On May 8, 2018 Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical- stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, reported upcoming presentations during two conferences, both to take place May 16 – 19, 2018; the 21st annual American Society of Gene and Cell Therapy 2018 conference being held in Chicago, IL, and the International Investigative Dermatology conference being held in Orlando, FL (Press release, Abeona Therapeutics, MAY 8, 2018, View Source;p=RssLanding&cat=news&id=2347659 [SID1234526221]).

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American Society of Gene and Cell Therapy
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Oral Presentations:

Phase 1/2 Clinical Trial for Recessive Dystrophic Epidermolysis Bullosa Using EB-101 (COL7A1 Gene-Corrected Autologous Keratinocytes)
Presenter: Jean Yuh Tang, M.D., Ph.D., Stanford University
Thursday, May 17, 2018
Time: 8:20 AM Central Time

Update on Phase 1/2 Gene Transfer Clinical Trial of Systemic Gene Transfer of scAAV9.U1a.hSGSH for MPSIIIA (Sanfilippo Syndrome)
Presenter: Kevin Flanigan, M.D., Nationwide Children’s Hospital
Friday, May 18, 2018
Time: 8:30 AM Central Time

A Phase 1/2 Clinical Trial of Systemic Gene Transfer of rAAV9.CMV.hNAGLU for MPS IIIB: Safety, Tolerability, and Preliminary Evidence of Biopotency
Presenter: Kevin Flanigan, M.D., Nationwide Children’s Hospital
Friday, May 18, 2018
Time: 4:30 PM Central Time

Symposium and Forum:

Assessing Neurodevelopment in Neurodegenerative Disease: The Use and Interpretation of Cognitive Scales
Presenter: Maria Escolar, M.D., Children’s Hospital of Pittsburgh of UPMC
Moderator: Barry Byrne, M.D., Ph.D., University of Florida
Thursday, May 17, 2018
Time: 7:00 AM Central Time

Tools and Technologies Forum
Co-Chair: S. Kaye Spratt, Ph.D., Abeona Therapeutics Inc.
Friday, May 18, 2018
Time: 5:45 PM – 7:45 PM Central Time

Poster Presentations:

Poster 547: Development of a Multi-Domain Responder Index for Clinical Trials with Multi-Domain Diseases
Presenter: Magdalena Tyrpien, Abeona Therapeutics Inc.
Thursday, May 17, 2018
Time: 5:15 PM Central Time

Poster 399: Identification of Novel AAV Capsids for Enhanced CNS Gene Transfer
Presenter: Daphne Chen, University of North Carolina, Chapel Hill
Thursday, May 17, 2018
Time: 5:15 PM Central Time

International Investigative Dermatology

View Source

Poster Presentation:

Poster 328: 50% Wound Healing Correlates with RDEB Patient Reported Outcomes in Pain, Itch, and Skin Durability
Friday, May 18, 2018
Time: 12:00 PM Eastern Time

On May 8, 2018 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the first quarter ended March 31, 2018 and recent company developments (Press release, Manhattan Pharmaceuticals, MAY 8, 2018, View Source [SID1234526205]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "2018 is off to a great start as we continue to advance our major pivotal programs and bolster our product pipeline, most recently with the addition of a BTK inhibitor program. As we move into the second quarter and the remainder of the year, we look forward to the announcement of topline data from our UNITY-CLL trial, completion of enrollment into the current cohorts of the UNITY-NHL study, final MS Phase 2 data, significant enrollment in our MS phase 3 program as well as filing decisions related to the Company’s first BLA/NDA later in the year." Mr. Weiss continued, "We believe 2018 will be a pivotal year for the Company as approval pathways across multiple indications become clearer and position us for future success."

First Quarter and Recent Highlights

BTK License: Entered into an exclusive global license agreement with Jiangsu Hengrui Medicine Co., Ltd. (or "Hengrui") to obtain worldwide rights, excluding Asia but including Japan, for the development of Hengrui’s Bruton’s Tyrosine Kinase (BTK) inhibitor program, including the lead candidate, TG-1701.
Umbralisib Lancet Publication: Results from the Phase 1 first-in-human study of umbralisib (TGR-1202), the Company’s novel once-daily PI3K delta inhibitor, were published in The Lancet Oncology.
TG-1601 Preclinical Data: Presented the first preclinical data from TG-1601, the Company’s novel BET inhibitor, at the 2018 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting.
Ublituximab Data in Multiple Sclerosis: Presented clinical and MRI data from the Phase 2 trial of ublituximab (TG-1101) in RMS at the 3rd Annual Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum 2018 and the American Academy of Neurology (AAN) 70th Annual Meeting.

