Latest ZIOPHARM Oncology Inc (ZIOP) company news
The biotech sector was in focus with regular pipeline updates. While Sarepta Therapeutics, Inc. SRPT and Solid Biosciences SLDB gained on positive data, Anika Therapeutics, Inc. ANIK and Ziopharm Oncology, Inc. ZIOP declined on dismal results.
Recap of Important Stories
Sarepta, Solid Biosciences Surge: The going has been good for the Duchenne muscular dystrophy (DMD) market. Shares of Sarepta surged significantly after the company reported impressive preliminary results from its phase I/IIa gene therapy trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with DMD. The biopsies performed at the end of day 90 showed robust micro-dystrophin expression in muscle measured by all methods and observed in all three patients. The patients enrolled also showed a significant decrease in serum creatine kinase (CK) levels, with a mean reduction of CK of over 87% at Day 60. (Read more:Sarepta's Stock Soars on Encouraging Gene Therapy Results).
Earlier, Solid Biosciences’ shares surged too after the company announced that the FDA has lifted the clinical hold on its phase I/II trial, IGNITE DMD, for its experimental candidate, SGT-001. Notably, SGT-001, Solid Biosciences’ lead candidate, is a microdystrophin gene transfer, which is being evaluated for the treatment of DMD. The clinical hold was lifted as the FDA acknowledged that Solid Biosciences has satisfactorily addressed all the issues raised. (Read more: Solid Surges as FDA Lifts Clinical Hold on DMD Drug).
Alexion Submits BLA for ALXN1210: Alexion Pharmaceuticals, Inc. ALXN announced that the company has submitted the Biologics License Application (BLA) to the FDA for approval of its experimental long-acting C5 complement inhibitor, ALXN1210, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). Since the submission is for a rare disease using a priority review voucher, the BLA is entitled to an eight-month review by the FDA instead of the standard 12-month review. Alexion’s lead drug, Soliris, is already approved for PNH and a tentative approval of ALXN1210 will give a significant boost to the company. ALXN1210 had earlier achieved non-inferiority to Soliris in complement inhibitor treatment-naive patients with PNH based on the co-primary endpoints of transfusion avoidance and normalization of LDH levels in a phase III study wherein ALXN1210 was administered intravenously every eight weeks.
Alexion carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank stocks here.
Anika Plunges on Disappointing Data: Shares of Anika Therapeutics have dropped in after-market trading after the company reported disappointing results from a phase III trial on Cingal, which is being conducted to support registration in the United States. The 16-02 trial compared Cingal, a combination of cross-linked HA and triamcinolone hexacetonide (TH), with TH alone and cross-linked HA in treating patients with osteoarthritis in the knee. The primary endpoint was a comparison of the pain reduction when treated with Cingal compared with only TH at 26 weeks. However, the results did not show any statistical significance even though Cingal achieved greater pain reduction numerically at every time point in the study. Cingal is Anika’s third-generation visco supplement, along with Orthovisc and Monovisc products, to treat pain associated with osteoarthritis of the knee.
Ziopharm Oncology Plunges As FDA Places Clinical Hold on CAR T Therapy: Shares of Ziopharm Oncology declined after the FDA placed a clinical hold on its phase I trial evaluating CD19-specific CAR-T therapies manufactured under point-of-care. The FDA has requested additional information relative to Chemistry, Manufacturing and Controls in support of the investigational new drug (IND) application for the trial. The therapy is being evaluated as an investigational treatment for patients with relapsed or refractory, CD19+ leukemias and lymphomas.
Amgen Got European Nod For Addition of Data to Blincyto: Amgen AMGN announced that the European Commission (EC) has granted a full marketing authorization for Blincyto (blinatumomab) based on encouraging overall survival (OS) data from the phase III study, TOWER, in adult patients with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Results showed that Blincyto demonstrated a superior improvement in median OS over standard-of-care (SOC) chemotherapy. Median OS was 7.7 months for Blincyto versus four months for SOC.
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology Index gained 1.02% over the last five trading sessions. Among the major biotech stocks, Regeneron gained 5.67%. Over the past six months, Celgene has lost 25.80%, while Amgen has gained 6.88%. (See the last biotech stock roundup here: Biotech Stock Roundup: AXON Soars, Amgen Drugs Get FDA, EC Nod)
What's Next in Biotech?
Stay tuned for regulatory updates and pipeline development news.
