On March 21, 2018 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a
clinical-stage, immuno-oncology biopharmaceutical company focused on the acquisition, development
and commercialization of novel treatments for patients with solid tumor cancers, reported the
completion of the dose escalation portion of the ongoing Phase 1 clinical trial of CK-301, a fully human
anti-PD-L1 antibody, in selected recurrent or metastatic cancers, and the initiation of the first dose
expansion cohort, which is evaluating an 800 mg dose of CK-301 administered every two weeks (Press release, Checkpoint Therapeutics, MAR 21, 2018, View Source [SID1234525087]).
James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "The completion of the dose
escalation portion of our Phase 1 trial marks an important milestone in the clinical development of our
anti-PD-L1 antibody, CK-301. Our focus now shifts to generating efficacy data in the dose expansion
portion of the trial through the enrollment of patients with tumor types believed to have a high potential
for objective response to anti-PD-L1 monotherapy. We look forward to reporting initial data from this
expansion cohort in the second half of 2018, and are targeting the initiation of our first registration trial
in first-line non-small cell lung cancer in the first quarter of 2019."

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Preliminary data from the dose escalation portion of the Phase 1 trial suggest that CK-301 is safe and well
tolerated across three fixed dose levels ranging from 200 mg to 800 mg administered every two weeks.
Treatment-related adverse events were mild to moderate and consistent with other approved PD-L1
antibodies. No dose-limiting toxicities have been reported and no patients have discontinued therapy to
date.

Based on these data, Checkpoint has commenced its first dose expansion cohort evaluating the fixed dose
of 800 mg, the highest dose tested in dose escalation, every two weeks in up to 40 checkpoint therapynaïve
patients with select tumor types associated with high clinical response rates to anti-PD-1/L1
monotherapies, with a priority on enrolling first-line non-small cell lung cancer patients whose tumors
have high PD-L1 expression.
Additional information on the trial can be found on www.clinicaltrials.gov using the identifier
NCT03212404.

About the Phase 1 CK-301 Trial

The Phase 1, first-in-human, open-label, multicenter trial is evaluating the safety and tolerability of
ascending doses of CK-301 in checkpoint therapy-naïve patients with selected recurrent or metastatic
cancers. Secondary endpoints include the evaluation or characterization of the pharmacokinetics,
immunogenicity and preliminary efficacy of CK-301. Following dose escalation, up to four dose expansion
cohorts may be enrolled to further characterize the safety and efficacy of CK-301 in specific patient subgroups. The trial is currently enrolling patients at sites across Australia, New Zealand and Thailand.

On March 21, 2018 Cernostics, a privately-held company focused on delivering next-generation cancer diagnostics and prognostics, reported that it has raised $2.5 million in Series A1 financing, led by Illumina Ventures (Press release, Cernostics, MAR 21, 2018, View Source [SID1234525085]). With this capital investment, Cernostics will be able to intensify its clinical and market development studies for the company’s TissueCypher Barrett’s Esophagus Assay — the first test of its kind to predict risk of development of esophageal cancer in patients with Barrett’s esophagus.

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Barrett’s esophagus, which affects more than three million Americans, occurs when chronic exposure to acid from the stomach causes the esophageal cell lining to deteriorate, creating an environment for cancer. Without treatment, Barrett’s can lead to esophageal adenocarcinoma, the fastest-rising cancer in the U.S. Today, the most common approach to managing Barrett’s is surveillance, involving regular endoscopic procedures with biopsy, monitoring for disease progression, and GERD-related drug therapy to control symptoms and prevent injury to the esophagus.

Traditional management of Barrett’s esophagus has left gaps in the diagnosis and grading of the disease. While early detection makes esophageal cancer preventable, both endoscopists and pathologists face challenges in determining which patients are truly at risk for progression of the disease. Cernostics’ TissueCypher Barrett’s Esophagus Assay was specifically designed to address these challenges by linking test performance to clinical outcome or progression to cancer, not level of dysplasia. By validating TissueCypher against progression to cancer, the test helps to mitigate challenges associated with sampling error and subjective diagnosis of histology grade.

Esophageal cancer is highly lethal. By selecting the right candidates for treatment, we have tools to prevent people from getting this cancer," said Jacques Bergman, MD PhD, Professor of Medicine and Endoscopy, Academic Medical Center, Amsterdam. "Cernostics TissueCypher test is transformative for the gastroenterological field because it provides physicians with the precise information necessary to make the right decision about patient care."

Through the utilization of the TissueCypher Barrett’s Esophagus Assay and currently available endoscopic treatments there is no reason that a patient with Barrett’s esophagus should ever receive a diagnosis of esophageal cancer. We have the precision medicine tools and technology to transform the care of these patients today," said Mike Hoerres, Cernostics’ CEO. "With this investment by Illumina Ventures, Cernostics will accelerate efforts with GI community leaders and stakeholders to develop robust, transformative clinical evidence on the value of TissueCypher. Furthermore, we have a tremendous opportunity to impact a wider variety of GI diseases through development of additional precision medicine testing based on the TissueCypher Image Analysis Platform.

