On August 7, 2018 RXi Pharmaceuticals Corporation (NASDAQ: RXII) a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform reported that it will report its financial results for the second quarter ended June 30, 2018, and provide a business update on Tuesday, August 14, 2018 after the close of the U.S. financial markets (Press release, RXi Pharmaceuticals, AUG 7, 2018, View Source [SID1234528489]).

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A live audio webcast will begin at 4:30 p.m. EDT. The webcast link is available under the "Investors – Events and Presentations" section of the Company’s website, www.rxipharma.com. The event may also be accessed by dialing toll-free in the United States: +1 844-376-4678. International participants may access the event by dialing: +1 209-905-5958.

An archive of the webcast will be available on the company’s website approximately two hours after the presentation.

On August 7, 2018 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported financial results for the second quarter ended June 30, 2018, and provided an update on recent clinical and corporate developments (Press release, Deciphera Pharmaceuticals, AUG 7, 2018, View Source [SID1234528505]).

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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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"The first half of 2018 was marked by exceptional progress, with data presented at the ASCO (Free ASCO Whitepaper) Annual Meeting in June demonstrating the potential of DCC-2618, our lead product candidate, in second- and third-line GIST patients, and supporting the planned Phase 3 trial, INTRIGUE, in second-line GIST patients," said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. "In addition, we observed continued robust clinical activity in heavily pretreated patients. For the balance of this year, we look forward to presenting additional data from the Phase 1 DCC-2618 study, as well as to the planned initiation of the INTRIGUE study."

Dr. Taylor continued, "In addition to our clinical progress, we also strengthened both our leadership team and balance sheet, and we are well positioned to advance our pipeline of novel kinase switch control inhibitors toward key milestones."

Clinical Programs

DCC-2618
At the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2018, Deciphera presented updated data from its ongoing Phase 1 clinical trial of DCC-2618 in patients with gastrointestinal stromal tumors (GIST). Highlights from the presentation included:
Initial objective response rates (ORR) and disease control rates (DCR) in second-and third-line GIST patients treated with DCC-2618 at ≥100mg daily exceeded previously published results of registrational trials for currently approved therapies, sunitinib in second-line patients and regorafenib in third-line patients.
Mutational profiling data across second-, third- and fourth-line GIST patients demonstrated the breadth of KIT mutations in GIST at baseline and the ability of DCC-2618 to reduce KIT mutant allele frequency.
Deciphera previously announced that following discussions with regulatory authorities in the United States and in Europe, it has designed the INTRIGUE trial as a randomized, multicenter, open-label, Phase 3 trial evaluating DCC-2618 vs. sunitinib in second-line GIST patients. The Company plans to initiate this trial later this year.
Deciphera completed enrollment in the three GIST cohorts in the expansion stage of the ongoing Phase 1 study, totaling 130 patients with second- through fourth-line plus GIST. In addition, enrollment is ongoing in the Company’s Phase 3 INVICTUS study in fourth-line and fourth-line plus GIST.
Deciphera will present an update on the GIST patients in the ongoing Phase 1 study as a Proffered Paper (oral) presentation at the ESMO (Free ESMO Whitepaper) 2018 Congress. The presentation titled "Initial Results of Phase 1 Study of DCC-2618, a Broad-spectrum KIT and PDGFRa Inhibitor, in Patients (pts) with Gastrointestinal Stromal Tumor (GIST) by Number of Prior Regimens" will be presented on October 19, 2018 in Munich.
In April 2018, the Company reported preclinical data at the Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) demonstrating that compared to the in vitro profiles of the FDA-approved kinase inhibitors imatinib, sunitinib, regorafenib, and midostaurin, and the investigational agent avapritinib (BLU-285), DCC-2618 demonstrated the broadest profile of inhibition of primary and secondary KIT mutations and primary PDGFRα mutations.
The Company also reported updated clinical data at the 2018 AACR (Free AACR Whitepaper) Annual Meeting demonstrating the safety and tolerability profile of DCC-2618 in 100 GIST patients treated at the recommended Phase 2 dose of 150 mg QD, which supports the selection of this dose for the ongoing pivotal, randomized Phase 3 INVICTUS study.
Rebastinib
Deciphera expects to initiate a company-sponsored open-label, multicenter Phase 1b study of rebastinib in combination with paclitaxel to assess safety, tolerability and pharmacokinetics in patients with locally advanced or metastatic solid tumors later this year.
DCC-3014
Deciphera continues to enroll patients in the Phase 1 dose escalation study of DCC-3014, a selective CSF1R immunokinase inhibitor, and expects to provide an update from this study later this year.
Corporate Updates

