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February 15, 2012 Niiki Pharma Breaks New Ground with Novel Cancer Therapy: Pharmaceutical International News

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Wednesday, February 15, 2012, 8:30am EST

A first-in-man cancer therapy in development by Niiki Pharma Inc. shows promise in targeting a new pathway known as GRP78. This target repairs damaged or deconstructed proteins that result from rampant tumor cell growth. Normal healthy cells have low levels of GRP78. Cancer cells require very high levels of GRP78 and become, in effect, "addicted" to GRP78 for their survival. Known as NKP-1339, Niiki Pharma's compound is an entirely new chemical entity that down regulates GRP78, thus shutting down the tumor cell's key survival mechanism.

"There is a growing body of medical literature that identifies GRP78 as a target for cancer therapies", Dr. Angela Ogden, MD, Chief Medical Officer at Niiki Pharma, explained in a recent interview with the Philadelphia Business Journal. "Nobody, other than Niiki, has a drug targeting GRP78. Now companies are starting to look at developing molecules to block it, but those are years away from getting into patients."

Anti-Tumor Activity and Favorable Safety Profile Leads to Phase IIa Clinical Trial

Niiki Pharma recently completed a Phase I trial and has determined the dose for a Phase II trial. The trial was conducted in patients with metastatic cancers that had failed all standard treatments. At the recommended Phase II dose, NKP-1339 treatment is generally well tolerated with manageable side effects.

Summary results of the Phase I trial show that anti-tumor activity, demonstrated by disease stability and/or tumor regression for 12-88+ weeks, was noted in patients with neuroendocrine tumors (NET), non-small cell lung cancer, sarcoma, colorectal and cancer of unknown primary. The effect of NKP-1339 on neuroendocrine tumors is noteworthy as there currently is no effective therapy available for the second most prevalent tumor type of the GI tract.

Promising Synergistic Activity with Other Anti-Cancer Agents

High levels of GRP78 in cancer cells have been correlated with drug resistance in numerous tumor types. Treatment with many anti-cancer agents further induces GRP78, which boosts the tumor cells resistance to the treatment. Anti-cancer agents that induce GRP78 include platinums, taxanes, anthracyclines, anti-metabolites, proteosome inhibitors and kinase inhibitors.

"We are quite pleased with the development of NKP-1339. The early activity that we have seen [NKP-1339 single agent] in neuroendocrine tumors naturally leads us to pursue further development in this orphan indication. We are also very excited about the development of NKP-1339 in combination with standard anticancer agents for several additional tumor types. In parallel to our clinical development, we are developing a companion diagnostic so that NKP-1339 therapy can be targeted to those who will derive the greatest benefit", added Dr. Ogden. NKP-1339 Phase I dose escalation trial results has been submitted for presentation at the American Society of Clinical Oncology (ASCO) 2012.

Read more: http://www.pharmaceutical-int.com/news/niiki-pharma-breaks-new-ground-with-novel-cancer-therapy.html

February 1, 2012 Niiki Pharma Forms Scientific Advisory Board with Leading U.S. Oncologists

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HOBOKEN, N.J. and PHILADELPHIA, Feb. 1, 2012 /PRNewswire/ -- Niiki Pharma Inc. announced today the formation of its Scientific Advisory Board. Daniel Haller M.D. will chair the advisory board, and will be joined by Lee Ellis M.D. and Matt Kulke M.D. The Board will provide clinical and strategic guidance to the company for its two first-in-class clinical stage anti-cancer compounds, NKP-1339 and NKP-2235. As the pipeline continues to mature, the Advisory Board will be expanded.

The initial focus of the board will be Niiki Pharma’s promising lead compound, NKP-1339. NKP- 1339, a small molecule that down-regulates GRP78, has recently completed the dose-escalation portion of its Phase I clinical trial.

"Although we have an increasing number of drugs for a diverse spectrum of diseases, new drugs that may have higher benefit and lower toxicity for our patients are always needed," said Dr. Haller. "This Board welcomes the opportunity to participate in the development of these agents."

Dr. Daniel Haller is internationally renowned in the field of oncology. He is currently Professor of Medicine in the Department of Medicine at the Abramson Cancer Center at the University of Pennsylvania. In addition to his leadership in clinical research throughout the NCI and cancer cooperative group system, Dr. Haller is currently co-chair of the NCI Gastrointestinal Intergroup. He served as Editor-in-Chief of the Journal of Clinical Oncology, the official journal of the American Society of Clinical Oncology (ASCO), was the Associate Editor of the Annals of Internal Medicine and was Editor-in-Chief of PDQ, the National Cancer Institute’s cancer information database.

