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Cellular Biomedicine Group Reports Third Quarter of 2019 Financial Results and Business Highlights

On November 6, 2019 Cellular Biomedicine Group Inc. (NASDAQ: CBMG) ("CBMG" or the "Company"), a biopharmaceutical firm engaged in the drug development of immunotherapies for cancer and stem cell therapies for degenerative diseases, reported its financial results and business highlights for the third quarter of 2019 (Press release, Cellular Biomedicine Group, NOV 6, 2019, View Source [SID1234550512]).

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"During the third quarter of 2019, we made great strides in both corporate and clinical progress. We started our U.S. expansion for research and clinical development in a new 22,000 square foot facility in Rockville, Maryland in October 2019. This milestone will allow us to foster strategic partnerships, develop new innovations and support continued development of CBMG’s cell therapy-based immune-oncology assets that have shown promise in early proof-of-concept trials in China," said Tony (Bizuo) Liu, Chief Executive Officer for the Company.

"We also had continued progress on the clinical side, with the initiation of our Phase II clinical trial in China of AlloJoin therapy for knee osteoarthritis (KOA). Additionally, our autologous stem cell therapy program for KOA, ReJoin, was accepted by the NMPA in China to begin a Phase II clinical trial. We are excited about our regenerative medicine programs as we are currently the only company that has received two clinical trial acceptances for any stem cell program in China."

Mr. Liu continued, "Presentations of our pre-clinical and clinical data at upcoming medical conferences later this year will demonstrate continued focus on our immune-oncology pipeline and we are proud to provide an update of our commitment to cancer immunotherapy."

Third Quarter 2019 and Other Recent Corporate Developments

New facility to expand research and development and to support clinical development in Rockville, MD
Initiation of AlloJoin therapy for multiple-site Knee Osteoarthritis Phase II clinical trial
ReJoin therapy received stem cell drug application acceptance for Phase II clinical trial by NMPA
Upcoming Clinical and Preclinical Presentations:

Society of Immunotherapy in Cancer ("SITC") 34th Annual Meeting
Poster Presentation
The Next Generation "Off-The-Shelf" Universal CAR For Adoptive Immunotherapy (Abstract ID: P229)
Friday, November 8, 2019 – 7:00 AM – 8:00 AM ET
Gaylord National Hotel & Convention Center, National Harbor, MD
American Society of Hematology ("ASH") 61st Annual Meeting and Exposition
Oral Presentation
Developing a Novel Anti-BCMA CAR-T For Relapsed or Refractory Multiple Myeloma (Submission ID: 125372)
Saturday, December 7, 2019 – 7:45 AM – 8:00 AM ET
Orange County Convention Center (OCCC), Orlando, FL
Financial Results for the Third Quarter of 2019

Net loss allocable to common stock holders for the quarter and nine months ended September 30, 2019 was $15.9 million and $37.3 million respectively, compared to $12.7 million and $30.4 million for the same periods in 2018.
General and administrative expenses for the quarter and nine months ended September 30, 2019 were $3.3 million and $10.0 million, respectively, compared to $3.3 million and $9.6 million for the same periods in 2018.
Research and development expenses for the quarter and nine months ended September 30, 2019 were $13.1 million and $28.2 million respectively, compared $6.5 million and $18.0 million for the same periods in 2018.
Net cash used in operating activities for the nine months ended September 30, 2019 was $28.1 million, compared to $19.4 million for the same period in 2018.
Cash balance was $29.0 million as of September 30, 2019, compared to $39.7 million as of June 30, 2019.
Conference Call and Webcast Information
The Company will host a conference call and webcast with the investment community on Wednesday, November 6th at 4:30 p.m. Eastern Time featuring remarks by Tony Liu, Executive Director, CEO and CFO of CBMG.

Live Call:

Toll-Free: 1-855-327-6838

International: 1-604-235-2082

Webcast:

View Source

Replay:

Toll-Free: 1-844-512-2921

International: 1-412-317-6671

Conference ID: 10007976

(Available approximately two hours after the completion of the live call until 11:59 p.m. ET on November 20, 2019)

4D Pharma plc: Preliminary safety and clinical observations from the Phase I / II study with Mrx0518 in combination with KEYTRUDA®

On November 6, 2019 4D Pharma plc (AIM: DDDD), a leading-edge pharmaceutical company in the development of live biotherapeutics, reported preliminary safety and clinical observations from an ongoing clinical phase -I / II study, in collaboration with MSD, a trade name of Merck & Co., Inc. of Kenilworth, NJUSA, in conjunction with its leading oncology candidate, Mrx0518, in combination with the MSD anti-PD-1 therapy with KEYTRUDA (pembrolizumab) in patients with advanced malignancies who previously responded and whose disease was subsequently co-administered PD-1 / PD-L1 inhibitors has progressed to evaluate (Press release, 4d Pharma, NOV 6, 2019, View Source;814041836.html [SID1234550511]). These are the first observations of an oncology study in humans with a live biotherapy product.

