On August 6, 2019 Oncternal Therapeutics, Inc., (Nasdaq: ONCT) a clinical-stage biotechnology company developing potential first-in-class product candidates for cancers with critical unmet medical need, reported that it has opened for enrollment its randomized Phase 2 study of cirmtuzumab, a ROR1-targeted monoclonal antibody, combined with ibrutinib in patients with chronic lymphocytic leukemia (CLL) (Press release, Oncternal Therapeutics, AUG 6, 2019, View Source [SID1234538212]). The decision to open Phase 2 of the company’s ongoing Phase 1/2 CIRLL (Cirmtuzumab and Ibrutinib targeting ROR1 for Leukemia and Lymphoma) clinical trial was triggered by favorable outcomes from the Part 1 dose-finding and Part 2 dose-confirming cohorts of the clinical trial, including an observed interim objective response rate (ORR) of 100% for the first 9 CLL patients with evaluable data receiving the recommended dosing regimen who have completed 12 weeks of cirmtuzumab plus ibrutinib treatment in Part 2, and a well-tolerated safety profile consistent with that seen with ibrutinib treatment alone.

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In June, the company presented data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, reporting that results from the first 12 patients with CLL treated in Part 1 of the Phase 1 portion of the study showed an observed interim objective response rate (ORR) of 91.7% for the combination of cirmtuzumab plus ibrutinib, including three patients with clinical or confirmed complete responses, and a well-tolerated safety profile consistent with that seen for ibrutinib treatment alone.

Included in the results presented at ASCO (Free ASCO Whitepaper) were preliminary results from six patients with mantle cell lymphoma (MCL), who were treated in a separate cohort of the CIRLL study. Data from this cohort will be presented at a future medical conference. One patient with MCL who had relapsed following an allogeneic stem cell transplant experienced a confirmed complete response (CR) after 3 months of cirmtuzumab plus ibrutinib treatment, including complete resolution of a large mediastinal mass. This CR appears to be durable, and has been confirmed after 6, 9 and 11 months of cirmtuzumab plus ibrutinib treatment.

The CIRLL clinical trial is supported by a grant from the California Institute for Regenerative Medicine (CIRM) and is being conducted in collaboration with the University of California at San Diego (UC San Diego).

"We are very pleased to be opening the randomized Phase 2 portion of the CIRLL study for patients with CLL and continue to be encouraged by the interim results from the study for both patients with CLL and patients with mantle cell lymphoma," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO.

About the Study
The CIRLL Study (CIRM-0001) is a Phase 1/2 clinical trial evaluating cirmtuzumab in combination with ibrutinib in patients with CLL or MCL. Part 1 of the study was a Phase 1 dose-finding portion designed to determine the Phase 2 dose, or recommended dosing regimen (RDR). Part 2 was a Phase 1b expansion cohort to confirm the RDR. Interim analyses were specified for Part 1 and Part 2. Part 3 of the study, which is now open for enrollment, is a Phase 2 study in which approximately 90 patients with CLL will be randomized to receive either ibrutinib alone or ibrutinib plus cirmtuzumab, with a primary endpoint of complete response rate. An interim assessment of the first 12 patients with CLL enrolled in Part 1 of the study was presented as a poster at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting. Additional information about the CIRM-0001 study and other clinical studies of cirmtuzumab may be accessed at ClinicalTrials.gov.

About Cirmtuzumab
Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of CLL and MCL, in a collaboration with the University of California San Diego School of Medicine and the California Institute for Regenerative Medicine. In addition, an investigator-initiated Phase 1 clinical trial of cirmtuzumab in combination with paclitaxel for women with metastatic breast cancer is being conducted at the UC San Diego School of Medicine. CIRM has also provided funding to support development programs for cirmtuzumab and a CAR-T product candidate that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors.