In June 2018, Deciphera announced the closing of an underwritten public offering of 4,945,000 shares at a public offering price of $40.00 per share, which included the exercise in full by the underwriters of their option to purchase up to 645,000 additional shares of common stock. Total net proceeds to Deciphera were approximately $185.3 million, after deducting underwriting discounts and commissions and other offering expenses.
In May 2018, the Company announced the appointment of Stephen B. Ruddy, Ph.D. as Chief Technical Officer. Dr. Ruddy brings to Deciphera more than 25 years of global pharmaceutical management and leadership experience in small-molecule and biologics development and manufacturing. He will be responsible for establishing and leading a world-class manufacturing and supply chain organization.
In May 2018, the Company also announced the appointment of Steven L. Hoerter, Chief Commercial Officer at Agios Pharmaceuticals, Inc., to its Board of Directors. Mr. Hoerter has more than 25 years of global pharmaceutical and biotechnology experience, having held senior positions at leading oncology companies. He will serve as an independent director and a member of the Nominating and Corporate Governance Committee.
Second Quarter 2018 Financial Results

Cash Position: As of June 30, 2018, cash and cash equivalents were $346.5 million compared to cash and cash equivalents of $196.8 million as of December 31, 2017. This increase was primarily related to proceeds obtained through the Company’s recent underwritten public offering offset by cash used in operating activities.
R&D Expenses: Research and development expenses for the second quarter of 2018 were $18.0 million compared to $8.4 million for the same period in 2017. The increase was primarily due to an increase in spending on the DCC-2618 program of $5.5 million as a result of clinical trial costs related to the pivotal Phase 3 INVICTUS study that began enrollment in January 2018 and the ongoing Phase 1 trial. Clinical costs also increased as a result of start-up activities related to the pivotal Phase 3 INTRIGUE study in second-line GIST, which is expected to be initiated in the second half of 2018. Manufacturing costs increased for DCC-2618 as a result of new process development to support anticipated greater drug requirements for commercialization as well as the manufacture of registration lots required to support the submission of a new drug application. Expenses related to our rebastinib program increased approximately $0.6 million primarily as a result of start-up activities related to our planned clinical trials. In addition, personnel-related, facility-related and other costs increased an aggregate of $3.6 million as the result of an increase in costs associated with an increase in headcount and incurred in connection with our early-stage drug discovery programs. Personnel costs for each of the second quarters of 2018 and 2017 included non-cash share-based compensation expense of $1.0 million and $0.2 million, respectively.
G&A Expenses: General and administrative expenses for the second quarter of 2018 were $4.5 million, compared to $2.2 million for the same period in 2017. The increase was primarily due to an increase in non-cash share-based compensation expense related to additional employee stock options and a higher value of our common stock and to an increase in legal and professional fees as a result of various advisory fees related to ongoing operations as a public company as well as costs incurred for pre-commercialization activities. Non-cash share-based compensation was $1.2 million and $0.4 million for each of the second quarters of 2018 and 2017, respectively.
Net Loss: For the second quarter of 2018, Deciphera reported a net loss of $21.7 million, or $0.65 per share, compared with a net loss of $10.6 million, or $0.91 per share for the same period in 2017.

On August 7, 2018 Johnson & Johnson (NYSE: JNJ) reported that it will participate in the Barclays Global Consumer Staples Conference on Thursday, Sept. 6th, at the InterContinental, Boston, MA (Press release, Johnson & Johnson, AUG 7, 2018, View Source [SID1234528658]). Jorge Mesquita, Executive Vice President, Worldwide Chairman, Consumer will represent the Company in a session scheduled at 12:45 p.m. (Eastern Time).

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This webcast will be available to investors and other interested parties by accessing the Johnson & Johnson website at www.investor.jnj.com.

A webcast replay will be available approximately two hours after the live webcast.

On August 7, 2018 ADC Therapeutics, an oncology drug discovery and development company that specializes in the development of proprietary antibody drug conjugates (ADCs), reported that the first patient has been dosed in its Phase II clinical trial intended to support the submission of Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) (Press release, ADC Therapeutics, AUG 7, 2018, View Source [SID1234596075]). The clinical trial is evaluating the efficacy and safety of ADCT-402 (loncastuximab tesirine) in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

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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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At the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, the Company presented interim Phase I data on ADCT-402 in 138 evaluable, heavily pre-treated lymphoma patients who had failed, or were intolerant to, any established therapy known to provide clinical benefit, with a median of three prior therapies. At the time, for the 49 response-evaluable patients in Part 1 of the study (dose escalation) with DLBCL who received ADCT-402 at doses greater than or equal to 120 μg/kg, the overall response rate (ORR) was 55 percent (27/49), with 18 patients achieving a complete response (37 percent) and 9 patients achieving a partial response (18 percent).

