On August 7, 2019 Athersys, Inc. (NASDAQ: ATHX) reported its financial results for the three months ended June 30, 2019 (Press release, Athersys, AUG 7, 2019, View Source [SID1234538300]).
Highlights of the second quarter of 2019 and recent events include:

Received Fast Track designation from the FDA for our clinical development program involving administration of MultiStem cell therapy for treatment of acute respiratory distress syndrome ("ARDS");

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Presented positive results from the MUST-ARDS study at the American Thoracic Society International Conference in May 2019;

Announced first patient enrolled in the ONE-BRIDGE study in Japan being conducted by our partner in Japan, HEALIOS K.K. ("Healios"), evaluating MultiStem cell therapy treatment of patients suffering from pneumonia-induced ARDS;

Advanced our ischemic stroke program, both through support of Healios’ TREASURE study in Japan and increased enrollment in our MASTERS-2 Phase 3 registrational study;

Entered into an open market sales agreement with Jefferies LLC, i.e., an ‘at-the-market’ equity program, providing additional flexibility and capital markets access as we progress to important milestones;

Hosted a successful investor day in New York City in May 2019 highlighting our capabilities and technologies, as well as progress made developing the MultiStem product platform;

Increased our media exposure as we were featured in video, podcasts and articles in the major publications Fortune and Forbes;

Participated in several events at the International Society of Cell and Gene Therapy Conference in June 2019, including podium presentations, a panel participation and chairing a session, further establishing the Company as a leader and significant contributor in the field of cell therapy;

Recognized revenues of $4.3 million and net loss of $9.7 million, or $0.06 net loss per share, for the quarter ended June 30, 2019; and

Ended the 2019 second quarter with $44.2 million of cash and cash equivalents.
"We continue to make steady progress in key areas, including advancing our Phase 3 clinical program for treating ischemic stroke, while supporting Healios in the advancement of its TREASURE study for stroke patients and ONE-BRIDGE study for ARDS patients in Japan," commented Dr. Gil Van Bokkelen, Chairman & CEO at Athersys. "Given our progress in multiple areas, including the recently announced Fast Track designation and promising results from our ARDS clinical program, we are exploring partnering opportunities in several important geographic areas, while we continue to advance our key initiatives and expand core capabilities. This includes continuing to advance our manufacturing activities and initiatives, that we believe will represent a significant competitive advantage as we move into commercialization.
"While we advance our programs through clinical development and toward commercialization, we have also taken prudent and appropriate steps to maintain a solid balance sheet, ensuring we have access to additional capital while we continue to maintain a long-term focus on creating value for our shareholders," concluded Dr. Van Bokkelen.

Second Quarter Results
Revenues were $4.3 million for the three months ended June 30, 2019 compared to $19.4 million for the three months ended June 30, 2018. Revenues from our collaboration with Healios were $4.2 million in the second quarter of 2019 compared to $18.8 million for the same period last year, which included the impact of the June 2018 collaboration expansion. Royalty revenue from RTI Surgical, Inc. ceased late in 2018 upon its decision to discontinue distribution of the licensed product. Our revenues are generally derived from license fees, manufacturing-related activities for Healios, other contract revenue from our collaborations and grant revenue.
Research and development expenses increased to $11.1 million for the three months ended June 30, 2019 from $10.1 million for the comparable period in 2018. The $1.0 million net increase is associated with increases in clinical trial and manufacturing process development costs of $0.7 million, personnel costs of $0.4 million, internal research supplies of $0.3 million and stock compensation costs of $0.2 million, with such increases partially offset by a decrease in license fees of $0.6 million. Included in our clinical expenses are costs associated with providing manufacturing services to Healios, which are invoiced to Healios in accordance with our collaboration agreements.
General and administrative expenses increased to $2.9 million for the three months ended June 30, 2019 from $2.4 million in the comparable period in 2018. The $0.5 million increase was due primarily to increased personnel costs, legal and professional fees, travel costs and stock compensation costs compared to the same period last year.
Net loss for the second quarter of 2019 was $9.7 million compared to net income of $6.9 million in the second quarter of 2018. The variance of $16.6 million is primarily attributable to the June 2018 expansion of the Healios collaboration.
In the six months ended June 30, 2019, net cash used in operating activities was $17.0 million compared to $1.3 million in the six months ended June 30, 2018, with 2018 being impacted by proceeds from the Healios collaboration expansion. At June 30, 2019, we had $44.2 million in cash and cash equivalents, compared to $51.1 million at December 31, 2018.
Conference Call
William (B.J.) Lehmann, President and Chief Operating Officer, and Laura Campbell, Senior Vice President of Finance, will host a conference call today to review the results as follows:

Date

August 7, 2019
Time

4:30 p.m. (Eastern Time)
Telephone access: U.S. and Canada

(877) 396-3286
Telephone access: International

(647) 689-5528
Access code

5087881
Live webcast

www.athersys.com, under the Investors section

We encourage shareholders to listen using the webcast link and to use the phone line if you intend to ask a question. A replay will be available at www.athersys.com under the Investors section approximately two hours after the call has ended. Shareholders may also call in for on-demand listening shortly after the completion of the call until 11:59 PM Eastern Time on August 14, 2019 by dialing (800) 585-8367 or (416) 621-4642 and entering Encore passcode 5087881.

