On November 11, 2016 Corvus Pharmaceuticals, Inc. (NASDAQ:CRVS), a clinical-stage biopharmaceutical company focused on the development and commercialization of novel immuno-oncology therapies, reported preliminary clinical safety and efficacy data from the dose-selection phase of its ongoing Phase 1/1b study of CPI-444 as a single agent and in combination with Genentech’s Tecentriq (atezolizumab), a fully humanized monoclonal antibody targeting protein programmed cell death ligand 1 (PD-L1) (Press release, Corvus Pharmaceuticals, NOV 11, 2016, View Source [SID1234516492]). CPI-444 is a selective and potent inhibitor of the adenosine A2A receptor. The data were presented today in a poster session by John Powderly II, M.D., founder and president of the Carolina BioOncology Institute, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 31ST Annual Meeting & Associated Programs in National Harbor, Maryland. The poster can be accessed online here. Schedule your 30 min Free 1stOncology Demo! "Although the data is early, we are seeing encouraging evidence of clinical activity with CPI-444 as a monotherapy and in combination with Tecentriq in patients with advanced refractory cancers," said Richard A. Miller an oncologist and co-founder, president and chief executive officer of Corvus. "We are excited about these preliminary data which show that several patients have achieved stable disease, one of the trial’s primary endpoints, with ongoing responses in cohorts receiving single agent and combination therapy. Tumor regression has been seen in patients who were naïve and refractory to prior treatments with anti-PD-1 or PD-L1 antibodies."
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Initial safety and efficacy data from the first 46 patients enrolled in the dose-selection phase of the Phase 1/1b trial with a median follow up of two months were presented at the conference. All patients had failed all approved therapies for their disease, with a median of four prior treatment regimens (range: 1-5). Fifty-two percent of patients were refractory to prior treatment with anti-PD-1/PD-L1 antibodies. Enrolled patients had the following cancers: non-small cell lung (NSCLC), N=10; triple negative breast (TNBC), N=10; bladder, N=6; renal, N=5; melanoma, N=7; colorectal, N=3; prostate, N=2; and head and neck, N=3. The primary endpoints of the study are response rate and duration of clinical benefit (defined as complete response, partial response or stable disease). Patients are treated until disease progression or evidence of grade 3 or 4 toxicity.
Results presented showed:
Of the 32 patients who reached the first efficacy assessment at two months, 12 have shown stable disease and 20 have shown disease progression. Fourteen patients have not yet reached the two-month assessment. Six of the patients with disease progression remain on treatment based on investigator judgement that there is clinical benefit. Overall, 32 patients continue to receive treatment in the study and 14 have discontinued therapy.
Of the 12 patients with stable disease, several have shown ongoing tumor regression (1-20 percent reduction of the volume of indicator tumor lesions) by CT scan but have not yet reached the criteria for partial response (>30 percent reduction in tumor size per RECIST criteria). Seven of the 12 patients with stable disease received CPI-444 as a single agent.
Of the 10 patients with NSCLC, seven received single agent CPI-444. Five of seven evaluable patients have stable disease at two or more months (three of whom received single agent therapy). Nine patients remain on treatment and one patient has discontinued.
Of the five patients with RENAL CANCER, four received single agent CPI-444. Four patients remain on treatment and three of four evaluable patients have stable disease.
Two of four evaluable patients with BLADDER CANCER showed stable disease at first assessment, both of whom received single agent CPI-444.
Of the ten patients with TNBC, one of seven evaluable patients showed stable disease. Eight patients remain on treatment.
One of four evaluable patients with MELANOMA has stable disease with regression of cutaneous tumor lesions; this patient received single agent CPI-444.
One of two evaluable patients with PROSTATE CANCER treated with combination therapy has stable disease and showed a decrease in prostate-specific antigen (PSA) at 29 weeks; this patient has gained weight and requires significantly less narcotics for pain management.
CPI-444 has been well tolerated to date, with one patient treated with combination therapy experiencing a possibly drug-related serious adverse event. This patient developed autoimmune hemolytic anemia that resolved upon discontinuation of therapy.
Patients receiving CPI-444 100 mg twice daily had sustained, complete blockade of A2A receptor activity in peripheral blood immune cells.
"The data generated in this trial confirms the value of the protocol design and could provide us with an efficient route to future registration trials of CPI-444, particularly as a monotherapy or in combination with anti-PD1/PD-L1 in patients who are refractory to previous treatment with PD1/PD-L1 antibodies," said Ginna G. Laport, M.D., vice president, Clinical Development, at Corvus. "This initial part of the trial identified the optimum dose of CPI-444 that is being used in the second part of the trial, which is currently enrolling patients."
