On January 3, 2019 Crescendo Biologics Ltd (Crescendo), the drug developer of novel, targeted T-cell enhancing therapeutics, reported that Takeda Pharmaceutical Company Limited (Takeda) is planning to evaluate the application of recently licensed Humabodies from Crescendo for the development of novel CAR-T therapeutics (Press release, Crescendo Biologics, JAN 3, 2019, View Source [SID1234553819]).

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This research will investigate the unique properties of the Humabody VHs for tumour targeting of CAR-Ts. Unlike single-chain variable fragments (scFvs), Humabody VHs do not require a light chain for high specificity and affinity, and can be easily configured for multi-specific target binding. Takeda’s decision to license these Humabodies provides an opportunity to evaluate a large number of different VHs directed to the same target, in order to identify a format that delivers both enhanced safety and functionality.

Crescendo’s global, strategic, multi-target collaboration and license agreement with Takeda was first announced in October 2016. Under this agreement, Crescendo’s proprietary transgenic platform and engineering expertise is being used to identify and configure Humabody-based therapeutics against certain targets selected by Takeda.

Dr Peter Pack, CEO of Crescendo, commented:
"We’re pleased to see the research Takeda is undertaking with our Humabody technology. Their desire to explore the Humabody technology in a CAR-T setting presents an exciting opportunity to evaluate whether Humabodies can address the issues that exist with other CAR-T targeting approaches."

On January 3, 2019 Ohr Pharmaceutical, Inc. ("Ohr") (Nasdaq: OHRP) reported that it has entered into a definitive merger agreement with NeuBase Therapeutics, Inc. ("NeuBase"), under which the stockholders of NeuBase would become the majority holders of the combined company (Press release, Ohr Pharmaceutical, JAN 3, 2019, View Source [SID1234556172]). The proposed merger will create a public company focused on advancing NeuBase’s peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) technology platform for the development of therapies to address severe and currently untreatable diseases caused by genetic mutations.

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Upon closing of the transaction, the combined company will change its name to "NeuBase Therapeutics, Inc." and propose its trading symbol on the NASDAQ be changed to "NBSE". The executive team of NeuBase will serve as the executive team of the combined company, led by Dietrich A. Stephan, Ph.D. as Chief Executive Officer.

"We are excited to enter into a definitive merger agreement with NeuBase, a company with a powerful technology and pipeline that has the potential to address multiple unmet medical needs across a range of serious genetic diseases," said Jason Slakter, M.D., Chief Executive Officer of Ohr Pharmaceutical. "Following a comprehensive review of strategic alternatives, Ohr’s Board of Directors concluded that the proposed transaction with NeuBase is in the best interest of our stockholders. The proposed merger will provide an opportunity to create value as an innovative, science-driven company with a proprietary technology platform utilizing advanced gene silencing techniques. We intend to hold a special meeting of Ohr shareholders in the first half of 2019 to vote on this merger."

"The proposed merger with Ohr signals the next stage of growth for NeuBase," added Dr. Dietrich Stephan, Chief Executive Officer of NeuBase Therapeutics. "The company’s new therapeutic modality has the potential to address a wide range of germline and somatic diseases caused by inappropriate expression and change-of-function mutations of genes. Our technology has significant potential advantages over currently available antisense and small molecule approaches to gene silencing, including high selectivity for targets, cell membrane and blood brain barrier permeability, early data indicating no immune response and a low cost of goods. These characteristics are essential for scalability in addressing a wide range of genetic diseases, including cancer. We are initially developing this exciting platform for RNA gene silencing in Huntington’s disease and myotonic dystrophy, with additional future, high value RNA silencing indications."

NeuBase’s modular PATrOL technology platform is being developed to treat a multitude of rare genetic diseases. The systemically-deliverable PATrOL therapies have the potential to improve upon current gene silencing treatments by combining the advantages of synthetic small molecule approaches with the precision of antisense technologies. NeuBase’s development is currently focused on severe neurological disorders such as Huntington’s disease and myotonic dystrophy, where blood-brain barrier penetration and broad tissue distribution are critical. In some cases, such as Huntington’s disease, systemic administration may ameliorate both CNS and non-CNS pathology, a benefit that current intrathecally administered therapies cannot achieve.

About the transaction
On a pro forma basis and based upon the number of shares of Ohr common stock to be issued in the merger, current Ohr stockholders will own approximately 20% of the combined company and NeuBase stockholders will own approximately 80% of the combined company, after accounting for the additional NeuBase financing transaction. The actual allocation will be subject to adjustment based on Ohr’s and NeuBase’s cash balance at the time of closing and the amount of the additional financing consummated by NeuBase at or before the closing of the merger. Certain members and affiliates of the board of directors and management of Ohr and Neubase have indicated an intent to invest in the additional NeuBase financing transaction.

