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]).

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"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.

On November 7, 2016 SignalRx Pharmaceuticals Inc., a clinical-stage company focused on developing more effective oncology drugs with designed multiple target-selected inhibition profiles, reported the publication of key research supporting the use of its clinical stage drug SF1126 alone and in combination with Sorafinib for the treatment of hepatocellular carcinoma (HCC) (Press release, SignalRx, NOV 7, 2016, http://www.ireachcontent.com/news-releases/signalrx-announces-publication-of-research-results-on-sf1126-as-a-first-in-class-dual-pi3kbrd4-inhibitor-for-treating-hcc-600328031.html [SID1234527326]). The research was published in the November issue of Molecular Cancer Therapeutics journal from the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (Mol Cancer Ther November 1 2016 15 (11) 2553-2562; DOI:10.1158/1535-7163.MCT-15-0976).

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Researchers at the University of California, San Diego School of Medicine and Moores Cancer Center, led by Dr. Donald Durden, Professor and Associate Director of Pediatric Oncology and senior scientific advisor for SignalRx, report results supporting the use of SF1126 as a novel therapeutic agent for the treatment of hepatocellular carcinoma (HCC), the most common kind of liver cancer and second most common cause of cancer death worldwide.

SF1126 is an anticancer agent shown to have an excellent therapeutic window with excellent tolerability and safety in Phase I clinical trials (Clinicaltrials.gov: NCT00907205). While most anti-cancer drugs only interact with a single cancer target, SF1126 inhibits two key cancer signaling molecules in liver cancer cells, phosphatidylinositol 3-kinase (PI3K) and bromodomain-containing 4 (BRD4). SF1126 represents a "first in class" approach to treat liver cancer by hitting two central signaling nodes of the liver cancer cell with only one therapeutic agent. The published work shows that this novel strategy kills liver cancer cells and prevents the growth of liver cancer tumors in mice.

Targeting two pathways with one drug can provide a significant therapeutic advantage since this approach also reduces the risk of severe "off-target" side effects resulting from the combination of side effects associated to each of the multiple drugs used. The treatment of HCC remains a challenge with Sorafenib as the only FDA-approved drug for liver cancer since it prolongs life for an average of only 2-3 months and can have significant side effects. Work from the Durden laboratory shows that using SF1126 with Sorafenib provides a dramatically improved anticancer effect by killing liver cancer cells in synergy.

In HCC, the deregulation of the PI3K/AKT/mTOR, Ras/Raf/MAPK and c-Myc signaling pathways are of prognostic significance. While Sorafenib blocks the Ras/Raf/MAPK pathway, it does not inhibit the PI3K/AKT/mTOR pathway or c-Myc activation. SF1126 controls c-Myc by inhibiting BRD4, which results in blockage of c-Myc production, and by inhibiting PI3K, which leads to enhanced c-Myc degradation. Hence, a combination of SF1126 with Sorafenib offers a new mechanism-driven mode of action to inhibit/treat HCC.

In particular, the research results published in Molecular Cancer Therapeutics demonstrate that:

SF1126 (pan PI3K/BRD4 inhibitor), as a single agent or in combination with Sorafenib, inhibits cancer cell proliferation (Hep 3B, Hep G2, SK-Hep1 and Huh7 HCC cell lines) by effectively inhibiting the PI3K/AKT/mTOR and Ras/Raf/MAPK pathways.
SF1126’s active moiety LY294002 binds to and blocks BRD4 interaction with the acetylated histone-H4 chromatin mark protein and displaces the BRD4 co-activator protein from the transcriptional start site of MYC in Huh7 and SK-Hep-1 HCC cell lines.
SF1126 blocks expression of c-Myc in HCC cells.
SF1126, either alone or in combination with Sorafenib, shows significant antitumor activity in vivo.
These published results establish SF1126 as a dual PI3K/BRD4 inhibitor and the first epigenetic/kinase inhibitor in the clinic. SF1126 has completed a Phase I clinical trial in humans with good safety profile, has received Orphan Drug Designation by the FDA, and is currently in a pediatric Phase I clinical trial in children with neuroblastoma.

Taken together, this published data strongly warrants additional clinical trials of SF1126 in advanced HCC as well as a combination Phase I trial with Sorafenib.

SignalRx is seeking partners for the clinical development of SF1126 as well as the acceleration of the company’s preclinical pipeline with novel and proprietary nM potent small molecules into first-in-man clinical trials.

