transthyretin amyloidosis (ATTR) program further demonstrating a high correlation between liver editing and reduction of the transthyretin (TTR) protein. ATTR is a systemic, debilitating and fatal disease caused by one of approximately 136 different inherited mutations in the TTR gene. The company found that a liver editing rate of only ~35-40 percent in NHPs is needed to achieve a therapeutically meaningful reduction of TTR, specifically a TTR protein reduction of >60 percent. The data also demonstrated the transient nature of Intellia’s proprietary modular LNP delivery system, which was rapidly cleared from circulation, with all CRISPR/Cas9 components undetectable within five days of administration. Furthermore, rates of editing were durable over a six-month period without re-dosing the animals.

These data included results from ongoing collaborations with researchers at Regeneron and the University of Porto in Portugal, where ATTR is endemic in certain populations. Today’s presentation, titled "Delivering on the therapeutic potential of CRISPR/Cas9: Development of an LNP-mediated genome editing therapeutic for the treatment of ATTR," was made by Yong Chang, Ph.D., vice president, safety pharmacology, Intellia. This presentation will be accessible through the Events and Presentations page of the Investor Relations section of Intellia’s website at www.intelliatx.com.

Data Update from Intellia’s Acute Myeloid Leukemia Program

In a presentation titled "Hunting novel WT1-specific T cell receptors for immune gene therapy of acute myeloid leukemia," Intellia and its research collaborator, OSR, led by Chiara Bonini, M.D., Ph.D., deputy director of the Division of Immunology, Transplantation and Infectious Diseases at San Raffaele Hospital and University, shared an update on the company’s lead ex vivo program in acute myeloid leukemia (AML). Researchers presented in vitro data showing that CRISPR/Cas9 editing resulted in over 90 percent knockout of endogenous T cell receptors (TCRs). Subsequent transduction of Wilms’ Tumor 1 (WT1)-specific transgenic TCRs led to high expression of the inserted TCR with over 95 percent purity in isolated cytotoxic T cells (CD8+ T cells). T cells were fully functional and specifically killed leukemic blast cells that expressed the WT1 antigen and HLA-A*02:01 allele. Several additional TCRs directed to multiple WT1 epitopes and human leukocyte antigen (HLA) alleles are under investigation, including undergoing in vitro and in vivo functional testing.

Intellia and OSR are collaborating to develop best-in-class CRISPR-edited T cells directed to a specific epitope of WT1, a tumor-associated antigen overexpressed across a wide range of different tumor types and a known driver of leukocyte blasts in hematological cancers. Intellia’s first cell therapy tumor target is WT1 for the treatment of AML and other potential hematological malignancies, as well as for solid tumors.

On October 18, 2018 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the company will host a conference call and live webcast on Thursday, November 1, 2018 at 8:00 a.m. ET to report its third quarter financial results and other business highlights (Press release, Agios Pharmaceuticals, OCT 18, 2018, http://investor.agios.com/news-releases/news-release-details/agios-webcast-conference-call-third-quarter-2018-financial [SID1234530260]).

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A live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The conference call can be accessed by dialing 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 5285068. The webcast will be archived and made available for replay on the company’s website beginning approximately two hours after the event

On October 18, 2018 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that the first patient has been dosed in a first-in-human phase 1 study assessing the safety and tolerability of DS-1205, a highly selective investigational AXL inhibitor, in combination with gefitinib in patients with metastatic or unresectable epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC) who have progressed on therapy with tyrosine kinase inhibitors (TKIs) (Press release, Daiichi Sankyo, OCT 18, 2018, View Source [SID1234530247]).

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For patients with metastatic EGFR-mutated NSCLC, targeted EGFR TKI therapies such as gefitinib, erlotinib, afatinib or osimertinib represent the first-line treatment of choice.3,4,5,6 However, patients eventually develop resistance to these treatments, typically experiencing disease progression within a year.3,4,5,6 Once a patient is resistant to all possible EGFR TKIs, there are limited treatment options, including chemotherapy, immunotherapy or investigational agents.3,6 Research suggests that inhibition of AXL, a receptor tyrosine kinase, may help delay or overcome the onset of resistance to the TKIs.1,2

"This first-in-human trial will build upon preclinical research to evaluate the potential of our AXL inhibitor, DS-1205, in combination with gefitinib in EGFR-mutated NSCLC that has progressed on TKI therapy," said Eric Slosberg, PhD, Head, Oncology Translational Development, Oncology Research and Development, Daiichi Sankyo. "We are evaluating for the first time in a clinical setting whether inhibition of the AXL pathway with DS-1205 may help prevent, delay or overcome the onset of resistance to EGFR TKIs, as we continue to research and develop new targeted therapy approaches for patients with metastatic NSCLC."

