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Deciphera Pharmaceuticals, Inc. Announces Third Quarter 2018 Financial Results

On November 8, 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 third quarter ended September 30, 2018 and provided an update on clinical and corporate developments (Press release, Deciphera Pharmaceuticals, NOV 8, 2018, View Source [SID1234530953]).

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"Recent clinical and corporate achievements support Deciphera’s transition to a late-stage, pre-commercial company," said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. "Data presented at ESMO (Free ESMO Whitepaper) have bolstered our confidence in DCC-2618’s potential to transform the current treatment paradigm for GIST patients. As we approach the completion of enrollment for our pivotal Phase 3 INVICTUS study in the coming weeks, we look forward to the data readout from that study expected next year. In addition, we remain on track to initiate our Phase 3 INTRIGUE study later this year. We are building our commercial capabilities for DCC-2618 in the United States and are continuing to invest in our clinical-stage pipeline, with the recent initiation of our Phase 1b/2 clinical study of our investigational agent rebastinib in combination with paclitaxel."

Clinical Programs

DCC-2618
At the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress in October 2018, Deciphera presented updated preliminary Phase 1 clinical study results of DCC-2618 in patients with gastrointestinal stromal tumors (GIST). Highlights from the presentation included:
Preliminary median progression free survival (mPFS) in second- and third-line GIST patients of 42 weeks and 40 weeks, respectively, that the Company believes demonstrates the potential for improved and durable clinical outcomes in patients with less advanced disease.
Updated objective response rates (ORR) and disease control rates (DCR) in second- and third-line GIST patients continue to exceed previously published results of registrational trials for currently approved therapies.
In fourth-line and fourth-line-plus GIST patients, for whom there are currently no approved therapies, the Company believes that the observed mPFS of 24 weeks demonstrates the potential for durable clinical outcomes in patients with advanced disease. Published studies have reported a mPFS of 4 to 6 weeks for similarly heavily pre-treated patients who did not receive an active therapy.
Deciphera expanded the ongoing Phase 1 study to include additional cohorts for patients with: various solid tumors, including melanoma; non-small cell lung cancer; germ cell cancer; penile cancer; soft tissue sarcoma; GIST or other solid tumor patients with renal impairment.
Deciphera will present preclinical data on the effects of the combination of DCC-2618 and MAPK pathway inhibitors on cell death and apoptosis in cellular assays of GIST and mastocytosis in a poster session at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Molecular Targets and Cancer Therapeutics Symposium on November 13, 2018.
Data in GIST patients from the ongoing Phase 1 study that were presented at the ESMO (Free ESMO Whitepaper) 2018 Congress in October 2018 will also be presented at the Annual Meeting of the Connective Tissue Oncology Society (CTOS) on November 15, 2018.
Deciphera previously announced that, following discussions with regulatory authorities in the United States and Europe, it is planning to initiate later this year a randomized, multicenter, open-label, pivotal Phase 3 INTRIGUE study evaluating DCC-2618 compared to sunitinib in second-line GIST patients.
Rebastinib
Deciphera initiated a Phase 1b/2 clinical study of rebastinib, the Company’s small molecule kinase switch control inhibitor of TIE2. In this two-part clinical study, rebastinib will be evaluated for the treatment of patients with advanced or metastatic solid tumors in combination with paclitaxel.
DCC-3014
Deciphera continues to enroll patients in the Phase 1 dose escalation study of DCC-3014, a selective small molecule kinase switch control inhibitor of CSF1R and expects to provide an update on this study later this year.
Corporate Updates

In September 2018, Deciphera appointed Daniel C. Martin as Chief Commercial Officer. Mr. Martin has more than 20 years of commercial experience within the biopharmaceutical industry with extensive background in oncology, including immuno-oncology.
In November 2018, Oliver Rosen, M.D., informed Deciphera that effective November 30, 2018, he would leave his position as the Company’s Chief Medical Officer to pursue another opportunity at an early-stage, private biotechnology company. The Company has commenced a search for his replacement.
"On behalf of the management team and board of directors, I want to thank Oliver for his many contributions to Deciphera over the past four and a half years," said Dr. Taylor. "During his tenure, Oliver was instrumental in developing DCC-2618 from a promising preclinical asset into a robust late-stage clinical program with a pivotal Phase 3 study expected to read out in 2019, a second Phase 3 study that is planned to initiate soon and a series of expansion studies in multiple indications. We wish Oliver all the best in his future endeavors."