Dr. Lee Ellis is an internationally recognized surgical oncologist, researcher, and leader in the field of translational oncology. He holds several positions at University of Texas MD Anderson Cancer Center, including Professor of Surgery and Cancer Biology; Director, Metastasis Research Center; Director, Colorectal Cancer Translational Research Program; and Chair, Ad Interim, Department of Cancer Biology. Dr. Ellis is vice-chair of the NCI Colon Cancer Task Force and serves on 8 editorial boards, including Journal of Clinical Oncology, Cancer Research and Clinical Cancer Research. Dr. Ellis has served in leadership roles in major cancer societies including ASCO, American Association for Cancer Research, and the Society for Surgical Oncology.

Dr. Matthew Kulke is a world expert in neuroendocrine (NET) tumors. He is Associate Professor of Medicine at Harvard Medical School and Dana-Farber Cancer Institute, where he leads the Carcinoid and Neuroendocrine Tumor Program. He has a leadership role in the Cancer and Leukemia Group B (CALGB) Gastrointestinal Cancer Committee, chairs the NCI NET Task Force and NCCN NET guidelines committees, and serves on the executive committee of the North American Neuroendocrine Tumor Society. Dr. Kulke has chaired multiple global pivotal studies exploring new therapies for NET.

About NKP-1339

NKP-1339 is a first-in-class small molecule anti-cancer compound that down-regulates GRP78, a known tumor survival and anti-apoptosis factor. In preclinical studies, NKP-1339 has demonstrated activity against multiple tumor types. In the Phase I trial, antitumor activity was seen against multiple cancers, including NET.

About NKP-2235

NKP-2235 is a first-in-class orally available small molecule that targets the endoplasmic reticulum with a unique pattern of cytotoxicity. NKP-2235 IND has been cleared and a Phase I trial will be initiated shortly.

NKP-1339 and NKP-2235 were discovered by Professor Bernhard Keppler, University of Vienna, Austria.

About Niiki Pharma Inc.

Niiki Pharma (www.niikipharma.com) is a development focused oncology company specializing in first-in-class cancer treatments directed at novel tumor targets and related companion diagnostics.

Contacts

For Media Relations: Carolyn Rhinebarger, carolyn@luminovas.com, +1-919-604-4053. For Investor Relations and Partnering: Ali Ardakani, ardakani@niikipharma.com, +1-201-238-2885

January 17, 2012 Niiki Pharma Announces the Completion of Phase I Dose Escalation for First-in-Man Anti-Cancer Agent NKP-1339

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HOBOKEN, N.J. and PHILADELPHIA, Jan. 17, 2012 /PRNewswire/ -- Niiki Pharma Inc. announced today that it has completed the dose escalation portion of the Phase I clinical trial of its lead product, NKP-1339. NKP-1339 is a first-in-class small molecule that down-regulates GRP78, a key tumor survival and anti-apoptosis factor.

The NKP-1339 single agent dose escalation Phase I trial was conducted in patients with advanced solid tumors resistant to multiple, standard therapies. NKP-1339 was administered intravenously once weekly for 3 weeks followed by one week of no treatment. Thirty-four patients were treated, of which 30 were evaluable for dose determination. The NKP-1339 Phase I trial is being led by Dr. Daniel Von Hoff, Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare in partnership with Translational Genomics Research Institute, and Dr. Howard Burris, at the Sarah Cannon Research Institute.

The NKP-1339 dose limiting toxicity was grade 2-3 nausea and grade 1-2 reversible renal insufficiency. At the recommended Phase II dose, NKP-1339 treatment was generally well tolerated and had manageable side effects. The most common drug-related side effects were grade 1 nausea, grade 1-2 vomiting and grade 1-2 fatigue. Infusional fever and chills were noted but prevented with steroid premedication.

"We reached our goal to determine the dose of NKP-1339 to take forward in future trials. Many toxicities commonly associated with other anticancer drugs, such as neutropenia, hepatotoxicity, neuropathy, rash, mucositis, diarrhea and alopecia, have not been seen to date," said Dr. Burris.

Anti-tumor activity, demonstrated by disease stability and/or tumor regression for 12-88+ weeks, was noted in patients with neuroendocrine tumors (NET), non-small cell lung cancer, sarcoma, colorectal and cancer of unknown primary. Three patients continue to receive NKP-1339 therapy on study.