Important observations:

Initial data from the first six patients show that the combination of Mrx0518 and KEYTRUDA is well tolerated.
Induction of clinically relevant response in two out of six patients with previous progressive disease
Three patients had to retire for illness-related reasons (two after the diagnosis of a progressive illness and one due to a disease-related adverse event).
The Phase I / II trial is an open-label study evaluating the safety and preliminary efficacy of Mrx0518 and KEYTRUDA in patients with renal cell carcinoma (RCC), melanoma, non-small cell lung cancer (NSCLC) and bladder cancer who have resistance against PD-1 / PD-L1 inhibitors. In preclinical studies, Mrx0518 has previously demonstrated significant efficacy as a monotherapy and as a combination therapy with the checkpoint therapy. Mrx0518 has also been shown to be an effective immune stimulant and capable of increasing the number of tumor infiltrating lymphocytes (TILs) that have been shown to be anticancer in preclinical cancer models.

Although the checkpoint therapies have proven to be effective anti-cancer treatments, they are not effective in all patients, and those who respond may develop resistance over time, leading to a loss of efficacy and progression of the disease Disease leads. The mode of action of Mrx0518 has the potential to reactivate the efficacy of checkpoint therapies, and this study was designed to investigate this effect in patients with advanced metastatic disease.

Patients eligible for the Phase I / II trial must have a progressive disease prior to enrollment, which has been confirmed by two residual scans. Part A of the study aims to enroll 12 patients, with the primary outcome being measurement of safety and tolerability. Part A will be performed over three weeks and patients will receive one cycle of KEYTRUDA, taking Mrx0518 orally twice a day (the "offer") .After completion of the first cycle, patients will be eligible for up to 35 cycles of KEYTRUDA in the drug combination (over about two years) or until disease progression occurs Patients routinely have restaging scans every nine weeks More information on the study can be found at the end of this announcement.

The clinical observations of the first six patients in Part A include:

Two patients showed partial response (according to RECIST version 1.11 criteria) with evidence of tumor disappearance and continue to participate in the study (one patient has been in the study for more than six months).
In another patient, the disease is stable and continues to participate in the study.
Two patients were excluded from the study because of a progressive disease.
One patient was excluded prior to the restaging scan for a serious disease-related adverse event.
The only patient currently tested for tumor biomarker showed signs of increased TIL after treatment (approval for biomarker assessment is optional).
No serious adverse drug reactions were noted.
Dr. Alex Stevenson, Chief Scientific Officer of 4D, said, "We are very encouraged by these early signs of activity in the combination of Mrx0518 and KEYTRUDA in patients with advanced disease. One third of patients who had previously stopped responding to PD-1 inhibitors and had a progressive disease have now shown clinical benefit. There is also a first indication in a patient suggesting that the treatment is capable of increasing the number of tumor infiltrating lymphocytes in a clinical setting, which is in line with our preclinical results. Although more work will be required to determine the robustness of the observed responses and their frequency in a larger number of patients, we are pleased to to report these initial results – the first from an oncological study with a live biotherapeutic. The open label trial continues and we will continue to review the data to optimize our future development strategy for Mrx0518 in these indications.

"These first results further support our ongoing investment in our oncology franchise. We plan to include Mrx0518 in additional clinical trials of various tumor types and settings, either in combination or as monotherapy. The next study with Mrx0518 in combination with radiotherapy for pancreatic cancer will start at MD Anderson before the end of this year. In addition, we continue to develop live biotherapeutic candidates with different modes of action for oncology applications. The first, MRx1299, is currently in GMP manufacturing development. "

Notes:
1 Criteria for assessing response to solid tumors

This notice contains inside information as defined in Article 7 of the Market Abuse Regulation (MAR) No 596/2014. The person responsible for making this announcement on behalf of the Company is Duncan Peyton . After the publication of this notice, this inside information will be considered publicly available.

ASH 2019 | CStone’s anti-PD-L1 antibody demonstrates a complete response rate of 31.8% in patients with relapsed or refractory extranodal natural killer/T-cell lymphoma

On November 6, 2019 CStone Pharmaceuticals ("CStone" or the "Company", HKEX: 2616) reported that an abstract on the CS1001-201 trial (Abstract #2833) was accepted by the 2019 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and has been published online yesterday on the meeting’s official website (Press release, CStone Pharmaceauticals, NOV 6, 2019, View Source;cstones-anti-pd-l1-antibody-demonstrates-a-complete-response-rate-of-31-8-in-patients-with-relapsed-or-refractory-extranodal-natural-killert-cell-lymphoma-300953456.html [SID1234550510]). Additionally, the Company will release further updated data from the CS1001-201 trial in a poster presentation at the conference.