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NEW YORK, NY / ACCESSWIRE / June 19, 2018 / Arrowhead Pharmaceuticals was seeing big gains in Monday's trading session after announcing that it has fully enrolled its phase 1 study of ARO-AAT, for alpha-1 antitrypsin deficiency (AATD). Shares of ZIOPHARM were dragging over 18% by the close after the FDA put a hold on its phase 1 study to evaluate CD19-specific CAR-T therapies.
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Arrowhead Pharmaceuticals, Inc.
ZIOPHARM Oncology, Inc.
Arrowhead Pharmaceuticals, Inc. shares closed up a little over 24% on about 5.5 million shares traded yesterday. The stock hit a brand new high of $14.00 on Monday after the company said it has fully enrolled its phase 1 study of ARO-AAT, the company's second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency called AATD. The company's CEO and President, Chris Anzalone, Ph.D., stated, "The ARO-AAT Phase 1 study provides the first readout on pharmacologic activity for a therapeutic leveraging our proprietary Targeted RNAi Molecule, or TRiM™, platform. We have escalated above a dose that we believe achieves maximal activity, and all doses to date appear to be generally well-tolerated." Arrowhead Pharmaceuticals is expecting to submit a late-breaking abstract with initial clinical data on ARO-AAT to the Liver Meeting®, the Annual Meeting of the American Association for the Study of Liver Disease (AASLD), this November.
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ZIOPHARM Oncology, Inc. shares closed down a little over 18% yesterday on about 5 million shares traded. The stock came just 6 cents shy of its 52-week low on Monday after announcing that the Food and Drug Administration has asked for more information before signing off on a phase 1 study of the company's "Sleeping Beauty" CAR-T for leukemia and lymphoma. The Phase 1 trial to evaluate CD19-specific CAR-T therapies manufactured under point-of-care has been placed on hold. The company's Chief Executive Officer Laurence Cooper, M.D., Ph.D stated, "We know what is needed to address the hold issues and are looking forward to responding to the agency in a timely manner. We are undertaking cutting-edge science and are on the verge of a paradigm shift based on our approach to very-rapidly manufacture CD19-specific T cells within two days using our non-viral approach to CAR-T therapy based on the Sleeping Beauty platform."
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After disclosing that the Food and Drug Administration is asking for more information before signing off on a phase 1 study of its "Sleeping Beauty" CAR-T for leukemia and lymphoma, shares in Ziopharm Oncology (NASDAQ: ZIOP) were tumbling 17.7% at 3 p.m. EDT today.
A shrinking cash stockpile on Ziopharm's balance sheet has management paring the research and development pipeline to maintain focus on two approaches. Unfortunately, there's uncertainty with both platforms now, following news of a delay in Sleeping Beauty's time line.
IMAGE SOURCE: GETTY IMAGES.
Last month the company said it's pausing plans to pursue a registration-ready trial of its most advanced therapy, Ad-RTS-hIL-12.
An IL-12 control therapy, Ad-RTS-hIL-12 boosts IL-12 expression in the tumor microenvironment to spark an immune-system response. The company's seen some intriguing early-stage responses to this therapy, but management postponed pivotal trials while it tackles "technical requirements related to chemistry and manufacturing control (CMC)."
Today, Ziopharm Oncology said that the FDA has not yet given it a green light to begin its Sleeping Beauty study. Ziopharm hopes to overcome delays in producing CAR-Ts that are associated with existing CAR-T treatments by allowing off-the-shelf production within one to two days. Existing, approved CAR-T's can take two to three weeks to be produced at outside laboratories.
Management didn't go into too much detail regarding what information the FDA is requesting before granting a go-ahead, except that it relates to CMC, but said they "know what is needed to address the hold issues and are looking forward to responding to the agency in a timely manner."
Responding in a timely manner is all well and good, but investors are right to be disappointed by today's disclosure. The uncertainty associated with pivotal trials of Ad-RTS-hIL-12 raised the stakes for Sleeping Beauty, and a trial start for the therapy this year was a major focus of excitement among bullish investors.
It's absolutely possible that this is a minor delay that's resolved quickly, but unfortunately, Ziopharm Oncology doesn't have the luxury of time. Exiting March, it boasted only $51 million in cash on the balance sheet; according to management, that's only enough to fund the company through the second quarter of 2019.
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Ziopharm Oncology Inc. said Monday the U.S. Food and Drug Administration has placed a clinical hold on a Phase 1 trial of a treatment for cancer. The trial sought to evaluate CD19-specific CAR-T therapies made with Sleeping Beauty technology as an investigational treatment for patients with relapsed or refractory, CD19 leukemias and lymphomas, the company said in a statement. Ziopharm specializes in developing therapies to treat a range of cancers, using Sleeping Beauty, a non-viral approach to genetically modify chimeric antigen receptor (CAR(+) ) and T-cell receptor (TCR(+) ) T cells, which target specific antigens in blood cancers and neoantigens in solid tumors.