"The preliminary read of NKP-1339 anti-tumor activity in this advanced cancer patient population shows evidence of activity in patients with NET, " commented Dr. Von Hoff.

"We are excited with the prospects for NKP-1339 in NET, an orphan indication which is presently highly underserved. NKP-1339 trials in this indication are in development. In addition the favorable NKP-1339 safety profile supports plans to evaluate NKP-1339 in combination regimens in other tumor types," said Angela Ogden, M.D., Chief Medical Officer at Niiki Pharma.

About NKP-1339

NKP-1339 is a first-in-class small molecule anti-cancer compound. NKP-1339 down-regulates GRP78, a key regulator of mis-folded protein processing and a tumor survival and anti-apoptosis factor. Up-regulation of GRP78 is associated with intrinsic and chemotherapy-induced resistance in many tumor types. In preclinical studies, NKP-1339 has demonstrated activity against multiple tumor types, including those resistant to other anti-cancer agents. NKP-1339 was discovered by Professor Bernhard Keppler, University of Vienna, Austria.

About Niiki Pharma Inc.

Niiki Pharma (www.niikipharma.com) is a development focused oncology company specializing in first-in-class cancer treatments directed at novel tumor targets and related companion diagnostics.

January 13, 2012 Niiki Pharma developing new weapons to battle cancer: Philadelphia Business Journal

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Philadelphia Business Journal
John George, January 13, 2012

Hooshmand Sheshbaradaran had a specific model in mind when he co-founded Niiki Pharma, a Philadelphia biopharmaceutical company whose focus is to develop new strategies for fighting cancer.

He didn't want the cumbersome layers of management found in Big Pharma companies, nor the limited scope of expertise that hampers some smaller biotech firms.

What he created was a semi-virtual company led by a small team of industry veterans. The seven-member staff has experience in all facets of drug development, having previously worked within the oncology divisions of global pharmaceutical companies such as Roche, Johnson & Johnson, Pharmacia, Pfizer and Bristol-Meyers Squibb.

"You need a broad base of experience to develop a drug," Sheshbaradaran said. "There's product licensing, research and development, manufacturing, marketing. Our team is not people who have thought about [getting a product to market], they've actually done it.… I like to say we are doing pharmaceutical company quality work with biotech agility and adding our own Niiki brand of cost-effectiveness."

With a staff of seven augmented by 14 consultants, the company maintains a low overhead and is able to allocate 80 cents of every dollar raised for drug-development expenses.

Sheshbaradaran, an Iranian-American raised in the United Kingdom, Sweden and United States, was inspired to spend his career in oncology drug development after he lost his mother to cancer when he was 24. He began his career at Pharmacia and worked at Roche then a small biotech firm before starting Niiki.

His company's name, Niiki, means "goodness" in Persian. Sheshbaradaran said the company's goal is to bring "Niiki" to cancer patients in need.

Since Niiki Pharma was incorporated in 2007, the company has raised $15 million.

Co-founder Ali Ardakani, Niiki's vice president of operations and corporate development, said the company raised $1 million in seed funding from Philadelphia-based biotech greenhouse operator BioAdvance and another $750,000 from Ben Franklin Technology Partners of Southeastern Pennsylvania. The remainder of the funds have come from angel investors, state and federal grants and the company's founders.

Niiki, which also has offices in Hoboken, N.J., received a $488,000 grant under last year's Qualifying Therapeutic Discovery Project program included in the health-care reform bill. "For a company like us," Ardakani said, "it made a big difference."

"Their team had done an exceptional job raising a significant amount of angel funding in what we all know is a terrible capital environment," said Barbara Schilberg, CEO of BioAdvance. "They also have done a great job managing outside consultants to get high-quality skills on an as-needed basis. The combination of the two shows the power of the semi-virtual model."

Howard Brooks, an Ernst & Young partner in the firm's life science practice, said in this difficult funding environment its not uncommon for early-stage biopharmaceutical companies to operate with small staffs and outsource activities such as research, manufacturing and even sales. "It's easier to get started and leverage efficiencies that are in place fro companies that specialize in those area," Brooks said.

Niiki Pharma recently formed a scientific advisory board led by Dr. Daniel G. Haller, a University of Pennsylvania professor of gastrointestinal oncology and former editor-in-chief of the "Journal of Clinical Oncology."

The four-year-old company has two new product candidates in development. Both were invented by Dr. Bernhard Keppler, an internationally known metalorganic chemist, and licensed from a German incubator company that held the rights to the experimental compounds.

