On May 14, 2018 CStone Pharmaceuticals (CStone) reported dosing of the first patient in a Phase I clinical trial in Australia for CS1003, an investigational anti-programmed death-1 (PD-1) monoclonal antibody (mAb) (Press release, CStone Pharmaceauticals, MAY 14, 2018, View Source [SID1234526640]). The open-label, first-in-human (FIH) study is initiated at Scientia Clinical Research Ltd in Australia and will investigate the safety, tolerability, and preliminary efficacy of CS1003 in patients with advanced solid tumors.

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"We are excited to announce initiation of clinical development for PD-1 inhibitor CS1003, our second pipeline candidate to enter Phase I studies in Australia after the CTLA-4 mAb CS1002 within one month," said Dr. Frank Jiang, Chief Executive Officer at CStone. "CS1003 is cross-reactive with human and mouse PD-1 which enables quick pre-clinical proof of concept experiments in combination with novel targets, leading to global first-in-combination potential. Because of this unique feature, CS1003 is critical to CStone’s combination strategy in cancer immunotherapy."

The launch of clinical studies for CS1003 is a key milestone for CStone, which now has three important checkpoint inhibitors for cancer therapy under clinical development. Alongside CS1001 (PD-L1) and CS1002 (CTLA-4), CS1003 will provide the backbone for CStone’s pipeline development of oncology combination therapies.

As noted by Dr. Jason Yang, Chief Medical Officer of CStone, "In preclinical studies, CS1003 demonstrated high affinity and selectivity for PD-1, as well as synergistic anti-tumor effects with multiple small-molecule drugs in animal models. The launch of this clinical program will allow us to gather important safety and efficacy data on CS1003. This will lay the foundation for future development of this molecule, in particular as the basis of combination therapies."

"We are excited to be enrolling our first patient treated with CS1003 and are hopeful that this novel immunotherapy drug will add to the available therapeutic options for advanced-stage tumors, either by itself or in combination with other drugs," said Dr. Charlotte Lemech, MBBS, BSc, FRACP, MD, lead investigator for this trial at Scientia Clinical Research Ltd.

The Phase Ia/Ib study includes a dose-escalation stage followed by a dose-expansion stage. Additional information on the trial can be found here.

About CS1003 and the PD-1/PD-L1 pathway

CS1003 is a humanized anti-PD-1 IgG4 monoclonal antibody developed by CStone using an internationally leading hybridoma platform. CS1003 has shown good tolerability and efficacy profile in preclinical in vivo studies.

PD-1, or programmed death-1, is an inhibitory checkpoint receptor expressed on T cells. Under normal circumstances, it binds with its ligands, programmed death ligand-1 or ligand 2 (PD-L1/PD-L2), inhibiting T cell and cytokine activation, serving to dampen the immune response in order to prevent damage to healthy tissues. However, studies have shown that PD-L1 can be abundantly expressed on the surface of many solid tumors as well as hematological malignancies. Cancer cells can therefore make use of the PD-1/PD-L1 pathway to successfully avoid immune system recognition and attack. Targeting of the PD-1/PD-L1 checkpoint by anti-tumor drugs can block the "tumor immune evasion mechanism" and restore anti-cancer immune ability in patients.

Currently, there are two anti-PD-1 antibodies approved globally: Opdivo (nivolumab) from Bristol-Myers Squibb and Keytruda (pembrolizumab) from Merck, Sharp & Dohme. Unlike other anti-PD-1 mAbs, CS1003 recognizes both human and murine PD-1, providing a unique competitive advantage during efficacy testing in syngeneic mouse tumor models particularly for development of effective combination therapies.

On May 14, 2018 GT Biopharma Inc. (OTCQB: GTBP)(Euronext Paris GTBP.PA) reported the promotion of Dr. Raymond W Urbanski MD, PhD to the position of President and Chief Medical Officer effective immediately. Dr. Urbanski will report to Shawn Cross, the Company’s Chief Executive Officer (Press release, GT Biopharma , MAY 14, 2018, View Source [SID1234539531]).

