Moleculin Biotech, Inc. (NASDAQ: MBRX) Breaking News - September 13, 2018

Moleculin’s Brain Cancer Drug Candidate Begins Patient Dosing at Clinical Trial Being Conducted at MD Anderson
 

Small molecule lead drug candidate blocks a critical target for tumors and crosses the blood brain barrier; begins first brain cancer patient dosing in clinical trial at MD Anderson Cancer Center

HOUSTON, TX - September 13, 2018  --In the ongoing challenge to combat the almost always deadly brain cancers, namely Glioblastoma and melanoma metastasized to the brain, the pharmaceutical company Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), has initiated a Phase 1 clinical trial of a new first-in-class cancer drug candidate, a small molecule compound discovered by Prof. Waldemar Priebe at The University of Texas MD Anderson Cancer Center and known as WP1066. The compound has been shown in animal models to both inhibit an important cell signaling protein STAT3 that is involved in cell growth and proliferation and considered critical to tumor development, while also stimulating an immune response. The first glioblastoma patient has received the initial doses of WP1066, which were apparently well tolerated, in the physician-sponsored IND (investigational new drug) study at MD Anderson Cancer Center.

Built from the chemical backbone of the active ingredient in propolis, a natural product of honey bees, WP1066 is the first anticancer agent with drug-like properties that consistently inhibits the activated form of STAT3 within cancer cells, a target that has been long-sought because of its broad range of tumor promoting effects.

Importantly, activated STAT3 supports the survival and proliferation of tumor cells, evasion of the immune response and metastasis to distant organs, as well as angiogenesis (growth of blood vessels) essential for tumor growth. Activated STAT3 is not only connected with directly supporting tumor activity, but also suppressing the immune system, making this target even more important to cancer therapy.

With the support of extensive preclinical studies demonstrating high antitumor activity and the critically important ability to cross the blood-brain barrier, WP1066 in this Phase 1 clinical trial will focus on treating aggressive brain tumors which all share a grim prognosis. The intent is to eventually treat up to 15 relapsed brain cancer patients over the next six to eight months. Phase 1 clinical trials typically focus on exploring safe and well tolerated doses, as well as evaluating initial signals of effectiveness. Each treatment is completed over three weeks.

“Treating the first brain tumor patient with WP1066 is the start of a very exciting and encouraging program for doctors treating the worst types of brain cancers. There has been very little progress in recent years toward improved therapies for glioblastoma and other aggressive primary or metastatic brain tumors. WP1066 has shown extremely promising results based on animal studies where we have seen inhibition of tumor growth and improvements in survival,” said Dr. Sandra Silberman, a world-renowned oncologist and Moleculin’s Chief Medical Officer. “This is based on the fact that although STAT3 has long been identified as an important target for treating tumors, for years most efforts have focused on attempts to indirectly inhibit STAT3 from upstream signaling, not from within the cancer cell itself. WP1066 appears to be unique in its ability in vitro and in animal models to consistently and directly inhibit the activated form of STAT3 and produce significant anticancer effects, including tumor growth inhibition and increased life span of treated animals.”

“This represents a major milestone for Moleculin,” commented Walter Klemp, Chairman and CEO. “There has been tremendous enthusiasm within the oncology community for targeting STAT3, a key molecular hub of multiple pathways promoting tumor growth. Although the industry has been struggling to find a way to target STAT3, we at Moleculin believe that most of these efforts have been mechanistically misguided and ended in failure because their approach would ultimately be ineffective at adequately blocking the activation of STAT3 and lack the necessary drug-like properties to succeed. The opportunity to test a unique STAT3 therapy in these patients is significant in supporting Moleculin’s mission to provide benefit for those who need new and better treatments.”

How WP1066 Works in Tumor Cells

WP1066 is a small molecule compound that can not only directly kill tumor cells, but also has the ability to overcome the tumor’s ability to evade the natural immune response, which would otherwise be working to eliminate the cancerous activity. This compound is a first in class drug candidate capable of down-regulating the activated form of STAT3, a target that has been long-sought because of its role in supporting the survival and growth of tumor cells.

The compound has been shown to prevent tumor progression and increase survival in a wide range of animal models by directly attacking tumors and blocking the cell signaling by STAT3 that supports tumor development and simultaneously suppressing regulatory T cells (Tregs), which then allows stimulation of an enhanced natural anti-tumor immune response. The compound’s dual functions have been shown to increase survival in a wide range of animal models, which have been documented in more than 50 peer-reviewed articles.

About Moleculin Biotech, Inc.

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on discoveries made at M.D. Anderson Cancer Center. Our clinical stage drugs are Annamycin, an anthracycline designed to avoid multidrug resistance mechanisms with little to no cardiotoxicity being studied for the treatment of relapsed or refractory acute myeloid leukemia, more commonly referred to as AML, and WP1066, an immuno-stimulating STAT3 inhibitor targeting primary brain tumors and brain metastases, pancreatic cancer and hematological malignancies. We are also engaged in preclinical development of additional drug candidates, including additional STAT3 inhibitors and compounds targeting the metabolism of tumors.

