Post by icemandios on Dec 8, 2015 3:57:38 GMT
Two for one in this post. First, this is a very nasty market on many levels. So one must be cautious and nimble, but faint heart never won fair maiden (or her money)....so:
TXMD
TheraputicsMD Announces Positive Top-Line Results from its Phase 3 Rejoice Trial in Postmenopausal Women with Vulvar and Vaginal Atrophy (VVA) Treated with 25 mcg, 10 mcg or 4 mcg of TX-004HR
Conference Call Scheduled for Today at 4:45 p.m. ET to Discuss Results
TherapeuticsMD, Inc. (NYSE MKT: TXMD), an innovative women’s healthcare company, today announced positive top-line results from its pivotal Phase 3 Rejoice Trial of TX-004HR, an investigational, applicator-free vaginal estradiol softgel, for the treatment of moderate to severe dyspareunia (vaginal pain during sexual intercourse), a symptom of vulvar and vaginal atrophy (VVA) due to menopause. VVA is a chronic condition affecting nearly half of postmenopausal women in the United States that can significantly impair their quality of life.
This Smart News Release features multimedia. View the full release here: www.businesswire.com/news/home/20151207006324/en/
TX-004HR was evaluated at 25 mcg, 10 mcg, and 4 mcg doses. The pre-specified four co-primary endpoints were the change from baseline to week 12 in the percentage of vaginal superficial cells, percentage of vaginal parabasal cells, vaginal pH, and in participants’ self-reported severity of dyspareunia as the most bothersome symptom of VVA.
Statistical Significance of Results for Co-Primary Endpoints
(Based on Mean Change from Baseline to Week 12 Compared to Placebo)
25 mcg
10 mcg
4 mcg
Superficial Cells
P < 0.0001
P < 0.0001
P < 0.0001
Parabasal Cells
P < 0.0001
P < 0.0001
P < 0.0001
Vaginal pH
P < 0.0001
P < 0.0001
P < 0.0001
Severity of Dyspareunia
P = 0.0001
P = 0.0001
P = 0.0255
The 25 mcg dose of TX-004HR demonstrated highly statistically significant results at the p ≤ 0.0001 level compared to placebo across all four co-primary endpoints. The 10 mcg dose of TX-004HR demonstrated highly statistically significant results at the p ≤ 0.0001 level compared to placebo across all four co-primary endpoints. The 4 mcg dose of TX-004HR also demonstrated highly statistically significant results at the p ≤ 0.0001 level compared to placebo for the endpoints of vaginal superficial cells, vaginal parabasal cells, and vaginal pH; the change from baseline compared to placebo in the severity of dyspareunia was at the p = 0.0255 level.
Statistical improvement over placebo was also observed for all three doses at the first assessment at week two and sustained through week 12. The pharmacokinetic data for all three doses demonstrated low systemic absorption, supporting the previous Phase 1 trial data. TX-004HR was well tolerated, and there were no clinically significant differences compared to placebo-treated participants with respect to adverse events. There were no drug-related serious adverse events reported. Additional presentations of this data are included at the end of this press release.
“We are extremely encouraged that all three doses of TX-004HR studied in the Rejoice Trial demonstrated positive results,” said TherapeuticsMD CEO Robert G. Finizio. “With efficacy observed as early as two weeks and the convenience of the applicator-free vaginal softgel, we believe that, if approved, TX-004HR has the potential to offer a highly differentiated, new treatment option that meets the needs of the millions of postmenopausal women with VVA who are suffering from pain during sexual intercourse. We look forward to sharing the Rejoice Trial results and to submitting a New Drug Application for TX-004HR to the Food and Drug Administration as soon as the first half of 2016.”
TX-004HR features SYMBODATM technology, which enables partial and complete solubilization of estradiol into medium-chain fatty acid oils often derived from coconut oil. This allows for the production of cohesive, stable formulations and provides content uniformity and accuracy of dosing strengths for TX-004HR.
“Nearly half of all postmenopausal women have VVA, yet few are treated with prescription therapy,” said TherapeuticsMD Chief Medical Officer Sebastian Mirkin, M.D. “The highly statistically significant efficacy results and safety profile from the Rejoice Trial are very promising. We are excited about the potential for TX-004HR to be a new treatment option with low systemic absorption for women with VVA suffering from moderate to severe dyspareunia.”
Safety and efficacy analyses of the Rejoice Trial data are ongoing. TherapeuticsMD plans to submit Rejoice Trial results for presentation at future scientific meetings and for publication in peer reviewed journals.