SignalRx’s novel dual inhibitors have a unique competitive advantage over combining separate agents in cancers where lethality requires simultaneous target inhibition for maximal effect with minimal side-effects. Because it provides a single pharmacodynamics profile the dual inhibition in a single molecule approach provides the optimal way to effect simultaneous target inhibition with significantly less toxicity than combinations of inhibitors.

On November 7, 2016 Caladrius Biosciences, Inc. (NASDAQ:CLBS) ("Caladrius" or the "Company"), a cell therapy company combining an industry-leading development and manufacturing services provider through its subsidiary PCT, LLC a Caladrius Company ("PCT") with a select therapeutic development pipeline, reported financial results for the three and nine months ended September 30, 2016 (Filing, Q3, Caladrius Biosciences, 2016, NOV 7, 2016, View Source [SID1234516370]).
Business and financial highlights for the third quarter of 2016 and recent weeks include:
•
Achieved total quarterly revenues of $9.3 million, up 58% compared with $5.9 million for the third quarter of 2015;
•
Received Fast Track designation from the U.S. Food and Drug Administration (FDA) for CLBS03 (autologous expanded regulatory T cells, or Tregs) for the treatment of type 1 diabetes (T1D), making it the first known therapeutic candidate for the treatment of T1D to receive the designation;
•
Completed enrollment of the first patient cohort of The Sanford Project: T-Rex Study, the Company’s Phase 2 clinical trial of CLBS03 for the treatment of recent-onset T1D in adolescents;
•
Received a favorable recommendation from the independent Data Safety Monitoring Board (DSMB) to proceed with enrollment of the second cohort of the T-Rex Study following the safety evaluation of the first cohort of 19 patients;
•
Entered into a new five-year strategic manufacturing services agreement under which PCT will produce SPEAR T-cell therapies for Adaptimmune Therapeutics plc; and
•
Entered into agreements with several accredited investors, including previous investors and strategic collaborator Sanford Health, to sell up to $25 million in common equity priced at market without warrants.
Management Commentary
"Throughout 2016, we consistently increased revenues at our PCT subsidiary and are poised to achieve our stated goal of 2016 annual revenue in excess of $30 million, which represents annual growth of more than 30%. In addition, year-to-date we secured over $50 million in strategic and/or committed financings, about half of which was non-dilutive with the remainder under favorable terms relative to the market rates for companies like ours. We also paid back to our lender over $9 million of long-term debt, significantly reduced our operating expenses and monetized non-core assets, all while advancing our key immune modulation program’s Phase 2 clinical study of CLBS03 to treat T1D," stated David J. Mazzo, Ph.D., Chief Executive Officer of Caladrius.
"We are particularly pleased with the progress of The Sanford Project: T-Rex Study in T1D. We completed enrollment of the first cohort of 19 patients and were delighted that the DSMB recommended continuation of the study based on a favorable safety assessment. We have begun enrolling patients into the second cohort of this 111-patient study earlier than originally expected and plan to reach the 50% enrollment mark in mid-2017. Enrollment of the 70th patient, which triggers an $8.4 million capital infusion under the terms of the September private placements, is expected to occur shortly thereafter. We continue to benefit from the support of Sanford Research. In addition to their equity investment, Sanford has agreed to extend operational support at their clinical sites for the remainder of the study. We continue to be excited by the promise of CLBS03, a novel therapeutic being developed to address the significant unmet medical need in this chronic disease that affects children and young adults. We have made significant achievements across a number of key areas that we believe position Caladrius for continued growth and success throughout the balance of 2016 and beyond," concluded Dr. Mazzo.
Third Quarter Financial Highlights
Total revenues for the third quarter of 2016 increased 58% to $9.3 million compared with $5.9 million for the third quarter of 2015 and increased 12% compared with $8.3 million for the second quarter of 2016. Gross margin on revenues was 8% in the third quarter of 2016 compared with 18% in the third quarter of 2015, directly impacted by the non-payment of approximately $600,000 in billings for work the Company performed and billed to a single customer during the third quarter of 2016, which customer is currently experiencing financial difficulties. Accordingly, the Company has delayed revenue recognition until such time as payment is reasonably assured. The Company continues to work with this client to obtain payment and will recognize any such receipts as revenue in the periods received.