The first product, NKP-1339, is an intravenous injection designed to target the GPR78 pathway. In normal cells, GRP78 proteins fix or get rid of malfunctioning proteins.

In cancer cells, Sheshbaradaran explained, GRP78 goes into overdrive and high jacks proteins cancer cells need to survive. The proteins fix the damage to cancer cells caused by chemotherapy. NKP-1339 is designed to reduce production levels of GRP78 proteins. That, in turn, causes tumor cells dependent on the proteins to start dying.

Dr. Angela K. Ogden, an oncologist and the chief medical officer at Niiki Pharma, said a growing body of medical literature is identifying GRP78 as a target for cancer therapies. "Nobody [other than Niiki] had developed a drug targeting GRP78," Ogden said. "Now companies are starting to look at developing molecules to block it, but those are years away from getting into patients."

Ogden said Niiki phase-I clinical trial tested NKP-1339 in patients with a variety of advanced cancers. They found the treatment produced limited and manageable side effects, such as moderate nausea and chills, but no skin rashes, hair loss or liver or heart problems — all side effects associated with existing cancer therapies.

Among their findings was positive response in several patients with neuroendocrine tumors associated with such cancers as pancreas and liver. Sheshbaradaran said the company expects to decide by mid-year what specific type of cancers it will target for phase-II testing.

Its second new drug candidate, NKP-2235, is a tablet that works, Sheshbaradaran said, by increasing the amount of stress in tumor cells so they "shut down their engines" and die.

In preclinical testing, the company said the therapy had shown the most promise in breast, cancer, lung and multiple myeloma cancers. The testing also demonstrated that NKP-2235 at low doses can prevent bone breakdown.

Read more: http://www.bizjournals.com/philadelphia/print-edition/2012/01/13/niiki-pharma-developing-new-weapons-to.html?page=all


From left: Dr. Angela K. Ogden, Ali Ardakani and Hooshmand Sheshbaradaran at University City Science Center. Photo Credits: John George, Philadelphia Business Journal

November 15, 2011 Niiki Pharma Reports on Synergistic Activity of Novel Anti-Cancer Agent NKP-1339 with Other Anti-Cancer Agents

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HOBOKEN, N.J. and PHILADELPHIA, Nov. 15, 2011 /PRNewswire/ -- Niiki Pharma Inc. presented the results of preclinical combination studies of its lead product, NKP-1339, at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics held in San Francisco, CA, November 12-16, 2011.

The data were presented as a poster, titled "NKP-1339 synergistic activity in both in vitro and in vivo preclinical models highlights the therapeutic opportunity for NKP-1339 combination trials with a broad range of anti-tumor agents."

Data summary:

  • In vitro combination studies were performed in breast, colon, lung, gastric, prostate, pancreatic and liver tumor cell lines. NKP-1339 was tested in combination with cisplatin, oxaliplatin, 5-FU, docetaxel, doxorubicin, gemcitabine, erlotinib or sorafenib as appropriate for each tumor cell type. Synergistic cytotoxicity was seen with all combinations tested.
  • In vivo gastric carcinoma xenograft model was tested with NKP-1339 in combination with cisplatin. The combination showed significant (P<0.05) tumor growth delay and extended survival when compared to the single agent activity for either compound.

The broad synergism of NKP-1339 across the tested anti-neoplastic agents is consistent with its proposed mechanism of action through inhibition of the GRP78 pathway.

"The synergy we have demonstrated in both in vitro and in vivo settings suggests that NKP-1339 will be effective in combination with these anti-cancer agents in the clinical setting. The favorable efficacy and safety profile observed to date in our ongoing single agent NKP-1339 Phase I trial warrants its investigation in combination studies for our next round of clinical trials," noted Angela Ogden MD, Chief Medical Officer at Niiki Pharma.

About NKP-1339

NKP-1339 is a first-in-class small molecule anti-cancer compound. NKP-1339 down-regulates the GRP78 pathway, a key regulator of mis-folded protein processing and a tumor survival factor. Up-regulation of GRP78 is associated with intrinsic and chemotherapy-induced resistance in many tumor types. In preclinical studies, single agent NKP-1339 has demonstrated activity against multiple tumor types, including those resistant to other anti-cancer agents.

NKP-1339 was discovered by Professor Bernhard K. Keppler, Dean of the Faculty of Chemistry at University of Vienna, Austria and President of Austrian Association of University Professors.

About Niiki Pharma Inc.