"It has been a privilege to be a part of Deciphera’s successful growth since 2014 led by the rapid development of DCC-2618," said Dr. Rosen. "I am proud of the progress the Company has made in translating the promise of its kinase switch control platform into an exciting pipeline of clinical-stage drug candidates designed to provide cancer patients with novel therapies that address unmet medical needs."

Third Quarter 2018 Financial Results

Cash Position: As of September 30, 2018, cash and cash equivalents were $320.9 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 June 2018 underwritten public offering, offset by cash used in operating activities. We expect our current cash and cash equivalents will enable us to fund our operating and capital expenditures and debt service payments into the second half of 2020.
R&D Expenses: Research and development expenses for the third quarter of 2018 were $20.6 million, compared to $9.8 million for the same period in 2017. The increase was primarily due to an increase in spending on the DCC-2618 program of $6.1 million as a result of clinical trial costs related to the pivotal Phase 3 INVICTUS study that began enrollment in January 2018. Clinical trial costs also increased due to start-up activities related to a second Phase 3 INTRIGUE study in second-line GIST, which is expected to be initiated by the end of 2018. In addition, chemistry, manufacturing and controls development and manufacturing costs for the DCC-2618 program increased as a result of process development activities to support anticipated drug requirements for commercialization and the manufacture of registration lots to support the submission of a new drug application. Expenses related to the rebastinib program increased $1.5 million, primarily due to start-up activities related to the Phase 1b/2 study of rebastinib in combination with paclitaxel, which initiated in October 2018. Personnel-related costs increased $2.7 million due to increased headcount in our research and development functions. Personnel-related costs for the third quarters of 2018 and 2017 included non-cash share-based compensation expense of $1.1 million and $0.5 million, respectively.
G&A Expenses: General and administrative expenses for the third quarter of 2018 were $5.3 million, compared to $2.4 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. Facility-related and other costs increased due to director and officer insurance costs and higher rent expense related to our new lease. Non-cash share-based compensation was $1.5 million and $0.6 million for the third quarters of 2018 and 2017, respectively.
Net Loss: For the third quarter of 2018, Deciphera reported a net loss of $24.4 million, or $0.65 per share, compared with a net loss of $12.0 million, or $1.04 per share, for the same period in 2017.

ArQule to Present Clinical and Preclinical Data for ARQ 751 at the 30th EORTC/AACR/NCI Symposium

On November 8, 2018 ArQule, Inc. (Nasdaq: ARQL) reported that it will be presenting clinical and preclinical data on the company’s next generation AKT inhibitor, ARQ 751, in three poster presentations at the 30th EORTC/AACR/NCI Symposium to be held from November 13 to 16, 2018 in Dublin, Ireland (Press release, ArQule, NOV 8, 2018, View Source [SID1234530928]).

Presentation Details

Title: A Phase 1 Dose Escalation Study of ARQ 751 in Adult Patients with Advanced Solid Tumors with AKT1, 2, 3 Genetic Alterations, Activating PI3K Mutations, PTEN-null, or Other Known Actionable PTEN Mutations
Abstract #:

395
Session:

Molecular Targeted Agents – PART II
Date:

Friday, November 16, 2018
Time:

10:00-14:00 PM CET
Location:

Exhibition Hall

Title:

Combination of the AKT inhibitor ARQ 751 with Immune Checkpoint Inhibitor and Other Therapeutic Agents
Abstract #:

371
Session:

Molecular Targeted Agents – PART II
Date:

Friday, November 16, 2018
Time:

10:00-14:00 PM CET
Location:

Exhibition Hall

Title:

Miransertib and ARQ 751 exhibit superior cell-death-inducing properties compared to other AKT inhibitors and can overcome resistance to other allosteric AKT inhibitors
Abstract #:

442
Session:

Molecular Targeted Agents – PART II
Date:

Friday, November 16, 2018
Time:

10:00-14:00 PM CET
Location:

Exhibition Hall

About ARQ 751
ARQ 751 is an orally bioavailable, selective small molecule inhibitor of the AKT serine/threonine kinase. The AKT pathway when abnormally activated is implicated in multiple oncogenic processes such as cell proliferation and apoptosis. This pathway has emerged as a target of potential therapeutic relevance for compounds that inhibit its activity, which has been linked to a variety of cancers as well as to select non-oncology indications. ARQ 751 is currently in a Phase 1 study in adult patients with refractory and/or metastatic tumors that harbor genetic alterations along the AKT pathway.