Rejoice Trial Design
The Rejoice Trial was a randomized, double-blinded, placebo-controlled, multicenter Phase 3 clinical trial designed to evaluate the safety and efficacy of three doses of TX-004HR — 25 mcg, 10 mcg and 4 mcg — compared to placebo for the treatment of moderate to severe dyspareunia in postmenopausal women with VVA. The co-primary efficacy endpoints are change from baseline to week 12 in the percentage of vaginal superficial cells, percentage of vaginal parabasal cells, vaginal pH, and severity of moderate to severe dyspareunia as the most bothersome symptom of VVA. The trial enrolled 764 postmenopausal women (40 to 75 years old) experiencing moderate to severe dyspareunia at approximately 89 sites across the United States and Canada. Trial participants were randomized to receive either TX-004HR at 25 mcg (n=190), 10 mcg (n=191), or 4 mcg (n=191) doses or placebo (n=192) for a total of 12 weeks, all administered once daily for two weeks and then twice weekly (approximately three to four days apart) for ten weeks.
Conference Call and Webcast
TherapeuticsMD will host a conference call today, during which management will discuss the top-line results of the pivotal Phase 3 Rejoice Trial. Details for the call are:
Date: December 7, 2015
Time: 4:45 p.m. ET
Telephone Access (US): (866) 665-9531
Telephone Access (International): (724) 987-6977
Access Code for All Callers: 98389089
Additionally, a live webcast can be accessed on the company’s website, www.therapeuticsmd.com, under the “Investors & Media” section.
About TX-004HR
TX-004HR is an investigational bio-identical 17β-estradiol vaginal drug product candidate being studied for the treatment of moderate to severe dyspareunia, a symptom of VVA, also known as genitourinary syndrome of menopause (GSM), in postmenopausal women. TX-004HR utilizes a unique applicator-free vaginal estradiol softgel capsule technology.
About Vulvar and Vaginal Atrophy (VVA)
An estimated 32 million women in the United States are currently suffering from symptoms of VVA1, and only 2.3 million (7%) are currently being treated with prescription therapy.2,3 The burden of VVA in the United States may increase due to aging of the population.4 Furthermore, due to increasing longevity,4 women may now suffer from VVA or other conditions related to decreased reproductive hormone levels for over one-third of their lives.
About TherapeuticsMD, Inc.
TherapeuticsMD, Inc. is an innovative healthcare company focused on developing and commercializing products exclusively for women. With its SYMBODA™ technology, TherapeuticsMD is developing advanced hormone therapy pharmaceutical products to enable delivery of bio-identical hormones through a variety of dosage forms and administration routes. The company’s clinical development pipeline includes two phase 3 products. The company also manufactures and distributes branded and generic prescription prenatal vitamins as well as over-the-counter vitamins under the vitaMedMD® and BocaGreenMD® brands. More information is available at the following websites: www.therapeuticsmd.com, www.vitamedmd.com and www.bocagreenmd.com.
Forward Looking Statements
This press release by TherapeuticsMD, Inc. may contain forward-looking statements. Forward-looking statements may include, but are not limited to, statements relating to TherapeuticsMD’s objectives, plans and strategies as well as statements, other than historical facts, that address activities, events or developments that the company intends, expects, projects, believes or anticipates will or may occur in the future. These statements are often characterized by terminology such as “believes,” “hopes,” “may,” “anticipates,” “should,” “intends,” “plans,” “will,” “expects,” “estimates,” “projects,” “positioned,” “strategy” and similar expressions and are based on assumptions and assessments made in light of management’s experience and perception of historical trends, current conditions, expected future developments and other factors believed to be appropriate. Forward-looking statements in this press release are made as of the date of this press release, and the company undertakes no duty to update or revise any such statements, whether as a result of new information, future events or otherwise. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties, many of which are outside of the company’s control. Important factors that could cause actual results, developments and business decisions to differ materially from forward-looking statements are described in the sections titled “Risk Factors” in the company’s filings with the Securities and Exchange Commission, including its most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q, as well as reports on Form 8-K, and include the following: the company’s ability to maintain or increase sales of its products; the company’s ability to develop and commercialize its hormone therapy drug candidates and obtain additional financing necessary therefor; the length, cost and uncertain results of the company’s clinical trials; the potential of adverse side effects or other safety risks that could preclude the approval of the company’s hormone therapy drug candidates; the company’s reliance on third parties to conduct its clinical trials, research and development and manufacturing; the availability of reimbursement from government authorities and health insurance companies for the company’s products; the impact of product liability lawsuits; the influence of extensive and costly government regulation; the volatility of the trading price of the company’s common stock and the concentration of power in its stock ownership. PDF copies of the company’s historical press releases and financial tables can be viewed and downloaded at its website: www.therapeuticsmd.com/pressreleases.aspx.
