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TG Therapeutics, Inc. Provides Business Update and Reports Third Quarter 2016 Financial Results

On November 7, 2016 TG Therapeutics, Inc. (NASDAQ:TGTX) reported its financial results for the third quarter ended September 30, 2016 and recent company developments (Press release, TG Therapeutics, NOV 7, 2016, View Source [SID1234516778]).

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Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer, stated, "The third quarter was a pivotal one for the company, particularly with the amendment of the GENUINE Phase 3 trial that will enable the rapid conclusion of the study while also maintaining the possibility for accelerated approval for TG-1101 in combination with ibrutinib. Importantly, with GENUINE enrollment wrapping up, we can dedicate our resources and focus to our proprietary UNITY program, which we believe offers the potential for highly active, well-tolerated therapy with certain pricing advantages over competitors. As a result, we see our value proposition rising as the concern around pharmaceutical pricing continues to grow." Mr. Weiss continued, "We see important value creating milestones over the next 12 months, including completion of enrollment of GENUINE expected by year end 2016 to be followed by top line data in the first half of 2017. During the course of 2017, we should also report early data from our UNITY-DLBCL study and our MS pivotal program should take full form supported by B-cell depletion data from our ongoing Phase 2 study in RRMS. With all these events lining up, we believe 2017 will be our most impactful year to date."

Third Quarter and Recent Highlights

ASH 2016: The Company looks forward to the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting where data presentations will include three oral presentations and three poster presentations. All clinical presentations will highlight the safety and efficacy for combinations of TGR-1202 with novel targeted agents including oral presentations with TGR-1202 in combination with ibrutinib and TGR-1202 in combination with ruxolitinib.

TGR-1202 Preclinical Differentiation: An October publication in Blood presented preclinical data describing the synergy of TGR-1202 with carfilzomib and the unique effects of the combination to silence c-Myc in various preclinical lymphoma and myeloma models. In addition, the publication described TGR-1202’s unique complimentary mechanism of inhibiting the protein kinase casein kinase-1 (CK1) epsilon, which may contribute to the silencing of c-Myc and explain TGR-1202’s clinical activity in aggressive lymphoma.

TGR-1202 + Carfilzomib: Based on the preclinical work on TGR-1202 published in Blood , a Phase 1/2 study of TGR-1202 and Carfilzomib in patients with relapsed or refractory lymphoma was launched.

GENUINE Study Amendment: The Company amended the ongoing GENUINE Phase 3 Clinical Trial by revising the primary endpoint to Overall Response Rate (ORR), with enrollment expected to be completed before year end 2016 and top-line data available in first half of 2017. The study is well powered to detect a difference in ORR, which, if successful, would potentially support a filing for accelerated approval.

Clinical Data Presentation of TG-1101 in NMO: During the 32nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis, the Company presented clinical data from the Phase Ib study of TG-1101 in patients with NMO with TG-1101 demonstrating rapid and effective depletion of B-cells during acute NMO relapse. The Company has an on-going Phase 2 study of TG-1101 in multiple sclerosis.

Orphan Drug Designations: The Company received Orphan Drug Designation for TGR-1202 for treatment of Chronic Lymphocytic Leukemia and for TG-1101 for treatment of Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD).

UNITY-DLBCL: Patient enrollment began for the registration-directed UNITY-DLBCL Phase 2b clinical study evaluating TG-1101 and TGR-1202 in combination compared to TGR-1202 monotherapy in patients with advanced relapsed/refractory DLBCL.
Key Remaining 2016 Milestones

Complete enrollment into the GENUINE Phase 3 clinical trial
Aggressively enroll into the Company’s registration directed trials, including the UNITY-CLL Phase 3, and the UNITY-DLBCL Phase 2b
Continue enrollment into the Phase 2 clinical trial in Multiple Sclerosis
Present clinical data from a variety of Phase 1 and 2 clinical trials at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2016, held in San Diego, CA
Financial Results for the Third Quarter 2016

Cash Position: Cash, cash equivalents, investment securities, and interest receivable were $60.7 million as of September 30, 2016.

R&D Expenses: Research and development (R&D) expenses were $21.8 million and $46.9 million for the three and nine months ended September 30, 2016, respectively, compared to $11.6 million and $32.5 million for the three and nine months ended September 30, 2015, respectively. Included in research and development expenses for the three and nine months ended September 30, 2016, are $10.2 million and $17.9 million, respectively, of manufacturing and CMC expenses for Phase 3 clinical trials and potential commercialization. The increase in R&D expenses for both the three and nine months ended September 30, 2016, is primarily due to the ongoing clinical development programs and related manufacturing costs for TG-1101 and TGR-1202.