CS1001-201 is a single-arm, multicenter Phase II clinical study designed to evaluate CS1001 monotherapy in relapsed or refractory extranodal natural killer (NK)/T-cell lymphoma (rr-ENKTL). The primary endpoint of the trial is objective response rate (ORR) assessed by an independent radiological review committee (IRRC); the secondary endpoints include investigator-assessed ORR, IRRC-assessed complete and partial response (CR, PR) rates, time to response, duration of response, progression-free survival, overall survival, and safety.

ENKTL is a subtype of mature T cell and NK cell lymphoma, with a higher incidence in Asia than in Europe or North America. ENKTL is an aggressive malignancy characterized by its rapid progression and poor prognosis. Patients with rr-ENKTL lack effective standard treatment after failing an L-asparaginase-based combination chemotherapy regimen. Studies to date have shown that Epstein-Barr virus (EBV) infection is linked to the pathogenic mechanisms of ENKTL, as EBV infection induces immune tolerance by upgrading PD-L1 expression in tumor cells, thus promoting tumor growth.

"In China, ENKTL accounts for approximately 6% of all lymphoma incidences, and those relapsed or refractory patients in particular have urgent unmet clinical needs. CS1001-201 is the first clinical trial worldwide investigating an anti-PD-L1 antibody in ENKTL patients, and promising initial antitumor activity has already been observed in the trial," said Dr. Frank Jiang, Chairman and CEO of CStone. "I am glad that the preliminary results from this trial will be announced for the first time at the 2019 ASH (Free ASH Whitepaper) Annual Meeting. CStone remains committed to addressing treatment gaps in China and around the world. We hope this trial will continue its rapid progress and soon produce more breakthroughs in the treatment of ENKTL."

CStone’s Chief Medical Officer, Dr. Jason Yang, noted: "The close association between ENKTL and the increased PD-L1 expression resulted by EBV infection suggests that blocking the PD-1/PD-L1 pathway could be an effective treatment for ENKTL patients. In the abstract published by ASH (Free ASH Whitepaper), CS1001 demonstrated an ORR of 40.9%, a complete and durable response rate of 31.8% and a benign safety profile. These results represent a major breakthrough in the treatment of rr-ENKTL and support the further development of CS1001 as a treatment for rr-ENKTL. We look forward to sharing encouraging additional data updates in the poster presentation at the 2019 ASH (Free ASH Whitepaper) Annual Meeting."

Results in the abstract published by ASH (Free ASH Whitepaper)

As of June 17, 2019, 29 patients were enrolled into the study. Among them, 22 (75.9%) patients had Stage IV of the disease at screening, 8 patients received 2 lines of prior treatments, and 6 patients received 3 or more lines of prior treatments. All of the patients received 1200 mg CS1001 intravenously every 3 weeks for up to 2 years, until disease progression, intolerance, etc. The median duration of follow-up was 5.55 (range, 0.69-12.19) months.

Demographics and baseline characteristics

15 (51.7%) of the 29 enrolled patients remained on treatment, and 14 (48.3%) had discontinued from the study treatment.
Reasons for discontinuations included disease progression (12 patients) and adverse events (AEs, 2 patients).
No discontinuations or deaths due to treatment-related AEs (TRAEs).
Preliminary efficacy data
CS1001 demonstrated promising antitumor activity in rr-ENKTL patients

Among the 22 efficacy-evaluable patients, the investigator-assessed ORR was 40.9%. 7 (31.8%) patients achieved complete and durable response.
2 (9.1%) patients achieved partial response (PR), and 1 additional patient achieved PR after pseudo-progression.
The duration of response (DoR) ranged from 0.03+ to 8.61+ months, and the median DoR was not reached.
The IRRC assessments were not available at the time of data cut-off.
Safety data

CS1001 was well tolerated in patients with rr-ENKTL

The median duration of treatment was 11.7 (range, 2.9 – 53.0) weeks.
25 (86.2%) patients reported treatment-emergent AEs (TEAEs).
21 (72.4%) patients reported treatment-related AEs (TRAEs), of which 3 (10.3%) had Grade ≥3 TRAEs.
Grade 5 AEs were reported in 2 patients, and none was assessed as related to CS1001.
Serious AEs were observed in 5 (17.2%) patients, and 1 of them (sinus node dysfunction) was assessed as related to CS1001 by the investigator.
Immune-related AEs (irAEs) were reported in 5 patients (17.2%). Except 1 Grade 3 rash, all irAEs were Grade 1 in severity.
TEAEs that led to permanent treatment discontinuation occurred in 2 (6.9%) patients; but no permanent discontinuation or death due to AEs were assessed as related to CS1001.
After data cut-off date, 3 additional patients reached the response assessment time point, of which 2 patients achieved CR, elevating the ORR to 44.0% (11/25) and CR rate to 36.0% (9/25). More details of the updated data will be reported at the 2019 ASH (Free ASH Whitepaper) Annual Meeting.