In a separate poster presentation (available online here), Corvus reported on the effects of treatment with CPI-444 on circulating blood immune cells and T-cell clonality. These results indicate:
Single agent treatment with CPI-444 leads to activation of T-cells in peripheral blood as well as increases in memory T-cells, key mediators of T-cell mediated immune responses.
Consistent with this observation, single agent CPI-444 leads to changes in the repertoire of T-cell clones in peripheral blood, consistent with induction of T-cell mediated immune responses. Limited changes in T-cell repertoires were seen in patients who progressed on treatment with either CPI-444 alone or in combination with atezolizumab.
Changes in T-cell repertoires were observed in anti-PD-1/PD-L1 treatment-naïve patients and in patients that were refractory to prior treatment with anti-PD-1/PD-L1 antibodies.
"The biomarker program is generating a wealth of information and we are encouraged by early data that suggest that CPI-444 treatment results in induction of T-cell mediated immune response in patients," said Ian McCaffery Ph.D., vice president, Translational Sciences, at Corvus. "Our goal is to understand the mechanisms of action and changes in patient immune status and these data suggest that we may be able to identify biomarkers to help define and identify the patients most likely to respond to CPI-444."
ABOUT THE PHASE 1/1B TRIAL
The Phase 1/1b trial is designed to examine the activity of CPI-444 as a single agent and in combination with Genentech’s Tecentriq (atezolizumab), an anti-PD-L1 antibody. Patients with non-small cell lung cancer, melanoma, renal cell cancer, triple-negative breast cancer, colorectal cancer, head and neck cancer, bladder cancer and prostate cancer who have failed all standard therapies are eligible.
The first part of the study (dose-selection) included four cohorts of 12 patients each (N=48) – three cohorts treated with single agent CPI-444 (100 mg twice daily for 14 days; 100 mg twice daily for 28 days; 200 mg once daily for 14 days) and one cohort treated with the combination (CPI-444 50 mg or 100 mg twice daily for 14 days combined with Tecentriq). A treatment cycle is 28 days. Based on biomarker analyses showing sustained, complete blockade of the adenosine A2A receptor in peripheral blood lymphocytes, and evidence of immune activation in circulating lymphocytes, an optimum single agent and combination dose of 100 mg twice a day for 28 days was selected. The second part of the study is currently evaluating CPI-444 as a single agent in five disease-specific cohorts, and CPI-444 in combination with Tecentriq in five additional matched disease-specific cohorts. Corvus expects that each of these 10 cohorts will initially enroll 14 patients, but each cohort may be expanded based on efficacy.
Author: [email protected]
10-Q – Quarterly report [Sections 13 or 15(d)]
NantKwest has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, NantKwest, NOV 10, 2016, View Source [SID1234516557]).
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RXi Pharmaceuticals Reports Third Quarter 2016 Financial Results and
Highlights Recent Corporate Developments
On November 10, 2016 RXi Pharmaceuticals Corporation (NASDAQ: RXII), a clinical-stage company developing innovative therapeutics that address significant unmet medical needs, reported its financial results for the third quarter ended September 30, 2016, and provided a business update (Filing, 8-K, RXi Pharmaceuticals, NOV 10, 2016, View Source [SID1234516723]).
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"In the third quarter of 2016, RXi has once again been able to keep its cash burn in line with our projections, while continuing the clinical development programs as planned," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He further added, "In recent weeks we announced an exclusive option to acquire MirImmune, Inc., a small biotech company that uses our proprietary RNAi technology in the field of cell therapy and immune-oncology. We are excited that this potential acquisition would allow RXi to embark on a significant path forward in the development of novel cancer therapeutics. In the past years our Company has shown, through its own clinical programs and external collaborations, that its uniquely structured RNAi compounds are effective in various animal and tissue culture models, as well as in the treatment of hypertrophic scarring, a human clinical model for fibrosis.
While we continue to work toward an acquisition of MirImmune, we expect to provide updates on most of our clinical and consumer health programs before the end of 2016, as well as an outlook on projects, timelines and milestones for the combined company in the first quarter of 2017. We continue to focus on building value for our shareholders by creating novel therapeutics, for a better life for patients."
The Company will host a conference call today at 4:30 p.m. EDT to discuss financial results and provide an update on the Company. The webcast link will be available under the "Investors – Event Calendar" section of the Company’s website, www.rxipharma.com. The event may also be accessed by dialing toll-free in the United States and Canada: +1 (888) 674-0219. International participants may access the event by dialing: +1 (862) 225-5367. An archive of the webcast will be available on the Company’s website approximately two hours after the presentation.
Select Third Quarter 2016 Financial Highlights
Cash Position
At September 30, 2016, the Company had cash, cash equivalents and short-term investments of approximately $4.4 million, compared with $10.6 million at December 31, 2015.