G&A Expenses: General and administrative (G&A) expenses were $3.2 million and $8.1 million for the three and nine months ended September 30, 2016, respectively, as compared to $2.3 million and $13.2 million for the three and nine months ended September 30, 2015, respectively. The period-over-period decrease in G&A expenses from the nine months ended September 30, 2015 relates primarily to non-cash compensation expenses related to equity incentive grants recognized during 2015. G&A expenses for the three months ended September 30, 2016 remained relatively flat compared to the second quarter of 2015, and we expect G&A expenses to remain relatively constant through the fourth quarter of 2016.

Net Loss: Net loss was $24.8 million and $54.6 million for the three and nine months ended September 30, 2016, respectively, compared to a net loss of $13.7 million and $45.3 million for the three and nine months ended September 30, 2015, respectively.

Financial Guidance: The Company believes its cash, cash equivalents, investment securities, and interest receivable of $60.7 million as of September 30, 2016 will be sufficient to fund the Company’s planned operations into the first half of 2018.

Portola Pharmaceuticals Reports Third Quarter 2016 Financial Results and Provides Corporate Update

On November 7, 2016 Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) reported a corporate update and reported its financial results for the third quarter ended September 30, 2016 (Press release, Portola Pharmaceuticals, NOV 7, 2016, View Source;p=RssLanding&cat=news&id=2220288 [SID1234516691]).

"We continue to focus on obtaining regulatory approval for our two lead programs in the United States and EU. Both programs have the potential to become the standard of care in two growth areas of thrombosis that have a high unmet medical need and limited treatment options. If approved, betrixaban, our oral Factor Xa inhibitor, would be the first anticoagulant indicated for extended VTE prophylaxis in the over 24 million medically ill patients admitted to the hospital annually in the G7. AndexXa, our much anticipated Factor Xa inhibitor antidote, would be the first agent approved to treat the growing number of patients admitted to the hospital with life-threatening anticoagulant-related bleeding. We have made important progress toward bringing both betrixaban and AndexXa to market. We submitted an NDA for betrixaban in the United States and plan to submit an MAA in the EU," said Bill Lis, chief executive officer of Portola. "Also, we continue to engage in positive, productive dialogue with the FDA to resolve outstanding questions regarding the Complete Response Letter for AndexXa, most of which are focused on manufacturing."

Recent Achievements, Upcoming Events and Milestones

Betrixaban – an oral Factor Xa inhibitor anticoagulant in development for the prevention of venous thromboembolism (VTE) in acute medically ill patients; designated Fast Track status by the U.S. Food and Drug Administration (FDA)

Submitted a New Drug Application (NDA) to the FDA seeking approval to market betrixaban for extended-duration prophylaxis of VTE in acutely ill medical patients with risk factors for VTE
Plan to submit a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) by year-end
Three abstracts on results of sub-analyses of the Phase 3 APEX Study conducted by the PERFUSE Study Group were accepted for oral and poster presentations at the upcoming American Heart Association Scientific Sessions 2016
AndexXa (andexanet alfa) – a Factor Xa inhibitor antidote in development for patients treated with a Factor Xa inhibitor when reversal of anticoagulation is needed due to life-threatening bleeding or when urgent surgery is required; designated a Breakthrough Therapy and an Orphan Drug by the FDA

Submitted an MAA, which completed the validation period and was accepted for review by the EMA
Continued productive dialogue with the FDA regarding the Complete Response Letter; Portola currently plans to resubmit the BLA in the second quarter of 2017
Presented a preliminary analysis of interim data from the ongoing Phase 3b/4 ANNEXA-4 Study in patients with acute major bleeding in a Late-Breaking Science Hot Line session at the European Society of Cardiology 2016 Congress with simultaneous publication online by The New England Journal of Medicine
Plan to present data from the ongoing Phase 2 study of andexanet alfa reversal of betrixaban in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2016
Cerdulatinib – an oral, dual Syk/JAK inhibitor in development to treat resistant or relapsed hematologic cancer patients

Continued to enroll patients in a Phase 2a study evaluating the safety and efficacy of cerdulatinib in patients with relapsed/refractory B-cell malignancies who have failed multiple therapies
Two abstracts were accepted for presentation at the upcoming ASH (Free ASH Whitepaper) Annual Meeting 2016
Third Quarter 2016 Financial Results
Collaboration revenue earned under Portola’s collaborations with Bristol-Myers Squibb Company and Pfizer, Bayer Pharma and Janssen Pharmaceuticals, and Daiichi Sankyo was $9.3 million for the third quarter of 2016 compared with $2.9 million for the third quarter of 2015. The increase in revenue was primarily due to $2.5 million received upon achievement of a milestone from the Bayer and Janssen Phase 3 agreement, $2.5 million received upon achievement of a milestone from the Daiichi Sankyo Phase 3 agreement, and incremental revenue of $2.0 million from three collaboration and license agreements executed in the first quarter of 2016 to develop and commercialize andexanet alfa in Japan.