When expressed by hematologic malignancies such as CLL and MCL, ROR1 acts as a receptor for the tumor growth factor Wnt5a. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to inhibiting Wnt5a activation, specifically targeting ROR1 expressing tumors, and this finding led to the discovery of the potent and highly selective antitumor activity of cirmtuzumab observed in preclinical studies. Oncternal believes ROR1 is an attractive target for cancer therapy because it is an oncofetal antigen – a protein not normally expressed in adults, but which confers a survival and fitness advantage when reactivated and expressed by tumor cells. Preclinical data indicate that when cirmtuzumab binds to ROR1, it blocks Wnt5a signaling, induces differentiation of the tumor cells, and inhibits tumor cell proliferation, migration and survival. Cirmtuzumab is in clinical development and has not been approved by the U.S. Food and Drug Administration for any indication.

On August 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 second quarter and six months ended June 30, 2019 (Press release, Cellular Biomedicine Group, AUG 6, 2019, View Source [SID1234538228]).

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"During the second quarter of 2019, we made strides in advancing CBMG’s clinical pipeline in China. This includes expansion to multiple sites, dose escalation and robust patient recruitment for our B cell maturation antigen (BCMA) CAR-T program targeting multiple myeloma (MM). We have finished enrolling patients for the first cohort in a dose escalation study and currently are enrolling patients for the second cohort. We are also expanding the study into multiple sites. We are moving the alpha-fetoprotein T-cell receptor (AFP-TCR-T) program forward and will start to screen and enroll patients in hepatocellular carcinoma (HCC). We are actively preparing for a multisite trial for our AlloJoin therapy for Knee Osteoarthritis (KOA)," commented Tony (Bizuo) Liu, Chief Executive Officer of CBMG.

Mr. Liu continued, "We continue to leverage the investigator initiated trial (IIT) process in China and plan to initiate these cancer clinical trials in the U.S. when we see positive proof of concept signals in the IIT studies in China. This allows us to prioritize and focus on developing clinical assets with the most potential and best chance to win both in China and globally."

Business Highlights for the Second Quarter and First Half 2019:

Advanced myriad of our immune-oncology (I/O) and regenerative medicine assets for the next stage in the translational medicine process, comprised of fine-tuning process development, multiple sites trial, increases in patient recruitment, and dose escalation;
Preparing to bring our I/O assets to the U.S. market.
Upcoming Clinical Milestones:

Present update of clinical data for CBMG’s anti-BCMA CAR-T in the fourth quarter of 2019
Financial Results for the Second Quarter and First Half 2019:

Net loss allocable to common stock holders for the quarter and six months ended June 30, 2019 was $12.1 million and $21.4 million respectively, compared to $9.2 million and $17.7 million for the same periods in 2018.
General and administrative expenses for the quarter and six months ended June 30, 2019 were $3.2 million and $6.6 million, respectively, compared to $3.1 million and $6.3 million for the same periods in 2018.
Research and development expenses for the quarter and six months ended June 30, 2019 were $9.1 million and $15 million respectively, compared $6.2 million and $11.4 million for the same periods in 2018, primarily due to the increased clinical development for our leading cell therapy targets.
Net cash used in operating activities for first half of 2019 was $18.7 million, compared $13.7 million for the same period in 2018.
Cash, cash equivalents and restricted cash was $56.7 million as of June 30, 2019, compared to $62.0 million as of March 31, 2019.
Conference Call and Webcast Information
The Company will host a conference call and webcast with the investment community on Tuesday, August 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: 10007371

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

On August 6, 2019 Conatus Pharmaceuticals Inc. (Nasdaq:CNAT) reported financial results for the quarter and six months ended June 30, 2019, and provided updates on its development programs (Press release, Conatus Pharmaceuticals, AUG 6, 2019, View Source [SID1234538254]).

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Program Updates
During the second quarter, Conatus announced that top-line results from the company’s ENCORE-LF clinical trial of emricasan did not meet its primary endpoint, results from the 24-week extension in the company’s ENCORE-PH clinical trial of emricasan did not meet predefined objectives, and the company is discontinuing further treatment of patients enrolled in the ENCORE-LF trial. Consequently, Conatus and its partner, Novartis, have no further development plans for emricasan. Conatus plans to complete its ongoing clinical trials of emricasan for which the company is responsible under the collaboration agreement and is in discussions with Novartis for the wind-down of the collaboration.