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Research and development (R&D) expenses for the third quarter of 2016 decreased 58% to $2.6 million compared with $6.3 million for the third quarter of 2015. The majority of current quarter expenses were dedicated to the Company’s immune modulation platform and, specifically, costs related to the T-Rex Study. The decline in R&D expenses over the prior year quarter was primarily related to the discontinuation of non-core R&D programs announced at the beginning of 2016 and related reductions in R&D staffing and departmental costs.
Selling, general and administrative (SG&A) expenses for the three months ended September 30, 2016 were $4.9 million, a small reduction from $5.1 million reported for the same period in 2015. This reflected significantly lower operational and compensation-related costs during the current year quarter compared with the prior year quarter, partially offset by higher equity-based compensation expenses compared with the prior year quarter.
The operating loss for the third quarter of 2016 was $6.9 million compared with an operating loss of $10.4 million for the third quarter of 2015, reflecting higher revenues and lower R&D expenses.
The Company reported a net loss attributable to Caladrius common stockholders for the third quarter of 2016 of $6.9 million, or $1.09 per share, compared with a net loss attributable to Caladrius common stockholders for the third quarter of 2015 of $11.4 million, or $2.06 per share.
Nine Month Financial Highlights
Total revenues for the nine months ended September 30, 2016 increased 68% to $25.1 million compared with $14.9 million for the first nine months of 2015. Gross margin for the first nine months of 2016 was 13% compared with 6% for the first nine months of 2015.
R&D expenses for the first nine months of 2016 decreased to $12.5 million compared with $20.7 million for the first nine months of 2015. The decline in R&D expenses over the first nine months of 2015 was primarily related to the discontinuation of non-core R&D programs announced at the beginning of 2016 and related reductions in R&D staffing and departmental costs.
SG&A expenses decreased to $16.1 million for the first nine months of 2016 compared with $25.0 million for the same period in 2015. This reflected operational and compensation-related cost reductions, as well as equity-based compensation expenses that were significantly below the prior year SG&A expense levels.
The operating loss for the first nine months of 2016 was $25.4 million compared with an operating loss of $54.1 million for the first nine months of 2015.
The net loss attributable to Caladrius common stockholders for the nine months ended September 30, 2016 was $26.7 million, or $4.45 per share, compared with a net loss attributable to Caladrius common stockholders for the nine months ended September 30, 2015 of $47.7 million, or $10.40 per share.
Balance Sheet and Cash Flow Highlights
As of September 30, 2016, Caladrius had cash and cash equivalents of $18.6 million, which included $10.6 million received from the previously announced equity financing in September 2016 and the payment of $3.0 million to Oxford Finance LLC for repayment of long-term debt. Net cash used in operating activities for the nine months ended September 30, 2016 was $20.3 million, compared with $30.5 million for the nine months ended September 30, 2015. Caladrius also invested $2.3 million in capital expenditures, primarily related to improvements to PCT’s Allendale, N.J. manufacturing facility.
2016 Financial Guidance
The Company updates its previous 2016 guidance as follows:
•
Consolidated 2016 Annual Revenues: affirms guidance to exceed $30 million or a greater than 30% increase compared with 2015.
•
Capital Improvements at PCT’s Allendale, N.J. Facility: affirms guidance that improvements will be completed in 2017, and updates guidance that approximately $2 million will be spent in calendar 2016. Overall cost to complete capital improvements in 2017, including the implementation of commercial grade quality systems, to be provided when 2017 guidance is announced.
•
CLBS03 Phase 2 Study Costs in 2016: affirms guidance of $6 million to $7 million.
•
Consolidated 2016 Operating Activities Cash Burn: affirms guidance of $25 million to $28 million, or between $5 million and $8 million in the fourth quarter of 2016, but with a trend toward the lower end of the range.

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.

On November 7, 2016 DelMar Pharmaceuticals, Inc. (NASDAQ: DMPI) ("DelMar" and the "Company"), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, reported that the Company and its research collaborators have presented data at two scientific conferences hosted by the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (Press release, DelMar Pharmaceuticals, NOV 7, 2016, View Source [SID1234516371]).

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AACR DNA Repair: Tumor Development & Therapeutic Response Conference. On Friday, November 4, 2016, DelMar and its collaborators from the University of British Columbia Prostate Cancer Research Center presented an abstract entitled: "Dissecting the Molecular Mechanism of Dianhydrogalactitol (VAL-083) in Cancer Treatment."
AACR New Horizons in Cancer Research: Delivering Cures through Cancer Research. Also, on Friday, November 4, 2016, DelMar and its collaborators from the University of Texas MD Anderson Cancer Center and the University of British Columbia presented an abstract entitled: "Assessment of dianhydrogalactitol in the treatment of relapsed or refractory non-small cell lung cancer."
The presentations can be accessed on DelMar’s website at View Source

Jeffrey Bacha, DelMar’s chairman & CEO, stated, "We continue to make great strides in understanding how VAL-083 targets cancer cells."