About CS1001

CS1001 is an investigational monoclonal antibody directed against PD-L1 being developed by CStone. Authorized by the U.S. based Ligand Corporation, CS1001 is developed by the OMT transgenic animal platform, which can generate fully human antibodies in one step. As a fully human, full-length anti-PD-L1 monoclonal antibody, CS1001 mirrors natural G-type immune globulin 4 (IgG4) human antibody, which can reduce the risk of immunogenicity and potential toxicities in patients, potentially representing a unique advantage over similar drugs.

CS1001 has completed a Phase I dose-escalation study in China, in which CS1001 showed good tolerability and produced sustained clinical benefits during Phase Ia and Ib stages of the study in multiple indications.

CS1001 is being investigated in a number of ongoing clinical trials, including one Phase I bridging study in the U.S. In China, its clinical program includes one multi-arm Phase Ib study, two pivotal Phase II studies, and three Phase III studies for several tumor types.

Artios Pharma, MD Anderson and ShangPharma Announce In-Licensing Agreement for DNA Damage Response Inhibitor

On November 6, 2019 Artios Pharma Limited (Artios), The University of Texas MD Anderson Cancer Center (MD Anderson) and ShangPharma Innovation (ShangPharma) reported the in-licensing by Artios of a small-molecule ATR inhibitor programme, developed jointly by MD Anderson and ShangPharma (Press release, Artios Pharma, NOV 6, 2019, View Source [SID1234550506]).

Under the agreement, Artios has exclusive rights to research, develop, manufacture and commercialize products globally. The lead candidate is expected to be ready for Investigational New Drug (IND) application by the second half of 2020.

"This programme has the potential to be a highly effective DNA damage response (DDR) targeted treatment in cancer. We look forward to advancing the work done by MD Anderson and ShangPharma for the benefit of cancer patients," said Dr. Niall Martin, Chief Executive Officer at Artios Pharma. "The addition of the ATR programme further supports our position as a leader in the DDR space and strengthens our growing portfolio of assets, which includes a leading Polθ programme, currently in candidate IND evaluation, and a large discovery stage platform of novel DNA repair nuclease inhibitors."

The ATR inhibitor programme is the result of an extensive collaboration between MD Anderson’s Therapeutics Discovery team and ShangPharma. Therapeutics Discovery is a multidisciplinary team created within MD Anderson to advance the next generation of cancer therapies to answer unmet oncology needs.

"Targeting DNA damage repair has the potential to provide an important therapeutic option for many patients in need of new treatments," said Philip Jones, Ph.D., vice president of Therapeutics Discovery at MD Anderson. "We are pleased Artios will leverage its unique expertise in this field to advance this novel therapy toward the clinic to improve outcomes for cancer patients."

ATR[1] is an important signalling protein in DNA double strand break repair and replication stress. Through inhibition of ATR, tumours bearing an ATM[2] deficiency can be selectively killed through a concept known as synthetic lethality. High levels of ATM mutations and protein loss have been characterised across many different tumour types, creating a significant opportunity for ATR inhibitors clinically. Based on clinical observations at MD Anderson, Therapeutics Discovery engaged with ShangPharma and its affiliate, ChemPartner, to develop small-molecule inhibitors of the DDR that could benefit patients across multiple cancer types.

"We are proud of the entire collaboration team, including ChemPartner, led by Sarah Lively, Ph.D., vice president of Innovation and New Technologies, for advancing the programme from early-stage research to formal drug discovery and development," said Walter Moos, Ph.D., CEO of ShangPharma Innovation. "We are pleased to transition this important programme to the capable development team at Artios, and we hope this ultimately provides an impactful therapy for those afflicted with cancer."

AcelRx Pharmaceuticals to Present at Credit Suisse 28th Annual Healthcare Conference

On November 6, 2019 AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for the use in medically supervised settings, reported that management will be presenting at the Credit Suisse 28th Annual Healthcare Conference to be held November 11 – 13 (Press release, AcelRx Pharmaceuticals, NOV 6, 2019, View Source [SID1234550499]). AcelRx management will provide an overview of the business and company updates during the live presentation and will be available for one-on-one meetings with investors.

Details of the event are as follows:

Credit Suisse 28th Annual Healthcare Conference
Date: Wednesday, November 13, 2019
Location: The Phoenician, Scottsdale, AZ
Presentation Time: 9:10 am local time (11:10 a.m. ET)

The conference will be webcast live and can be accessed through the Investors page at www.acelrx.com. For those not available to listen to the live broadcast, a replay will be archived for 90 days and available through the Investors page on www.acelrx.com.