For more information about the Company, please visit http://www.moleculin.com.

Forward-Looking Statements
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the ability of WP1066 to show activity in brain tumor patients and the ability to enroll and treat patients within the time period discussed. These statements relate to future events, future expectations, plans and prospects. Although Moleculin Biotech believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin Biotech has attempted to identify forward-looking statements by terminology including ''believes,'' ''estimates,'' ''anticipates,'' ''expects,'' ''plans,'' ''projects,'' ''intends,'' ''potential,'' ''may,'' ''could,'' ''might,'' ''will,'' ''should,'' ''approximately'' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission (“SEC”) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Contacts
Joe Dorame, Robert Blum or Joe Diaz
Lytham Partners, LLC
602-889-9700
mbrx@lythampartners.com

SOURCE:  Moleculin Biotech, Inc.
 

Moleculin Biotech, Inc. Reports Financial Results for the Second Quarter Ended June 30, 2018
 

HOUSTON, TX - August 13, 2018  -- Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"),  clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the MD Anderson Cancer Center, today announced its financial results for the second quarter ended June 30, 2018. Additionally, the Company announced potential upcoming milestones and recent corporate developments.

Management Discussion

Walter Klemp, Chairman and CEO of Moleculin, said, “During the second quarter, and the first half of 2018, we continued to make measurable progress in achieving important milestones in our three core disruptive technologies and six oncology drug candidates. We are successfully executing our strategic plan to advance our innovative cancer treatment solutions through the regulatory process and work our way toward accelerated FDA approvals by focusing on significant unmet needs. Our recent accomplishments include:

receiving Polish National Office approval to begin our second Phase I/II clinical trial in Poland to study Annamycin for treatment of relapsed or refractory acute myeloid leukemia (“AML”);
qualified a second and a third U.S. Annamycin clinical site with a fourth, we believe, to come in the near term;
commencing treatment for the first patient enrolled in the Annamycin U.S. Clinical trial at The University Hospitals Cleveland Medical Center, which includes the Seidman Cancer Center and the Cleveland Clinic;
announcing the opening of enrollment for a physician-sponsored clinical trial of WP1066 for the treatment of glioblastoma and brain metastases in adults;
initiating operations in Australia to benefit from potential rebates of up to 43.5% of qualified R&D expenditures and speed up preclinical development;
commencing preclinical toxicology testing of WP1732, a fully water-soluble STAT3 inhibitor we believe, based on preclinical testing, has the potential to be a breakthrough discovery for rare and difficult to treat cancers through our new subsidiary in Australia; and
submitting a request to Polish authorities for clinical trial authorization (“CTA”) for our STAT3 inhibitor, WP1220, for the treatment of Cutaneous T-Cell Lymphoma (“CTCL”) which, if approved, will give us our third drug in clinic.
In our mission to develop breakthrough treatments for rare and difficult cancers, we have developed unique attributes in certain compounds that we believe will: inhibit STAT3 – a prevalent indicator in many cancers; avoid heart damage, eliminate multi-drug resistance with little to no cardiotoxicity; and inhibit metabolic activity by blocking the energy supply cancer cells require and inducing immune system function to target unique and highly metastatic tumors. We believe our three highly differentiated technologies have the potential to effectively attack high profile acute cancer states, ranging from AML; brain tumors; deadly forms of skin cancer; to pancreatic cancer. We currently have two oncology drugs in clinical trials, Annamycin and WP1066, a physician-sponsored trial, with the possibility of two others commencing clinical trials in 2019. This is only possible because of the tireless dedication and the hard work of all our associates at Moleculin. They have driven the momentum that we’ve achieved to this point while remaining fiscally responsible in our development process. We are highly focused on leveraging our successes to potentially change the treatment for cancer and excited about the opportunities ahead.”

“With total R&D and total operating expense of $4.2 million and $5.5 million in the quarter, respectively, it should be noted that we had some one-time R&D charges in the quarter. Specifically, we expensed approximately $2.3 million related to the production of additional drug product for our Annamycin clinical trials, including product for the expansion beyond the currently planned Phase I/II clinical trial, and the initiation of pre-clinical work on WP1732 in Australia. We believe that the latter will generate Australian income tax credits next calendar year for which cash should be received by our Australian subsidiary in 2019. Furthermore, we incurred $1 million in R&D expense related to finalizing the acquisition of the license to the non-skin rights of the WP1066 portfolio,” stated Jonathan P. Foster, executive vice president and chief financial officer of Moleculin. He continued, “We expect our R&D expense to be lower going forward on a quarterly basis extending our cash on hand into the second quarter of 2019.”