Total operating expenses for the third quarter of 2016 were $100.8 million compared with $58.5 million for the same period in 2015. Total operating expenses for the third quarter of 2016 included $7.8 million in stock-based compensation expense compared with $6.1 million for the same period in 2015.

Research and development expenses were $87.2 million for the third quarter of 2016 compared with $48.4 million for the third quarter of 2015. The increase in R&D expenses was primarily due to a $27.3 million impairment charge for AndexXa manufacturing expenses that Portola prepaid to CMC Biologics. These expenses were intended to be credited against future batch manufacturing costs for the Line C, or 6×2,000 liter, manufacturing process. This impairment charge was triggered by the Company’s decision to focus on Line A/B manufacturing at CMC Biologics and Gen 2 manufacturing at Lonza, and suspend Line C manufacturing at CMC Biologics.

Selling, general and administrative expenses for the third quarter of 2016 were $13.6 million compared with $10.1 million for the same period in 2015 as the Company increased headcount to support its growth and increased commercial launch preparation activities for AndexXa in advance of the PDUFA date in August 2016.

For the third quarter of 2016, we reported a net loss of $92.9 million dollars, or a net loss per share of $1.64, compared with a net loss of $55.2 million dollars, or a net loss per share of $1.05, for the same period in 2015. Net loss for the third quarter of 2016 included the $27.3 million impairment charge. Shares used to compute net loss per share attributable to common stockholders were approximately 56.5 million for the third quarter of 2016 compared with approximately 52.6 million for the same period in 2015.

As of September 30, 2016, cash, cash equivalents and investments totaled $274.6 million compared with cash, cash equivalents and investments of $460.2 million as of December 31, 2015.

Current Report

On November 7, 2016 Horizon Pharma plc (NASDAQ: HZNP), a biopharmaceutical company focused on improving patients’ lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, reported its third-quarter 2016 financial results today and confirmed its full-year 2016 GAAP net sales, non-GAAP adjusted net sales and adjusted EBITDA guidance, as updated on October 25, 2016, following the completion of the acquisition of Raptor Pharmaceutical Corp (Press release, Horizon Pharma, NOV 7, 2016, View Source [SID1234516657]).

"We delivered strong results in the third quarter as we continued to execute on our long-term strategy of building a more-diversified, sustainable biopharmaceutical company anchored by a growing mix of orphan medicines," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "We have made several strategic decisions this year to put Horizon Pharma on a strong path forward, including securing formulary status with two major PBMs for our primary care medicines and completing two significant acquisitions in rare diseases."
Financial Highlights

(in millions except for per
share amounts and
percentages) Q3 16 Q3 15 %
Change YTD 16 YTD 15 %
Change
Net sales (1)
$ 208.7 $ 226.5 (8 ) $ 670.8 $ 512.5 31
Non-GAAP adjusted net sales (1)
273.7 226.5 21 735.8 512.5 44
Net (loss) income
(5.9 ) 3.3 NM (36.3 ) 15.5 NM
Non-GAAP net income
115.5 69.8 65 248.1 151.4 64
Adjusted EBITDA
141.2 131.1 8 334.3 239.7 39
Loss (earnings) per share – diluted
(0.04 ) 0.02 NM (0.23 ) 0.10 NM
Non-GAAP earnings per share – diluted
0.70 0.42 67 1.51 0.98 54

(1) On Sept. 26, 2016, Horizon Pharma agreed to pay Express Scripts $65 million as part of a litigation settlement, which was recorded as a one-time reduction to GAAP net sales for the three and nine months ended Sept. 30, 2016, in accordance with U.S. Generally Accepted Accounting Principles (GAAP). The exclusion of the $65 million settlement from GAAP net sales is the only adjustment reflected in third-quarter and year-to-date non-GAAP adjusted net sales.

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Company Highlights

● Third-quarter 2016 GAAP net sales, including the previously announced $65 million litigation settlement with Express Scripts as a one-time reduction, were $208.7 million, a decrease of 8 percent compared to the third quarter of 2015, primarily attributable to the settlement. Non-GAAP adjusted net sales excluding the $65 million settlement were $273.7 million, an increase of 21 percent compared to the third quarter of 2015, driven by growth across each of the Company’s business units: Orphan, Rheumatology and Primary Care.

● Medicines for rare diseases, which include RAVICTI , ACTIMMUNE , KRYSTEXXA and BUPHENYL , represented 35 percent of total non-GAAP adjusted net sales in the third quarter of 2016, an increase from 29 percent of total net sales in the third quarter of 2015.

● Third-quarter 2016 GAAP net loss was $5.9 million or a diluted loss per share of $0.04; and non-GAAP net income was $115.5 million or non-GAAP diluted earnings per share of $0.70.

● On October 25, 2016, Horizon Pharma completed the acquisition of Raptor Pharmaceutical Corp., which was a significant step in advancing the Company’s strategy to expand its rare disease business with the addition of two orphan medicines, PROCYSBI (cysteamine bitartrate) delayed-release capsules and QUINSAIR (aerosolized form of levofloxacin). More than half of the Company’s medicines now treat patients with rare diseases.

