Regulus has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Regulus, 2017, NOV 7, 2017, View Source [SID1234521806]).

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On November 7, 2017 Curis, Inc. (NASDAQ:CRIS), a biotechnology company focused on the development and commercialization of innovative and effective drug candidates for the treatment of human cancers, reported its financial results for the third quarter ended September 30, 2017 (Press release, Curis, NOV 7, 2017, View Source [SID1234521629]).

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"We are pleased to announce the acceptance of our Investigational New Drug (IND) application for CA-4948, an inhibitor of IRAK4 kinase, which we expect to enter the clinic soon for the treatment of lymphomas with specific mutations," said Ali Fattaey, President and CEO. "In addition, we are continuing to enroll patients in the Phase 1 trial of CA-170, an oral, small molecule, dual immune checkpoint inhibitor, to determine the recommended dose and most promising areas of future development for this molecule. We look forward to presenting additional preliminary data from the ongoing Phase 1 trial at the upcoming 2017 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Meeting. In our CUDC-907 program, we expect to present data supporting its clinical benefit in patients with MYC-altered DLBCL at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) conference in December, and are planning for our discussions regarding these results with the FDA. And, on the financial front, our recently completed offering of common stock in September provides us operating capital to support these programs well into 2019."

Third Quarter 2017 Financial Results

Curis reported a net loss of $15.5 million, or $0.11 per share, on both a basic and diluted basis for the third quarter of 2017, as compared to a net loss of $28.3 million, or $0.21 per share, on both a basic and diluted basis for the same period in 2016. Curis reported a net loss of $45.3 million, or $0.31 per share, on both a basic and diluted basis for the nine months ended September 30, 2017, as compared to a net loss of $49.1 million, or $0.38 per share on both a basic and diluted basis for the same period in 2016. The net loss for the prior year period includes a non-cash in-process research and development charge of $18.0 million related to the amendment of Curis’s license agreement with Aurigene.

Revenues for the third quarter of 2017 were $2.4 million, as compared to $1.8 million for the same period in 2016. Revenues for the nine months ended September 30, 2017 were $6.6 million, as compared to $5.2 million for the same period in 2016. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge.

Operating expenses were $16.9 million for the third quarter of 2017, as compared to $29.5 million for the same period in 2016.

Operating expenses for the nine months ended September 30, 2017 were $49.3 million, as compared to $52.4 million for the same period in 2016, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily relates to amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.1 million for both the third quarter of 2017 and 2016. Cost of royalty revenues for the nine months ended September 30, 2017 and 2016 were $0.3 million for both periods.

In-Process Research and Development Expense. No in-process research and development expenses were recorded for the nine months ended September 30, 2017 as compared to $18.0 million recorded during the third quarter of 2016 associated with the issuance of 10,208,333 shares of Curis common stock to Aurigene as consideration for the rights granted under the terms of the September 2016 amendment to our collaboration agreement.

Research and Development Expenses. Research and development expenses were $13.4 million for the third quarter of 2017, as compared to $6.8 million for the same period in 2016. The increase was primarily due to a payment to Aurigene of $3.8 million for an exclusivity option in September 2017 and increased direct spending related to clinical activities of CA-170 and increased employee-related expenses primarily due to additional headcount. Research and development expenses were $38.2 million for the nine months ended September 30, 2017 as compared to $22.4 million for the same period in 2016.­

General and Administrative Expenses. General and administrative expenses were $3.4 million for the third quarter of 2017 as compared to $4.7 million for the same period in 2016. The decrease in general and administrative expenses was driven primarily by a one-time stock-based compensation modification expense incurred in 2016 and lower legal, professional, consulting and other administrative expenses. General and administrative expenses were $10.8 million for the nine months ended September 30, 2017, as compared to $11.7 million for the same period in prior 2016.

Other expense, net was $1.0 million for the third quarter of 2017, as compared to $0.6 million for the same period in 2016. Other expense, net primarily consisted of interest expense related to Curis Royalty’s (a wholly owned subsidiary of Curis) debt obligations. Other expense, net was $2.7 million and $1.8 million for the nine months ended September 30, 2017 and 2016, respectively.

As of September 30, 2017, Curis’s cash, cash equivalents, marketable securities and investments totaled $69.2 million and there were approximately 164.0 million shares of common stock outstanding. On a fully-diluted basis, which includes 17.0 million options, there were 181.0 million shares outstanding.

Immuno-oncology (CA-170: PD-L1 / VISTA antagonist program; Aurigene collaboration):

In September 2017, Curis presented preliminary data from the dose escalation stage of the CA-170 Phase 1 trial at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress.