The proposed transaction has been approved by the board of directors of both companies. The merger is subject to the approval of Ohr shareholders at a special meeting of shareholders, which is expected to occur in the first half of 2019, along with the satisfaction or waiver of other customary conditions.

This communication does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.

Roth Capital Partners, LLC is acting as financial advisor to Ohr for the transaction and Troutman Sanders LLP is serving as legal counsel to Ohr. Allele Capital Partners, LLC at Tribal Capital Markets, LLC is acting as financial advisor and Paul Hastings LLP is serving as legal counsel to NeuBase.

Additional Information about the Merger and Where to Find It
In connection with the Merger, the Company intends to file relevant materials with the Securities and Exchange Commission (the "SEC"), including a registration statement on Form S-4 that will contain a prospectus, joint proxy and information statement. Investors and security holders of the Company and NeuBase are urged to read these materials when they become available because they will contain important information about the Company, NeuBase and the Merger. The joint proxy statement, information statement, prospectus, and other relevant materials (when they become available), and any other documents filed by the Company with the SEC, may be obtained free of charge at the SEC web site at www.sec.gov. In addition, investors and security holders may obtain free copies of the documents filed with the SEC by the Company by directing a written request to: Ohr Pharmaceutical, Inc., 800 Third Avenue, 11th Floor, New York, NY, Attention: Corporate Secretary. Investors and security holders are urged to read the joint proxy statement, prospectus and the other relevant materials when they become available before making any voting or investment decision with respect to the Merger.

This communication shall not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. No offering of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended.

Participants in the Solicitation
The Company and its directors and executive officers and NeuBase and its directors and executive officers may be deemed to be participants in the solicitation of proxies from the stockholders of the Company in connection with the proposed transaction. Information regarding the special interests of these directors and executive officers in the merger will be included in the joint proxy statement/prospectus referred to above. Additional information regarding the directors and executive officers of the Company is also included in the Company’s Annual Report on Form 10-K for the year ended September 30, 2018 and the proxy statement for the Company’s 2018 Annual Meeting of Stockholders. These documents are available free of charge at the SEC web site (www.sec.gov) and from the Company, Attn: Corporate Secretary, at the address described above.

Ohr Pharmaceutical financial results for the fiscal year ended September 30, 2018:

For the fiscal year ended September 30, 2018, the Company reported a net loss of approximately $13.2 million, or ($0.23) per share, compared to a net loss of approximately $23.8 million, or ($0.53) per share in the fiscal year ended September 30, 2017.
For the fiscal year ended September 30, 2018, total operating expenses were approximately $13.9 million, consisting of $3.6 million in general and administrative expenses, $4.3 million of research and development expenses, $1.1 million in depreciation and amortization, $0.7 million in loss on impairment of goodwill, $5.3 million in loss on impairment of intangible assets, and $1.2 million in gain on settlement of liabilities. This compares to total operating expenses of $23.8 million in the fiscal year ended September 30, 2017, comprised of approximately $5.3 million in general and administrative expenses, $17.4 million in research and development expenses, $1.2 million in depreciation and amortization, and $0.1 million in gain on settlement of liabilities.
At September 30, 2018, the Company had cash and cash equivalents of approximately $3.8 million, compared to cash and equivalents of approximately $12.8 million at September 30, 2017.

On January 3, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported its participation at the 37th Annual J.P. Morgan Healthcare Conference (Press release, Kura Oncology, JAN 3, 2019, View Source [SID1234532393]). Troy Wilson, Ph.D., J.D., President and Chief Executive Officer, is scheduled to present an overview of the company on Thursday, January 10, 2019 at 9:30 a.m. PT / 12:30 p.m. ET, followed by a Q&A session at 10:00 a.m. PT / 1:00 p.m. ET. The conference will be held from January 7-10, 2019 in San Francisco.

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Live audio webcasts of the presentation and Q&A session will be available in the Investors section of Kura’s website at www.kuraoncology.com, with an archived replay available for 30 days following the event.

On January 3, 2019 Aadi Bioscience, Inc. (Aadi), a clinical stage biopharmaceutical company focused on treating diseases driven by mTOR activation, reported that its drug TARZIFYX (sirolimus albumin-bound nanoparticles for injectable suspension, ABI-009) has received Breakthrough Therapy Designation status from the FDA for the indication of Advanced (metastatic or locally advanced) Malignant PEComa (perivascular epithelioid cell tumor) (Press release, Aadi, JAN 3, 2019, View Source [SID1234532410]). The designation was granted based on data provided to the FDA from Aadi’s ongoing phase 2 registration trial (NCT02494570) in Advanced Malignant PEComa. In October 2018, Aadi also received Fast Track Designation from the FDA for TARZIFYX for the same indication. Aadi received orphan drug designation for TARZIFYX for the treatment of PEComa in December 2017.