● To provide long-term durability for its primary care medicines, the Company has secured formulary status with two leading Pharmacy Benefit Managers (PBMs) that represent approximately 35 percent of covered lives in the United States. The Company remains in active discussions and negotiations with other PBMs and payers with the goal of further increasing access to its medicines. The Company is investing in the expansion of its managed care organization to support its broader contracting strategy with PBMs and payers.

● The Company will present data on both KRYSTEXXA and RAYOS at the upcoming American College of Rheumatology meeting November 11-16, 2016, in Washington D.C. This is the first medical meeting in three years where KRYSTEXXA will have a significant clinical and commercial presence, which will continue to expand the awareness of KRYSTEXXA as an important treatment option for refractory chronic gout patients.
Horizon Pharma Confirms 2016 Full-Year Guidance

● Confirmed full-year 2016 net sales guidance on a GAAP basis of approximately $980 to $985 million, which includes the previously announced $65 million settlement with Express Scripts as a one-time reduction and includes the acquisition of Raptor Pharmaceutical Corp. Confirmed net sales guidance on a non-GAAP adjusted basis of approximately $1.045 to $1.050 billion, which excludes the $65 million settlement with Express Scripts.

● Confirmed full-year 2016 adjusted EBITDA guidance of $450 to $460 million, which includes the acquisition of Raptor Pharmaceutical Corp.

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Third-Quarter Business Unit Net Sales Results

(in millions except for percentages) Q3 16 Q3 15 %
Change YTD 16 YTD 15 %
Change
Orphan
$ 71.4 $ 66.1 8 $ 211.2 $ 139.6 51
RAVICTI (1)
42.2 33.4 26 118.6 52.4 126
ACTIMMUNE
24.9 28.7 (13 ) 80.5 79.4 1
BUPHENYL (1)
4.3 4.0 10 12.1 7.8 55
Rheumatology
40.5 12.8 217 101.0 31.7 219
KRYSTEXXA (2)
25.6 - NM 61.6 - NM
RAYOS
13.4 11.7 15 36.0 29.2 24
LODOTRA
1.5 1.1 37 3.4 2.5 35
Primary Care
161.8 147.6 10 423.6 341.2 24
PENNSAID 2%
80.2 43.9 83 207.9 91.6 127
DUEXIS
47.6 56.9 (16 ) 122.8 130.0 (6 )
VIMOVO
32.8 46.8 (30 ) 89.7 119.6 (25 )
MIGERGOT (2)
1.2 - NM 3.2 - NM
Litigation settlement (3)
(65.0 ) - NM (65.0 ) - NM

Total GAAP net sales (3)
$ 208.7 $ 226.5 (8 ) $ 670.8 $ 512.5 31

Total non-GAAP adjusted net sales (3)
$ 273.7 $ 226.5 21 $ 735.8 $ 512.5 44

(1) RAVICTI and BUPHENYL were acquired on May 7, 2015.
(2) KRYSTEXXA and MIGERGOT were acquired on January 13, 2016.
(3) On Sept. 26, 2016, Horizon Pharma agreed to pay Express Scripts $65 million as part of a litigation settlement, which was recorded as a one-time reduction to GAAP net sales for the three and nine months ended Sept. 30, 2016, in accordance with U.S. GAAP. The exclusion of the $65 million settlement from GAAP net sales is the only adjustment reflected in third-quarter and year-to-date non-GAAP adjusted net sales.

● Orphan Business Unit: RAVICTI sales in the third quarter of 2016 were $42.2 million, an increase of 26 percent compared to the third quarter of 2015. RAVICTI was launched in Canada in the fourth quarter of 2016. ACTIMMUNE sales in the third quarter of 2016 were $24.9 million. Following the acquisition of Raptor Pharmaceutical Corp. on October 25, 2016, the Company added to its Orphan Business Unit PROCYSBI for the treatment of nephropathic cystinosis, a rare metabolic disorder, and QUINSAIR for the management of chronic pulmonary infections for patients with cystic fibrosis. QUINSAIR is not approved in the United States.
ACTIMMUNE Phase 3 Trial in Friedreich’s ataxia and Phase 1 Trial in Oncology
In its pipeline, the Company continues to expect topline data in late December from the Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in FA (STEADFAST) Phase 3 clinical trial. There are an estimated 3,700 diagnosed patients in the United States with Friedreich’s ataxia (FA) and the Company believes an indication for ACTIMMUNE in FA, if approved, could represent a $500 million to $1 billion peak annual net sales opportunity. In the Phase 1 dosing trial evaluating ACTIMMUNE as a combination therapy for certain cancers, the first six-patient cohort was completed in May, the second six-patient cohort was completed in September, and the third six-patient cohort is now enrolling.