Following evidence in Phase 1 of tumor shrinkage, multiple patients remaining on CA-170 treatment for extended periods, and compelling signals for biomarkers of immune modulation in patient blood and tumor samples, Curis’s partner Aurigene announced a decision to fund a jointly-designed Phase 2 study at sites in India.

Precision oncology (CA-4948, IRAK4 inhibitor program):

Curis announces U.S. FDA acceptance of the IND to test CA-4948 in a Phase 1 trial in patients with hematologic malignancies, and in particular those with MYD88 gene mutations. A Phase 1 trial is expected to begin in the fourth quarter of 2017.

Precision oncology (CUDC-907: MYC-altered DLBCL program):

In August 2017, Curis reported data from the interim analysis of the Phase 2 trial of CUDC-907 in patients with MYC-altered diffuse large B-cell lymphoma (DLBCL). The company is preparing for discussions with the FDA regarding these results.

CA-327 (an orally bioavailable inhibitor of PDL1 and TIM3 immune checkpoints):

Curis expects to file an IND in the first half of 2018.

Upcoming Activities

Curis expects that it will make presentations at the following conferences through December 2017:

An oral presentation on preliminary data from the ongoing Phase 1 trial of CA-170 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 32nd Annual Meeting (Nov. 8-12, 2017) in Oxon Hill, Maryland

Poster presentation on oral small molecule combination therapy targeting PD-L1, VISTA and Tim-3 checkpoints at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 32nd Annual Meeting (Nov. 8-12, 2017) in Oxon Hill, Maryland

Presentation as part of an analyst-moderated fireside chat at the Cowen IO NEXT Summit (Nov. 10, 2017) in Oxon Hill, Maryland

CUDC-907 poster presentation at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Dec. 9-12, 2017) in Atlanta

Overview of Company’s pipeline at the Guggenheim Securities Healthcare Conference (Dec. 13, 2017) in Boston

Conference Call Information

Curis management will host a conference call today, Nov. 7, 2017, at 8:30 a.m. EST to discuss these financial results and provide a corporate update.

To access the live conference call, please dial (877) 868-1829 from the United States or (253) 237-1135 from other locations shortly before 8:30 a.m. EST. The conference ID number is 6198027. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.

On November 7, 2017 Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on oncology, reported seven presentations at the upcoming Connective Tissue Oncology Society (CTOS) and the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meetings to be held in Maui, Hawaii from November 8-11 and in National Harbor, Maryland, from November 8-12, 2017 respectively (Press release, Immune Design, NOV 7, 2017, View Source [SID1234521650]).

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Selected highlights from these presentations include:

CTOS

A Phase 2 study of CMB305 and Atezolizumab in NY-ESO-1+ soft tissue sarcoma: interim analysis of immunogenicity, tumor control and survival (Abstract ID #2785040, Presenter: Sant P. Chawla, M.D, Director of the Cancer Center of Southern California, medical oncologist at Cedars-Sinai Comprehensive Cancer Center, adjunct associate professor at Stanford University and the University of Texas, M.D. Anderson Cancer Center; Encore Presentation)

Interim analysis (n=36) in the randomized Phase 2 study showed patients receiving the combination of CMB305 and atezolizumab experienced greater clinical benefit and immune response than those who received atezolizumab alone, notwithstanding that patients in the combination arm had more advanced disease.
The trend of greater clinical benefit on the combination arm remained consistent in the full study population (n=88), including partial responses on the combination arm and none on the atezolizumab-only arm.
Association of NY-ESO-1 expression with baseline immunity and clinical outcomes in soft tissue sarcoma patients treated with LV305 or CMB305 (Abstract ID #2804760, Presenter: Seth M. Pollack, M.D, Fred Hutchinson Cancer Research Center, Assistant Professor, Division of Oncology, University of Washington)

The cancer-testes antigen, NY-ESO-1, is highly expressed in certain soft tissue sarcoma subtypes. 48 soft tissue sarcoma patients receiving the NY-ESO-1-target cancer vaccine, CMB305 or its prime-only component, LV305, were pooled for analysis.
Despite a poorer prognosis expected in patients with tumors with high levels of NY-ESO-1 expression, patients with high expression levels treated with either LV305 or CMB305 had a trend toward improved clinical outcomes as compared to those in the lower quartiles.
SITC

Anti-NY-ESO-1 immune response and survival benefit after LV305 therapy in patients with advanced sarcoma and other solid tumors (Poster #P109, Presenter: Jan H. ter Meulen, M.D., Dr. Habil., DTM&H, Immune Design)