Anticipated Milestones

Anticipated Milestone Potential Timeframe
Announcement that our Investigational New Drug (IND) for Annamycin has become effective and that we may begin clinical trials Accomplished
Initial IRB (Institutional Review Board) approvals and site initiations of various clinical sites participating in our Phase I/II clinical trial of Annamycin Accomplished and ongoing through Second Half of 2018
Establishment of a new Recommended Phase 2 Dose for Annamycin Second Half of 2018
A clinician sponsored IND for WP1066 for treatment of adult brain tumors moving forward Accomplished; Now open for enrollment
Announcement of initial clinical data for Annamycin trial 2018
Announcement of further benefits of our sponsored research agreement with MD Anderson Accomplished and Ongoing into 2019
Announce CTA for WP1220 for the treatment of cutaneous T-cell lymphoma (CTCL) 2018
(CTA Filed)
Announce WP1122 move into preclinical work 2018
Announce WP1732 move into preclinical work Accomplished
Announce IND for WP1732 submitted First Half of 2019
Announce a fourth drug approved for clinical trial 2019
Second Quarter Highlights and Recent Corporate Developments

Moleculin Seeks Approval from Polish Regulatory Agency for Skin Cancer Clinical Trial - August 9, 2018, the Company announced its submission of a request to Polish authorities for a CTA for its STAT3 inhibitor, WP1220, for the treatment of CTCL which, if approved, will give the Company its third drug in clinic. Published research supports the belief that Cutaneous T-Cell Lymphoma, a deadly form of skin cancer, may be highly dependent on the upregulation of the activated form of STAT3. The Company believes WP1220 may be ideally suited as a topical agent to inhibit STAT3 and therefore could potentially become a valuable new drug for the treatment of CTCL. A request for CTA in Poland is the equivalent of a request for Investigational New Drug status in the U.S.

Moleculin Announces Enrollment Opens for Brain Tumor Trial of WP1066 - July 31, 2018, the Company announced enrollment opened for a physician-sponsored clinical trial of WP1066 for the treatment of glioblastoma and brain metastases in adults. This is the first investigator-initiated trial of WP1066, an important milestone. The goal of this clinical research study is to find the highest tolerable dose of WP1066 that can be given to patients with recurrent (has returned after treatment) cancerous brain tumors or melanoma that has spread to the brain. The safety of this drug will also be studied. WP1066 is designed to target the STAT3 pathway in cancer cells, which independent research has shown allows these cells to survive and proliferate, increases new blood vessels to the tumor, causes the cancer cells to move throughout the body and brain, and reduces the ability of the immune system to effectively combat tumor development. In addition, the Company believes that WP1066 may also have the potential to stimulate a natural anti-tumor immune response.

Moleculin Expects to Meet FDA IND Filing Requirements for its Pancreatic Cancer Drug Candidate with Development Work in Australia - July 18, 2018, the Company announced it began preclinical toxicology testing of its WP1732, a fully water-soluble STAT3 inhibitor with the potential to be a breakthrough discovery for rare and difficult to treat cancers through its new subsidiary in Australia. By utilizing its subsidiary in Australia and the attractive R&D tax credits it offers, it can accelerate the preclinical work of WP1732 and maintain a strong cash balance. The Company believes this will allow it to complete its IND-enabling work and meet FDA submission requirements before year-end, which should allow it to complete the IND filing during 2019, while also reducing the Company’s total cost of development.

Moleculin Expands Operations to Australia; Taps R&D Incentive Program Capped at $20,000,000 AUD Turnover - July 11, 2018, the Company announced it had formed Moleculin Australia Pty. Ltd., a wholly-owned subsidiary to oversee preclinical development in Australia. For companies like Moleculin with less than $20,000,000 AUD group turnover, it can amount to a rebate of up to 43.5% of qualified R&D expenditures. The Australian subsidiary provides a great opportunity to speed up preclinical development and reduce the overall cost of continued drug development efforts.

Moleculin Selected for the Russell Microcap Index - June 26, 2018, the Company announced it was selected to be added to the Russell Microcap® Index effective after the U.S. market opened on June 25, 2018, when the Russell Investments reconstituted its comprehensive set of U.S. and global equity indexes. Membership in the Russell Microcap® Index, which remains in place for one year, means automatic inclusion in the appropriate growth and value style indexes.

Moleculin Announces $2.3 Million Registered Direct Offering Priced At-the-Market - June 21, 2018, the Company announced that it entered into a definitive agreement with institutional investors for a registered direct offering of securities with gross proceeds of approximately $2.3 million, which was completed on June 22, 2018.

Moleculin Receives Approval for Leukemia Clinical Trial - June 20, 2018, the Company announced it received Polish National Office approval to begin its second Phase I/II clinical trial to study Annamycin for the treatment of relapsed or refractory AML. Consent from the Polish National Office was the final step required to allow recruitment of patients for this important trial.

Moleculin Targets accelerated FDA approval of WP1732; Pursues Development for Ocular Tumors - June 12, 2018, the Company announced that it entered into an agreement with the Jagiellonian University in Krakow, Poland, for the development of its STAT3 inhibitor, WP1732, for the treatment of ocular tumors. The Company believes the water-soluble nature of WP1732 could make it an ideal candidate for targeting these unique and highly metastatic tumors.