About Miransertib
Miransertib (ARQ 092) is an orally available, selective, pan-AKT (protein kinase B) inhibitor that potently inhibits AKT1, 2 and 3 isoforms. Dysregulation of AKT has been implicated in a variety of rare overgrowth diseases and cancers; however, there are currently no approved inhibitors of AKT. AKT inhibitors, either as single agent or combination therapy, show significant promise in molecularly defined patient populations. Miransertib is currently in a Phase 1/2 company-sponsored study for PIK3CA-Related Overgrowth Spectrum (PROS), a Phase 1 study for ultra-rare Proteus syndrome conducted by the National Institutes of Health (NIH/NHGRI), and a Phase 1b study in combination with the hormonal therapy, anastrozole, in patients with advanced endometrial cancer with AKT and PI3K mutations. Miransertib has been granted Rare Pediatric Disease Designation and Fast Track Designation by the U.S. Food and Drug Administration (FDA), as well as Orphan Designation by the FDA and European Medicines Agency in the rare overgrowth disease, Proteus syndrome.

BioLineRx Reports Significant Progress Across Oncology Programs and Provides Third Quarter Financial Update

On November 8, 2018 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported its financial results for the third quarter ended September 30, 2018 and provided a corporate update (Press release, BioLineRx, NOV 8, 2018, View Source;p=irol-newsArticle&ID=2376172 [SID1234530927]).

Highlights and achievements during the third quarter 2018 and subsequent period:

Reported positive results from lead-in period of Phase 3 GENESIS trial in stem-cell mobilization; data from first lead-in patient cohort prompted Data Monitoring Committee to recommend early continuation to randomized placebo-controlled part 2 of trial;
Presented data from ongoing Phase 2a COMBAT/KEYNOTE-202 pancreatic cancer study in collaboration with Merck at the ESMO (Free ESMO Whitepaper) 2018 Congress demonstrating that BL-8040 in combination with KEYTRUDA (pembrolizumab) showed encouraging disease control and overall survival in patients with metastatic pancreatic cancer; compelling pharmacodynamic data also demonstrated T-cell infiltration into tumors and a reduction of the tumor immuno-suppressive microenvironment;
Based on COMBAT/KEYNOTE-202 results, announced expansion of immuno-oncology collaboration with Merck to include a triple combination arm investigating the safety, tolerability and efficacy of BL-8040, KEYTRUDA and chemotherapy;
Entered into agreement with Biokine Therapeutics to increase the Company’s economic stake in BL-8040 to 80% from the previous level of 60%;
Initiated Phase 1/2a multicenter, open-label clinical study in the UK and Israel for AGI-134, a novel immunotherapy evoking a direct anti-tumor response and vaccine effect for the treatment of solid tumors;
"We made significant progress during the third quarter and subsequent period advancing clinical development of both of our oncology programs – BL-8040 and AGI-134," said Philip Serlin, Chief Executive Officer of BioLineRx. "The Phase 3 GENESIS study in stem-cell mobilization is now advancing in the randomized placebo-controlled part 2 of the trial. We are also rapidly moving forward in our expanded collaboration with Merck in pancreatic cancer, on the basis of the encouraging results recently presented at ESMO (Free ESMO Whitepaper), with an additional cohort adding chemotherapy to the BL-8040/KEYTRUDA combination. Further, we are very pleased to have initiated the first-in-human clinical study for AGI-134, our unique immunotherapy cancer vaccine. These achievements follow BL-8040’s very promising results in relapsed/refractory AML that were presented at the recent EHA (Free EHA Whitepaper) Congress during the second quarter, showing significantly improved overall survival compared to historical data."

"BL-8040 is currently being evaluated in eight Phase 2 or Phase 3 clinical trials in multiple oncology indications, four of which are being run under collaborations with global pharma companies. In addition, based on the very promising data seen in relapsed/refractory AML, we intend to further pursue this indication and we continue to evaluate the optimal clinical development pathway going forward. The broad dataset from BL-8040’s robust clinical development program was the motivation behind our recent decision to significantly increase our economics in BL-8040, and sets the stage for a catalyst-rich 2019," Mr. Serlin concluded.