Immune response and long term overall survival data in 24 sarcoma patients, who have received multiple lines of therapy and have been followed for at least two years, shows a median progression free survival of 4.67 months and a median overall survival that has not yet been reached.
Exploratory analysis of biomarker data demonstrates that patients with baseline anti-NY-ESO-1 antibodies or induced immune response on LV305 appear to have improved survival outcomes.
G100 and ZVex-based combination immunotherapy induces near complete regression of established glioma tumors in mice (Poster #P256, Presenter: Tina Chang Albershardt, Ph.D., Immune Design)

"Prime/pull" immunization consisting of systemic administration of ZVex vector with-antigen and intratumoral administration of G100, respectively, resulting in a >90% reduction of established gliomas.
We believe these are among the best data showing control of glioma in the orthotopic mouse model using therapeutic cancer vaccines.
Transduction of MAGE-A1, A3, A4, A10 and IL-12 by ZVex, a dendritic cell targeting platform induces robust multi-antigen T-cell immune responses without antigenic interference or immunodominance (Poster #P127, Presenter: Jardin Leleux, Ph.D., Immune Design)

A multigenome ZVex vector expressing the cancer testis antigens MAGE-A1, 3, 4, 10 and the cytokine IL12 induces balanced T-cell responses against all four antigens.
These data demonstrate that the multigenome vector platform can overcome antigenic competition, a common problem encountered with virally vectored vaccines.
Intratumoral expression of IL12 using the ZVex dendritic cell-targeting lentiviral vector exerts potent anti-tumor effects via induction of multiple immune effectors, including CD8 T cell responses (Poster #P401, Presenter: Tina Chang Albershardt, Ph.D., Immune Design)

Extending previously published observations, these data show that the potent intratumoral effects of ZVex-IL12 are mediated by multiple immune effector cells, including CD8 T-cells.
These data provide further preclinical validation of the vector platform for intratumoral applications.
Public NY-ESO-1 specific TCRs as novel biomarkers for immune monitoring of NY-ESO-1 positive cancer patients (Poster #P58 and oral presentation, presenter: Hailing Lu, M.D., Ph.D., Immune Design)

These data extend previously published observations of an association of NY-ESO-1- specific, shared TCR Vβ-CD3 sequences with survival in NY-ESO-1-expressing cancer patients.
The findings may support use of public TCR as a vaccine response biomarker for NY-ESO-1 cancer patients that could augment or replace established, yet more cumbersome, T-cell ELISPOT assays.

On November 7, 2017 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported company highlights and financial results for the third quarter ended September 30, 2017 (Press release, Ignyta, NOV 7, 2017, View Source [SID1234521678]). The company is issuing this press release in lieu of conducting a conference call.

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"In addition, during the quarter we continued to advance our pipeline of precision medicines targeted at the molecular and immunological drivers of cancer, and we strengthened our balance sheet through an equity offering that provides us with additional resources to continue developing meaningful new cancer therapies for patients."
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"We are pleased with the continued development and regulatory progress of our lead product candidate, entrectinib—an investigational, CNS-active, potent, and selective tyrosine kinase inhibitor being developed for tumors that harbor TRK or ROS1 fusions—as we approach the expected submission of two NDAs and a PMA in 2018," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "In addition, during the quarter we continued to advance our pipeline of precision medicines targeted at the molecular and immunological drivers of cancer, and we strengthened our balance sheet through an equity offering that provides us with additional resources to continue developing meaningful new cancer therapies for patients."

Company Highlights

Entrectinib

Regulatory Updates: Orphan Drug Designation and PRIME

In July 2017, we announced that FDA granted orphan drug designation to entrectinib for "treatment of NTRK fusion-positive solid tumors."

In October 2017, we announced that the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation for entrectinib in the treatment of NTRK fusion-positive, locally advanced or metastatic solid tumors in adult and pediatric patients who have either progressed following prior therapies or who have no acceptable standard therapy. Entrectinib is the only TRK inhibitor to have been granted PRIME designation, which is analogous to the Breakthrough Therapy Designation from the U.S. FDA that entrectinib received earlier in 2017.

Development and Clinical Data Updates Towards Dual TRK and ROS1 NDA Submissions

In September 2017, we announced completion of enrollment of the efficacy data sets for both the TRK tissue-agnostic (i.e., fusion-positive solid tumor) cohort and the ROS1 NSCLC cohort to support dual NDA submissions in 2018.