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"I am pleased to announce the promotion of Dr. Urbanski to President and Chief Medical Officer. The combination of Ray’s experience as a practicing physician and subsequently experience in industry, where he has served in key leadership positions including serving as the Chief Medical Officer of one of Pfizer’s business units and as the Chief Medical Officer of Mylan. His training, depth of knowledge and experience as well as his organizational acumen has been invaluable as we prepare GT Biopharma for our next stage of growth," said Shawn M. Cross, Chairman and Chief Executive Officer of GT Biopharma. "In addition to pushing forward our pre-clinical and clinical product candidates, Ray has played a critical role in advancing other company initiatives including recruiting experienced members to our scientific advisory board and board of directors, implementing internal processes and procedures, which are less visible but very important, as progress towards certain goals including a NASDAQ up-listing, among others. In short, I am delighted to have Ray as a senior member of our leadership team."

Since joining the company in October 2017 Dr. Urbanski has been instrumental in driving key milestones and initiatives including the transitioning the first TriKE IND from the University of Minnesota to GT Biopharma while engaging the FDA in preparation for human clinical testing to begin in 2H 2018; implementing processes to expedite the identification and development of future tumor antigen targets; driving forward our Bi-specific Antibody Drug Conjugate platform which included the formation of our Antibody-Drug Conjugate Clinical Advisory Board. Dr. Urbanski has also been a major factor in developing a strong working relationship with the University of Minnesota, Masonic Cancer Center, the epicenter of innovation for the TriKE and TetraKE platforms.

Dr. Urbanski also represents the company at key international meetings such as ASH (Free ASH Whitepaper) and the upcoming ASCO (Free ASCO Whitepaper) conferences, attending investor conferences and recruiting top tier Scientific Advisory Board members and consultants.

On May 14, 2018 Intrexon Corporation (NYSE: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported Lt. General (Ret.) Thomas P. Bostick, PhD, PE, Dist.M. ASCE, NAE, Intrexon’s Chief Operating Officer, and Thomas E. Shrader, PhD, CFA, Vice President, Communications and Strategy, will participate in a fireside chat session at the Bank of America Merrill Lynch 2018 Healthcare Conference at Encore at the Wynn, Las Vegas, Nevada on Tuesday, May 15th, at 5:00 p.m. Pacific Time (Press release, Intrexon, MAY 14, 2018, View Source [SID1234526562]).

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On May 14, 2018 Molecular Templates, Inc. (Nasdaq:MTEM) ("Molecular"), a clinical-stage oncology company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer, reported financial results for the first quarter of 2018 (Press release, Molecular Templates, MAY 14, 2018, View Source [SID1234526587]). As of March 31, 2018, cash and cash equivalents totaled $49.3 million. Molecular’s current cash balance is expected to fund operations into late 2019.

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"We are very pleased with the clinical results we have generated to date for MT-3724, which we expect to enter Phase II studies in relapsed/refractory DLBCL patients in the second half of 2018," said Eric Poma, Ph.D., CEO and CSO of Molecular Templates. "In the next twelve months, we expect our clinical pipeline to expand significantly as we file INDs for MT-4019 as well as our ETBs targeting HER2 and PD-L1."

Company Highlights and Upcoming Milestones

Corporate

At the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in April 2018, preclinical data were presented for Molecular’s ETBs targeting PD-L1 (which incorporates Molecular’s Antigen Seeding Technology – a differentiated immune-oncology approach) and HER2.
On March 2, 2018, Molecular closed a $10 million debt facility with Perceptive Advisors. The proceeds were used to repay an existing debt facility with Silicon Valley Bank and will support Molecular’s build out of its GMP manufacturing facility, to support Molecular’s own pipeline as well as partnerships. The first tranche of $5 million was received in 1Q18 and the second tranche of $5 million is due to be received in 3Q18.
MT-3724

MT-3724 (an ETB targeting CD20) is in an ongoing Phase Ib expansion study intended to better define the single agent overall response rate in heavily pre-treated diffuse large B-cell lymphoma (DLBCL) patients with additional updates expected in 2Q18.
Molecular also expects to initiate Phase II combination studies with MT-3724 in earlier lines of DLBCL in 2H18.
MT-4019