Niiki Pharma is a development focused oncology company specializing in first-in-class cancer treatments directed at novel cellular targets and related companion diagnostics.

Links

Niiki Pharma Inc. www.niikipharma.com
Professor Bernhard K. Keppler, University of Vienna http://anorg-chemie.univie.ac.at

September 20, 2011 Niiki Pharma Announces Positive Interim Data from Ongoing Clinical Trial of Novel Anti-Cancer Agent NKP-1339

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HOBOKEN, N.J. and PHILADELPHIA, Sept. 20, 2011 /PRNewswire/ -- Niiki Pharma Inc. announced interim results from the ongoing Phase I clinical trial of its lead product, NKP-1339. NKP-1339 is a first-in-class transferrin targeted small molecule that down-regulates GRP78, a key regulator of mis-folded protein processing and a tumor survival factor.

NKP-1339 Phase I Interim Data

The Phase I trial is conducted in patients with metastatic solid tumors resistant to standard therapies. Previous to enrollment in the trial, all patients had received multiple standard therapies and exhibited disease progression on their last treatment. Of the first 24 patients enrolled, six patients (25%) exhibited anti-tumor activity, demonstrated by disease stability and/or tumor regression for at least 12 weeks.

Of the responding six patients, one patient with carcinoid tumor, a non-pancreatic neuroendocrine tumor (non-pNET), has tumor regression and continues on NKP-1339 therapy for more than 70 weeks. The five other patients have experienced stable disease of up to 24 weeks with NKP-1339 treatment. These patients have the following tumor types: one gastrinoma non-pNET, one colorectal cancer, two non-small cell lung cancers and one cancer of unknown primary.

NKP-1339 treatment has been well tolerated to date with mild manageable side effects. The most common drug-related side effects are grade 1-2 fever and mild flu-like symptoms, which can be prevented with standard medications. The maximum tolerated dose has not been reached and NKP-1339 dose escalation continues.

The trial is being led by Dr. Daniel Von Hoff, Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare in partnership with Translational Genomics Research Institute (TGen), and Dr. Howard Burris, Director of Drug Development at the Sarah Cannon Research Institute (SCRI).

"We need new treatments for patients with non-pNET. The NKP-1339 anti-tumor activity observed in these two non-pNET patients is significant. This activity, together with mild-side effect profile, makes NKP-1339 a potentially promising new treatment for this disease," said Dr. Daniel Von Hoff.

"It is gratifying to see someone whose tumor has been resistant to other therapies do well with this promising investigational therapy. The patient has received NKP-1339 for more than a year and continues to benefit," added Dr. Howard Burris.

"We are pleased by these interim results demonstrating the single agent anti-tumor activity of NKP-1339 in patients with advanced cancers refractory to standard treatments," said Dr. Angela Ogden, Chief Medical Officer for Niiki Pharma Inc.

"The preliminary results of this ongoing trial, showing the anti-tumor activity and the safety profile of NKP-1339, support our preclinical studies that the drug targets tumors selectively and is active against different tumors. We are delighted to see years of research come to fruition in collaboration with Niiki Pharma's team," said Professor Keppler, University of Vienna, inventor of NKP-1339.

About NKP-1339

NKP-1339 is a first-in-class transferrin targeted ruthenium-based anti-cancer compound. Transferrin receptor is significantly over-expressed in many tumor types. The intracellular target pathways of NKP-1339 include down-regulation of GRP78, a key regulator of mis-folded protein processing and a tumor survival factor. GRP78 is the cause of resistance in many tumor types. In preclinical studies, NKP-1339 has demonstrated activity against multiple tumor types, including those resistant to other anti-cancer agents, including platinum-containing agents, anthracyclines and anti-tubulins.

NKP-1339 was invented by Professor Bernhard K. Keppler, currently the Dean of the Faculty of Chemistry at University of Vienna, Austria, and Head of the Research Platform "Translational Cancer Therapy Research", a multi-disciplinary collaboration between University of Vienna and the Medical University of Vienna. Professor Keppler is a member of the Vienna Board of Trustees for Innovative and Interdisciplinary Cancer Research, the current President of the Austrian Association of University Professors, and a founding member of the Central European Society of Anticancer-Drug Research. He is on the Editorial Boards of several scientific journals, including: Anticancer Research, Chemical Monthly, Bioinorganic Chemistry and Applications, Journal of Inorganic Biochemistry (2005-2009) and Current Chemical Biology.