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● Rheumatology Business Unit: KRYSTEXXA sales in the third quarter of 2016 were $25.6 million, an increase of 29 percent sequentially compared to the second quarter of 2016. KRYSTEXXA patient infusions and benefit investigations, which are the leading indicator of new patient starts, continue to increase and the Company is investing in additional commercial support, education and outreach efforts to accelerate growth. RAYOS sales in the third quarter of 2016 were $13.4 million, an increase of 15 percent compared to the third quarter of 2015.

● Primary Care Business Unit: Total sales growth for the primary care business unit increased approximately 10 percent compared to the third quarter of 2015, driven by strong performance of PENNSAID 2%. Sales of PENNSAID 2% in the third quarter of 2016 were $80.2 million, an increase of 83 percent compared to the third quarter of 2015. DUEXIS and VIMOVO sales in the third quarter of 2016 were $47.6 million and $32.8 million, respectively.
Third-Quarter 2016 Financial Results
Note: For additional detail and reconciliation of non-GAAP financial measures to the most directly comparable GAAP financial measures, please refer to the tables at the end of this release.

● Gross Profit: Under U.S. GAAP in the third quarter of 2016, the gross profit ratio was 59.2 percent compared to 73.0 percent in the third quarter of 2015. The non-GAAP gross profit ratio in the third quarter of 2016 was 91.6 percent compared to 92.1 percent in the third quarter of 2015.

● Operating Expenses: On a GAAP basis in the third quarter of 2016, total operating expenses were 69.4 percent of GAAP net sales. Research & development (R&D) expenses were 6.1 percent of GAAP net sales, sales & marketing (S&M) expenses were 34.8 percent of GAAP net sales and general & administrative (G&A) expenses were 28.5 percent of GAAP net sales. Non-GAAP total operating expenses in the third quarter of 2016 were 39.9 percent of non-GAAP adjusted net sales. Non-GAAP R&D expenses were 3.8 percent of non-GAAP adjusted net sales, non-GAAP S&M expenses were 24.0 percent of non-GAAP adjusted net sales, and non-GAAP G&A expenses were 12.1 percent of non-GAAP adjusted net sales.

● Income Tax Rate: The income tax rate in the third quarter of 2016 on a GAAP basis was 82.5 percent and on a non-GAAP basis was 9.0 percent. The income tax rate for the first nine months of 2016 on a GAAP basis was 46.8 percent and on a non-GAAP basis was 14.6 percent.

● Net (Loss) Income: On a GAAP basis in the third quarter of 2016, net loss was $5.9 million and non-GAAP adjusted net income was $115.5 million.

● EBITDA: In the third quarter of 2016, EBITDA was $58.2 million, or 27.9 percent of GAAP net sales. Adjusted EBITDA in the third quarter of 2016 was $141.2 million, or 51.6 percent of non-GAAP adjusted net sales, compared to $131.1 million, or 57.9 percent of net sales in the third quarter of 2015.

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● (Loss) Earnings per Share: On a GAAP basis in the third quarter of 2016, diluted loss per share was $0.04 and in the third quarter of 2015, diluted earnings per share was $0.02. Non-GAAP diluted earnings per share in the third quarter of 2016 and 2015 were $0.70 and $0.42, respectively, representing growth of 66.7 percent. Weighted average shares outstanding used for calculating GAAP diluted loss per share and non-GAAP diluted earnings per share in the third quarter of 2016 were 161.0 million and 164.9 million, respectively.
Cash Flow Statement and Balance Sheet Highlights

● On a GAAP basis in the third quarter of 2016, operating cash flow was $128.8 million. Non-GAAP operating cash flow was $133.8 million in the third quarter of 2016. On a GAAP basis, operating cash flow in the first nine months of 2016 was $230.3 million compared to operating cash flow in the first nine months of 2015 of $59.2 million. On a non-GAAP basis, operating cash flow in the first nine months of 2016 was $259.8 million compared to operating cash flow in the first nine months of 2015 of $167.2 million.

● The Company had cash and cash equivalents of $549.3 million as of September 30, 2016. Cash and cash equivalents as of June 30, 2016 were $424.5 million.

● Total principal amount of debt outstanding was $1.270 billion as of September 30, 2016, which was composed of $395 million in senior secured term loans due 2021, $475 million in 6.625 percent senior notes due 2023, and $400 million of 2.5 percent exchangeable senior notes due 2022. Net debt at September 30, 2016 was $721 million.
On October 25, 2016, the Company completed a private offering of senior notes and borrowed incremental term loans under its existing senior secured credit facility to partially fund the acquisition of Raptor Pharmaceutical Corp., repay Raptor’s debt and pay related fees and expenses. Following the issuance of this new debt, the new total principal amount of debt outstanding is $1.945 billion, which is composed of $770 million in senior secured term loans due 2021; $475 million in 6.625 percent senior notes due 2023, $300 million in 8.75 percent senior notes due 2024, and $400 million of 2.5 percent exchangeable senior notes due 2022.