References
1 Kingsberg SA, Wysocki S, Magnus L, et al. Vulvar and vaginal atrophy in postmenopausal women: Findings from the Revive (REal Women’s VIews of Treatment Options for Menopausal Vaginal ChangEs) survey. J Sex Med. 2013;10:1790-1799.
2 Mac Bride MB, Rhodes DJ, Shuster LT. Vulvovaginal atrophy. Mayo Clin Proc. 2010;85:87-94.
3 North American Menopause Society. The role of local vaginal estrogen for treatment of vaginal atrophy in postmenopausal women: 2007 position statement of The North American Menopause Society. Menopause. 2007; l4(3 Pt 1):355-69.
4 US Census Bureau. Age and Sex Composition: 2010. 2011 May. Report No.: C2010BR-03.
View source version on businesswire.com: www.businesswire.com/news/home/20151207006324/en/
TherapeuticsMD
Investors
Dan Cartwright, 561-961-1900
Chief Financial Officer,
Dan.Cartwright@TherapeuticsMD.com
or
Media
Elliot Fox, 212-257-6724
Director, Media & Engagement, WCG
efox@w2ogroup.com
Copyright Business Wire 2015
Source: Business Wire (Dec 7, 2015 16:10:00 EST)
News by QuoteMedia
www.quotemedia.com
And then there is this:
ZIOP
ZIOPHARM Announces Presentation of Data From CD19-Specific CAR+ T-Cell Therapy Programs at ASH Annual Meeting
– Results demonstrate survival benefit in long-term follow-up
infusing autologous genetically modified T cells after HSCT–
– Results demonstrate survival benefit in follow-up infusing donor-derived genetically modified T cells after allogeneic HSCT, including haploidentical HSCT –
– Next-generation Sleeping Beauty CD19-specific CAR+ T-cell trial initiated –
BOSTON, Dec. 7, 2015 (GLOBE NEWSWIRE) -- ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, today announced that results from its CD19-specific CAR T-cell therapy programs were presented at the 57th American Society of Hematology (ASH) Annual Meeting in Orlando, Florida. The presentation (Abstract 862), titled "Pre-Emptive Donor Lymphocyte Infusion with CD19-Directed, CAR-Modified T Cells Infused after Allogeneic Hematopoietic Cell Transplantation for Patients with Advanced CD19+ Malignancies" was presented by lead author Partow Kebriaei, M.D., Associate Professor, Department of Stem Cell Transplantation and Cellular Therapy, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, in an oral session, and is available at www.ziopharm.com. The investigational therapies infusing T cells genetically modified to express a CD19-specific chimeric antigen receptor (CAR), described in the presentation, were exclusively licensed to ZIOPHARM and its collaboration partner, Intrexon Corporation (NYSE:XON), through an agreement with MD Anderson.
"This study, which was initially designed to test the safety and tolerability of the Sleeping Beauty platform, a system for the non-viral genetic modification of cells, appears to show a survival benefit with a doubling of overall survival compared to historical controls," said Dr. Richard Champlin, Chair, Department of Stem Cell Transplantation and Cellular Therapy at MD Anderson. "This is the measure of clinical success for a post-transplant therapy, where the contribution of either treatment is difficult to discern in early follow-up. Also promising was the absence of GvHD among HLA-mismatched CAR+ T-cell recipients, even at large doses, which supports infusing third-party T cells."
In the two separate trials described in Dr. Kebriaei's presentation, patient-derived (autologous) or donor-derived (allogeneic) CAR-modified T cells targeting CD19 were administered to recipients with advanced CD19+ malignancies after hematopoietic stem-cell transplantation (HSCT). The studies employed the Sleeping Beauty system, a cost-effective non-viral two plasmid electroporation method to stably express CAR in T cells.
Seven patients with advanced non-Hodgkin lymphoma (NHL) were treated with autologous cells, all of whom remained alive and six of whom remained in complete remission at a median 25.5 months of follow-up translating to a three-year progression free survival (PFS) of 83% and a three-year overall survival (OS) of 100%.