On August 7, 2019 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing novel small molecule therapeutics to treat unmet needs in autoimmunity and cancer, reported its second quarter 2019 financial results and corporate highlights (Press release, Kezar Life Sciences, AUG 7, 2019, View Source [SID1234538299]).

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"I’m proud of the tremendous progress made by the team over the quarter, culminating with the first-in-patient data reported with KZR-616," said John Fowler, Chief Executive Officer. "The broad therapeutic potential demonstrated thus far by KZR-616 reinforces our view that selective immunoproteasome inhibition can target multiple autoimmune diseases. To that end, in addition to having initiated the Phase 2 portion of the MISSION study in lupus nephritis patients, we are pleased to announce that the Phase 2 PRESIDIO and MARINA trials have commenced in four additional autoimmune indications—dermatomyositis, polymyositis, autoimmune hemolytic anemia, and immune thrombocytopenia. Furthermore, we remain on-track to nominate the first oncology clinical candidate from our novel protein secretion program before the end of the year."

Recent Clinical and Business Highlights

KZR-616 (Selective Immunoproteasome Inhibitor)

MISSION Study

The Phase 1b/2 MISSION study in systemic lupus erythematosus (SLE) patients with and without nephritis is currently ongoing.

We reported promising data with KZR-616 in patients with SLE at the European League Against Rheumatism (EULAR) 2019 Annual Meeting. Initial data were reported from 3 cohorts of the open-label dose escalation portion of the trial (Phase 1b). The study met its objectives by establishing the safety and tolerability of KZR-616 and identifying doses to advance into our Phase 2 clinical trials.

Enrollment to the Phase 1b portion of the MISSION study is ongoing and updated data from cohorts testing step-up dosing to a 60 mg dose are expected in Q4 2019.

The Phase 2 portion of the MISSION study, which is evaluating KZR-616 for the treatment of lupus nephritis (LN), was initiated.

PRESIDIO Study

We recently initiated the PRESIDIO study (NCT04033926). This is a Phase 2 randomized, double-blind, placebo-controlled, crossover, multicenter study to evaluate the safety, tolerability, efficacy, PK and PD of treatment with KZR-616 in patients with active dermatomyositis (DM) or polymyositis (PM). The trial is expected to enroll 24 patients with either DM or PM.

MARINA Study

We recently initiated the MARINA study (NCT04039477). MARINA is a Phase 2 randomized, dose-blind, multicenter study to evaluate the safety and efficacy of KZR-616 in the treatment of patients with autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). The trial is expected to enroll 40 patients with either AIHA or ITP.

Protein Secretion Program (Sec61 translocon modulation)

Our research and discovery efforts targeting the protein secretion pathway as a potential therapy for oncology indications is progressing well, and we remain on track to nominate a first clinical candidate before the end of the year.

Management Update

Kezar announced today that Niti Goel, MD, Chief Medical Officer, will be departing the company following a transition period ending October 1, 2019.

"On behalf of the Executive Team and Board of Directors, I would like to sincerely thank Niti for her contributions during her tenure at Kezar," said John Fowler, Chief Executive Officer. "Niti’s exceptional skill designing innovative clinical trials and her unwavering commitment to patients has positioned KZR-616 for success in our Phase 2 studies and will be missed."

Financial Results

Cash, cash equivalents and marketable securities totaled $93.4 million as of June 30, 2019, compared to $107.4 million as of December 31, 2018. The decrease in cash, cash equivalents and marketable securities was primarily attributable to cash used by the company in operations to advance its clinical stage programs as well as preclinical research and development.

Research and development expenses for the second quarter of 2019 increased by $1.7 million to $6.9 million from $5.2 million in the second quarter of 2018. This increase was primarily related to advancing both the KZR-616 clinical program across indications and the protein secretion preclinical program.

General and administrative expenses for the second quarter of 2019 increased by $0.7 million to $2.4 million from $1.7 million in the second quarter of 2018. The increase was primarily due to an increase in personnel expenses and costs related to operating as a public company.

Net loss for the second quarter of 2019 was $8.7 million, or $0.46 per basic and diluted common share, compared to a net loss of $6.8 million, or $3.31 per basic and diluted common share, for the second quarter of 2018.