Moleculin’s Breakthrough Discovery of a New Molecule for Cancer Treatment Advances to Development Agreement with the University of Iowa - June 06, 2018, the Company announced that it entered into an agreement with The University of Iowa Pharmaceuticals for the development of a formulation for WP1732. The Company believes WP1732 represents a major expansion of its STAT3 inhibition capability by providing a highly soluble alternative that is ideally suited for IV administration. This agreement marks the beginning of creating a preclinical package to submit to the FDA in order to request Investigational New Drug status.

Moleculin Invited to Present to International BioForum 2018 Conference - May 24, 2018, the Company announced that its CEO, Walter Klemp, was asked to address the 2018 BioForum Conference in Łódź, Poland regarding the Polish-American Innovation Bridge: Bringing validated innovations from USA to Poland.

Moleculin to Begin Clinical Trials at UMC Southwest Cancer Center - May 16, 2018, the Company announced that a second U.S. site, located in Lubbock, Texas, has qualified for its clinical trial to study Annamycin for the treatment of relapsed or refractory AML. UMC Southwest Cancer Center qualified as the second U.S. site for Moleculin’s clinical trial of Annamycin. Dr. Sanjay Awasthi, Division Chief of Hematology/Oncology at Texas Tech University will serve as the site’s Principal Investigator.

Moleculin Announces Engagement with Voisin Consulting Life Sciences to Expand Annamycin Clinical Trial - May 03, 2018, the Company announced it has engaged Voisin Consulting Life Sciences (“VCLS”), as an additional regulatory consulting firm and contract research organization to prepare for expansion of its clinical trial to study Annamycin for the treatment of relapsed or refractory AML. VCLS headquartered in Paris, France will evaluate Australia and selected Western European countries for the potential expansion of clinical sites for the Company's AML clinical trial.

Moleculin Announces New Data for Immuno-Stimulating Drug to be Presented at International Conference - April 26, 2018, the Company announced Dr. Waldemar Priebe, Chair of the Company's Scientific Advisory Board, has been selected to present findings on Moleculin's STAT3 inhibitor and immune-stimulating agent, WP1066, at the Global Academic Programs (“GAP”) 2018 in Stockholm, Sweden from May 15 to 17, 2018. The annual GAP Conference provides a forum for faculty from MD Anderson and its Sister Institutions to develop collaborations and exchange research results and ideas. The GAP 2018 Conference is being sponsored by a prestigious list of major pharmaceutical companies, including Roche, Bayer, Bristol-Meyers Squibb, AstraZeneca, Novartis, Merck, and Pfizer.

Moleculin Enters Agreement with BSP Pharmaceuticals for its Leukemia Drug Candidate - April 24, 2018, the Company announced it entered into an agreement with BSP Pharmaceuticals S.p.A to expand production capacity for Annamycin. BSP Pharmaceuticals S.p.A., based in Latina, Italy will begin preparations for commercial scale production of the Annamycin drug product. BSP has a solid track record for supplying liposomal formulations to large pharmaceutical companies.

Moleculin Announces Patients Treated in FDA Approved Phase I/II Annamycin Clinical Trial - April 04, 2018, the Company announced that patients have successfully begun treatment in its U.S. Phase I/II clinical trial of Annamycin for the treatment of relapsed or refractory AML. The first patient enrolled in Moleculin's Annamycin clinical trial was treated at The University Hospitals Cleveland Medical Center Seidman Cancer Center on March 28, 2018.

Financial Results for the Second Quarter Ended June 30, 2018

Research and Development Expense. Research and development (“R&D”) expense was $4.2 million and $0.5 million for the three months ended June 30, 2018 and 2017, respectively. The increase of approximately $3.7 million mainly represents an increase of approximately: $2.3 million associated with producing additional drug product for the Company’s Annamycin clinical trials and with pre-clinical work on WP1732 in anticipation of filing an IND in 2019, $1.0 million accrued expense related to the HPI Option Repurchase Payment, $0.2 million related to an increase in R&D associated headcount costs; and $0.2 million related to various other expenses.

General and Administrative Expense. General and administrative expense was $1.2 million and $0.8 million for the three months ended June 30, 2018 and 2017, respectively. The increase of approximately $0.4 million was mainly attributable to the increase in headcount and associated payroll costs of $0.3 million, and $0.1 million of stock-based compensation. Such increases are due to the increased corporate activity as the Company enters clinical trials and increases its pre-clinical work on WP1732.

Net Loss. The net loss for the three months ended June 30, 2018 was $5.1 million, which included non-cash income of $0.3 million on the gain in fair value of the Company's warrant liability, which was offset by noncash charges for $0.3 million related to stock-based compensation and other stock-based expenses.