Karyopharm Reports Third Quarter 2016 Financial Results and Highlights Recent Progress

On November 7, 2016 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the third quarter 2016 and commented on recent accomplishments and clinical development plans for its lead, novel, oral Selective Inhibitor of Nuclear Export (SINE) compound selinexor (KPT-330), and KPT-8602, its second-generation SINE compound (Filing, Q3, Karyopharm, 2016, NOV 7, 2016, View Source [SID1234516628]).

"During the third quarter, we communicated our planned development and regulatory approval path for oral selinexor as a treatment for patients with multiple myeloma (MM)," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "We believe this plan, based on the positive selinexor-dexamethasone efficacy emerging from STORM and the selinexor-Velcade (bortezomib)-dexamethasone combination data from STOMP, provides a path to FDA and EMA filings. At the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) 2016 Annual Meeting, we will be highlighting twenty-one abstracts, including key presentations featuring maturing data from both STORM and STOMP, new clinical data in acute myeloid leukemia (AML), including selinexor in combination with chemotherapies in patients with newly diagnosed and relapsed/refractory AML, and preliminary data from a Phase 1 study of KPT-8602 in patients with relapsed/refractory MM."

Dr. Kauffman continued, "Looking ahead to the remainder of 2016, we are focused on executing the STORM trial expansion which will add approximately 120 additional patients with penta-refractory disease. We expect to report top-line data from this expanded cohort in early 2018, and, assuming a positive outcome, we intend to use this data to support accelerated approval for selinexor in MM. The trial design for the planned Phase 3 BOSTON study evaluating selinexor in combination with bortezomib and dexamethasone in patients with MM previously treated with one to three regimens, moves selinexor into much earlier lines of therapy and is currently being finalized to include feedback from the FDA. We remain on track to commence this pivotal study in early 2017."

Third Quarter 2016 and Recent Highlights:
Selinexor in Multiple Myeloma

• Reporting updated STORM data at ASH (Free ASH Whitepaper) 2016. Karyopharm is scheduled to present updated clinical data from the ongoing Phase 2b STORM study at the upcoming ASH (Free ASH Whitepaper) 2016 annual meeting in early December. In an oral presentation titled, "Selinexor and Low Dose Dexamethasone in Patients with Lenalidomide, Pomalidomide, Bortezomib, Carfilzomib and Anti-CD38 Ab Refractory MM STORM Study," Dan T. Vogl, MD, MSCE, Assistant Professor of Medicine, Perelman School of Medicine, University of Pennsylvania, will present data demonstrating that selinexor in combination with low-dose dexamethasone achieved an overall response rate (ORR) of 21% across all evaluable patients in the study. The ORRs were 21% in patients with quad-refractory disease and 20% in patients with penta-refractory disease, all based on Independent Review Committee (IRC) adjudication. The side effect profile for selinexor was consistent with previous trials, with low rates of Grade >3 non-hematologic toxicity, Grade >4 infections (1.3%) and sepsis (1.3%). Patients with quad-refractory disease have documentation that they have previously received two PIs (bortezomib (Velcade) and carfilzomib (Kyprolis)) and two IMiDs (lenalidomide (Revlimid) and pomalidomide (Pomalyst)), and their disease is refractory to at least one PI, at least one IMiD, alkylating agents and glucocorticoids, and has progressed following their most recent therapy. Patients with penta-refractory myeloma have quad-refractory disease that is also refractory to an anti-CD38 monoclonal antibody, such as daratumumab (Darzalex) or isatuximab.

• Expanding STORM Study to Include 120 Additional Patients with Penta-refractory MM. The Company believes that there are currently no available therapies with known activity in patients with penta-refractory myeloma, and that this represents a growing unmet medical need. Therefore, Karyopharm has expanded the STORM study to include approximately 120 additional patients with penta-refractory MM and expects to report top-line data from the expanded cohort in early 2018. Assuming a positive outcome, Karyopharm intends to use the data from the expanded STORM study to support accelerated approval for selinexor in MM.