The primary endpoint of the Phase II, multi-center, open-label, single-arm trial is the ORR in patients treated with ADCT-402, as confirmed by central review. Secondary endpoints include assessments of duration of response, complete response rate, relapse-free survival, progression-free survival and overall survival, as well as safety, pharmacokinetics and health-related quality of life. The trial will enroll approximately 140 patients with relapsed or refractory DLBCL at multiple centers in the USA and Europe.

"We are pleased to have dosed the first patient in our registrational Phase II clinical trial evaluating ADCT-402 in patients with DLBCL who have relapsed and have refractory disease after two or more multi-agent treatment regimens. Our Phase I clinical trial of ADCT-402 in non-Hodgkin lymphoma showed significant activity in patients with DLBCL and an acceptable safety profile," said Jay Feingold, MD, PhD, Chief Medical Officer and Senior Vice President of Clinical Development at ADC Therapeutics. "Unfortunately, there is no effective treatment for patients with multiple relapsed and refractory DLBCL, so we are excited about the potential to improve outcomes in these patients with ADCT-402 in a single-arm trial. We anticipate reporting results from the Phase II trial in the third quarter of 2019 and are hopeful that the data will support our submission of a BLA to the FDA."

Alex Spira, MD, PhD, FACP, Director of Virginia Cancer Specialists Research Institute and Clinical Assistant Professor of Oncology at Johns Hopkins School of Medicine, added, "Patients with DLBCL who have relapsed or are refractory after second-line chemotherapy face a very poor prognosis. There is a

significant unmet need for an effective new treatment option for this patient population, and we believe ADCT-402 has the potential to help impact patient outcomes in this disease."

For more information about the Phase II clinical trial, please visit www.clinicaltrials.gov (identifier NCT03589469).

ADC Therapeutics also plans to initiate multiple combination studies with ADCT-402 in the fourth quarter of 2018.

About Diffuse Large B-Cell Lymphoma (DLBCL)

Non-Hodgkin lymphoma (NHL) is the seventh most common type of cancer in the U.S., and accounted for an estimated 4.3 percent of new cancer cases in 2017.1 Diffuse large B-cell lymphoma (DLBCL) accounts for nearly one-third (32.5 percent) of NHL.2 The most common initial treatment for patients with DLBCL is chemo-immunotherapy. Response to initial treatment is high, but more than half of patients do not have long-term disease control.3 The current standard of care for relapsed DLBCL is additional chemotherapy, which can be followed by stem cell transplantation (SCT). The prognosis for relapsed patients is poor, especially for those with chemotherapy-refractory disease with a short interval between remission and relapse or those who relapse after high-dose therapy and SCT. There is a significant unmet need for an effective treatment for patients with relapsed or refractory DLBCL.

About ADCT-402

ADCT-402 is an antibody drug conjugate (ADC) composed of a humanized monoclonal antibody that binds to human CD19, conjugated through a linker to a pyrrolobenzodiazepine (PBD) dimer toxin. Once bound to a CD19-expressing cell, ADCT-402 is internalized into the cell where enzymes release the PBD-based warhead. CD19 is a clinically validated target for the treatment of B-cell malignancies. The PBD-based warhead has the ability to form highly cytotoxic DNA interstrand cross-links, blocking cell division and resulting in cell death. Preliminary data from a Phase I clinical trial in relapsed or refractory B-cell non-Hodgkin lymphoma demonstrate ADCT-402 has significant activity in patients with diffuse large B-cell lymphoma (DLBCL). ADCT-402 is also being evaluated in an ongoing Phase I clinical trial in patients with relapsed or refractory B-cell lineage acute lymphoblastic leukemia (B-ALL). The U.S. Food and Drug Administration has granted orphan drug designation to ADCT-402 for the treatment of DLBCL and mantle cell lymphoma.

On August 7, 2018 BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) reported financial results for the second quarter ended June 30, 2018 (Press release, BioCryst Pharmaceuticalsa, AUG 7, 2018, http://ir.biocryst.com/news-releases/news-release-details/biocryst-reports-second-quarter-2018-financial-results [SID1234528490]).