Expected significant milestones through end of 2019:

Initiation of Phase 2 triple combo pancreatic cancer trial of BL-8040, KEYTRUDA and chemotherapy under collaboration with Merck by the end of 2018;
Potential interim results from Phase 2 AML consolidation study in mid-2019;
Initial safety results from part 1 of Phase 1/2a trial for AGI-134 in second half of 2019;
Top-line results from the Phase 2 triple combo pancreatic cancer trial of BL-8040, KEYTRUDA and chemotherapy under collaboration with Merck toward the end of 2019;
Top-line results from one or more of the solid tumor trials under collaboration with Genentech during 2019.
Financial Results for the Third Quarter Ended September 30, 2018

Research and development expenses for the three months ended September 30, 2018 were $5.0 million, a decrease of $0.6 million, or 11.1 %, compared to $5.6 million for the three months ended September 30, 2017. The decrease resulted primarily from higher expenses associated with drug product development and manufacturing for AGI-134 in the 2017 period. Research and development expenses for the nine months ended September 30, 2018 were $14.6 million, an increase of $1.3 million, or 9.6 %, compared to $13.3 million for the nine months ended September 30, 2017. The increase in the 2018 period resulted primarily from higher expenses associated with BL-8040, including the GENESIS and COMBAT trials; preparations for initiation of the AGI-134 clinical trial; and BL-1230.

Sales and marketing expenses for the three months ended September 30, 2018 were $0.3 million, similar to the comparable period in 2017. Sales and marketing expenses for the nine months ended September 30, 2018 were $1.1 million, a decrease of $0.1 million, or 6.7%, compared to $1.2 million for the nine months ended September 30, 2017. The decrease resulted primarily from one-time legal fees related to AGI-134 paid in the 2017 period.

General and administrative expenses for the three months ended September 30, 2018 were $0.9 million, a decrease of $0.3 million, or 22.7%, compared to $1.2 million for the three months ended September 30, 2017. The decrease resulted from a decrease in fees paid for consulting services. General and administrative expenses for the nine months ended September 30, 2018 were $2.9 million, a decrease of $0.2 million, or 5.9%, compared to $3.0 million for the nine months ended September 30, 2017. The decrease also resulted from a decrease in fees paid for consulting services.

The Company’s operating loss for the three months ended September 30, 2018 amounted to $6.2 million, compared with an operating loss of $7.1 million for the corresponding 2017 period. The Company’s operating loss for the nine months ended September 30, 2018 amounted to $18.6 million, compared with an operating loss of $17.6 million for the corresponding 2017 period.

Non-operating income (expenses) for the three and nine months ended September 30, 2018 primarily relate to fair-value adjustments of warrant liabilities on the Company’s balance sheet and the capital gain from realization of the investment in iPharma. Non-operating income (expenses) for the three and nine months ended September 30, 2017 primarily relate to fair-value adjustments of warrant liabilities on the Company’s balance sheet.

Net financial income amounted to $0.1 million for the three months ended September 30, 2018, similar to the comparable period in 2017. Net financial income for both periods relates primarily to gains recorded on foreign currency hedging transactions and investment income earned on bank deposits. Net financial income amounted to $0.4 million for the nine months ended September 30, 2018, compared to net financial income of $0.9 million for the nine months ended September 30, 2017. Net financial income for the 2018 period primarily relates to investment income earned on bank deposits, offset by losses recorded on foreign currency hedging transactions. Net financial income for the 2017 period relates primarily to gains recorded on foreign currency hedging transactions and investment income earned on bank deposits.

The Company’s net loss for the three months ended September 30, 2018 amounted to $6.3 million, compared with a net loss of $7.2 million for the corresponding period. The Company’s net loss for the nine months ended September 30, 2018 amounted to $17.3 million, compared with a net loss of $17.0 million for the corresponding 2017 period.

The Company held $35.0 million in cash, cash equivalents and short-term bank deposits as of September 30, 2018.

Net cash used in operating activities was $19.1 million for the nine months ended September 30, 2018, compared with net cash used in operating activities of $14.2 million for the nine months ended September 30, 2017. The $4.9 million increase in net cash used in operating activities during the nine-month period in 2018, compared to the nine-month period in 2017, was the result of increased research and development expenses in the 2018 period, as well as a decrease in accounts payable and increase in prepaid expenses and other receivables.

Net cash provided by investing activities was $16.0 million for the nine months ended September 30, 2018, compared to net cash used in investing activities of $19.5 million for the nine months ended September 30, 2017. The changes in cash flows from investing activities relate primarily relate primarily to investments in, and maturities of, short-term bank deposits, as well as the investment in Agalimmune in 2017 and the realization of the investment in iPharma in 2018.