Liquidity and Capital Resources

As of June 30, 2018, the Company had $11.7 million in cash and cash equivalents. On June 22, 2018, the Company completed the sale to institutional investors for a registered direct offering of securities for the sale of 1,092,636 shares of the Company’s common stock, at a purchase price of $2.105 per share. Concurrently with the sale of the common shares, the Company also sold warrants to the investors 710,212 shares of common stock. The Company sold the common shares and warrants for aggregate gross proceeds of approximately $2.3 million. Subject to certain beneficial ownership limitations, the warrants will be initially exercisable on the six-month anniversary of the issuance date at an exercise price equal to $2.02 per share of common stock, subject to adjustments as provided under the terms of the warrants. The warrants are exercisable for five years from the initial exercise date. The closing of the sales of these securities under the agreement occurred on June 22, 2018. The Company believes that its existing cash and cash equivalents as of June 30, 2018 will be sufficient to fund its planned operations into the second quarter of 2019. Such plans are subject to change depending on clinical enrollment progress and use of drug product.

About Moleculin Biotech, Inc.

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on discoveries made at MD Anderson Cancer Center. Our clinical stage drugs are Annamycin, an anthracycline designed to avoid multidrug resistance mechanisms with little to no cardiotoxicity being studied for the treatment of relapsed or refractory acute myeloid leukemia, more commonly referred to as AML, and WP1066, an immuno-stimulating STAT3 inhibitor targeting primary brain tumors and brain metastases, pancreatic cancer and hematological malignancies. We are also engaged in preclinical development of additional drug candidates, including additional STAT3 inhibitors and compounds targeting the metabolism of tumors.

For more information about the Company, please visit http://www.moleculin.com.

Forward-Looking Statements
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the potential for Annamycin to demonstrate safety and efficacy in AML patients in clinical trials in the United States and in Poland, and the timeframe in which such trials are completed,; the ability of MD Anderson to successfully enroll patients in the Phase 1 clinical trial for WP1066, the timeframe in which such trial is completed, and the ability of WP1066 to show safety and efficacy in patients with glioblastoma or melanoma that has metastasized to the brain; the potential for WP1220 to become an effective treatment for CTCL and the ability of the Company to obtain Polish regulatory approvals to commence clinical trials to study WP1220 for CTCL; the ability and timeline pursuant to which the Company is able to prepare the preclinical data necessary for an IND for WP1732; and the potential for WP1122 to become an effective treatment for brain tumors or the ability of a WP1220 analog to become a safe and effective drug for pancreatic cancer in humans. These statements relate to future events, future expectations, plans and prospects. Although Moleculin Biotech believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin Biotech has attempted to identify forward-looking statements by terminology including ''believes,'' ''estimates,'' ''anticipates,'' ''expects,'' ''plans,'' ''projects,'' ''intends,'' ''potential,'' ''may,'' ''could,'' ''might,'' ''will,'' ''should,'' ''approximately'' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission (“SEC”) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Contacts
Joe Dorame, Robert Blum or Joe Diaz
Lytham Partners, LLC
602-889-9700
mbrx@lythampartners.com

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Moleculin Announces Enrollment Opens for Brain Tumor Trial of WP1066
 

HOUSTON, TX - July 31, 2018  -- Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, today announced enrollment has opened for a physician-sponsored clinical trial of WP1066 for the treatment of glioblastoma and brain metastases in adults.

“We have been eagerly awaiting the beginning of this physician sponsored clinical trial,” commented Walter Klemp, Chairman and CEO of Moleculin. “The trial, which is now listed on clinicaltrials.gov and being conducted at MD Anderson Cancer Center, is now open for enrollment. This is our first investigator initiated trial of WP1066 and an important milestone. Given the unique potential of WP1066, we expect more trials to follow.”

The goal of this clinical research study is to find the highest tolerable dose of WP1066 that can be given to patients with recurrent (has returned after treatment) cancerous brain tumors or melanoma that has spread to the brain. The safety of this drug will also be studied.

WP1066 is designed to target the STAT3 pathway in cancer cells, which makes these cells divide, increases new blood vessels to the tumor, causes the cancer cells to move throughout the body and brain, and avoids them being detected by the immune system. We believe that targeting this pathway may cause the immune system to kill the cancer cells.

Forward-Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the ability to receive the benefit of tax credits in Australia, the timing of the completion of the IND-enabling work on WP1732, and the ability to secure IND status for and conduct clinical trials with WP1732. These statements relate to future events, future expectations, plans and prospects. Although Moleculin Biotech believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin Biotech has attempted to identify forward-looking statements by terminology including ''believes,'' ''estimates,'' ''anticipates,'' ''expects,'' ''plans,'' ''projects,'' ''intends,'' ''potential,'' ''may,'' ''could,'' ''might,'' ''will,'' ''should,'' ''approximately'' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission (“SEC”) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Contacts
Joe Dorame, Robert Blum or Joe Diaz
Lytham Partners, LLC
602-889-9700
mbrx@lythampartners.com

Source: Moleculin Biotech, Inc.

Moleculin Expects to Meet FDA IND Filing Requirements for its Pancreatic Cancer Drug Candidate with Development Work in Australia
 

HOUSTON, TX - July 18, 2018  -- Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, today announced it has begun preclinical toxicology testing of its WP1732, a fully water-soluble STAT3 inhibitor through its new subsidiary in Australia.