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Aadi’s Phase 2 registration trial for Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT), a rare form of sarcoma, has completed enrollment. Aadi previously received agreement from the FDA that this open label study in 30 efficacy evaluable patients with a primary endpoint of overall response rate could support the submission of an NDA for approval to treat this rare disease.

"We are extremely pleased with the progress of the AMPECT study and recognition of the high unmet need in this patient population via the Breakthrough Designation. Our small, but dedicated team is gearing up for NDA filing in 2019," said Neil Desai, Ph.D., Founder and Chief Executive Officer of Aadi. "There has been no prior clinical trial in this rare disease and standard chemotherapies used in sarcoma treatment have very limited activity in this patient population."

mTOR inhibition is relevant in a number of disease states and has not been fully exploited in oncology, cardiovascular disease, CNS diseases, mitochondrial disease, diseases of ageing, etc. Due to the unique properties of Aadi’s drug including effective CNS penetration, it is being tested in nine ongoing clinical studies across these indications with encouraging results even outside of oncology in early studies in pulmonary hypertension and surgically refractory epilepsy. Aadi’s ongoing and planned clinical trials include Oncology (pediatric tumors, advanced sarcoma, first-line treatment of advanced colorectal cancer, newly diagnosed and recurrent glioblastoma, advanced neuroendocrine tumors), Cardiovascular indications (pulmonary arterial hypertension), CNS indications (surgically refractory epilepsy) and Mitochondrial disease (Leigh Syndrome).

On January 3, 2019 Marker Therapeutics, Inc. (NASDAQ: MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported a year-end update in five clinical trials using the Company’s therapeutic products, LAPP and MAPP multi-antigen targeted T cell (MultiTAA) therapies and TPIV200, its Folate Receptor Alpha (FRα) peptide cancer vaccine product candidate (Press release, Marker Therapeutics, JAN 3, 2019, View Source [SID1234532426]).

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"We are pleased to provide an update on our progress in advancing clinical trials using our therapeutic platform," stated Peter L. Hoang, President & CEO of Marker Therapeutics. "With our MultiTAA cell therapies, we continue to build on the size and depth of our patient dataset. These updates now bring our total reported number of patients to 72, up from 57 in our previously reported results. I believe this represents one of the most extensive sets of clinical results in cell therapy for cancer treatment and illustrates the potential safety and clinical effects of MultiTAA T cells for patients suffering from a number of terrible cancers."

"In our vaccine program, we continue to demonstrate our commitment to excellence in our clinical execution," Mr. Hoang continued. "Last year when I joined the company, I expressed that we would work to improve our clinical efficiency, and I believe that the completion of enrollment of our FRV-004 study in ovarian cancer over six months ahead of schedule reflects our dedication to that objective. In fact, we have now completed enrollment of our last two clinical trials significantly ahead of projections, reflecting the commitment of our management and clinical team to execute multi-center studies effectively."

The Company reported clinical updates in three Baylor College of Medicine (BCM)-sponsored Phase I/II clinical trials using its MultiTAA T cell therapies, LAPP and MAPP:

Lymphoma

In the Phase I/II clinical trial in lymphoma, BCM has now treated 15 patients with active disease who have failed an average of four lines of prior therapy. Of these patients, five patients experienced transient disease stabilization followed by disease progression. Four patients have ongoing stable disease of between 9 to 24+ months following infusion of the MultiTAA-specific T cells, while the remaining six have all had complete and durable responses (4 months to 60+ months), as assessed by PET imaging.

No relapses observed to date for any patient entering a complete response (CR).
Patients with active disease who have ongoing complete responses after infusion of MultiTAA cells are now between 1 and 5 years in CR (ongoing).
Several patients with stable disease show potential for durable disease stabilization, with two patients observed to have stable disease for over 9 months and 24 months, respectively.
Responses in all six patients who entered a CR were associated with an expansion of infused T cells as well as the induction of antigen spreading.
None of the treated patients developed cytokine release syndrome, neurotoxicity or any other infusion-related adverse events.
BCM has also treated 17 patients who had developed a CR following their last treatment (adjuvant therapy) for lymphoma, and all but two of these patients remain in remission (3-42 months post-infusion). Monitoring has continued on all patients previously reported, and none of these patients have yet relapsed with disease. Average duration of remission for patients with a continuing complete response (CCR) is over 26 months (ongoing), versus 16 months (ongoing) as of the last patient update.

Multiple Myeloma

Marker Therapeutics also provided an update to the BCM-sponsored Phase I/II clinical trial in multiple myeloma. Results were presented at an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2018 Annual Meeting.