In October 2017, at the WCLC, we announced updated interim results from our clinical trials, including the STARTRK-2 trial, of entrectinib. In this interim analysis (based upon an enrollment cut-off of December 31, 2016 and data cut-off of September 13, 2017):

Entrectinib demonstrated a 78% confirmed objective response rate (ORR; by Investigator; 95% CI: 60.0, 90.7) and a 69% confirmed ORR (by Blinded Independent Central Review, or BICR; 95% CI: 50.0, 83.9) in 32 patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) that harbored ROS1 fusions;
Entrectinib demonstrated compelling durability in these patients, with a median duration of response (mDOR) of 28.6 months (by BICR; 95% CI: 6.8, 34.8; median follow-up of 12.9 months) and a median progression-free survival (mPFS) of 29.6 months (by BICR; 95% CI: 7.7, 36.6; median follow-up of 8.5 months); and
Of the patients evaluated, 11 had CNS metastases at baseline as assessed by investigator, and 83% (5 out of 6; by BICR) of the patients with BICR-confirmed measurable CNS metastases at presentation had confirmed intracranial RECIST responses to treatment with entrectinib.
Entrectinib remained well tolerated, with more than 200 patients treated at the recommended Phase 2 dose, with mostly Grade 1-2 reversible treatment-related adverse events. The program is tracking towards dual NDA submissions in TRK and ROS1 in 2018, if supported by clinical data, with an anticipated U.S. commercial launch in both indications in 2019.

RXDX-105

In September 2017, at the ESMO (Free ESMO Whitepaper) conference, we announced new Phase 1b clinical data on RXDX-105—an investigational, VEGFR-sparing, potent RET inhibitor—in which a preliminary ORR of 75 percent was observed in patients with non-KIF5B-RET fusions, with six of eight patients achieving a confirmed partial response. In contrast, those with KIF5B-RET fusions (14 patients) did not demonstrate RECIST responses. RXDX-105 continued to be well tolerated, with the most common treatment-related adverse events being Grade 1 or 2 and reversible with dose modifications. The most common Grade 3 treatment-related adverse events (> 5%) were rash (10%), hypophosphatemia (7%) and elevated ALT (7%).

RXDX-106

In October 2017, at the AACR (Free AACR Whitepaper) Tumor Immunology and Immunotherapy meeting, we presented new data highlighting the immuno-oncological efficacy of RXDX-106—a novel immunomodulatory agent with potent anti-tumor activity, alone and in combination with checkpoint inhibitors, that in preclinical models has demonstrated immunomodulatory effects in the tumor microenvironment (TME) through TYRO3, AXL, and MER (TAM) receptor tyrosine kinase (RTK) inhibition. The data presented demonstrated immune-mediated, single-agent anti-tumor activity of RXDX-106 in multiple tumor models. The anti-tumor effect was further enhanced by combination therapy with immune checkpoint inhibitors, potentially by reversing immunosuppression of innate immunity in the TME. The data also suggested that RXDX-106 has a novel mechanism of enhancing overall immune function by activating both innate and adaptive immunity, as observed by treatment-mediated changes in relevant cytokine levels and immune cell biomarkers, and regulating cross-talk between immune and cancer cells. These promising early findings support further development of RXDX-106 to potentially treat a wide variety of cancers.

Financing Transaction

In October 2017, the company raised aggregate gross proceeds of $160.0 million after issuing 10.0 million shares of its common stock in an underwritten public offering at a price to the public of $16.00 per share.

Third Quarter 2017 Financial Results

For the third quarter of 2017, net loss was $28.6 million, or $0.51 per share, compared with $23.3 million, or $0.56 per share, for the third quarter of 2016.

Ignyta did not record any revenue for the third quarter of 2017 or for the third quarter of 2016.

Research and development expenses for the third quarter of 2017 were $21.7 million, compared with $16.6 million for the third quarter of 2016. This increase was due to an increase in external clinical development costs and the chemistry, manufacturing and control costs associated with entrectinib and our other product candidates, and increased facilities costs of $1.1 million due to the expansion of our leased facilities space.

General and administrative expenses for the third quarter of 2017 were $6.5 million, compared with $6.1 million for the third quarter of 2016. This increase was due to an increase in our facilities costs, as described above, and an increase in outside services expenses, due to an increase in pre-launch commercial activities, which was partially offset by a reduction in depreciation expense.

At September 30, 2017, we had cash, cash equivalents and available-for-sale securities totaling $144.8 million—which does not include the $150 million of net proceeds from the October financing—and current and long-term debt of $32.0 million. At December 31, 2016, we had cash, cash equivalents and available-for-sale securities totaling $133.0 million and current and long-term debt of $32.0 million.

On November 7, 2017 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that the following presentations by Company management at upcoming investor conferences will be webcast (Press release, ImmunoGen, NOV 7, 2017, View Source [SID1234521679]):

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Jefferies 2017 London Healthcare Conference
November 15, 2017 at 10:40am GMT
Evercore ISI Biopharma Catalyst/Deep Dive Conference
November 30, 2017 at 3:30pm ET
A webcast of each presentation will be accessible through the Investors section of the Company’s website, www.immunogen.com. Following the live webcast, a replay will be available at the same location for approximately two weeks.