Remaining 2018 Milestones

Present top-line overall response rate data from the UNITY-CLL Phase 3 trial of ublituximab plus umbralisib in front line and relapsed/refractory Chronic Lymphocytic Leukemia (CLL).
Prepare and potentially file the Company’s first BLA and/or NDA.

Complete enrollment in the current arms of the UNITY-NHL trial, including the Follicular Lymphoma, Marginal Zone Lymphoma, and Diffuse Large B-Cell Lymphoma cohorts.
Present updated clinical data from ongoing oncology trials and final results from the Phase 2 trial of ublituximab in Multiple Sclerosis (MS) at major medical meetings during 2018.

Financial Results for the First Quarter 2018

Cash Position: Cash, cash equivalents, investment securities, and interest receivable were $109.2 million as of March 31, 2018. Pro-forma cash, cash equivalents, investment securities, and interest receivable as of March 31, 2018 (excluding our second quarter 2018 operations) are approximately $123.3 million, after giving effect to $14.1 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the second quarter of 2018.

Other R&D Expenses: Other research and development (R&D) expense (not including non-cash compensation) was $32.2 million for the three months ended March 31, 2018 compared to $20.4 million for the three months ended March 31, 2017. Included in other research and development expense for the three months ended March 31, 2018 was $14.5 million of clinical trial expense and $9.6 million of manufacturing and CMC expenses for Phase 3 clinical trials and potential commercialization. The current period increase in Other R&D expenses is primarily due to the ongoing clinical development programs and related manufacturing costs for TG-1101 and TGR-1202.

●Other G&A Expenses: Other general and administrative (G&A) expense (not including non-cash compensation) was $2.1 million for the three months ended March 31, 2018 as compared to $1.3 million for the three months ended March 31, 2017. Other G&A expenses for the three months ended March 31, 2018 remained relatively flat compared to the first quarter of 2017, and we expect Other G&A expenses to increase modestly through the remainder of 2018.

●Net Loss: Net loss was $41.5 million for the three months ended March 31, 2018, compared to a net loss of $27.7 million for the three months ended March 31, 2017. Excluding non-cash items the net loss for the three months ended March 31, 2018 was approximately $33.2 million.

●Financial Guidance: Net cash utilized for operating activities during the three months ended 2018 was approximately $28.0 million. The Company believes its cash, cash equivalents, investment securities, and interest receivable on hand as of March 31, 2018, inclusive of the proceeds raised subsequent to the first quarter, will be sufficient to fund the Company’s planned operations through mid-2019.

Conference Call Information

The Company will host an investor conference call today, May 8, 2018, at 8:30am ET, to discuss the Company’s first quarter 2018 financial results and provide a business outlook for the remainder of 2018.

In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics First Quarter 2018 Earnings Call. A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

On May 8, 2018 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported first quarter 2018 financial results (Press release, Loxo Oncology, MAY 8, 2018, View Source [SID1234526204]).

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"In the first quarter, we delivered against all of our planned corporate goals. We completed the rolling NDA submission for larotrectinib, enrolled well on the LOXO-292 Phase 1 trial and moved LOXO-305 toward a clinical start in the second half of 2018. We also entered into an important collaboration with Illumina to develop a companion diagnostic test for larotrectinib and LOXO-292," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "We look forward to providing a comprehensive update on the Phase 1 study of LOXO-292 at ASCO (Free ASCO Whitepaper) on June 2nd."