Nineteen patients with advanced acute lymphoblastic leukemia and NHL were treated with allogeneic T cells. One-year PFS among these patients was 53% and one-year OS was 63%. Among eight patients who received donor-derived T cells after haploidentical HSCT, all remained alive and six remained in complete remission after a median of 5.2 months of follow up, translating to a one-year PFS of 75% and OS of 100%.
No infusion related or late toxicity was observed in any recipients. A mild elevation in cytokines was observed, without cytokine storm. Autologous and allogeneic cells survived an average of 201 and 51 days, respectively.
A graft-versus-host disease (GvHD) rate of 11% was observed among recipients receiving donor-derived CAR+ T cells which did not differ from controls. Because of this low rate of GvHD, a complication associated with allogeneic HSCT, administration of up to 108/m2 genetically modified haploidentical T cells was possible. This lays the groundwork for infusing HLA-mismatched CAR+ T cells as an off-the-shelf broadly-accessible therapeutic.
"Sleeping Beauty, which is among the fastest technologies to go from bench to bedside, was a critical innovation of this trial," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM. "In addition to demonstrating safety and durable responses, it provides us a pathway for testing new CARs, co-expressing CAR with other molecules, and to do so at relatively low cost in the setting of both patient-derived and third-party derived products. We look forward to the new trial initiated at MD Anderson using Sleeping Beauty-modified T cells expressing a next-generation CD19-specific CAR in active CD19+ lymphoid malignancies."
Results from the haploidentical population were also presented separately in an oral presentation, titled "Donor-derived CD19-specific CAR+ T-cell therapy after haploidentical hematopoietic stem-cell transplantation," by Dr. Cooper, at the Haplo2015 Symposium, December 3, 2015, in Orlando, Florida. The presentation is available at www.ziopharm.com.
About ZIOPHARM Oncology, Inc.:
ZIOPHARM Oncology is a Boston, Massachusetts-based biotechnology company employing novel gene expression, control and cell technologies to deliver safe, effective and scalable cell- and viral-based therapies for the treatment of cancer. The Company's synthetic immuno-oncology programs, in collaboration with Intrexon Corporation (NYSE:XON) and the MD Anderson Cancer Center, include chimeric antigen receptor T cell (CAR-T) and other adoptive cell based approaches that use non-viral gene transfer methods for broad scalability. The Company is advancing programs in multiple stages of development together with Intrexon Corporation's RheoSwitch Therapeutic System® technology, a switch to turn on and off, and precisely modulate, gene expression in order to improve therapeutic index. The Company's pipeline includes a number of cell-based therapeutics in both clinical and preclinical testing which are focused on hematologic and solid tumor malignancies.
Forward-Looking Safe-Harbor Statement:
This press release contains certain forward-looking information about ZIOPHARM Oncology, Inc. that is intended to be covered by the safe harbor for "forward-looking statements" provided by the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding the progress, timing and results of preclinical and clinical trials involving the Company's drug candidates, and the progress of the Company's research and development programs. All of such statements are subject to certain risks and uncertainties, many of which are difficult to predict and generally beyond the control of the Company, that could cause actual results to differ materially from those expressed in, or implied by, the forward-looking statements. These risks and uncertainties include, but are not limited to: whether chimeric antigen receptor T cell (CAR T) approaches, Ad-RTS-IL-12, TCR and NK cell-based therapies, or any of our other therapeutic candidates will advance further in the pre-clinical or clinical trials process and whether and when, if at all, they will receive final approval from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies and for which indications; whether chimeric antigen receptor T cell (CAR T) approaches, Ad-RTS-IL-12, TCR and NK cell-based therapies, and our other therapeutic products will be successfully marketed if approved; the strength and enforceability of our intellectual property rights; competition from other pharmaceutical and biotechnology companies; and the other risk factors contained in our periodic and interim SEC reports filed from time to time with the Securities and Exchange Commission, including but not limited to, our Annual Report on Form 10-K for the fiscal year ended December 31, 2014, and our Quarterly Reports on Form 10Q for the quarters ended March 31, 2015, June 30, 2015 and September 30, 2015. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof, and we do not undertake any obligation to revise and disseminate forward-looking statements to reflect events or circumstances after the date hereof, or to reflect the occurrence of or non-occurrence of any events.
Trademarks
RheoSwitch Therapeutic System® (RTS®) technology is a registered trademark of Intrexon Corporation.
CONTACT: Lori Ann Occhiogrosso
ZIOPHARM Oncology, Inc.