• Reporting Updated STOMP data at ASH (Free ASH Whitepaper) 2016. Karyopharm is also scheduled to present updated clinical data from the ongoing Phase 1b STOMP study at ASH (Free ASH Whitepaper) 2016. In an oral presentation titled, "Selinexor in Combination with Bortezomib and Dexamethasone Demonstrates Significant Activity in Patients with Refractory MM Including Proteasome-Inhibitor Refractory Patients," Nizar Bahlis, MD, Assistant Professor of Hematology, Southern Alberta Cancer Research Institute, will present data demonstrating that selinexor in combination with Velcade (bortezomib) and dexamethasone (SVd) achieved an ORR of 77% across all evaluable patients in the study. All 10 patients with non-refractory disease responded (5 patients with a very good partial response (VGPR) and 5 patients with a partial response (PR)) for an ORR of 100%. Twelve of the 22 patients in the SVd combination arm had MM previously refractory to a proteasome inhibitor, typically bortezomib or carfilzomib. Seven of these 12 patients responded (1 complete response and 6 PRs) for an ORR of 58%. Only one patient (4.5%) had progressive disease, suggesting that this regimen induces rapid and potent myeloma control, even amongst patients with MM that is refractory to one or more proteasome inhibitors. Side effects were generally less than those observed with the individual drugs, and only one case of neuropathy (Grade 1, 4.5%) was reported. Similar high levels of activity are observed with the combination of selinexor and carfilzomib with dexamethasone, including in patients with MM that is refractory to one or more proteasome inhibitors; results from the Phase 1/2 study of this combination will also be reported at ASH (Free ASH Whitepaper). Together, these data indicate that treatment with selinexor in combination with proteasome inhibitors leads to high levels of anti-MM activity, including in patients with proteasome-inhibitor refractory disease.

• Initiating Pivotal Phase 3 BOSTON Study in Early 2017. Based on the robust data from the SVd arm of the STOMP study, Karyopharm plans to initiate a pivotal randomized Phase 3 study, known as the BOSTON (Bortezomib, Selinexor and dexamethasone) study, which will evaluate SVd compared to bortezomib and low-dose dexamethasone (Vd) in patients with MM who have had one to three prior lines of therapy. Karyopharm has identified the combination dose of selinexor (100mg weekly), bortezomib (1.3 mg/m2 weekly given sub-cutaneously for 4 of 5 weeks) and dexamethasone (40mg weekly) to be used in the BOSTON study and expects that the study will enroll approximately 360 patients. Based on feedback from the FDA, the protocol is currently being finalized and the Company remains on track to commence the BOSTON study in early 2017.

• Karyopharm to Host Dinner Reception and Webcast at ASH (Free ASH Whitepaper) 2016 Focusing on Multiple Myeloma. On Monday, December 5, 2016, Karyopharm will host an investor and analyst dinner reception, which will feature a moderated panel discussion with recognized thought leaders in the treatment of MM, updated selinexor data in MM, and a live Q&A session. The event will take place during the ASH (Free ASH Whitepaper) 2016 annual meeting and interested parties can access a live webcast of the event beginning December 5, 2016 at 8:15 p.m. PT by going to the "Investors" section of the company’s website at View Source
Selinexor in Acute Myeloid Leukemia (AML)

• Reporting Clinical Data from Phase 2 SAIL Study at ASH (Free ASH Whitepaper) 2016. Updated clinical data from the Phase 2 SAIL study evaluating selinexor in combination with Ara-C and idarubicin in heavily pretreated patients with relapsed or refractory AML will be highlighted in an oral presentation by Walter Fiedler, MD, University Medical Center Hamburg. The SAIL data demonstrate that the selinexor, Ara-C and idarubicin combination achieved compelling response rates and has the potential to be an effective AML treatment option and serve as a bridge to stem cell transplantation in this patient population.

• Other Selinexor Combination Studies Selected for Oral and Poster Presentations at ASH (Free ASH Whitepaper) 2016. Three additional abstracts were selected for presentation at ASH (Free ASH Whitepaper), including one oral presentation highlighting data from a clinical trial evaluating the combination of selinexor with high-dose cytarabine and mitoxantrone in patients with AML (Amy Wang, University of Chicago) and two poster presentations (Bhavana Bhatnagar, Ohio State University and Kendra Sweet, Moffitt Cancer Center). These presentations highlight early-stage clinical data demonstrating the feasibility and tolerability of selinexor in combination with other standard of care agents in patients with AML, including in elderly patients, as well as early signs of clinical activity, including response rates that are superior to published data using standard chemotherapy regimens.
Selinexor in Solid Tumors

• Reported Updated SIGN data at ESMO (Free ESMO Whitepaper) 2016. Updated clinical data from the Phase 2 SIGN study evaluating selinexor for the treatment of gynecological cancers were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 annual meeting. In this study, single-agent selinexor demonstrated robust clinical benefit and favorable tolerability in patients with heavily pretreated gynecologic cancers, including a 49% disease control rate (DCR = PR plus stable disease for >3 months) in ovarian cancer and 45% in endometrial cancer. Selinexor-associated adverse events were found to be manageable with supportive care and dose modifications as demonstrated by the number of patients who have remained on study after achieving disease control, with some continuing treatment for longer than 12 months.

KPT-8602

• Reporting Phase 1 KPT-8602 Clinical Data at ASH (Free ASH Whitepaper) 2016. Clinical data from a Phase 1/2 study evaluating KPT-8602, Karyopharm’s second-generation SINE compound, will be presented at ASH (Free ASH Whitepaper) 2016 by Frank Cornell, MD, Vanderbilt Ingram Cancer Center. These data demonstrate that oral KPT-8602 is well tolerated in heavily pretreated patients with relapsed or refractory MM and shows early signs of encouraging efficacy.