MT-4019 (an ETB candidate designed to target CD38-expressing myeloma cancer cells) is progressing through IND enabling studies.
Takeda and Molecular are evaluating CD38 ETBs and could potentially select a drug candidate for development by the end of 3Q18. If the two companies do not select a joint candidate for development, Molecular anticipates filing an IND application for MT-4019 in 3Q18 and initiating a Phase I clinical trial in 2H18.
Research

Molecular expects to file an IND application for an ETB targeting HER2 in 4Q18.
Molecular expects to file an IND application for an ETB targeting PD-L1 (with antigen seeding) in 1Q19.
Several other ETB candidates are in pre-clinical development, targeting both solid and hematological cancers.
Takeda Multi-Target Collaboration

In December 2017, Takeda selected two targets for further research using Molecular’s ETBs. This triggered $4 million in milestone payments, which were paid by Takeda in 2Q18.
Financial Results

The net loss attributable to common shareholders for the first quarter was $8.7 million, or $0.32 per basic and diluted share. This compares with a net loss attributable to common shareholders of $1.6 million, or $7.56 per basic and diluted share for the same period in 2017.

Revenues for the first quarter of 2018 were $0.5 million, compared to $1.9 million for the same period in 2017. Revenues for the first quarter of 2018 and 2017 were comprised of grant revenue from the Cancer Prevention & Research Institute of Texas, and revenues from collaborative research and development agreements. Total research and development expenses for the first quarter of 2018 were $6.7 million, compared with $1.1 million for the same period in 2017. Total general and administrative expenses for the first quarter of 2018 were $2.9 million, compared with $1.8 million for the same period in 2017.

On May 14, 2018 Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies based on its novel, universal Antibody-Coupled T-cell Receptor (ACTR) technology platform, reported financial results and provided a corporate update for the first quarter ended March 31, 2018 and recent activities (Press release, Unum Therapeutics, MAY 14, 2018, View Source [SID1234526563]).

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"Following our successful initial public offering in April 2018 and concurrent private placement, we are in a strong financial position to continue developing our proprietary, universal ACTR technology platform and rapidly advancing our pipeline of cellular immunotherapies through clinical development," said Chuck Wilson, CEO of Unum. "We are currently evaluating the potential of ACTR in combination with different tumor-targeting antibodies, in three ongoing multi-center Phase I trials, ATTCK-20-2 and ATTCK-20-03 evaluating ACTR087 and ACTR707, respectively, in combination with rituximab in patients with CD20+ r/r Non-Hodgkin Lymphoma (NHL), and ATTCK-17-01 evaluating ACTR087 in combination with SEA-BCMA in patients with r/r multiple myeloma. We expect to report preliminary data from these three trials late this year. In the second half of 2018 we also look forward to filing an IND and initiating clinical development of ACTR707 in combination with trastuzumab for the treatment of patients with HER2+ advanced cancers, our first solid tumor product candidate."

Recent Business Highlights and Outlook

Successfully Completed IPO and Concurrent Private Placement: In April, 2018, Unum successfully completed an initial public offering (IPO) of 5,985,000 shares of common stock at a public offering price of $12.00 per share, including the exercise by the underwriters of 215,000 shares of their overallotment option, raising $66.8 million in net proceeds. In addition, with a private placement concurrent with the IPO, Seattle Genetics, Inc. purchased $5.0 million shares of common stock at the initial public offering price. The proceeds from the IPO and the concurrent private placement will be used primarily to advance Unum’s four lead ACTR development candidates.

Initiated Cohort Expansion Phase of ATTCK-20-2 Phase I trial; Plans to Expand Clinical Development: In May 2018, Unum initiated the cohort expansion phase of the ATTCK-20-2 trial evaluating safety and anti-lymphoma activity of ACTR087 at the preliminary recommended phase 2 dose (RP2D) level used in combination with rituximab in patients with CD20+ r/r NHL. Unum expects to report updated data, including preliminary data from this phase of the ATTCK-20-2 trial, in the fourth quarter of 2018.