617-259-1987
locchiogrosso@ziopharm.com
David Pitts
Argot Partners
212-600-1902
david@argotpartners.com
Source: GlobeNewswire (Dec 7, 2015 17:15:23 EST)
News by QuoteMedia
www.quotemedia.com
TXMD
TheraputicsMD Announces Positive Top-Line Results from its Phase 3 Rejoice Trial in Postmenopausal Women with Vulvar and Vaginal Atrophy (VVA) Treated with 25 mcg, 10 mcg or 4 mcg of TX-004HR
Conference Call Scheduled for Today at 4:45 p.m. ET to Discuss Results
TherapeuticsMD, Inc. (NYSE MKT: TXMD), an innovative women’s healthcare company, today announced positive top-line results from its pivotal Phase 3 Rejoice Trial of TX-004HR, an investigational, applicator-free vaginal estradiol softgel, for the treatment of moderate to severe dyspareunia (vaginal pain during sexual intercourse), a symptom of vulvar and vaginal atrophy (VVA) due to menopause. VVA is a chronic condition affecting nearly half of postmenopausal women in the United States that can significantly impair their quality of life.
This Smart News Release features multimedia. View the full release here: www.businesswire.com/news/home/20151207006324/en/
TX-004HR was evaluated at 25 mcg, 10 mcg, and 4 mcg doses. The pre-specified four co-primary endpoints were the change from baseline to week 12 in the percentage of vaginal superficial cells, percentage of vaginal parabasal cells, vaginal pH, and in participants’ self-reported severity of dyspareunia as the most bothersome symptom of VVA.
Statistical Significance of Results for Co-Primary Endpoints
(Based on Mean Change from Baseline to Week 12 Compared to Placebo)
25 mcg
10 mcg
4 mcg
Superficial Cells
P < 0.0001
P < 0.0001
P < 0.0001
Parabasal Cells
P < 0.0001
P < 0.0001
P < 0.0001
Vaginal pH
P < 0.0001
P < 0.0001
P < 0.0001
Severity of Dyspareunia
P = 0.0001
P = 0.0001
P = 0.0255
The 25 mcg dose of TX-004HR demonstrated highly statistically significant results at the p ≤ 0.0001 level compared to placebo across all four co-primary endpoints. The 10 mcg dose of TX-004HR demonstrated highly statistically significant results at the p ≤ 0.0001 level compared to placebo across all four co-primary endpoints. The 4 mcg dose of TX-004HR also demonstrated highly statistically significant results at the p ≤ 0.0001 level compared to placebo for the endpoints of vaginal superficial cells, vaginal parabasal cells, and vaginal pH; the change from baseline compared to placebo in the severity of dyspareunia was at the p = 0.0255 level.
Statistical improvement over placebo was also observed for all three doses at the first assessment at week two and sustained through week 12. The pharmacokinetic data for all three doses demonstrated low systemic absorption, supporting the previous Phase 1 trial data. TX-004HR was well tolerated, and there were no clinically significant differences compared to placebo-treated participants with respect to adverse events. There were no drug-related serious adverse events reported. Additional presentations of this data are included at the end of this press release.
“We are extremely encouraged that all three doses of TX-004HR studied in the Rejoice Trial demonstrated positive results,” said TherapeuticsMD CEO Robert G. Finizio. “With efficacy observed as early as two weeks and the convenience of the applicator-free vaginal softgel, we believe that, if approved, TX-004HR has the potential to offer a highly differentiated, new treatment option that meets the needs of the millions of postmenopausal women with VVA who are suffering from pain during sexual intercourse. We look forward to sharing the Rejoice Trial results and to submitting a New Drug Application for TX-004HR to the Food and Drug Administration as soon as the first half of 2016.”
TX-004HR features SYMBODATM technology, which enables partial and complete solubilization of estradiol into medium-chain fatty acid oils often derived from coconut oil. This allows for the production of cohesive, stable formulations and provides content uniformity and accuracy of dosing strengths for TX-004HR.
“Nearly half of all postmenopausal women have VVA, yet few are treated with prescription therapy,” said TherapeuticsMD Chief Medical Officer Sebastian Mirkin, M.D. “The highly statistically significant efficacy results and safety profile from the Rejoice Trial are very promising. We are excited about the potential for TX-004HR to be a new treatment option with low systemic absorption for women with VVA suffering from moderate to severe dyspareunia.”
Safety and efficacy analyses of the Rejoice Trial data are ongoing. TherapeuticsMD plans to submit Rejoice Trial results for presentation at future scientific meetings and for publication in peer reviewed journals.