About the DS-1205 and Gefitinib Combination Study

The multicenter phase 1, open-label, two-part study will enroll patients with metastatic or unresectable EGFR-mutated NSCLC who have T790M mutation-negative tumors and have experienced disease progression during treatment with erlotinib, gefitinib or afatinib, or developed disease progression while on osimertinib. The first part of the study (dose escalation) will assess the safety, tolerability, pharmacokinetics and efficacy of DS-1205 in combination with gefitinib to determine the recommended dose for expansion. The second part of the study (dose expansion) will evaluate the safety, tolerability, pharmacokinetics and efficacy of the combination therapy. Study endpoints include safety, pharmacokinetics, objective response rate, duration of response, disease control rate, time to response, progression-free survival, overall survival and exploratory biomarker analyses. The study is expected to enroll approximately 60 patients at approximately 20 sites in Japan and other countries. For more information on the clinical trial, visit ClinicalTrials.gov.

Unmet Need in EGFR-Mutated Metastatic NSCLC

Lung cancer is the most common cancer in the world and the leading cause of cancer deaths.7, 8 There were approximately 1.8 million new cases of lung cancer reported globally and 1.69 million deaths from lung cancer in 2012.8 NSCLC is the most common form of lung cancer, accounting for approximately 80 to 85 percent of all cases. It is difficult to treat, particularly in advanced disease, and the five-year survival rate for metastatic NSCLC is only one percent.9

In the past decade, significant improvements in treatment have occurred due to the development of targeted therapies, such as EGFR TKIs, for advanced NSCLC.3 EGFR mutations have been reported in a wide range of NSCLC patients (from 10 to 50 percent), and frequency may vary based on ethnicity and other factors.3,10

While the majority of patients with EGFR-mutated tumors respond to EGFR TKIs, resistance eventually develops in more than half of these patients, and they typically experience disease progression within a year.11 Resistance may be acquired or intrinsic.3 The most common mechanism for acquired resistance to gefitinib, erlotinib or afatinib involves a secondary EGFR mutation called T790M, which may be treated with the EGFR TKI osimertinib.3,4,5,6 However, patients who experience disease progression following treatment with gefitinib, erlotinib or afatinib and whose tumors lack the T790M mutation, and patients who experience disease progression following osimertinib treatment have limited options such as chemotherapy, immunotherapy or investigational treatments.5,6

Researchers are exploring specific therapeutic strategies to overcome acquired resistance to EGFR TKIs.3 Studies have demonstrated that up-regulation of AXL, a receptor tyrosine kinase, may develop in some patients with EGFR-mutated NSCLC who experience disease progression on TKI therapy, and this up-regulation might be a mechanism of resistance to EGFR TKI treatment.11,12,13 Accordingly, inhibition of AXL might restore sensitivity to EGFR TKI treatment.11 While preclinical research has demonstrated that inhibition of AXL by DS-1205 restored sensitivity to erlotinib and the addition of DS-1205 to TKI therapy at the beginning of treatment delayed the onset of resistance to gefitinib, erlotinib or osimertinib, it is as yet to be confirmed that DS-1205 will work similarly in humans.1,2

Abnormal expression and activation of AXL have been correlated with poor prognosis, increased growth and metastasis, and drug resistance in many cancers including NSCLC.12,13

About DS-1205

Part of the investigational Breakthrough Science pipeline of Daiichi Sankyo Cancer Enterprise, DS-1205 is an investigational highly selective AXL inhibitor currently being evaluated in a phase 1 clinical study in combination with gefitinib for metastatic or unresectable EGFR-mutated NSCLC. DS-1205 is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.