“Based on preclinical testing, we believe the discovery of WP1732, a fully water-soluble STAT3 inhibitor, has the potential to be a breakthrough discovery for rare and difficult to treat cancers. As a result of our preclinical testing, we have received multiple requests to commence clinical trials and we are pleased to be taking the next steps in preparing for the appropriate clinical work,” commented Walter Klemp, Chairman and CEO of Moleculin. “ By utilizing our subsidiary in Australia and the attractive R&D tax credits it offers, we can accelerate the preclinical work of WP1732 and maintain a strong cash balance. We believe this will allow us to complete our IND-enabling work and meet FDA submission requirements before year-end while also reducing our total cost of development.”

About Moleculin Biotech, Inc.

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on discoveries made at M.D. Anderson Cancer Center. Our clinical stage drugs are Annamycin, an anthracycline designed to avoid multidrug resistance mechanisms with little to no cardiotoxicity being studied for the treatment of relapsed or refractory acute myeloid leukemia, more commonly referred to as AML, and WP1066, an immuno-stimulating STAT3 inhibitor targeting primary brain tumors and brain metastases, pancreatic cancer and hematological malignancies. We are also engaged in preclinical development of additional drug candidates, including additional STAT3 inhibitors and compounds targeting the metabolism of tumors.

For more information about the Company, please visit http://www.moleculin.com.

Forward-Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the ability to receive the benefit of tax credits in Australia, the timing of the completion of the IND-enabling work on WP1732, and the ability to secure IND status for and conduct clinical trials with WP1732. These statements relate to future events, future expectations, plans and prospects. Although Moleculin Biotech believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin Biotech has attempted to identify forward-looking statements by terminology including ''believes,'' ''estimates,'' ''anticipates,'' ''expects,'' ''plans,'' ''projects,'' ''intends,'' ''potential,'' ''may,'' ''could,'' ''might,'' ''will,'' ''should,'' ''approximately'' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission (“SEC”) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Contacts
Joe Dorame, Robert Blum or Joe Diaz
Lytham Partners, LLC
602-889-9700
mbrx@lythampartners.com

Source: Moleculin Biotech, Inc.

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About Moleculin Biotech:

Moleculin Biotech, Inc. is preclinical and clinical-stage pharmaceutical company focused on the development of game-changing anti-cancer drug candidates, many of which are based on discoveries made at M.D. Anderson Cancer Center, the world’s largest cancer research facility headquartered within the world’s largest medical center. Each of our projects represents a breakthrough discovery and a highly disruptive technology.

Our lead product candidate is Annamycin, a Phase II clinical stage anthracycline for the treatment of relapsed or refractory acute myeloid leukemia, more commonly referred to as AML.

Unlike current therapies that risk cardiotoxicity and can have their effectiveness limited due to multidrug resistance, Annamycin appears capable of avoiding both of these problems and has already demonstrated the ability to save lives in clinic. We are now preparing to seek accelerated approval for this game-changing drug.

We also have two active pre-clinical small molecule portfolios, one of which is focused on the modulation of hard-to-target cell signaling mechanisms and appears capable stimulating the patient’s natural immune system while also attacking tumors directly. The other portfolio targets the metabolism of tumors and exploits a unique approach for crossing the blood brain barrier for the treatment of glioblastoma and other central nervous system malignancies.

Company Overview

Moleculin Biotech, Inc. is a preclinical and clinical-stage pharmaceutical company focused on the development of anti-cancer drug candidates, many of which are based on discoveries made at M.D. Anderson Cancer Center. Our lead product candidate is Annamycin, a Phase II clinical stage anthracycline for the treatment of relapsed or refractory acute myeloid leukemia, more commonly referred to as AML. We also have two active pre-clinical small molecule portfolios, one of which is focused on the modulation of hard-to-target tumor cell signaling mechanisms and the recruitment of the patient's own immune system. The other portfolio targets the metabolism of tumors.

Company Profile

Moleculin Biotech, Inc. is a preclinical and clinical-stage pharmaceutical company focused on the development of anti-cancer drug candidates, many of which are based on discoveries made at M.D. Anderson Cancer Center. Our lead product candidate is Annamycin, a Phase II clinical stage anthracycline for the treatment of relapsed or refractory acute myeloid leukemia, more commonly referred to as AML. We also have two active pre-clinical small molecule portfolios, one of which is focused on the modulation of hard-to-target tumor cell signaling mechanisms and the recruitment of the patient's own immune system. The other portfolio targets the metabolism of tumors.

Business Description

Moleculin Biotech, Inc. is a preclinical and clinical-stage pharmaceutical company focused on the development of anti-cancer drug candidates, many of which are based on discoveries made at M.D. Anderson Cancer Center. Our lead product candidate is Annamycin, a Phase II clinical stage anthracycline for the treatment of relapsed or refractory acute myeloid leukemia, more commonly referred to as AML. We also have two active pre-clinical small molecule portfolios, one of which is focused on the modulation of hard-to-target tumor cell signaling mechanisms and the recruitment of the patient's own immune system. The other portfolio targets the metabolism of tumors.