Rejoice Trial Design
The Rejoice Trial was a randomized, double-blinded, placebo-controlled, multicenter Phase 3 clinical trial designed to evaluate the safety and efficacy of three doses of TX-004HR — 25 mcg, 10 mcg and 4 mcg — compared to placebo for the treatment of moderate to severe dyspareunia in postmenopausal women with VVA. The co-primary efficacy endpoints are change from baseline to week 12 in the percentage of vaginal superficial cells, percentage of vaginal parabasal cells, vaginal pH, and severity of moderate to severe dyspareunia as the most bothersome symptom of VVA. The trial enrolled 764 postmenopausal women (40 to 75 years old) experiencing moderate to severe dyspareunia at approximately 89 sites across the United States and Canada. Trial participants were randomized to receive either TX-004HR at 25 mcg (n=190), 10 mcg (n=191), or 4 mcg (n=191) doses or placebo (n=192) for a total of 12 weeks, all administered once daily for two weeks and then twice weekly (approximately three to four days apart) for ten weeks.
Conference Call and Webcast
TherapeuticsMD will host a conference call today, during which management will discuss the top-line results of the pivotal Phase 3 Rejoice Trial. Details for the call are:
Date: December 7, 2015
Time: 4:45 p.m. ET
Telephone Access (US): (866) 665-9531
Telephone Access (International): (724) 987-6977
Access Code for All Callers: 98389089
Additionally, a live webcast can be accessed on the company’s website, www.therapeuticsmd.com, under the “Investors & Media” section.
About TX-004HR
TX-004HR is an investigational bio-identical 17β-estradiol vaginal drug product candidate being studied for the treatment of moderate to severe dyspareunia, a symptom of VVA, also known as genitourinary syndrome of menopause (GSM), in postmenopausal women. TX-004HR utilizes a unique applicator-free vaginal estradiol softgel capsule technology.
About Vulvar and Vaginal Atrophy (VVA)
An estimated 32 million women in the United States are currently suffering from symptoms of VVA1, and only 2.3 million (7%) are currently being treated with prescription therapy.2,3 The burden of VVA in the United States may increase due to aging of the population.4 Furthermore, due to increasing longevity,4 women may now suffer from VVA or other conditions related to decreased reproductive hormone levels for over one-third of their lives.
About TherapeuticsMD, Inc.
TherapeuticsMD, Inc. is an innovative healthcare company focused on developing and commercializing products exclusively for women. With its SYMBODA™ technology, TherapeuticsMD is developing advanced hormone therapy pharmaceutical products to enable delivery of bio-identical hormones through a variety of dosage forms and administration routes. The company’s clinical development pipeline includes two phase 3 products. The company also manufactures and distributes branded and generic prescription prenatal vitamins as well as over-the-counter vitamins under the vitaMedMD® and BocaGreenMD® brands. More information is available at the following websites: www.therapeuticsmd.com, www.vitamedmd.com and www.bocagreenmd.com.
Forward Looking Statements
This press release by TherapeuticsMD, Inc. may contain forward-looking statements. Forward-looking statements may include, but are not limited to, statements relating to TherapeuticsMD’s objectives, plans and strategies as well as statements, other than historical facts, that address activities, events or developments that the company intends, expects, projects, believes or anticipates will or may occur in the future. These statements are often characterized by terminology such as “believes,” “hopes,” “may,” “anticipates,” “should,” “intends,” “plans,” “will,” “expects,” “estimates,” “projects,” “positioned,” “strategy” and similar expressions and are based on assumptions and assessments made in light of management’s experience and perception of historical trends, current conditions, expected future developments and other factors believed to be appropriate. Forward-looking statements in this press release are made as of the date of this press release, and the company undertakes no duty to update or revise any such statements, whether as a result of new information, future events or otherwise. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties, many of which are outside of the company’s control. Important factors that could cause actual results, developments and business decisions to differ materially from forward-looking statements are described in the sections titled “Risk Factors” in the company’s filings with the Securities and Exchange Commission, including its most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q, as well as reports on Form 8-K, and include the following: the company’s ability to maintain or increase sales of its products; the company’s ability to develop and commercialize its hormone therapy drug candidates and obtain additional financing necessary therefor; the length, cost and uncertain results of the company’s clinical trials; the potential of adverse side effects or other safety risks that could preclude the approval of the company’s hormone therapy drug candidates; the company’s reliance on third parties to conduct its clinical trials, research and development and manufacturing; the availability of reimbursement from government authorities and health insurance companies for the company’s products; the impact of product liability lawsuits; the influence of extensive and costly government regulation; the volatility of the trading price of the company’s common stock and the concentration of power in its stock ownership. PDF copies of the company’s historical press releases and financial tables can be viewed and downloaded at its website: www.therapeuticsmd.com/pressreleases.aspx.