BOSTON, June 18, 2018 (GLOBE NEWSWIRE) -- Ziopharm Oncology, Inc. (ZIOP) today announced the U.S. Food and Drug Administration (FDA) placed on clinical hold a Phase 1 trial to evaluate CD19-specific CAR-T therapies manufactured under point-of-care and requested additional information in support of the investigational new drug (IND) application for the trial.
Ziopharm, Precigen, Inc., a wholly-owned subsidiary of Intrexon Corporation (XON) and The University of Texas MD Anderson Cancer Center, the IND sponsor, are seeking to conduct a clinical trial to evaluate CAR+ T cells manufactured with Sleeping Beauty technology as an investigational treatment for patients with relapsed or refractory, CD19+ leukemias and lymphomas. CAR+ T cells very-rapidly manufactured with the Sleeping Beauty platform for this third-generation trial are designed to co-express CD19-specific chimeric antigen receptor, or CAR, membrane-bound interleukin 15 and a safety switch. The FDA has requested additional information relative to Chemistry, Manufacturing and Controls. Ziopharm and its partners will address the FDA’s requests, and the initiation of this trial may be delayed.
“We know what is needed to address the hold issues and are looking forward to responding to the agency in a timely manner,” said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. “We are undertaking cutting-edge science and are on the verge of a paradigm shift based on our approach to very-rapidly manufacture CD19-specific T cells within two days using our non-viral approach to CAR-T therapy based on the Sleeping Beauty platform.”
Ziopharm believes this feedback from the FDA does not affect timelines for the Company’s planned trial at the National Cancer Institute using the Sleeping Beauty platform to target solid tumors infusing TCR-modified T cells. The second-generation clinical trial evaluating Sleeping Beauty-manufactured CD19-specific CAR-T cells continues enrolling and infusing patients at MD Anderson.
About Ziopharm Oncology, Inc.
Ziopharm Oncology is a Boston-based biotechnology company focused on the development of next-generation immunotherapies utilizing gene- and cell-based therapies to treat patients with cancer. In partnership with Precigen Inc., a wholly-owned subsidiary of Intrexon Corporation (XON), Ziopharm is focused on the development of two platform technologies designed to deliver safe, effective and scalable cell- and viral-based therapies for the treatment of multiple cancer types: Controlled IL-12 and Sleeping Beauty for genetically modifying T cells. The Company's lead asset, Ad-RTS-hIL-12 plus veledimex, has demonstrated in clinical trials the potential to control interleukin-12, leading to an infiltration of T cells that fight brain cancer. The Company also is advancing therapies using Sleeping Beauty, a non-viral approach to genetically modify chimeric antigen receptor (CAR+) and T-cell receptor (TCR+) T cells, which target specific antigens in blood cancers and neoantigens in solid tumors. Sleeping Beauty uses the Company's so-called point-of-care technology, a shortened manufacturing process which potentially can be developed as a decentralized manufacturing process based in hospitals. These programs are being advanced in collaboration with Precigen and with MD Anderson Cancer Center, the National Cancer Institute and Merck KGaA, Darmstadt, Germany.
This press release contains certain forward-looking statements within the meaning of 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 Company's business and strategic plans, the progress and timing of the development of the Company's research and development programs, including the timing for initiating its first in-human trial using its very rapid manufacturing process and other clinical trials using the Sleeping Beauty technology. All 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: the Company’s ability to address FDA’s clinical hold concerns, the FDA’s review of our other Sleeping Beauty programs, changes in the Company's financial condition and cash needs, funding or other strategic opportunities that become available to the Company, the Company's ability to finance its operations and business initiatives and obtain funding for such activities; whether chimeric antigen receptor T cell (CAR-T) approaches, Ad-RTS-hIL-12, TCR and NK cell-based therapies, or any of other product candidates will advance further in the preclinical research or clinical trial 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-hIL-12, TCR and NK cell-based therapies, and the Company's other therapeutic products it develops will be successfully marketed if approved; the strength and enforceability of the Company's intellectual property rights; competition from other pharmaceutical and biotechnology companies; as well as other risk factors contained in the Company's periodic and interim reports filed from time to time with the Securities and Exchange Commission, including but not limited to, the risks and uncertainties set forth in the "Risk Factors" section of the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2017 and subsequent reports that the Company may file with the Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof, and the Company does 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.