Recent Highlights

Companion Diagnostic (CDx) Partnership

Global Development and Commercialization Partnership with Illumina: On April 10, 2018, Loxo Oncology announced a global strategic partnership with Illumina to develop and commercialize a CDx version of Illumina’s TruSight Tumor 170 for NTRK fusions and RET fusions/mutations as a Class III FDA-approved companion diagnostic in conjunction with larotrectinib and LOXO-292, respectively. The companies are also planning to broaden the clinical utility of the full panel by seeking approval for the rest of the assay content as a Class II cancer genomic profiling test. More information can be found here.
Larotrectinib

Completion of New Drug Application (NDA) Rolling Submission to FDA: On March 26, 2018, Loxo Oncology completed the rolling submission of an NDA to the U.S. Food and Drug Administration (FDA) for larotrectinib for the treatment of adult and pediatric patients with locally advanced or metastatic solid tumors harboring an NTRK gene fusion. More information can be found here.
The Lancet Oncology Publication: On March 29, 2018, The Lancet Oncology published results for larotrectinib in the treatment of pediatric patients with TRK fusion cancer. The publication included results from the ongoing pediatric Phase 1/2 trial. In this trial, larotrectinib induced an objective response rate (ORR) of 93 percent in pediatric patients with TRK fusion-positive solid tumors. The publication can be found here.
LOXO-292

Annals of Oncology Publication: On April 18, 2018, the Annals of Oncology published a manuscript illustrating the preclinical profile of LOXO-292, preclinical work supporting LOXO-292’s selective inhibition of RET, and continued evidence of clinical proof-of-concept. The publication included two patient cases who both presented with RET-altered, multikinase (MKI) inhibitor-resistant cancers. One patient harbored RET M918T-mutant medullary thyroid cancer with an acquired RET V804M gatekeeper resistance mutation, and the other patient harbored KIF5B-RET fusion-positive non-small cell lung cancer with brain metastases. The latter case was previously reported on in October 2017 in a presentation at the International Association for the Study of Lung Cancer 18th World Conference on Lung Cancer. Due to clinical urgency, both were treated with LOXO-292 on intra-patient dose escalation single patient protocols. Both patients achieved RECIST confirmed partial responses. This represents the first clinical report of the predicted RET V804M gatekeeper mutation arising in a patient previously treated with MKIs, and the first successful treatment of a patient in that setting. In this two-patient dataset, LOXO-292 was well-tolerated with no adverse events attributed to LOXO-292. The publication can be found here.
LOXO-292 Oral Presentation Accepted at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting: On April 4, 2018, Loxo Oncology announced that LOXO-292 interim clinical data from the ongoing Phase 1 clinical trial will be presented in an oral presentation at the ASCO (Free ASCO Whitepaper) Annual Meeting held June 1 – 5, 2018 in Chicago, Illinois. The presentation is entitled "A Phase 1 Study of LOXO-292, A Potent and Highly Selective RET Inhibitor, in Patients with RET-Altered Cancers." Loxo Oncology will host a conference call and live webcast on Saturday, June 2, 2018 at 4:00 p.m. CT to discuss the clinical data after they are presented at ASCO (Free ASCO Whitepaper). Information on how to access the call and webcast can be found below.
Upcoming Milestones

Larotrectinib (TRK)
FDA is expected to accept the filing of the New Drug Application in the first half of 2018
Marketing Authorisation Application submission by Bayer in the European Union is expected in 2018
Presentation of updated TRK fusion clinical data is expected in the second half of 2018
LOXO-195 (next-generation TRK)
Presentation of updated clinical data is expected in the second half of 2018
LOXO-292 (RET)
Presentation of updated clinical data at the ASCO (Free ASCO Whitepaper) Annual Meeting
LOXO-305 (BTK)
Initiation of a Phase 1 clinical trial is expected in the second half of 2018
First Quarter 2018 Financial Results

As of March 31, 2018, Loxo Oncology had aggregate cash, cash equivalents and investments of $735.6 million, compared to $626.2 million as of December 31, 2017. Loxo Oncology received the remaining $150 million of the $400 million upfront payment related to the Bayer collaboration in the first quarter of 2018.

Revenue from the collaboration agreement was $38.4 million for the first quarter of 2018, compared to none for the first quarter of 2017. This represents $42.9 million in revenue recognized from the $400 million upfront payment from the Bayer collaboration offset by $4.4 million, Loxo Oncology’s share of the joint larotrectinib co-promotion costs. Loxo Oncology recognizes revenue from the upfront payment on a proportional performance basis utilizing a calculation based on quarterly research and development spending associated with larotrectinib and LOXO-195, relative to cumulative and forecasted research and development spending on larotrectinib and LOXO-195 over the course of the collaboration agreement. As a result, the quarterly revenue recognized for the upfront payment varies from quarter to quarter. A supporting schedule that shows the different components of revenue from the collaboration agreement is included with the attached financial statements.