References
1 Kingsberg SA, Wysocki S, Magnus L, et al. Vulvar and vaginal atrophy in postmenopausal women: Findings from the Revive (REal Women’s VIews of Treatment Options for Menopausal Vaginal ChangEs) survey. J Sex Med. 2013;10:1790-1799.
2 Mac Bride MB, Rhodes DJ, Shuster LT. Vulvovaginal atrophy. Mayo Clin Proc. 2010;85:87-94.
3 North American Menopause Society. The role of local vaginal estrogen for treatment of vaginal atrophy in postmenopausal women: 2007 position statement of The North American Menopause Society. Menopause. 2007; l4(3 Pt 1):355-69.
4 US Census Bureau. Age and Sex Composition: 2010. 2011 May. Report No.: C2010BR-03.
View source version on businesswire.com: www.businesswire.com/news/home/20151207006324/en/
TherapeuticsMD
Investors
Dan Cartwright, 561-961-1900
Chief Financial Officer,
Dan.Cartwright@TherapeuticsMD.com
or
Media
Elliot Fox, 212-257-6724
Director, Media & Engagement, WCG
efox@w2ogroup.com
Copyright Business Wire 2015
Source: Business Wire (Dec 7, 2015 16:10:00 EST)
News by QuoteMedia
www.quotemedia.com
And then there is this:
ZIOP
ZIOPHARM Announces Presentation of Data From CD19-Specific CAR+ T-Cell Therapy Programs at ASH Annual Meeting
– Results demonstrate survival benefit in long-term follow-up
infusing autologous genetically modified T cells after HSCT–
– Results demonstrate survival benefit in follow-up infusing donor-derived genetically modified T cells after allogeneic HSCT, including haploidentical HSCT –
– Next-generation Sleeping Beauty CD19-specific CAR+ T-cell trial initiated –
BOSTON, Dec. 7, 2015 (GLOBE NEWSWIRE) -- ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, today announced that results from its CD19-specific CAR T-cell therapy programs were presented at the 57th American Society of Hematology (ASH) Annual Meeting in Orlando, Florida. The presentation (Abstract 862), titled "Pre-Emptive Donor Lymphocyte Infusion with CD19-Directed, CAR-Modified T Cells Infused after Allogeneic Hematopoietic Cell Transplantation for Patients with Advanced CD19+ Malignancies" was presented by lead author Partow Kebriaei, M.D., Associate Professor, Department of Stem Cell Transplantation and Cellular Therapy, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, in an oral session, and is available at www.ziopharm.com. The investigational therapies infusing T cells genetically modified to express a CD19-specific chimeric antigen receptor (CAR), described in the presentation, were exclusively licensed to ZIOPHARM and its collaboration partner, Intrexon Corporation (NYSE:XON), through an agreement with MD Anderson.
"This study, which was initially designed to test the safety and tolerability of the Sleeping Beauty platform, a system for the non-viral genetic modification of cells, appears to show a survival benefit with a doubling of overall survival compared to historical controls," said Dr. Richard Champlin, Chair, Department of Stem Cell Transplantation and Cellular Therapy at MD Anderson. "This is the measure of clinical success for a post-transplant therapy, where the contribution of either treatment is difficult to discern in early follow-up. Also promising was the absence of GvHD among HLA-mismatched CAR+ T-cell recipients, even at large doses, which supports infusing third-party T cells."
In the two separate trials described in Dr. Kebriaei's presentation, patient-derived (autologous) or donor-derived (allogeneic) CAR-modified T cells targeting CD19 were administered to recipients with advanced CD19+ malignancies after hematopoietic stem-cell transplantation (HSCT). The studies employed the Sleeping Beauty system, a cost-effective non-viral two plasmid electroporation method to stably express CAR in T cells.
Seven patients with advanced non-Hodgkin lymphoma (NHL) were treated with autologous cells, all of whom remained alive and six of whom remained in complete remission at a median 25.5 months of follow-up translating to a three-year progression free survival (PFS) of 83% and a three-year overall survival (OS) of 100%.
Nineteen patients with advanced acute lymphoblastic leukemia and NHL were treated with allogeneic T cells. One-year PFS among these patients was 53% and one-year OS was 63%. Among eight patients who received donor-derived T cells after haploidentical HSCT, all remained alive and six remained in complete remission after a median of 5.2 months of follow up, translating to a one-year PFS of 75% and OS of 100%.