The best successes in reducing cancer deaths have been achieved through the use of precision molecular tools to identify cancer early, combined with effective early intervention, such as with cervical and colon cancer screening programs." said Tom Willis, Partner at Illumina Ventures. "The genomics revolution is ushering in a next generation of these tests, such as TissueCypher, and Illumina Ventures is seeking to enable their success.

With today’s investment from Illumina Ventures, Cernostics has raised a total of $13 million, including investments from UPMC Enterprises, Novitas Capital, Geisinger Health System, the Pittsburgh Life Sciences Greenhouse, and Ben Franklin Technology Partners of Northeastern PA.

On March 21, 2018 Avelas Biosciences, Inc., a clinical stage oncology-focused platform technology company that is developing products to advance a new standard-of-care for cancer surgery and therapeutic intervention, reported that Carmine N. Stengone, President and Chief Executive Officer, will present at the Needham & Company 17th Annual Healthcare Conference on Wednesday, March 28, 2018, at 1:30 p.m. EDT (10:30 a.m. PDT) in New York City (Press release, Avelas Biosciences, MAR 21, 2018, View Source [SID1234525080]).

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On March 21, 2018 Transgene (Paris:TNG), a biotech company that designs and develops virus-based immunotherapies, reported its financial results for 2017 and provided an update on the progress of its clinical pipeline, preclinical pipeline and its technology platforms (Press release, Transgene, MAR 21, 2018, View Source [SID1234524933]).

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Philippe Archinard, Chairman and Chief Executive Officer of Transgene, commented:
"In 2017, Transgene made significant progress across all aspects of its business and launched seven clinical trials. These studies are designed to demonstrate the potential of our therapeutic vaccine and oncolytic virus immunotherapeutics in combination with ICIs. As a result, we remain on track to communicate t clinical data on five products before the end of 2018.

"With the launch of our Invir.IOTM platform, we also confirmed our positioning at the forefront of innovation in the field of oncolytic viruses, an area of immense promise. With Invir.IOTM, we design novel virotherapies against cancer with multiple and complementary modes of action to better control the tumor microenvironment and to attack tumors much more effectively. The signings of collaborative agreements with BioInvent and Randox are the first steps of the acknowledgement of this potential. They cumulate with our ongoing clinical developments of Pexa-Vec and TG6002.

"Our expertise in both therapeutic vaccines and oncolytic viruses, confirmed by recent achievements, allows us to look at 2018 with confidence."

Clinical Pipeline Review

TG4010: combination trial with nivolumab (ICI); collaborations with Bristol-Myers Squibb

TG4010 is a therapeutic vaccine being developed for the treatment of advanced-stage non-squamous non-small cell lung cancer (NSCLC). TG4010’s mechanism of action, excellent safety profile and existing clinical data make it a very suitable candidate for combinations with other therapies.

Treating lung cancer remains an important medical need despite recent progress. There is a clear need to increase both the number of patients responding to treatment (response rate) and the duration of response to achieve better patient outcomes. New positive clinical data from the ICI pembrolizumab in first-line lung cancer patients were recently announced. As a result, we expect most first-line patients, particularly in the US, to receive an ICI as a monotherapy or in combination with chemotherapy depending on the level of PD-L1 expression of the patients’ tumor cells.
Transgene, with its company-sponsored first-line trial assessing TG4010 in combination with nivolumab and chemotherapy, is well positioned both in Europe and in the US to prosper in this changed environment. We continue to believe that the addition of TG4010 could be crucial in improving outcomes for first-line patients.

TG4010
+ Opdivo (ICI)
(nivolumab)
+ chemotherapy
Phase 2

Non-small cell lung cancer (NSCLC) – 1st line

Trial of TG4010 in combination with nivolumab and with chemotherapy in patients
whose tumor cells express low or undetectable levels of PD-L1; sponsor: Transgene

Collaboration deal signed in April 2017 with Bristol-Myers Squibb. BMS is supplying
Opdivo (nivolumab)
First patient treated in January 2018; trial recruiting in Europe and in the US
First results expected in 2H 2018
TG4010
+ Opdivo (ICI)
(nivolumab)
Phase 2

Non-small cell lung cancer (NSCLC) – 2nd line

Trial of TG4010 in combination with nivolumab, which is being provided by Bristol-Myers
Squibb, in a collaborative agreement with UC Davis Medical Center (USA); principal
investigator: Dr. Karen Kelly; sponsor: UC Davis

First patient treated in March 2017; trial’s 4 sites open in California
The use of ICIs in first-line therapy had led to slower recruitment into this second
line trial, as patients previously treated with ICIs are excluded. Results from the
interim analysis of this study are now expected in 2H 2018.
Pexa-Vec: ongoing Phase 3 trial, ongoing Phase 2 combination trials

Pexa-Vec is Transgene’s lead oncolytic virus drug candidate. It is designed to selectively target and destroy cancer cells through intracellular viral replication (oncolysis), and by stimulating the body’s immune response against cancer cells. Its mechanism of action and tolerability profile make it an appropriate candidate for use in combinations.