In June 2019, Conatus announced that the company was implementing a restructuring plan in order to extend its resources, which included reducing staff and suspending development of its inflammasome disease candidate, CTS-2090. The company also announced that it had engaged Oppenheimer & Co., Inc., as its financial advisor to assist in the exploration and evaluation of strategic alternatives to enhance shareholder value. There can be no assurance of a successful outcome from these efforts, or of the form or timing of any such outcome.

Financial Results
The net loss for the second quarter of 2019 was $0.7 million compared with $4.5 million for the second quarter of 2018. The net loss for the first six months of 2019 was $5.4 million compared with $9.5 million for the first six months of 2018.

Total revenues consisted of collaboration revenue related to the company’s collaboration with Novartis. Total revenues were $10.8 million for the second quarter of 2019 compared with $8.8 million for the second quarter of 2018. The increase of $2.0 million was primarily due to a net cumulative catch-up in revenue recognized under the Novartis agreement, partially offset by lower revenues under the Novartis agreement due to lower emricasan-related research and development expenses.

Total revenues were $17.8 million for the first six months of 2019 compared with $18.5 million for the first six months of 2018. The decrease of $0.7 million was primarily due to lower revenues under the Novartis agreement due to lower emricasan-related research and development expenses, partially offset by a net cumulative catch-up in revenue recognized under the Novartis agreement.

Research and development expenses were $8.6 million for the second quarter of 2019 compared with $10.7 million for the second quarter of 2018. Research and development expenses were $17.9 million for the first six months of 2019 compared with $22.8 million for the first six months of 2018. These decreases were primarily due to lower emricasan-related research and development expenses and lower personnel costs, partially offset by recognition of severance and noncash stock compensation costs for research and development employees related to the restructuring plan announced in June 2019.

General and administrative expenses were $3.1 million for the second quarter of 2019 compared with $2.6 million for the second quarter of 2018. General and administrative expenses were $5.6 million for the first six months of 2019 compared with $5.3 million for the first six months of 2018. These increases were primarily due to recognition of severance and noncash stock compensation costs for general and administrative employees related to the restructuring plan announced in June 2019.

Cash, cash equivalents and marketable securities were $28.7 million at June 30, 2019, compared with $40.7 million at December 31, 2018. The company is projecting a year-end 2019 net balance of cash, cash equivalents and marketable securities of between $10 million and $15 million.

On August 6, 2019 Dova Pharmaceuticals, Inc. (Nasdaq: DOVA), a pharmaceutical company focused on acquiring, developing, and commercializing drug candidates for diseases where there is a high unmet need, reported its operating and financial results for the second quarter ended June 30, 2019 (Press release, Dova Pharmaceuticals, AUG 6, 2019, View Source [SID1234538197]).

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"The last few months have been a transformational period in Dova’s history, with FDA approval and launch of our thrombopoietin receptor agonist (TPO-RA) DOPTELET for ITP, European approval of DOPTELET for severe thrombocytopenia in adult patients with CLD, and an expanded co-promotion partnership with Salix for CLD. These accomplishments have strengthened our position as a growing leader in the treatment of thrombocytopenia," said Dr. David Zaccardelli, President and Chief Executive Officer of Dova. "Beyond ITP, we look forward to results of our further indication expansion activities, with top-line data from our Phase 3 trial of DOPTELET for the treatment of chemotherapy induced thrombocytopenia (CIT) expected in the first half of 2020."

Second Quarter and Subsequent Highlights

DOPTELET ITP Approval – In late June, the FDA approved a supplemental New Drug Application (sNDA) that expanded the use of DOPTELET to include the treatment of thrombocytopenia in adult patients with ITP who have had an insufficient response to a previous treatment.