Research and Development Expenses
Research and development expenses for the quarter ended September 30, 2016 were $1.5 million, compared with $1.7 million for the quarter ended September 30, 2015. Research and development expenses decreased from the prior year’s quarter primarily due to manufacturing expenses for the RXI-109 drug product completed in the second half of 2015, offset by increases in manufacturing and clinical trial-related costs for Samcyprone. Research and development expenses further decreased due to a reduction in stock-based compensation expense related to the full vesting of stock options granted in 2012.
General and Administrative Expenses
General and administrative expenses for the quarter ended September 30, 2016 were $0.7 million as compared with $0.8 million for the quarter ended September 30, 2015. The decrease in general and administrative expenses was primarily due to a decrease in stock-based compensation expense related to the full vesting of stock options granted in 2012, offset by an increase in expenses related to the use of outside professional services due to the Company’s increased business development activities in line with its key corporate initiatives.
Net Loss
Net loss for the quarter ended September 30, 2016 was $2.2 million, compared with $2.5 million for the quarter ended September 30, 2015. Net loss decreased from the prior year’s quarter primarily due to the change in research and development expenses, as discussed above.
Select Business and Corporate Highlights
RXi has developed a robust self-delivering RNAi (sd-rxRNA) therapeutic platform where drug-like and delivery properties are built directly into the compound itself. There is no need for delivery vehicles for local applications and our self-delivering platform has been shown to be compatible with various systemic systems. Due to the fact these compounds can be designed to selectively block the expression of any target in the genome, our technology is applicable to a broad spectrum of therapeutic areas.
In March 2015, MirImmune, Inc., a privately-held company focused on the development of next generation immunotherapies for the treatment of cancer, entered into an exclusive license agreement for use of RXi’s sd-rxRNA technology in developing innovative cell-based cancer immunotherapies. Since that time, MirImmune’s progress in cell therapy using RXi’s technology has formed a strong foundation for therapeutic development in the immuno-oncology space.
Last month, RXi entered into an exclusive option to acquire MirImmune, Inc., in consideration for a number of shares equal to 19.99% of the then outstanding shares of common stock of RXi, plus additional potential consideration contingent on MirImmune reaching certain milestones.
Upon RXi’s exercise of its option to acquire MirImmune, the acquisition would not only allow the Company to create value for our shareholders, it more importantly sets the stage for what could potentially be a transformational change in the way we treat patients with various malignancies.
The Company’s goal, through internal research and external partnerships, is to develop more tolerable treatments resulting in better quality of life and extended survival for patients. This approach is a key first step into the field of cell-based therapies, where sd-rxRNA has numerous advantages over other RNAi technologies.
Provectus Biopharmaceuticals Reports Third Quarter 2016 Financial Results
On November 10, 2016 Provectus Biopharmaceuticals, Inc. (OTCQB:PVCT, www.provectusbio.com), a clinical-stage oncology and dermatology biopharmaceutical company ("Provectus" or the "Company"), reported its financial results for the quarter ended September 30, 2016 (Press release, Provectus Pharmaceuticals, NOV 10, 2016, https://www.pvct.com/pressrelease.html?article=20161110.1 [SID1234516712]).
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Third Quarter Results and Balance Sheet Highlights
Our cash and cash equivalents were $5,178,076 at September 30, 2016, compared with $4,891,313 at June 30, 2016. As of December 31, 2015, cash and equivalents were $14,178,902.
Shareholders’ equity at September 30, 2016 was $5,309,712. This compares to shareholders’ equity of $9,140,166 at June 30, 2016, and $16,316,941 as of December 31, 2015.
For additional information regarding Provectus’ results of operations and financial condition for the third quarter ended September 30, 2016, please see Provectus’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 9, 2016.
Management will host its 2016 third quarter business update conference call on Monday, November 14, 2016 at 4 pm Eastern Standard Time. Management will provide a business update on PV-10 and PH-10 to the investment community and answer questions from investors.
Those who wish to participate in the conference call may telephone 877-407-4019 from the U.S. International callers may telephone 201-689-8337 approximately fifteen minutes before the call. A webcast will also be available at www.provectusbio.com.
A digital replay will be available by telephone approximately two hours after the completion of the call until March 4, 2017 and may be accessed by dialing 877-660-6853 from the U.S. or 201-612-7415 for international callers, and using the Conference ID # 13648197.
ProNAi Therapeutics Reports Third Quarter 2016 Results
On November 10, 2016 ProNAi Therapeutics, Inc. (NASDAQ: DNAI), a clinical stage drug development company focused on advancing targeted therapeutics for the treatment of patients with cancer, reported its financial and operational results for the third quarter of 2016 (Press release, ProNAi Therapeutics, NOV 10, 2016, View Source [SID1234516692]).