These data will also inform the strategy for a planned multi-center Phase II clinical trial exploring ACTR T cells used in combination with rituximab in patients with CD20+ r/r NHL who received prior CD19 CAR T cell therapy.

In addition, Unum intends to file a protocol amendment to the ATTCK-20-2 trial in the second half of 2018 to explore ACTR087 in combination with an alternative rituximab dosing regimen from that currently being studied. Preclinical experiments have shown that the level of ACTR T cell activity depends upon the amount of the co-administered antibody. As such, ACTR087 safety and anti-tumor activity in combination with rituximab in CD20+ r/r NHL may be even further optimized by an alternative rituximab regimen. Testing the alternative regimen will complement the clinical data being generated to support additional clinical trials with the combination.

Initiated Patient Enrollment and Dosing in ATTCK-17-01 Phase I trial: In the first quarter, Unum initiated patient enrollment ATTCK-17-01, a Phase I, multi-center, open-label clinical trial designed to test the safety, tolerability, and anti-myeloma activity of ACTR087 used in combination with SEA-BCMA in patients with r/r multiple myeloma. Unum is currently enrolling and dosing patients in this trial and expects to report preliminary data in the fourth quarter of 2018.

Continued Enrollment in ATTCK-20-03 Phase I trial: In the fourth quarter of 2017, Unum initiated patient enrollment in a Phase I, multi-center, open-label clinical trial called ATTCK-20-03, evaluating the safety, tolerability, and anti-lymphoma activity of ACTR707 used in combination with rituximab in patients with CD20+ r/r NHL. Unum has completed enrollment in the first dose level of this ongoing dose escalation study and expects to report preliminary data from the trial in the fourth quarter of 2018.

On Track to File IND for First Solid Tumor ACTR Product Candidate in the Second Half of 2018: Unum is on track to file an IND in the second half of 2018 for ACTR707 in combination with trastuzumab for the treatment of patients with HER2+ advanced cancers.
First Quarter 2018 Financial Results

Collaboration Revenue: Collaboration revenue recognized during the three months ended March 31, 2018 and 2017 of $2.2 million and $1.8 million, respectively, reflects the recognition of a portion of the $25.0 million upfront payment received from Seattle Genetics under Unum’s collaboration agreement as well as reimbursements of research and development costs by Seattle Genetics. Effective January 1, 2018, Unum adopted the new revenue recognition standard, ASC 606, which changed the manner in which the Company recognizes revenue from this collaboration agreement.

R&D Expenses: Research and development expenses were $8.1 million for the three months ended March 31, 2018, compared to $7.0 million for the same period last year. The increase reflects higher clinical trial costs for the three active Phase I clinical trials, as well as increased personnel-related costs, materials and facility-related costs related to scaling manufacturing processes, and increased consultant costs. This was partially offset primarily by a decrease in consulting and manufacturing costs incurred for the Phase I clinical trial of ACTR087 in combination with rituximab as there was no production activity in the first quarter of 2018.

G&A Expenses: General and administrative expenses for the three months ended March 31, 2018 were $1.1 million, compared to $0.9 million for the prior year period.

Net Loss: Net loss attributable to common stockholders was $6.8 million, or $0.66 per share, for the three months ended March 31, 2018, and $6.0 million, or $0.58 per share, for the three months ended March 31, 2017.

Cash, Cash Equivalents and Marketable Securities: As of March 31, 2018, Unum had cash, cash equivalents, and marketable securities of $32.4 million. This amount does not include the approximately $66.8 million in net proceeds from its IPO in April 2018, $5.0 million from the concurrent private placement, and available borrowings under its loan and security agreement of $15.0 million. The Company believes that the net proceeds from the IPO and concurrent private placement, together with its existing cash, cash equivalents, and marketable securities, will fund operating expenses and capital expenditure requirements through at least December 2019, without considering available borrowings under its loan and security agreement.