DOPTELET ITP Launch – In mid-July, DOPTELET was launched commercially for the ITP indication. A sales force of approximately 60 sales professionals with extensive Hematology/

Oncology experience, are now calling on roughly 6,000 HCPs that represent 96% of the ITP patient potential in the United States.

DOPTELET Differentiating Characteristics in ITP –

◦
DOPTELET is the only oral TPO-RA approved for use in adult patients with ITP that does not have a boxed warning for hepatotoxicity and does not require routine liver enzyme monitoring.

DOPTELET is the only oral TPO approved for use in adult patients with ITP without food-type restrictions.

DOPTELET, as an oral tablet, does not require weekly visits to a health care provider’s office for subcutaneous injections.

DOPTELET increased platelet counts to >50,000 in 66% of ITP patients by Day 8 of treatment in a Phase 3 trial.

DOPTELET ITP Publications – Dova presented four abstracts on the safety and efficacy of avatrombopag from studies in patients with ITP at the 65th International Society on Thrombosis and Haemostasis (ISTH) Annual Scientific Meeting, held July 6-10, 2019, in Melbourne, Australia. These included efficacy data in patients who had previously received another TPO-RA, and the results of a dosing model study.

DOPTELET CLD MAA – In late June, the EC granted marketing authorization for DOPTELET for the treatment of severe thrombocytopenia in adult patients with CLD who are scheduled to undergo an invasive procedure. Further, Dova continues to advance partnering discussions to commercialize DOPTELET in markets outside the US.

Salix Partnership – Dova strategically expanded its co-promotion partnership for CLD in the United States with Salix. Starting on July 1, 2019, in addition to the gastroenterology, colorectal surgery, and proctology segments, Salix has the exclusive right to co-promote DOPTELET for the CLD indication to the hepatology and interventional radiology segments. Dova will continue to pay Salix a commission based on a percentage of net sales in these specialties, which beginning on July 1, 2019, is expected to be in the mid-thirties. In addition, the co-promotion agreement was extended to September 2023.

Phase 3 CIT Clinical Trial – Dova continues to enroll patients in a Phase 3 trial evaluating the safety and efficacy of DOPTELET for the treatment of CIT and plans to report top-line results in the first half of 2020.

DOPTELET CLD Launch Highlights

Net product sales for DOPTELET were $3.5 million for the second quarter.
◦
Inventory held by specialty pharmacies in Dova’s contracted network decreased by approximately $450,000 from March 31, 2019 to June 30, 2019.
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Prescription referrals increased approximately 7% in the second quarter of 2019 compared to the first quarter of 2019.
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As expected, sales were negatively affected by competitive pricing pressure and payer restrictions in the second quarter of 2019, which Dova is addressing with the new pricing for DOPTELET implemented mid-July 2019.

Early in the second quarter of 2019, a new marketing strategy for the CLD indication was launched, including revised product positioning, messaging, and a new action-oriented brand campaign.

From launch through June 30, 2019, a total of 1,368 health care professionals have prescribed DOPTELET to their patients, with an increasing number of repeat prescribers.

More than 9,200 calls were conducted, reaching more than 4,000 unique health care providers during the second quarter of 2019.

For prescriptions in the second quarter of 2019 that have gone through the adjudication process with payers, 77% of those prescriptions were approved. On average, the time to decision for a referral was 7.4 business days in the second quarter of 2019.

Second Quarter Financial Results

Net product sales for DOPTELET were $3.5 million for the second quarter of 2019. Dova recognizes revenue when products are delivered to its specialty pharmacy partners.

Dova reported a net loss of $17.1 million for the second quarter of 2019, compared to a net loss of $19.7 million for the same period in 2018.

Cost of product sales for the second quarter of 2019 were $0.4 million, which consisted of the cost of inventory, royalty payments and certain distribution and overhead costs.

Research and development expenses were $4.5 million in the second quarter of 2019, compared to $4.5 million for the same period in 2018.