Advanced liver cancer remains an important medical need. The approaches currently being developed are aimed at increasing treatment response rates and improving overall survival. The registration of nivolumab in the US as a second-line treatment, together with existing data, suggest that patients could also respond positively to an ICI as a first-line treatment.
Transgene’s development of Pexa-Vec as a first-line treatment combined with both the current standard of care (sorafenib) and nivolumab, could play an important role in the future treatment of hepatocellular carcinoma.

Pexa-Vec
+ sorafenib
(PHOCUS)
Phase 3

Advanced liver cancer (hepatocellular carcinoma – HCC) – 1st line

Clinical trial being conducted by Transgene’s partner, SillaJen (sponsor)
Ongoing recruitment. First patient treated in Europe in April 2017
Trial recruitment authorized in China (July 2017)
First data readout expected in 2019
Pexa-Vec
+ Opdivo (ICI)
(nivolumab)
Phase 2

Advanced liver cancer (hepatocellular carcinoma – HCC) – 1st line

Principal investigator: Prof. Olivier Rosmorduc (AP-HP, Paris); sponsor: Transgene
First patient treated in July 2017; several active trial sites
First data readout expected in 2H 2018
Pexa-Vec is also being developed for the treatment of other solid tumors.
Ongoing trials are expected to deliver results in 2018.

Pexa-Vec
+ metronomic
cyclophosphamide
Phase 1/2a

HER2 negative breast cancer and soft tissue sarcoma (METROmaJX)

Principal investigator: Prof. Antoine Italiano (Institut Bergonié, Bordeaux);
sponsor: INCa
Positive results from the Phase 1 part were presented at ESMO (Free ESMO Whitepaper) 2017 (Sept. 2017)
Recruitment ongoing
Pexa-Vec
+ Yervoy (ICI)
(ipilimumab)
Phase 1

Solid tumors (ISI-JX)

Coordinating investigator: Dr. Aurélien Marabelle; sponsor: Centre Léon Bérard, Lyon
First patient treated in February 2017
Recruitment ongoing
Pexa-Vec
Neo-adjuvant

Solid tumors

Principal investigator: Prof. Alan Anthoney; sponsor: University of Leeds (UK)
Recruitment completed
Our partner SillaJen has also published clinical data that further demonstrate the potential of Pexa-Vec. It presented a poster at ASCO (Free ASCO Whitepaper) GU featuring the results of a clinical trial in patients with renal cell carcinoma and liver metastases (n=17). The disease control rate reached 76%, with one complete response, confirming that Pexa – Vec, administered intravenously as a monotherapy, can induce antitumor activity.

TG4001: trial in combination with avelumab (ICI); collaboration agreement with Merck KGaA and Pfizer

TG4001 is a therapeutic vaccine that has demonstrated good tolerability, a significant HPV clearance rate and promising efficacy results. Its mechanism of action and good safety profile make TG4001 a good vaccine candidate to be combined with other therapies.

TG4001
+ Bavencio (ICI)

(avelumab)
Phase 1/2

HPV-positive head and neck cancer – 2nd line

Clinical collaboration agreement with Merck KGaA and Pfizer, for the supply of
avelumab for the trial
Principal investigator: Prof. Christophe Le Tourneau (Institut Curie, Paris);
sponsor: Transgene
First patient treated in September 2017; several sites recruiting
First results expected in 2H 2018
TG6002: first-in-human trial started

A new-generation oncolytic immunotherapy, TG6002 has been designed by Transgene to combine the mechanism of oncolysis (targeted breakdown of cancer cells) with local production of the chemotherapy (5-FU) directly in the tumor. This approach aims at attacking solid tumors on several fronts while avoiding the chemotherapy-associated side effects.

TG6002
Phase 1

Glioblastoma

Principal investigator: Prof. Ahmed Idbahi (AP-HP, Paris), with the support of INCa
(French national cancer institute); sponsor: AP-HP
First patient treated in October 2017
TG6002
Phase 1

Gastro-intestinal adenocarcinoma

First IND request filed for this trial; sponsor: Transgene
TG1050: First part of the results featured at the AASLD in October 2017

TG1050 is a therapeutic vaccine designed for the treatment of chronic hepatitis B. TG1050’s technology (T101) is being developed in China through the joint-venture between Transgene and Tasly Biopharmaceutical Technology.