Annamycin

annamycinAnnamycin is an anthracycline intended for the treatment of relapsed or refractory AML. The therapy of combining two chemotherapeutic drugs, which always includes an anthracycline, in inducing a remission of leukemic cells (called “induction therapy”) has not improved since it was first used in the 1970s and we estimate that this induction therapy has the same cure rate of about 20% as at that time. Currently, the only viable long term option for acute leukemia patients is a bone marrow transplant, which is successful in a significant number of patients. However, in order to qualify for a bone marrow transplant, patients must first undergo induction therapy.

One of the leading anthracyclines used for induction therapy in acute leukemia patients is doxorubicin, which has reported over $700 million in annual revenues. Despite the importance and success of approved anthracyclines like doxorubicin, they are all unfortunately cardiotoxic, which can result in damage to the heart and limit the dosage amount that may be administered to patients. Additionally, the tumor cells being treated often have or develop resistance to the first line anthracycline, often through what is called “multidrug resistance” making them capable of purging themselves of the current anthracyclines and limiting the effectiveness of the therapy. Consequently, there remains no effective therapy for these patients and most will succumb quickly to their leukemia. This is where we believe Annamycin can be a complete game-changer.

Annamycin is a unique liposome formulated anthracycline (also referred to in literature as “L-Annamycin”) that has been designed to eliminate cardiotoxicity and avoid the multidrug resistance mechanisms that often defeat current anthracyclines. It has been tested in 6 clinical trials and 114 patients without any reporting of cardiotoxicity and in 2 of those clinical trials focused on leukemia, it showed fewer dose-limiting toxicities than are normally experienced with doxorubicin (one of the leading first-line anthracyclines used for induction therapy).

Annamycin demonstrated efficacy in 8 of 16 patients in a Phase I study in adult relapsed or refractory AML patients, with 6 of 14 patients completely clearing leukemic blasts. A 30 patient dose-ranging Phase I/II study in acute lymphocytic leukemia demonstrated a similar efficacy profile, with 3 of 10 patients treated with the maximum tolerable dose clearing their leukemic blasts to a level sufficient to qualify for a bone marrow transplant. One of these patients went on to receive a successful curative bone marrow transplant.

We believe Annamycin is better than the currently approved induction therapy drugs in four key ways: (i) it has demonstrated clinical activity in a patient population for whom there are currently no effective therapies, (ii) it appears to be capable of avoiding the “multi-drug resistance” mechanisms that often limit the effectiveness of currently approved anthracyclines; (iii) it has been shown to be non-cardiotoxic in animal models, when compared with doxorubicin and no events of cardiotoxicity have been reported from the use of Annamycin in 114 patients; and (iv) in laboratory studies using AML cell lines, it has been shown to be more potent than the leading approved drug.

Based on initial conversations with the FDA, because of this serious unmet medical need, we believe Annamycin may qualify for a “Special Protocol Assessment” providing for accelerated approval based on our planned Phase II clinical trial. We also believe Annamycin will qualify for Orphan Drug status, which could entitle us to market exclusivity of up to 7 and 10 years from the date of approval of a New Drug Application (NDA) and Marketing Authorization (MA), in the US and the European Union (EU), respectively.

This resistance to therapy is often the result of molecular “pumps” in tumor-cell membranes that actively expel chemotherapy drugs from the interior. This allows tumor cells to avoid the intended toxic effects of the drug.

As MDR begins to counteract chemotherapy drugs, it can require higher and higher doses to kill tumor cells, yet the unwanted side effects of the drugs, like cardiotoxicity, ultimately prevent such increases in dosing.

Two kinds of pumps commonly responsible for multidrug resistance in cancer are P-glycoprotein (P-gp) and the so-called multidrug resistance–associated protein (MRP). Because of their role in MDR, they have been the targets of several anticancer efforts. Unfortunately, efforts to block the activity of these pumps have resulted in serious side effects because they also play an important role in normal cell function.

Our most advanced therapy, Annamycin, represents a much-needed breakthrough in the battle against multidrug resistance. Its unique design prevents it from being recognized by MDR pumps, allowing Annamycin to avoid the multidrug resistance mechanisms that often defeat current therapies.

Multidrug Resistance

cellMultidrug Resistance (MDR) refers to mechanisms by which many cancers develop resistance to chemotherapy drugs and is a major factor in the failure of many forms of chemotherapy. It affects patients with a variety of blood cancers and solid tumors, including breast, ovarian, lung, and lower gastrointestinal tract cancers. Tumors usually consist of mixed populations of malignant cells, some of which are drug-sensitive while others are drug-resistant. Chemotherapy kills drug-sensitive cells, but leaves behind a higher proportion of drug-resistant cells. As the tumor begins to grow again, chemotherapy may fail because the remaining tumor cells are now able to recognize the chemotherapy and reject it at the cellular level, thus rendering it resistant to the therapy.