Selling, general and administrative expenses were $15.5 million in the second quarter of 2019, compared to $18.6 million for the same period in 2018. The second quarter of 2018 saw higher expense levels as a result of the full sales staff being onboard for most of the second quarter of 2018 while in 2019 the full sales staff for the launch of DOPTELET for the ITP indication were hired at the end of the second quarter and early third quarter.

As of June 30, 2019, Dova had $76.8 million in cash and equivalents compared to $104.6 million as of December 31, 2018. Additionally, on May 6, 2019, Dova entered into an amended and restated loan agreement with Silicon Valley Bank that extended the interest only period of the existing $20 million loan facility by 12 months and provided additional potential borrowings of $30 million upon achieving certain clinical and revenue milestones. In July 2019, subsequent to the quarter end, Dova drew $10 million on this loan agreement upon approval in ITP.

Company to Host Conference Call

Dova will host a conference call today, August 6, 2019 at 9:00 a.m. ET to discuss second quarter ended June 30, 2019 financial results as well as recent operational highlights. A question-and-answer session will follow Dova’s remarks.

To participate on the live call, please dial 866-550-8145 (domestic) or +1-430-775-1344 (international) and provide the conference ID 7784728 five to 10 minutes before the start of the call.

A live audio webcast of the call will also be available via the "Investor Relations" page of the Dova website, www.dova.com. Please log on through Dova’s website approximately 10 minutes before the scheduled start time. A replay of the webcast will be archived on Dova’s website for 90 days following the call.

Indication and Important Safety Information
INDICATION
DOPTELET (avatrombopag) is indicated for the treatment of thrombocytopenia in adult patients with:

Chronic liver disease who are scheduled to undergo a procedure.

Chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.
IMPORTANT SAFETY INFORMATION FOR DOPTELET
Warnings and Precautions
DOPTELET is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic and thromboembolic complications in patients with chronic liver disease or chronic immune thrombocytopenia. Portal vein thrombosis has been reported in patients with chronic liver disease, and thromboembolic events (arterial and venous) have been reported in patients with chronic immune thrombocytopenia treated with TPO receptor agonists.
In clinical trials, 0.2% (1/430) of patients with chronic liver disease treated with DOPTELET developed a treatment-emergent event of portal vein thrombosis. In clinical trials in patients with chronic immune thrombocytopenia, 7% (9/128) of patients treated with DOPTELET developed a thromboembolic event.
Consider the potential increased thrombotic risk when administering DOPTELET to patients with known risk factors for thromboembolism, including genetic prothrombotic conditions (Factor V Leiden, Prothrombin 20210A, Antithrombin deficiency or Protein C or S deficiency).
DOPTELET should not be administered to patients with chronic liver disease or chronic immune thrombocytopenia in an attempt to normalize platelet counts. Follow the dosing guidelines to achieve target platelet counts. Monitor patients receiving DOPTELET for signs and symptoms of thromboembolic events and institute treatment promptly.
Contraindications: None
Drug Interactions
Dose adjustments are recommended for patients with chronic immune thrombocytopenia taking moderate or strong dual CYP2C9 and CYP3A4 inducers or inhibitors.
Adverse Reactions
The most common adverse reactions (≥3%) in patients with chronic liver disease were: pyrexia, abdominal pain, nausea, headache, fatigue, and peripheral edema.

The most common adverse reactions (≥10%) in patients with chronic immune thrombocytopenia were: headache, fatigue, contusion, epistaxis, upper respiratory tract infection, arthralgia, gingival bleeding, petechiae, and nasopharyngitis.

On August 6, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported that Andy Boral, M.D., Ph.D., Blueprint Medicines’ Chief Medical Officer, will present a company overview at the 2019 Wedbush PacGrow Healthcare Conference in New York, NY on Tuesday, August 13, 2019 at 9:45 a.m. ET (Press release, Blueprint Medicines, AUG 6, 2019, View Source [SID1234538213]).

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A live webcast of the presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcast will be archived on Blueprint Medicines’ website for 30 days following the presentation.