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"Building on the clear direction from our shareholders and a strong conviction in the medical community that BCX7353 is a highly differentiated asset which can deliver enormous value to patients and shareholders, we have made substantial progress advancing our prophylactic and acute BCX7353 clinical programs, and significantly strengthened our balance sheet," said Jon P. Stonehouse, BioCryst’s President and Chief Executive Officer.

"Enrollment in the ZENITH-1 trial has completed, and we look forward to reporting Part 1 results later this quarter. Enrollment in the APeX-2 and APeX-S trials continues to go extremely well, and we are confident that we will report top-line safety and efficacy in the second quarter of next year. We believe we have the programs, the focused commitment of an experienced team and the financial resources to deliver significant value to patients and shareholders with our existing portfolio and we are excited about the clinical and regulatory milestones ahead of us in the next 12 months," Stonehouse added.

Second Quarter 2018 Financial Results

For the three months ended June 30, 2018, total revenues were $12.5 million, compared to $3.1 million in the second quarter of 2017. The increase in revenue was primarily associated with the recognition of $7.0 million of deferred revenue and a $5.0 million milestone, both associated with the European Medicines Agency’s (EMA) approval of peramivir (ALPIVABTM). These revenues were partially offset by lower collaboration revenue under U.S. Government development contracts.

Research and Development (R&D) expenses for the second quarter of 2018 increased to $21.0 million from $15.8 million in the second quarter of 2017, primarily due to increased spending on the Company’s hereditary angioedema (HAE) and preclinical programs, as well as additions in R&D personnel. These increases were partially offset by decreased activity under U.S. Government development contracts.

General and administrative (G&A) expenses for the second quarter of 2018 increased to $9.5 million, compared to $2.8 million in the second quarter of 2017. The increase was primarily due to a $4.9 million reserve recorded for concern regarding the collectability of the EMA approval milestone, as well as incurred merger-related costs. As previously disclosed, BioCryst and Seqirus are engaged in a formal dispute resolution that involves many items under the contract including, but not limited to, the EMA approval milestone.

Interest expense was $2.2 million in the second quarter of 2018, compared to $2.1 million in the second quarter of 2017. Also, a $619,000 mark-to-market gain on the Company’s foreign currency hedge was recognized in the second quarter of 2018, as compared to a $400,000 mark-to-market loss in the second quarter of 2017. These changes result from periodic changes in the U.S. dollar/Japanese yen exchange rate. During the second quarters of 2018 and 2017, the Company also realized currency gains of $889,000 and $921,000, respectively, from the exercise of a U.S. Dollar/Japanese yen currency option within its foreign currency hedge.

Net loss for the second quarter of 2018 was $18.5 million, or $0.19 per share, compared to a net loss of $16.9 million, or $0.21 per share, for the second quarter 2017.

Cash, cash equivalents and investments totaled $122.1 million at June 30, 2018, and reflect a decrease from $159.0 million at December 31, 2017. Net operating cash use for the second quarter 2018 was $18.4 million, and the first six months of 2018 was $41.3 million.

Year to Date 2018 Financial Results

For the six months ended June 30, 2018, total revenues were $16.5 million, compared to $12.5 million in the first half of 2017. The increase in revenue was primarily associated with the recognition of $7.0 million of deferred revenue and a $5.0 million milestone payment, both associated with the EMA approval of peramivir. These revenues were offset by infrequent revenue events that occurred in 2017 that did not recur in 2018. Those 2017 events were the recognition of $4.1 million of royalty revenue from Japanese government stockpiling of RAPIACTA and a $2.0 million payment for the Canadian regulatory approval of RAPIVAB. The increase in revenues was partially offset by lower collaboration revenue under U.S. Government development contracts.

R&D expenses increased to $39.5 million from $32.5 million in the first half of 2017, primarily due to increased spending on our HAE and preclinical programs. These increases were partially offset by a decrease in the Company’s peramivir and galidesivir development spending in 2018.

G&A expenses for the first half of 2018 increased to $17.1 million, compared to $5.9 million in the first half of 2017. The increase was primarily due to approximately $6.4 million of merger-related costs associated with the Company’s failed merger with Idera Pharmaceuticals, Inc. (Idera) and a $4.9 million reserve for collectability of the EMA approval milestone of peramivir.