Management & Business Advisors

WALTER V. KLEMP
Founder, Chairman, CEO, and Business Advisory Board Member

Walter Klemp has been our Co-Founder, Chairman and CEO since 2007. Mr. Klemp has 29 years of experience in start-up and high-growth companies, the past nine of which have been spent developing FDA-approved dermatology therapy devices and topical compounds.

Mr. Klemp was also President and CEO of Zeno Corporation from 2004 to 2010, where he successfully developed and marketed a number of dermatology devices and drugs from concept through FDA approval.

Previously, Mr. Klemp served as Founder, CEO and Chairman of Drypers Corporation, a publicly traded multinational consumer products company, from 1987 to 2000. At Drypers, Mr. Klemp developed growth strategies, orchestrated mergers and acquisitions, and grew the company from start-up to $400 million in annualized sales and to a #1 ranking on the INC 500. Notably, he has overseen nearly $750 million in public and private financings throughout his career.

DONALD PICKER, PHD
President and Chief Operating Officer

Donald Picker, PhD, joined the Moleculin team in 2009 with over 35 years of drug development experience. At Johnson Matthey, Dr. Picker was responsible for the development of Carboplatin, one of the world’s leading cancer drugs, acquired by Bristol-Myers Squibb and with annual sales of over $500 million. He also oversaw the development of Satraplatin and Picoplatin, third-generation platinum drugs currently in late-stage clinical development.

Dr. Picker has significant experience in dermatological pharmaceutical discovery and development as well, having led projects for topical therapies in psoriasis, atopic dermatitis and acne.

ROBERT SHEPARD, MD, FACP
Chief Medical Officer

Dr. Robert Shepard has extensive research credentials in hematology and oncology and is board certified in oncology, hematology and internal medicine. He has a wide array of experience in translational medicine and clinical research and has been actively involved in oncology research since 1970, responsible for the complete clinical development of several drugs and immune therapies for biopharmaceutical companies, including serving as the consulting Chief Medical Officer for six companies. Dr. Shepard is a Magna Cum Laude graduate of Harvard University in biochemical sciences and molecular biophysics and studied in the Harvard-M.I.T. Health Sciences program. He held fellowships in hematology and oncology at the Tufts-New England Medical Center where he conducted laboratory research in leukemias, myeloma and myelodysplasia, as well as fellowship in pharmacology and molecular genetics at the Dana-Farber Cancer Center and Harvard Medical School. Dr. Shepard holds academic appointments at Harvard University, Tufts University and the University of Virginia.

LOUIS PLOTH JR.
Chief Financial Officer

Louis Ploth has over 30 years of domestic and international business experience in public and private companies ranging from start-ups to mid-size public corporations. Mr. Ploth has served as a Board Member, Chief Financial Officer, Chief Accounting Officer, VP of Business Development and General Manager of Operations.

Mr. Ploth has managed several public offerings, private placements and venture rounds accumulating more than $100 million. He has also completed various corporate transactions including an out-licensing agreement with deal terms of $20 million in up-front and first year payments, $37.5 million in milestone payments and high double-digit escalating royalties.

Mr. Ploth has experience with product in-licensing, royalty stream repurchasing, financial aspects of product clinical development, product out-licensing and product launch.

LORI H. BISSON
Business Advisory Board Member

Lori Bisson is a Co-Founder of Moleculin, a Director and our acting CFO. Ms. Bisson brings a wealth of experience in consumer products and regulated environments from her tenure with both Zeno Corporation and Drypers Corporation. She also serves as the CFO for a medical device company.

Previously, Ms. Bisson was CFO of Zeno Corporation from 2005 to 2008. She also served as CFO of Gulfstream Trading, Ltd., a privately held $1.5 billion oil trading company.

Ms. Bisson received her CPA license while working as an auditor with Arthur Andersen.

New hope for acute leukemia patients…

In a Phase II clinical trial, Annamycin was given to patients who had failed an average of five previous induction therapy attempts, and 30% of those patients cleared enough of their leukemic cells to qualify for a bone marrow transplant. This kind of performance, if repeated in a larger clinical trial, could make Annamycin a potential blockbuster new drug. And, Annamycin’s unique lack of cardiotoxicity could be a game-changer for pediatric patients.

Tackling the most difficult cancers…

By reducing the activity of certain tumor supporting transcription factors, tumor cells lose their ability to survive and reproduce. By increasing the activity of other transcription factors, WP1066 actually calls the patient’s natural immune system into action. Importantly, this has been shown in some of the most difficult cancers to treat, such as pancreatic cancer and metastatic melanoma.

Breaking through to brain tumors…

Brain tumors are among the most difficult cancers to treat, largely because of something called the “blood brain barrier”. WP1122 was specifically designed to slip past the blood brain barrier, using the same chemical trickery that converts morphine into heroin. We compared WP1122 to the current standard of care, temozolomide, in live human brain tumors transplanted into mice and demonstrated that WP1122 is capable of outperforming temozolomide.
 

SOURCE: http://www.moleculin.com/

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