About NKP-1339 Phase I Trial Principal Investigators

Dr. Daniel Von Hoff, M.D., F.A.C.P., is the Physician-in-Chief and Distinguished Professor at Virginia G. Piper Cancer Center Clinical Trials, at Scottsdale Healthcare in partnership with TGen in Phoenix, Arizona, and the Chief Scientific Officer for US Oncology. He was appointed to and served six years on President Bush's National Cancer Advisory Board and was past President of the American Association for Cancer Research (AACR) a former Board member of the American Society of Clinical Oncology (ASCO), and the 2010 recipient of the David A. Karnofsky Memorial Award by ASCO for his outstanding achievements in cancer research.

Dr. Howard Burris, M.D., F.A.C.P., is the Director of Drug Development for SCRI, a Nashville, Tennessee, global clinical research organization focusing on advancing therapies and accelerating drug development in areas such as oncology, cardiology and gastroenterology and a member of Tennessee Oncology. He is also a member of the ASCO Ethics, Clinical Practice and Program Committees, Board member of Gilda's Club, Past-President and Trustee for the Southern Association of Oncology, and Editor of the Oncology Report.

About Niiki Pharma Inc.

Niiki Pharma Inc. is an oncology therapeutics development company, which is focused on development of targeted first-in-class treatments for cancer.

Links

Niiki Pharma Inc. www.niikipharma.com
Dr. Daniel Von Hoff, Tgen www.tgen.org
Dr. Howard Burris, SCRI www.sarahcannonresearch.com
Professor Bernhard K. Keppler, University of Vienna http://anorg-chemie.univie.ac.at

June 1, 2011 First-in-class novel anti-cancer agent in development by Niiki Pharma Shows Promising Phase I Results, data to be Presented at American Society for Clinical Oncology (ASCO) 2011 Meeting, Chicago, June 4-8

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HOBOKEN, N.J. and PHILADELPHIA, June 1, 2011 /PRNewswire/ -- Niiki Pharma announced today that it will present interim data from the ongoing Phase I clinical study for its lead product, NKP-1339, at the 2011 American Society for Clinical Oncology meeting in Chicago, IL. NKP-1339 is a novel transferrin targeted ruthenium based anti-cancer compound. The intracellular targets of NKP-1339 include GRP78, a key regulator of mis-folded protein processing. In preclinical studies, in vivo and in vitro activity has been demonstrated against multiple tumor types, including those resistant to other anti-cancer agents.

The NKP-1339 Phase I trial is a dose-ascending trial and determines the safety, tolerability, maximum tolerated dose (MTD) and pharmacokinetics of NKP-1339. A total of 16 patients with metastatic solid tumors resistant to standard therapies have been treated to date at six different dose levels. NKP-1339 treatment has been generally well tolerated, with the most common drug related side effects of grade 1-2 fever and mild flu-like symptoms. MTD has not been reached and NKP-1339 dose escalation continues. Signs of anti-tumor activity (stable disease greater than or equal to four months; tumor regression) have been observed. A patient with a neuroendocrine tumor (NET) of the small intestine has been on NKP-1339 therapy for over 13 months with a continuing minor response. The patient remains on NKP-1339 therapy. Another patient with the metastatic gastrinoma NET exhibited six months stable disease, and two patients with metastatic non-small cell lung cancer exhibited four months stable disease.

The NKP-1339 Phase I trial is being led by Dr. Daniel Von Hoff, Translational Genomics Research Institute, Arizona, and Dr. Howard Burris, Sarah Canon Research Institute, Tennessee. Reflecting on NKP-1339's novel mechanism of action, Dr. Von Hoff noted "Resistance to anti-cancer therapy remains a major challenge in treatment of patients with metastatic cancer. First-in-class drugs, like NKP-1339, can take us one step further to address this high unmet need." While Dr. Burris commented, "The results to date indicate that NKP-1339 could be a promising new agent that would enhance our anti-cancer armamentarium."

About Niiki Pharma

Niiki Pharma Inc. is an oncology therapeutics development company with a pipeline of novel targeted anti-cancer agents. For more information, visit www.niikipharma.com.

About Translational Genomics Research Institute

The Translational Genomics Research Institute (TGen) is dedicated to conducting groundbreaking research with diseases such as cancer, neurological disorders and diabetes. For more information, visit www.tgen.org

About Sarah Canon Research Institute

Sarah Cannon Research Institute (SCRI) is a global clinical research organization focusing on advancing therapies and accelerating drug development in areas such as oncology, cardiology and gastroenterology. For more information, visit http://sarahcannonresearch.com.