Third Quarter 2016 Financial Results
Cash, cash equivalents and investments as of September 30, 2016, including restricted cash, totaled $176.9 million, compared to $166.2 million as of June 30, 2016. The increased cash balance includes the net proceeds from the sales of common stock through the Company’s At-the-Market (ATM) financing facility through September 30, 2016 of approximately $31.5 million dollars.

Subsequent to the close of the quarter, in October, the Company sold additional shares of common stock through the same ATM facility for additional net proceeds of approximately $15.4 million. In total, Karyopharm sold 5,243,914 shares of common stock for gross proceeds of $48.2 million and net proceeds of approximately $46.9 million in September and October combined. As of October 31, 2016, the Company has 41,262,146 shares outstanding and 47,215,794 fully diluted shares inclusive of outstanding stock options and restricted stock units.

For the quarter ended September 30, 2016, research and development expense was $19.9 million compared to $25.9 million for the quarter ended September 30, 2015. For the quarter ended September 30, 2016, general and administrative expense was $5.9 million compared to $4.8 million for the quarter ended September 30, 2015.

Karyopharm reported a net loss of $25.4 million, or $0.69 per share, for the quarter ended September 30, 2016, compared to a net loss of $30.4 million, or $0.85 per share, for the quarter ended September 30, 2015. Net loss includes stock-based compensation expense of $5.6 million and $3.5 million for the quarters ended September 30, 2016 and September 30, 2015, respectively.

Financial Outlook
Karyopharm expects to end 2016 with at least $170.0 million in cash, cash equivalents and investments. Based on current operating plans, Karyopharm expects that its existing cash and cash equivalents will fund its research and development programs and operations through the end of 2018, including through the data readout for the expanded STORM cohort, completion of enrollment for the BOSTON study and advancement of the SOPRA, SADAL and SEAL clinical studies to their next data inflection points.

Kura Oncology Reports Third Quarter 2016 Financial Results

On November 7, 2016 Kura Oncology, Inc., (Nasdaq:KURA) a clinical stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported third quarter 2016 financial results and recent business highlights (Press release, Kura Oncology, NOV 7, 2016, View Source;p=RssLanding&cat=news&id=2220292 [SID1234516570]).

"During the third quarter, we have continued to advance our tipifarnib development program," said Troy Wilson, Ph.D., J.D., President and CEO of Kura Oncology. "Our Phase 2 HRAS solid tumor trial is progressing and the two patients from stage 1 with partial responses have now been on study for 15 months and 8 months, which is encouraging given the relatively limited clinical benefit these patients observed on prior therapy. We have focused the second stage of the trial on patients with HRAS mutant squamous cell head and neck cancer, and we look forward to additional results in this patient population."

"In addition, our trials in PTCL and lower-risk MDS are ongoing, and we recently initiated our planned Phase 2 trial for tipifarnib in patients with CMML," stated Dr. Wilson. "We believe each of these Phase 2 studies has a strong scientific and clinical rationale, and they provide multiple potential opportunities for registration-enabling studies."

Dr. Wilson continued, "We are preparing to advance our ERK inhibitor, KO-947, into clinical testing and are encouraged by the consistent and compelling activity we have observed in preclinical models of cancers with mutations or dysregulation of the MAPK pathway as well as by our identification of potential biomarkers to guide development. I am very pleased we are on track to submit an IND before year-end."

Upcoming Clinical and Preclinical Activities for Kura Oncology Programs

Submission of the investigational new drug (IND) for KO-947 is anticipated in the fourth quarter of 2016.

Nomination of a development candidate for the menin-MLL program is anticipated in the fourth quarter of 2016.

Presentation of preclinical data for KO-947 and menin-MLL program at EORTC in Munich in November 2016.

Initiation of a Phase 1 study for KO-947 is anticipated in the first half of 2017.

Topline data from the Phase 2 study of tipifarnib in PTCL is anticipated in the first half of 2017.

Additional data from the Phase 2 study of tipifarnib in HRAS mutant tumors is anticipated in the first half of 2017.
Financial Results for the Third Quarter 2016

Cash, cash equivalents and short-term investments totaled $74.6 million as of September 30, 2016, compared with $85.7 million as of December 31, 2015. Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2018.

Research and development expenses for the third quarter of 2016 were $5.3 million, compared to $4.6 million for the third quarter of 2015.

General and administrative expenses for the third quarter of 2016 were $1.7 million, compared to $1.8 million for the third quarter of 2015.

Net loss for the third quarter of 2016 was $6.9 million, or $0.37 per share, compared to a net loss of $6.1 million, or $0.57 per share, for the third quarter of 2015.