No infusion related or late toxicity was observed in any recipients. A mild elevation in cytokines was observed, without cytokine storm. Autologous and allogeneic cells survived an average of 201 and 51 days, respectively.
A graft-versus-host disease (GvHD) rate of 11% was observed among recipients receiving donor-derived CAR+ T cells which did not differ from controls. Because of this low rate of GvHD, a complication associated with allogeneic HSCT, administration of up to 108/m2 genetically modified haploidentical T cells was possible. This lays the groundwork for infusing HLA-mismatched CAR+ T cells as an off-the-shelf broadly-accessible therapeutic.
"Sleeping Beauty, which is among the fastest technologies to go from bench to bedside, was a critical innovation of this trial," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM. "In addition to demonstrating safety and durable responses, it provides us a pathway for testing new CARs, co-expressing CAR with other molecules, and to do so at relatively low cost in the setting of both patient-derived and third-party derived products. We look forward to the new trial initiated at MD Anderson using Sleeping Beauty-modified T cells expressing a next-generation CD19-specific CAR in active CD19+ lymphoid malignancies."
Results from the haploidentical population were also presented separately in an oral presentation, titled "Donor-derived CD19-specific CAR+ T-cell therapy after haploidentical hematopoietic stem-cell transplantation," by Dr. Cooper, at the Haplo2015 Symposium, December 3, 2015, in Orlando, Florida. The presentation is available at www.ziopharm.com.
About ZIOPHARM Oncology, Inc.:
ZIOPHARM Oncology is a Boston, Massachusetts-based biotechnology company employing novel gene expression, control and cell technologies to deliver safe, effective and scalable cell- and viral-based therapies for the treatment of cancer. The Company's synthetic immuno-oncology programs, in collaboration with Intrexon Corporation (NYSE:XON) and the MD Anderson Cancer Center, include chimeric antigen receptor T cell (CAR-T) and other adoptive cell based approaches that use non-viral gene transfer methods for broad scalability. The Company is advancing programs in multiple stages of development together with Intrexon Corporation's RheoSwitch Therapeutic System® technology, a switch to turn on and off, and precisely modulate, gene expression in order to improve therapeutic index. The Company's pipeline includes a number of cell-based therapeutics in both clinical and preclinical testing which are focused on hematologic and solid tumor malignancies.
Forward-Looking Safe-Harbor Statement:
This press release contains certain forward-looking information about ZIOPHARM Oncology, Inc. that is intended to be covered by the safe harbor for "forward-looking statements" provided by the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding the progress, timing and results of preclinical and clinical trials involving the Company's drug candidates, and the progress of the Company's research and development programs. All of such statements are subject to certain risks and uncertainties, many of which are difficult to predict and generally beyond the control of the Company, that could cause actual results to differ materially from those expressed in, or implied by, the forward-looking statements. These risks and uncertainties include, but are not limited to: whether chimeric antigen receptor T cell (CAR T) approaches, Ad-RTS-IL-12, TCR and NK cell-based therapies, or any of our other therapeutic candidates will advance further in the pre-clinical or clinical trials process and whether and when, if at all, they will receive final approval from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies and for which indications; whether chimeric antigen receptor T cell (CAR T) approaches, Ad-RTS-IL-12, TCR and NK cell-based therapies, and our other therapeutic products will be successfully marketed if approved; the strength and enforceability of our intellectual property rights; competition from other pharmaceutical and biotechnology companies; and the other risk factors contained in our periodic and interim SEC reports filed from time to time with the Securities and Exchange Commission, including but not limited to, our Annual Report on Form 10-K for the fiscal year ended December 31, 2014, and our Quarterly Reports on Form 10Q for the quarters ended March 31, 2015, June 30, 2015 and September 30, 2015. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof, and we do not undertake any obligation to revise and disseminate forward-looking statements to reflect events or circumstances after the date hereof, or to reflect the occurrence of or non-occurrence of any events.
Trademarks
RheoSwitch Therapeutic System® (RTS®) technology is a registered trademark of Intrexon Corporation.
CONTACT: Lori Ann Occhiogrosso
ZIOPHARM Oncology, Inc.
617-259-1987
locchiogrosso@ziopharm.com
David Pitts
Argot Partners
212-600-1902
david@argotpartners.com
Source: GlobeNewswire (Dec 7, 2015 17:15:23 EST)
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