Research and development expenses were $32.0 million for the first quarter of 2018 compared to $20.2 million for the first quarter of 2017. This increase was primarily due to expanded larotrectinib development activities including clinical costs, as well as additional development expenses related to our other programs, and higher employment costs primarily due to increased headcount. These numbers are net of 50/50 cost-sharing with Bayer for larotrectinib and LOXO-195 development costs. Loxo Oncology recognized research and development-related stock-based compensation expense of $4.3 million during the first quarter of 2018 as compared to $2.4 million for the first quarter of 2017.

General and administrative expenses were $12.2 million for the first quarter of 2018 compared to $4.8 million for the first quarter of 2017. The increase was primarily due to additional headcount and associated employment costs and general and administrative professional fees. Loxo Oncology recognized general and administrative-related stock-based compensation expense of $5.4 million during the first quarter 2018 compared to $1.6 million for the first quarter of 2017.

Net loss was $3.6 million for the first quarter of 2018, compared to $24.5 million for the first quarter of 2017. This decrease in net loss is primarily driven by the revenue recognized from the $400.0 million upfront payment from the Bayer collaboration, the larotrectinib and LOXO-195 development reimbursement from the Bayer collaboration, offset by increases in operating expenses.

Non-GAAP net loss was $36.8 million for the first quarter of 2018, compared to $20.6 million for the first quarter of 2017. This non-GAAP net loss measure, more fully described below under "Non-GAAP Financial Measures," excludes the recognition of collaboration revenue related to the Bayer upfront payment and share-based compensation expenses. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

Earnings Conference Call Information
Loxo Oncology will host a conference call today at 8:00 a.m. ET to discuss the first quarter 2018 financial results and company updates. To participate in the conference call, please dial (877) 930-8065 (domestic) or (253) 336-8041 (international) and refer to conference ID 1979239. A replay will be available shortly after the conclusion of the call and archived on the company’s website for 30 days following the call.

ASCO Conference Call and Webcast Information
Loxo Oncology will be hosting a conference call and live webcast with slides and Q&A on Saturday, June 2, 2018 at 4:00 p.m. CT to discuss the interim LOXO-292 clinical data after they are presented at ASCO (Free ASCO Whitepaper). To participate in the conference call, please dial (877) 930-8065 (domestic) or (253) 336-8041 (international) and refer to conference ID 3597058. A live webcast of the presentation will be available at View Source A replay will be available shortly after the conclusion of the call and archived on the company’s website for 30 days following the call.

On May 8, 2018 Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, reported that it will participate in a fireside chat at the Bank of America Merrill Lynch 2018 Health Care Conference on Tuesday, May 15, 2018, at 8 a.m. PT (11:00 a.m. ET) in Las Vegas, Nevada (Press release, Editas Medicine, MAY 8, 2018, View Source;p=RssLanding&cat=news&id=2347637 [SID1234526203]).

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A live webcast of the presentation will be available on the Investors & Media section of the Editas Medicine website at www.editasmedicine.com. An archived replay will be available for approximately 30 days following the presentation.

On May 8, 2018 bluebird bio, Inc. (Nasdaq: BLUE) reported that members of the management team will present at the following upcoming investor conferences (Press release, bluebird bio, MAY 8, 2018, View Source [SID1234526202]):

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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

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Deutsche Bank’s 43rd Annual Health Care Conference, Wednesday, May 9, at 10:00 a.m. ET at the Intercontinental Hotel, Boston, Massachusetts.
Bank of America Merrill Lynch 2018 Healthcare Conference, Tuesday, May 15, at 2:20 p.m. PT at the Encore at Wynn Hotel, Las Vegas, Nevada.
To access the live webcasts of bluebird bio’s presentations, please visit the "Events & Presentations" page within the Investors and Media section of the bluebird bio website at View Source Replays of the webcasts will be available on the bluebird bio website for 90 days following the conferences.