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"Over the past few months we’ve successfully re-established ProNAi as a clinical stage oncology company advancing promising drug candidates that target the DNA Damage Response (DDR) network. This is a particularly compelling target space in oncology, highlighted by growing enthusiasm for the PARP inhibitors, which also inhibit a component of the DDR network," said Dr. Nick Glover, President and CEO of ProNAi Therapeutics. "Our candidates seek to leverage the DDR network beyond PARP with ‘next generation’ DDR therapeutics targeting Chk1 and Cdc7, and we plan to conduct broad clinical development programs for these assets supported by robust research with the goal of efficiently determining their potential utility for the treatment of a variety of oncology indications."
During the quarter, ProNAi reported it had licensed the exclusive worldwide rights to PNT737, a highly selective, orally available, small molecule inhibitor of Checkpoint kinase 1 (Chk1). PNT737 is being investigated in two Phase 1 clinical trials being conducted at the Royal Marsden NHS Foundation Trust and other centers in the United Kingdom. ProNAi anticipates expanding on the current clinical program underway for PNT737, including into the United States, with the expectation of filing an Investigational New Drug application in the second half of 2017.
To support broader studies in well-defined patient populations, ProNAi plans to conduct research designed to explore markers of sensitivity to PNT737 that may facilitate patient selection and to identify additional therapeutic combination opportunities. A possible development path for PNT737 is the treatment of tumors carrying mutations in genes known to contribute to DNA damage and genomic instability – a key hallmark of cancer. The significant and persistent DNA damage caused by these mutations, coupled with Chk1 inhibition, may result in death of the cancer cells, a synergistic effect referred to as ‘synthetic lethality’. Similarly, excessive DNA damage can be induced with other DDR targeting agents such as PARP inhibitors, as well as certain chemotherapies or radiation, highlighting the potential for synergies between these modalities and Chk1 inhibition.
ProNAi is also advancing PNT141, a Cdc7 inhibitor that regulates DNA replication and the DDR network in a different, potentially complementary way to PNT737. Inhibiting both Chk1 and Cdc7 simultaneously may be advantageous and presents the potential for novel combination strategies for PNT737 and PNT141.
Third Quarter 2016 Financial Results (all amounts reported in U.S. currency)
Research and development expenses increased to $12.3 million for the three months ended September 30, 2016 from $8.3 million for the three months ended September 30, 2015. Research and development expenses increased to $28.1 million for the nine months ended September 30, 2016 from $18.3 million for the nine months ended September 30, 2015. These increases in 2016 were primarily due to a $7.0 million upfront fee due to CRT Pioneer Fund LP (CPF) for the exclusive license of PNT737, the recognition of a $2.0 million fee that will be due upon the successful transfer of two ongoing clinical trials in accordance with the license agreement and a $0.9 million upfront fee paid to Carna Biosciences, Inc. for the exclusive license of PNT141. The remaining increase was attributable to increased personnel-related costs and a non-recurring $2.4 million restructuring charge related to estimated close-out expenses for PNT2258. These were partially offset by a decrease in third-party manufacturing and clinical trial costs.
General and administrative expenses increased to $3.0 million for the three months ended September 30, 2016 from $2.7 million for the three months ended September 30, 2015. General and administrative expenses increased to $10.8 million for the nine months ended September 30, 2016 from $6.1 million for the nine months ended September 30, 2015. These increases in 2016 were primarily due to increased personnel-related costs and fees incurred in support of activities as a public company and corporate growth, costs pertaining to business development activities and a $0.4 million non-recurring restructuring charge.
Total operating expenses for the three months ended September 30, 2016 were $15.3 million compared to $11.0 million for the three months ended September 30, 2015. Total operating expenses for the nine months ended September 30, 2016 were $38.8 million compared to $24.4 million for the nine months ended September 30, 2015. Total operating expenses included non-cash stock based compensation of $1.3 million and $4.0 million for the three and nine months ended September 30, 2016 and of $1.3 and $1.9 for the three and nine months ended September 30, 2015, respectively.
For the three months ended September 30, 2016, ProNAi incurred a net loss of $15.2 million compared to a net loss of $18.5 million for the three months ended September 30, 2015. For the nine months ended September 30, 2016, ProNAi incurred a net loss of $38.6 million compared to a net loss of $41.8 million for the nine months ended September 30, 2015. During the three and nine months ended September 30, 2015, net loss included a non-cash charge related to the change in fair value of preferred stock warrants of $7.5 million and $17.4 million.
At September 30, 2016, ProNAi had $122.7 million in cash and cash equivalents compared to $150.2 million in cash and cash equivalents at December 31, 2015. Subsequent to the end of the quarter, ProNAi paid the $7.0 million upfront fee due to CPF for the exclusive license of PNT737.
At September 30, 2016, there were 30,350,560 shares of common stock issued and outstanding and stock options to purchase 6,629,163 shares of common stock issued and outstanding.