The data demonstrate that, upon treatment, VAL-083 rapidly forms cross-links on the DNA of cancer cells leading to cell cycle arrest in the S/G2 phase and lethal double-strand DNA breaks. This mechanism is distinct from other alkylating agents used in the treatment of cancer such as temozolomide, nitrosoureas or platinum-based chemotherapy. Because VAL-083’s mechanism differs from the others it is not subject to the same resistance mechanisms and therefore may be used to treat patients whose tumors are resistant to other therapies.

"We are leveraging clinical validation from prior NCI-sponsored research with a modern understanding of VAL-083’s unique anti-cancer mechanism to diversify our product development portfolio into new indications such as lung and ovarian cancer," added Mr. Bacha.

About VAL-083
VAL-083 is a "first-in-class," small-molecule chemotherapeutic. In more than 40 Phase I and II clinical studies sponsored by the U.S. National Cancer Institute, VAL-083 demonstrated clinical activity against a range of cancers including lung, brain, cervical, ovarian tumors and leukemia both as a single-agent and in combination with other treatments.

VAL-083 has received an orphan drug designation in Europe for the treatment of malignant gliomas and the U.S. FDA Office of Orphan Products has granted an orphan designation to VAL-083 for the treatment of glioma, medulloblastoma and ovarian cancer.

The Company has completed a successful end of Phase II meeting with the US FDA and plans to advance VAL-083 into a pivotal clinical trial for GBM patients following bevacizumab failure. DelMar presented data from its Phase I/II clinical trial in refractory GBM at the 2016 American Association of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual meeting demonstrating that the median survival of 22 patients receiving an assumed therapeutic dose of VAL-083 (≥20mg/m2) was 8.35 months following bevacizumab (Avastin) failure compared to published literature where survival of approximately two to five months has been reported.

DelMar’s advanced development program will feature a single multi-center randomized Phase III study measuring survival outcomes compared to a "physicians’ choice" control, which, if successful, would serve as the basis for a New Drug Application (NDA) submission for VAL-083. The control arm will consist of a limited number of salvage chemotherapies currently utilized in the treatment of Avastin-failed GBM. The final pivotal trial design will be confirmed with the FDA following further discussions with the Company’s clinical advisors.

In addition to the pivotal trial, DelMar also plans to initiate two separate Phase II clinical trials in earlier-stage GBM patients.

In collaboration with the University of Texas MD Anderson Cancer Center: A non-comparative, biomarker-driven, Phase II study to determine if treatment of MGMT-unmethylated recurrent GBM with VAL-083 or CCNU improves overall survival at 9 months, compared to historical control in bevacizumab naïve patients. (clinicaltrials.gov identifier: NCT02717962)
In collaboration with Sun-Yat Sen University and Guangxi Wuzhou Pharmaceutical (Group) Co.: A single arm Phase II clinical trial to confirm the tolerability of DelMar’s dosing regimen in combination with radiotherapy (XRT) and to explore the activity of VAL-083 in newly diagnosed MGMT-unmethylated GBM patients whose tumors are known to express high levels of MGMT.
DelMar believes that data from these clinical trials, if successful, will form the basis of a new paradigm in the treatment for all GBM patients who fail, or whose tumors exhibit features that make them unlikely to respond to currently available chemotherapy.

In addition to its clinical research in GBM, DelMar believes that its research supports a unique mechanism of action for VAL-083 and that these data support the potential of VAL-083 as a new chemotherapy that may offer improved outcomes in the treatment of GBM and other solid tumors in patients whose tumors have failed or exhibit features that make them resistant to or unlikely to respond to current standard-of-care chemotherapy.

The company and its collaborators from the University of Texas MD Anderson Cancer Center recently presented data at the 11th Biennial Ovarian Cancer Research Symposium demonstrating that VAL-083 was able to overcome cisplatin-resistance in ovarian cancer cell lines with known p53 mutations and displays synergy with both cisplatin and AstraZeneca’s PARP inhibitor Olaparib against ovarian cancer in vitro.