Net cash provided by financing activities was $2.8 million for the nine months ended September 30, 2018, compared to net cash provided by financing activities of $37.7 million for the nine months ended September 30, 2017. The decrease in cash flows from financing activities reflects the public offering completed in April 2017.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, November 8, 2018 at 10:00 a.m. EDT. To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0685 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until November 10, 2018; please dial +1-888-326-9310 from the U.S. or +972-3-925-5925 internationally.

BioCryst to Present at Upcoming Investor Conferences

On November 8, 2018 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported that the company will present at the Jefferies London Healthcare Conference on Thursday, November 15, 2018 at 3:00 a.m. ET and the Piper Jaffray 30th Annual Healthcare Conference in New York on Tuesday, November 27 at 3:30 p.m. ET (Press release, BioCryst Pharmaceuticals, NOV 8, 2018, http://ir.biocryst.com/news-releases/news-release-details/biocryst-present-upcoming-investor-conferences [SID1234530926]).

Links to a live audio webcast and replay of these presentations may be accessed in the Investors section of BioCryst’s website at http://www.biocryst.com.

Loxo Oncology Reports Third Quarter 2018 Financial Results

On November 8, 2018 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company developing highly selective medicines for patients with genomically defined cancers, reported third quarter 2018 financial results (Press release, Loxo Oncology, NOV 8, 2018, View Source [SID1234530925]).

"In the third quarter we made significant progress across our pipeline," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "At ESMO (Free ESMO Whitepaper), larotrectinib investigators provided a comprehensive program update that included durability and additional response data in patients with TRK fusion cancers. Medical meeting updates for LOXO-292 in September and October showed encouraging ongoing durability of response for patients with RET-driven cancers. Taken together, these data have increased our conviction around a foundational company thesis—that selective, purpose-built medicines offer the best opportunity for durable efficacy and a manageable safety profile. In the fourth quarter, we look forward to LOXO-305, our fourth program, entering the clinic."

Recent Highlights

Larotrectinib

Conference presentations
European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress: On October 21, 2018, updated clinical data for larotrectinib were presented at ESMO (Free ESMO Whitepaper). The oral presentation provided approximately one year of additional follow-up for the primary dataset, the 55 patients with TRK fusion cancer described in the larotrectinib New England Journal of Medicine publication from February 2018. In addition, the update included data for a supplementary dataset, an additional 67 patients with TRK fusion cancer who were subsequently enrolled across the larotrectinib development program. Response evaluations were based on investigator assessment. As of a data cut-off date of July 30, 2018, in the primary dataset (n=55), the overall response rate (ORR) was 80% (44/55) (95% CI: 67-90%) and in the supplementary dataset (n=67), the ORR was 81% (44/54) (95% CI: 69-91%). Across both datasets, the ORR was 81% (88/109) (95% CI: 72-88%). The ORR analyses for the supplementary and integrated datasets included nine patients with unconfirmed partial responses awaiting confirmatory response assessments, but did not include 13 patients who were awaiting an initial response assessment and continuing on study. Median duration of response (DOR) had not been reached in either the primary dataset or supplementary dataset, with median follow-up of 17.6 months and 7.4 months, respectively. Larotrectinib was well tolerated, with the majority of adverse events recorded as grade 1 or 2. The most common treatment-emergent adverse events occurring in 15% or more of patients in the trial were fatigue, dizziness, nausea, constipation, anemia, increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), cough, diarrhea, vomiting, pyrexia, dyspnea, headache, myalgia and peripheral oedema. See the presented data here.
Annual Meeting of the American Thyroid Association (ATA): On October 4, 2018, clinical data for patients with TRK fusion thyroid cancer enrolled in the larotrectinib development program were presented in an oral presentation at ATA. See the presented data here.
International Association for the Study of Lung Cancer (IASLC) 19th World Conference on Lung Cancer: On September 24, 2018, clinical data for patients with TRK fusion non-small cell lung cancer (NSCLC) enrolled in the larotrectinib development program were presented in a poster presentation at the IASLC World Conference on Lung Cancer. The poster can be found here.
Molecular Analysis for Personalised Therapy 2018 Congress: On September 15, 2018, Ventana Medical Systems, Inc., a member of the Roche Group, and Loxo Oncology, presented a co-authored poster on the analytical validation of Ventana’s pan-TRK IHC assay at the Molecular Analysis for Personalised Therapy 2018 Congress. These data, in addition to other recently published evidence, suggest an annual incidence of approximately 2,500 to 3,000 cases of TRK fusion cancer in the United States. The poster can be found here.
Integrative Therapies Program for Children with Cancer (ITPCC): On September 13, 2018, clinical data for children and adolescents with TRK fusion metastatic thyroid carcinoma enrolled in the larotrectinib development program were presented at the ITPCC conference. The poster can be found here.
Publications
Targeted Oncology Publication: On October 2, 2018, a manuscript was published online in Targeted Oncology detailing the potential effectiveness of TRK inhibition, including larotrectinib treatment, in patients with tumors harboring NTRK gene fusions, and the need for effective testing strategies. The publication can be found here.
British Journal of Cancer Publication: On September 17, 2018, a case report was published in the British Journal of Cancer detailing a patient with TRK fusion high-grade glioma treated with larotrectinib. The publication can be found here.
Cancer Publication: On September 11, 2018, a manuscript was published online in Cancer detailing the treatment of children with locally advanced TRK fusion sarcoma who were treated preoperatively with larotrectinib and underwent subsequent surgical resection. The publication can be found here.
JCO Precision Oncology Publication: On August 2, 2018, a case report was published in JCO Precision Oncology detailing an adolescent patient with a TRK fusion undifferentiated sarcoma treated with larotrectinib. The publication can be found here.
European Marketing Authorization Application (MAA): On August 27, 2018, Loxo Oncology and Bayer announced that Bayer had submitted an MAA for larotrectinib to the European Medicines Agency (EMA). More information can be found here.
LOXO-195