Ten patients with active disease were treated, including:
One patient with a CR durable for approximately 29 months before relapse, was subsequently given a second treatment infusion of MultiTAA T cells, resulting in stable disease for 3 months (ongoing) after the second treatment.
Two patients achieved partial responses (PR) of between 14 and 22 months (ongoing) as of last follow-up.
All seven remaining patients experienced stabilization of disease following infusion of MultiTAA cells initially. Three patients developed transient disease stabilization of between 3-7 months with subsequent progression, and four patients have ongoing stable disease.
Eight patients were treated in remission, with a median follow up of 21 months. Only one patient has relapsed to date.
Correlative studies show significant expansion of MultiTAA T cells, as well as significant evidence of epitope spreading with expansion of endogenous T cells specific for tumor-associated antigens that were not targeted by the MultiTAA product.
MultiTAA therapy appears to be safe and well-tolerated, with no incidence of cytokine release syndrome, neurotoxicity or any other serious adverse events related to the therapy.
Acute Lymphoblastic Leukemia

Marker Therapeutics also reported initial results from the BCM-sponsored Phase I clinical trial in acute lymphoblastic leukemia (ALL). In this study, patients were treated with MultiTAA T cells as a maintenance therapy for patients in CR post-allogeneic stem cell transplant. Leukemic relapse remains the major cause of treatment failure in hematopoietic stem cell transplant (HSCT) recipients.

10 patients have been enrolled and treated in this clinical trial, with eight patients evaluable for response. To date, all but one remains in CR, with patients ranging from 1 to 22 months in CCR (ongoing). Because of the highly refractory nature of these patients, the length of CCRs and the low rate of relapse amongst these patients, the Company believes that these early results are promising and may represent meaningful clinical benefit.
Marker Therapeutics also reported key updates from clinical studies of TPIV200, its Folate Receptor Alpha (FRα) peptide cancer vaccine product candidate.

Ovarian Cancer

Marker Therapeutics reported that it had completed enrollment in its Phase II study in ovarian cancer (Study FRV-004), using TPIV200 as a maintenance therapy for patients in their first remission after surgery and platinum-based chemotherapy. Marker has enrolled, randomized, and treated 120 patients at 17 clinical sites. The study completed enrollment six months faster than anticipated. The Company expects to reach its planned interim analysis trigger of 50 patients who have progressed by the end of the second quarter of 2019, with interim data reported by year end.

Enrollment of this study was completed over six months ahead of schedule, reflecting ongoing management initiatives to improve and enhance clinical operations efficiency.
Marker had previously projected the initiation of its interim analysis to begin in Q4 2018, triggered by the 50th patient to progress following treatment. Despite faster than expected enrollment of patients in this study, as of the end of December fewer than 50 patients had progressive disease. As a result, Marker now expects to reach its planned interim analysis trigger by end of the second quarter of 2019, with interim data reported by year end.
Triple Negative Breast Cancer

Marker Therapeutics also reported initial findings from its interim analysis of its dose-finding study (Study FRV-002) in patients with triple negative breast cancer, using TPIV200 as a maintenance therapy for patients in remission following first-line therapy. The four-arm study included low and high dose TPIV200 with or without cyclophosphamide.

Of 27 patients evaluated to date for immunogenicity, 26 showed significant immune response to the vaccine treatment. Of 80 patients treated at 11 clinical sites, 11 have shown disease progression to date following treatment with TPIV200.
"These additional clinical results strongly augment our existing, previously disclosed patient dataset. In patients who were treated for active disease in lymphoma, we continue to see long-lived, ongoing complete responses that are now durable beyond five years and have yet to observe a patient who achieves a CR subsequently relapse," said Dr. Richard Kenney, Acting Chief Medical Officer of Marker Therapeutics. "Notably, in the adjuvant lymphoma patients we have also not seen any additional relapses, with several patients now beyond four years in their continuing complete response. While the median progression-free survival has not yet been reached in any of these trials, observationally it appears that the time to progression for patients receiving MultiTAA T cell therapy may compare favorably with results reported in CD19 and BCMA-targeted CAR-T studies in lymphoma and multiple myeloma, without inducing the toxicities normally associated with gene-modified adoptive cell therapies."

"Given the highly refractory nature of the patients with acute lymphoblastic leukemia treated, we believe the preliminary results appear to be very promising, with only one patient having relapsed to date," continued Dr. Kenney. "These early results may indicate that MultiTAA therapy may be able to drive clinical benefit for these patients without the need for donor-lymphocyte infusions (DLIs), and the associated risk of graft versus host disease (GvHD). Finally, our clinical sites have been very supportive of our Phase II vaccine studies in ovarian and breast cancer, and their rapid enrollment is a credit to our Principal Investigators and clinical investigative sites, as well as our clinical operations team. We are pleased with the progress in building our clinical development infrastructure and believe we can leverage that experience to drive our upcoming MultiTAA T cell studies efficiently."