TG1050

+ Standard of care
Phase 1/1b

Chronic hepatitis B

First part of the results featured at the AASLD (October 2017)
Good safety profile confirmed (single and multiple doses)
Initiation in January 2018 of a clinical study in China assessing T101
Full results available in 2H 2018
Invir.IOTM: New Industry Leading OV Platform

In September 2017, Transgene announced the launch of Invir.IO a patented cutting-edge technology platform designed to generate a new generation of multifunctional oncolytic viruses capable of enhancing the tumor micro-environment’s modulation. Novel oncolytic virus therapeutics based on Invir.IO have the capacity to incorporate several transgenes encoding for a range of specific anticancer weapons. By using this approach and working alongside external collaborators to provide access to clinically relevant transgenes, Transgene aims to develop OV therapeutics that can transform the treatment of cancer.

Transgene has already demonstrated that oncolytic viruses from the Invir.IO platform attack tumors on several fronts and, in addition to the remarkable oncolytic properties of Vaccinia viruses, may:

induce immunogenic death of cancer cells, and
allow the expression of several anticancer weapons such as cytokines, chemokines, enzymes, monoclonal antibodies or mini-antibodies (SdAbs, single-domain antibodies) in the tumor.
Transgene is currently evaluating 10 preclinical Invir.IO OVs to identify the most appropriate candidates to take into clinical development to address a number of priority indications.

To complement its in-house expertise, Transgene signed two collaborative research agreements in 2017 to gain access to partners’ transgene sequences, coding for anticancer agents, to be incorporated into an Invir.IO patented oncolytic virus owned by Transgene. The resulting oncolytic viruses have the potential to be significantly more effective than the combination of these agents administered separately:

Incorporating an anti-CTLA-4 antibody from BioInvent alone or together with other anticancer weapons. The local expression of anti-CTLA-4 antibodies in the tumor should reduce Treg mediated-immunosuppression in the tumor, thus increasing antitumor activity. This approach is expected to have a much better safety profile compared to the systemic use of anti-CTLA-4 antibodies;
Incorporating one or more SdAbs generated by Randox in order to combine the effects of oncolytic viruses with the therapeutic properties of SdAbs, which will be expressed directly in the tumor micro-environment, in order to directly or indirectly stimulate effector cells in solid tumors.
In 2018, Transgene plans to invest approximately two thirds of its preclinical research spending on its Invir.IO platform and OV candidates. Transgene expects to obtain preclinical proof of concept for its lead candidates OVs in 2018 and to be in a position to initiate the first clinical trials with Invir.IO designed virotherapies in 2019.

Research Collaboration with Servier

In June 2017, Transgene signed a collaboration agreement with Servier to generate an original engineering process of allogeneic CAR-T cell therapies by applying Transgene’s viral vectorization technology and capabilities. The aim is to improve the engineering performance of the process by reducing the number of steps.Transgene received €1 million as upfront payment. The collaboration also generates R&D service fees as well as potential success fees.

Intellectual Property

In 2017, Transgene filed several patent applications on new technologies, including Invir.IOTM developments.More than 20 patents were granted and ensure the protection of Transgene’s innovations.

Key Financials for 2017

Net cash burn for 2017 was reduced to €28.1 million compared to €30.6 million in 2016.
Cash available at year-end 2017: €41.4 million, compared to €56.2 million at the end of 2016. This cash balance includes €13.5 million (net) raised from a private placement concluded in November 2017.
Net operating expenses of €36.0 million in 2017, compared to €33.0 million in 2016.
Net loss of €32.2 million in 2017, compared to a loss of €25.2 million in 2016.
"We were able to reduce our cash burn in 2017 despite accelerating our development plan, starting numerous clinical trials, and making a planned milestone payment to SillaJen of €3.8 million. Operating costs remain under good control which has allowed the Company to allocate most of its financial resources to strategic clinical and preclinical operations. Our current cash resources, including the funds we raised in the private placement in November 2017, will allow us to deliver a number of important clinical milestones in the second half of 2018," said Jean-Philippe Del, Vice President, Finance.The financial statements for 2017 as well as management’s discussion and analysis are attached to this press release (Appendices A and B).

On March 21, 2018 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer and wet age-related macular degeneration, reported that Charles Theuer, M.D, Ph.D., President and CEO, will present a corporate update at the 17th Annual Needham Healthcare Conference at 2:30 pm EDT on Wednesday, March 28, 2018, at the Westin Grand Central Hotel in New York, NY (Press release, Tracon Pharmaceuticals, MAR 21, 2018, View Source [SID1234524941]).

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To access a live webcast of the presentation, please visit the "Events and Presentation" section within the "Investors" section of the TRACON Pharmaceuticals website at www.traconpharma.com. A replay of the webcast will be available on the website for 60 days following the event.