On May 14, 2018 Aradigm Corporation (NASDAQ: ARDM) (the "Company") reported financial results for the first quarter and three months ended March 31, 2018 (Press release, Aradigm, MAY 14, 2018, View Source [SID1234526609]).

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First Quarter 2018 Financial Results

The Company recorded $1.5 million in revenue in the first quarter of 2018 compared with $1.7 million in revenue in the first quarter of 2017. The Company recognized $1.3 million in contract revenue – related party, $29,000 in government contract revenue and $129,000 in government grant revenue for the first quarter of 2018, as compared to $1.6 million in contract revenue – related party, $39,000 in government contract revenue and $31,000 in government grant revenue for the first quarter of 2017.

Total operating expenses for the first quarter of 2018 were $5.3 million, compared with total operating expenses of $4.5 million for the first quarter of 2017. The increase in expenses was related to the FDA Advisory Committee Meeting. General and administrative costs were unchanged. Research and development expenses increased $800,000. In the first quarter of 2018, our research and development expenses were higher due to higher consulting costs related to the FDA meeting and the submission fees for our MAA application as well as severance expenses. This increase in expenses was offset by lower costs for clinical expense and lower employee related expenses due to a reduction in headcount. The receipt of a tax incentive in Australia offset a portion of the research and development expenses in the first quarter of 2017.

Net loss for the first quarter of 2018 was $4.8 million or $0.32 per share, compared with a net loss of $3.7 million or $0.25 per share in the first quarter of 2017. The increase in net loss resulted primarily from an increase in operating expenses of $800,000 related to the FDA meeting in January and the submission of the MAA to EMA in March for approval of Linhaliq. A decrease in revenue of $200,000 and an increase in interest expense of $100,000 related to the convertible notes was recorded.

Liquidity and Capital Resources and Related Matters

As of March 31, 2018, the Company’s cash and cash equivalents totaled $1.4 million.

In January, Aradigm received a Complete Response Letter (CRL) from the FDA regarding the New Drug Application (NDA) for Linhaliq as a treatment for non-cystic fibrosis bronchiectasis (NCFBE) patients with chronic lung infections with Pseudomonas aeruginosa (P. aeruginosa).

The CRL states that the FDA has determined that it cannot approve the NDA in its present form and provides specific reasons for this action along with recommendations needed for resubmission; the areas of concern include clinical data, human factor validation study and product quality.

The Aradigm Board of Directors approved temporary measures on February 9, 2018 intended to preserve the Company’s cash resources.

In April Aradigm raised $2.0 million through the issuance of bridge notes and obtained commitments for additional monthly funding through September of 2018 totaling $5 million. This $7.0 million along with the cash balance of $1.4 million will be sufficient to fund operations through the third quarter of 2018.

Aradigm is pursuing potential alternatives to resolve our cash position in the short term as well as developing strategic options that would provide for our long term viability. We feel it is very important to bring Linhaliq to commercialization in as many geographies as possible to allow patients suffering from non-cystic fibrosis bronchiectasis (NCFBE) to receive the benefits of Linhaliq. Patients, patient advocacy groups and key opinion leaders have expressed support as we work towards this goal. The MAA was filed in early March and validated for review in late March and that is the first step in achieving regulatory approval in Europe.

About Non-Cystic Fibrosis Bronchiectasis

NCFBE is a severe, chronic and rare disease characterized by abnormal dilatation of the bronchi and bronchioles, frequently associated with chronic lung infections. It is often a consequence of a vicious cycle of inflammation, recurrent lung infections, and bronchial wall damage. NCFBE represents an unmet medical need with high morbidity and mortality that affects more than 150,000 people in the U.S. and over 200,000 people in Europe. There is currently no drug approved for the treatment of this condition

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 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported that members of the management team will present at the following upcoming investor conferences (Press release, Deciphera Pharmaceuticals, MAY 14, 2018, View Source [SID1234526560]):

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UBS Global Healthcare Conference on Monday, May 21, 2018 at 8:30 AM ET. The conference is being held at the Grand Hyatt New York in New York City.
Jefferies Global Healthcare Conference on Thursday, June 7, 2018 at 1:30 PM ET. The conference is being held at the Grand Hyatt New York in New York City.
A live webcast of the events will be available on the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay of the webcasts will be archived on the Company’s website for 90 days following the presentation.

On May 14, 2018 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology, reported new scientific advisors in immuno-oncology and autoimmunity (Press release, Intellia Therapeutics, MAY 14, 2018, View Source [SID1234526584]). The advisors hail from prestigious international institutions and collectively have both scientific and clinical expertise in cell therapies in these areas.

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"The advisors we’ve assembled include researchers and physicians who are luminaries in their fields," said Intellia President and Chief Executive Officer John Leonard, M.D. "Pursuing both in vivo and ex vivo pipelines, Intellia has a broad spectrum approach to genome editing in a variety of therapeutic applications using our CRISPR/Cas9 technology. We look forward to working alongside these experts and benefiting from their deep experience and insight, to drive development of our wholly owned ex vivo programs in immuno-oncology and autoimmunity."

The following scientific leaders and clinicians are Intellia’s advisors on ex vivo cell therapy in immuno-oncology:

Evren Alici, M.D., Ph.D., assistant professor and group leader, hematology, Karolinska Institutet, Sweden

Chiara Bonini, M.D., Ph.D., full professor, Università Vita-Salute San Raffaele; deputy director, Division of Immunology, Transplantation and Infectious Diseases, Ospedale San Raffaele; and head, Experimental Hematology Unit, Ospedale San Raffaele, Italy

Daniel DeAngelo, M.D., Ph.D., associate professor of medicine, Harvard Medical School, and director, clinical and translational research, adult leukemia, Dana Farber Cancer Institute, United States

Saar Gill, M.D., Ph.D., assistant professor of medicine, Center for Cellular Immunotherapies, University of Pennsylvania, United States

Johanna Olweus, M.D., Ph.D., full professor and head, Department of Cancer Immunology, University of Oslo, and director, K.G. Jebsen Center for Cancer Immunotherapy, Norway

E. John Wherry, Ph.D., Richard and Barbara Schiffrin President’s Distinguished Professor of Microbiology, and director, Institute for Immunology, University of Pennsylvania, United States

Juan Carlos Zúñiga-Pflücker, Ph.D., professor and chair, Department of Immunology, University of Toronto, and senior scientist, Sunnybrook Research Institute, Canada
These experts are Intellia’s advisors on ex vivo cell therapy in autoimmunity:

Laurence Turka, M.D., chief scientific officer, Rheos Medicines; deputy director, Immune Tolerance Network; and professor of surgery and medicine (part-time), Massachusetts General Hospital and Harvard Medical School, United States

Kathryn Wood, D.Phil., F.Med.Sci., emeritus professor of immunology, Nuffield Department of Surgical Sciences, University of Oxford, United Kingdom

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.