LOXO-195 Orphan Drug Designation (ODD): In October, the U.S. Food and Drug Administration (FDA) granted ODD to LOXO-195 for the treatment of solid tumors with neurotrophic tyrosine receptor kinase (NTRK)-fusion proteins that have developed acquired resistance to prior TRK inhibitor therapy. The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides to Loxo Oncology certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.
LOXO-292

Annual Meeting of the ATA: On October 6, 2018, updated interim clinical data for LOXO-292 from the global Phase 1/2 LIBRETTO-001 trial in patients with RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer were presented at the Annual Meeting of the ATA. The data presented were based on a July 19, 2018 data cut-off date and included the 29 patients with RET-mutant MTC and the nine patients with RET fusion-positive thyroid cancer who were included in the LOXO-292 presentation at the 2018 ASCO (Free ASCO Whitepaper) Annual Meeting. With 3.5 months of additional follow-up since the ASCO (Free ASCO Whitepaper) presentation, LOXO-292 demonstrated encouraging, early evidence of durable activity. Sixteen of 17 (94%) responding RET-mutant MTC patients remained on therapy and in response (median follow-up of 7.6 months for all 29 patients; median follow-up of 8.4 months for responding patients). Seven of seven (100%) responding RET fusion-positive thyroid remained on therapy and in response (median follow-up of 7.6 months for all nine patients; median follow-up of 8.5 months for responding patients). In RET-mutant MTC, the overall response rate was 59% (17/29) (95% CI: 39-77%) and the confirmed overall response rate was 56% (15/27) (95% CI: 35-75%). Of nine patients with RET fusion-positive thyroid cancer, the confirmed overall response rate was 78% (7/9) (95% CI: 40-97%). Of the 82 patients in the safety analysis, most treatment-emergent adverse events were Grade 1 in severity and judged by the investigator as not related to LOXO-292. See the presented data here.
IASLC 19th World Conference on Lung Cancer: On September 25, 2018, updated interim clinical data for LOXO-292 from the global Phase 1/2 LIBRETTO-001 trial in patients with RET fusion-positive NSCLC were presented at the IASLC World Conference on Lung Cancer. The data presented were based on a July 19, 2018 data cut-off date and included the 38 patients with RET fusion-positive NSCLC who were initially included in the LOXO-292 presentation at the 2018 ASCO (Free ASCO Whitepaper) Annual Meeting. With 3.5 months of additional follow-up since the ASCO (Free ASCO Whitepaper) presentation, LOXO-292 demonstrated encouraging, early evidence of durable activity, with 25 of 26 (96%) responding RET fusion-positive NSCLC patients remaining on therapy and 24 of 26 (92%) remaining in response (median follow-up of 8.5 months for all 38 patients; median follow-up of 9.5 months for responding patients). The overall response rate was 68% (26/38) (95% CI: 51-83%) and the confirmed overall response rate was 68% (25/37) (95% CI: 50-82%). Of the 82 patients in the safety analysis, most treatment-emergent adverse events were Grade 1 in severity and judged by the investigator as not related to LOXO-292. See the presented data here.
LOXO-292 Breakthrough Therapy Designations: The FDA granted three Breakthrough Therapy Designations to LOXO-292:
for the treatment of patients with metastatic RET fusion-positive non-small cell lung cancer who require systemic therapy and have progressed following platinum-based chemotherapy and an anti-PD-1 or anti-PD-L1 therapy;
for the treatment of patients with RET-mutant medullary thyroid cancer who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment options; and for
for the treatment of patients with advanced RET fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment options.
More information can be found here and here.
LOXO-292 Orphan Drug Designation: In October, the FDA granted ODD to LOXO-292 for the treatment of pancreatic cancer. The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides to Loxo Oncology certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.
LOXO-305