Interest expense was $4.4 million in the first half of 2018, compared to $4.2 million in the first half of 2017. Also, a $1.2 million mark-to-market loss on the Company’s foreign currency hedge was recognized in the first half of 2018, as compared to a $1.9 million mark-to-market loss in the first half of 2017. These changes result from periodic changes in the U.S. dollar/Japanese yen exchange rate. During 2018 and 2017, the Company also realized currency gains of $889,000 and $921,000, respectively, from the exercise of a U.S. Dollar/Japanese yen currency option within its foreign currency hedge.

Net loss for the first half of 2018 was $44.2 million, or $0.45 per share, compared to a net loss of $31.1 million, or $0.40 per share, for the first half 2017.

Clinical Development Update & Outlook

On August 6, 2018, BioCryst announced it had received Fast Track Designation by the U.S. Food and Drug Administration (FDA) for BCX7353 for the prevention of angioedema attacks in patients with HAE.

On August 6, 2018, BioCryst announced the full exercise of the underwriters’ option to purchase additional shares and the completion of its public offering resulting in the sale of 10,454,546 shares of its common stock at a price of $5.50 per share. The net proceeds from this offering are approximately $53.5 million, after deducting underwriting discounts and commissions and other estimated offering expenses.

On July 11, 2018, BioCryst announced it had completed enrollment in all three cohorts of its ZENITH-1 clinical trial, a proof-of-concept Phase 2 clinical trial liquid formulation of BCX7353 for treatment of acute HAE attacks.

On July 25, 2018, BioCryst announced that results from the Phase 2, APeX-1 trial of BCX7353 for the prevention of attacks in patients with HAE were published in the July 26th issue of The New England Journal of Medicine.

On July 20, 2018, BioCryst entered into a $30 million secured loan facility with MidCap Financial Trust as administrative agent and lender (MidCap), pursuant to the terms and conditions of that certain Amended and Restated Credit and Security Agreement. The Credit Agreement replaces the Credit and Security Agreement dated as of September 23, 2016.

On July 10, 2018, BioCryst announced that it had terminated the previously announced merger agreement with Idera following the Company’s stockholders’ failure to approve the adoption of the merger agreement. Pursuant to the merger agreement, the Company reimbursed Idera$6 million in July.

On June 25, 2018, BioCryst announced that the Company had reached agreement on the design of a Phase 3 trial and regulatory requirements for marketing authorization of BCX7353 for HAE with the Pharmaceuticals and Medical Devices Agency in Japan.

On May 24, 2018, BioCryst announced that the EMA Committee for Orphan Medicinal Products issued a positive opinion on BioCryst’s application for orphan designation of BCX7353 for the treatment of HAE. In addition, the United Kingdom’s Medicines and Healthcare products Regulatory Agency has granted a Promising Innovative Medicine designation to BCX7353.
Financial Outlook for 2018

Based upon development plans, merger-related incurred costs from the recently terminated merger agreement with Idera and awarded government contracts, BioCryst expects its 2018 net operating cash use to be in the range of $85 to $105 million, and its 2018 operating expenses to be in the range of $90 to $110 million. The Company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the Company’s stock, as well as by the vesting of the Company’s outstanding performance-based stock options.

Conference Call and Webcast

BioCryst’s leadership team will host a conference call and webcast Tuesday, August 7, 2018 at 11:00 a.m. Eastern Time to discuss these financial results and recent corporate developments. To participate in the conference call, please dial 1-877-303-8027 (United States) or 1-760-536-5165 (International). No passcode is needed for the call. The webcast can be accessed live or in archived form in the "Investors" section of the Company’s website at www.BioCryst.com. An accompanying slide presentation may also be accessed via the BioCryst website. Please connect to the website at least 15 minutes prior to the start of the conference call to ensure adequate time for any software download that may be necessary.

About BCX7353

Discovered by BioCryst, BCX7353 is a novel, oral, once-daily, selective inhibitor of plasma kallikrein currently in development for the prevention and treatment of angioedema attacks in patients diagnosed with HAE. BCX7353 was generally safe and well tolerated in the Phase 2 APeX-1 clinical trial. BioCryst is currently conducting the Phase 3 APeX-2 clinical trial and the long-term safety APeX-S clinical trial, both evaluating two dosage strengths of BCX7353 administered orally once-daily as a preventive treatment to reduce the frequency of attacks in patients with HAE. BioCryst is also conducting the ZENITH-1 clinical trial. ZENITH-1 is a proof-of-concept Phase 2 clinical trial testing an oral liquid formulation of BCX7353 for the treatment of acute angioedema attacks.