Society of Hematologic Oncology (SOHO) Annual Meeting: On September 12, 2018, preclinical characterization data for LOXO-305 were presented at the SOHO Annual Meeting. The poster can be found here.
Third Quarter 2018 Financial Results

As of September 30, 2018, Loxo Oncology had aggregate cash, cash equivalents and investments of $647.6 million, compared to $626.2 million as of December 31, 2017.

Revenue from the collaboration agreement was $42.5 million for the third quarter of 2018, compared to none for the third quarter of 2017. This represents $52.9 million in revenue recognized from the $400.0 million upfront payment from the Bayer collaboration offset by $10.5 million, Loxo Oncology’s share of the joint larotrectinib co-promotion costs in the same period.

Revenue from the collaboration agreement was $123.5 million for the nine months ended September 30, 2018, compared to none for the nine months ended September 30, 2017. This represents $147.0 million in revenue recognized from the $400.0 million upfront payment from the Bayer collaboration offset by $23.5 million, Loxo Oncology’s share of the joint larotrectinib co-promotion costs in the same period. 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 $56.9 million for the third quarter of 2018 compared to $64.8 million for the third quarter of 2017. This decrease was primarily due to a non-recurring charge related to the $40.0 million asset acquisition of the BTK inhibitor program from Redx in the third quarter of 2017, offset by expanded development expenses across the LOXO-292 and LOXO-305 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 $4.2 million during the third quarter of 2018 as compared to $2.1 million for the third quarter of 2017.

Research and development expenses were $130.5 million for the nine months ended September 30, 2018 compared to $109.3 million for the nine months ended September 30, 2017. This increase was primarily due to expanded development expenses across the LOXO-292 and LOXO-305 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 $14.3 million during the nine months ended September 30, 2018 as compared to $8.0 million for the nine months ended September 30, 2017.

General and administrative expenses were $15.9 million for the third quarter of 2018 compared to $9.7 million for the third quarter of 2017. The increase was primarily due to additional headcount, associated employment costs, and general and administrative professional fees. Loxo Oncology recognized general and administrative-related stock-based compensation expense of $6.9 million during the third quarter of 2018 compared to $3.1 million for the third quarter of 2017.

General and administrative expenses were $43.8 million for the nine months ended September 30, 2018 compared to $21.0 million for the nine months ended September 30, 2017. The increase was primarily due to additional headcount, associated employment costs, and general and administrative professional fees. Loxo Oncology recognized general and administrative-related stock-based compensation expense of $18.8 million during the nine months ended September 30, 2018 compared to $6.7 million for the nine months ended September 30, 2017.

Net loss was $27.1 million and $42.3 million for the three and nine months ended September 30, 2018, respectively, compared to $73.3 million and $128.2 million for the three and nine months ended September 30, 2017, respectively. This decrease in net loss was 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 $68.8 million and $156.2 million for the three and nine months ended September 30, 2018, respectively, compared to $28.1 million and $73.6 million for the three and nine months ended September 30, 2017, respectively. 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 and Webcast Information
Loxo Oncology will host a conference call today at 8:00 a.m. ET to discuss the third quarter 2018 financial results and company updates. A live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the company’s website at www.loxooncology.com. The conference call can be accessed by dialing (877) 930-8065 (domestic) or (253) 336-8041 (international) and referring to conference ID 8379404. The webcast will be archived and made available for replay on the company’s website beginning approximately two hours after the event.