Total shares outstanding were 19.1 million as of June 30, 2019. Additionally, there were outstanding options to purchase 2.9 million shares of common stock at a $8.02 weighted average exercise price as of June 30, 2019.

About KZR-616

KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Nonclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Phase 1a clinical trial results in healthy volunteers provide evidence that KZR-616 potentially avoids adverse effects caused by currently marketed non-selective proteasome inhibitors, which we believe prevent them from being utilized as a chronic treatment in autoimmune disorders. Phase 2 trials have commenced for the treatment of lupus nephritis (MISSION study), dermatomyositis and polymyositis (PRESIDIO study), and autoimmune hemolytic anemia and immune thrombocytopenia (MARINA study).

About Protein Secretion

We are conducting research and discovery efforts targeting protein secretion pathways as potential therapies for oncology and immuno-oncology indications. In mammalian cells, the secretion of proteins such as cytokines and the expression of cell surface transmembrane proteins such as cytokine receptors involve a process called cotranslational translocation. For most proteins, this process occurs via the Sec61 translocon, a highly conserved multi-subunit protein complex found in the membrane of the endoplasmic reticulum of all cells. Inhibition of the Sec61 translocon with small molecules blocks the secretion of some or all proteins, which can result in several physiologic outcomes, including altered cellular function, inhibition of cytokine release and/or cell death. We believe this platform has the potential to yield oral small molecule alternatives to currently marketed biologic therapeutics, to act as cytotoxic anti-cancer agents or to block the secretion of novel targets of interest in immuno-oncology or inflammation.

On August 7, 2019 Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the second quarter ended June 30, 2019 (Press release, Syndax, AUG 7, 2019, View Source [SID1234538298]). In addition, the Company provided a clinical and business update. As of June 30, 2019, Syndax had $80.5 million in cash, cash equivalents and short-term investments.

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"The FDA’s recent clearance of our IND application for SNDX-5613, a potent, highly selective, oral Menin inhibitor, marks an important milestone not only for Syndax, but also for patients suffering with acute leukemias," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "The Menin-MLL interaction has been strongly implicated in the development of MLL-r and NPM1 mutant leukemias. We look forward to initiating the clinical program and anticipate establishing a safe dose that provides appropriate target coverage in patients."

Dr. Morrison added, "We look forward to the near-term completion of E2112, the Phase 3 registration trial of entinostat plus exemestane in HR+, HER2- breast cancer, which we anticipate will occur either in the fourth quarter of 2019 or the first half of 2020. A positive survival benefit at either assessment will enable us to file an NDA with the FDA and take us one step closer to improving outcomes for patients with this difficult to treat disease."

Pipeline Updates

Entinostat

In May 2019, Syndax announced that the E2112 trial passed its fourth interim overall survival (OS) analysis and will continue as planned until either an OS benefit is observed, or the final target number of events occur. E2112 is Syndax’s NCI-sponsored, ECOG-ACRIN led Phase 3 registration trial of entinostat, a Class I selective HDAC inhibitor, plus exemestane in advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer.

The Company continues to anticipate the final E2112 interim OS analysis in 4Q19. If necessary, the final OS assessment, which will be triggered when the trial reaches a total of 410 death events, is expected to be conducted in 2Q20. A positive OS assessment at any point would enable the Company to file for full regulatory approval. The E2112 trial design was informed by the Phase 2b ENCORE 301 trial, the results of which led to entinostat’s Breakthrough Therapy designation in HR+, HER2- breast cancer, in which patients receiving the entinostat/exemestane combination demonstrated a strong OS benefit.

SNDX-5613

In July 2019, Syndax announced that the U.S. Food and Drug Administration (FDA) cleared the Company’s Investigational New Drug (IND) application to begin a Phase 1/2 trial for SNDX-5613, a highly selective Menin inhibitor. The Company will refer to the clinical development of SNDX-5613 as the AUGMENT Program. The Phase 1/2 open-label trial will assess orally administered SNDX-5613 in adults with relapsed/refractory (R/R) acute leukemias. The Phase 1 dose escalation portion of the study will evaluate the safety, tolerability and pharmacokinetics of SNDX-5613, and will seek to establish a recommended Phase 2 dose. The Phase 2 portion will evaluate efficacy, as defined by Complete Response rate (per International Working Group response criteria), across three expansion cohorts: MLL-rearranged (MLL-r) acute lymphoblastic leukemia (ALL), MLL-r acute myeloid leukemia (AML), and NPM1 mutant AML. The Company expects to report initial clinical data from the trial in 2020.

SNDX-6352

Enrollment continues in the Phase 1 dose escalation trial of SNDX-6352, Syndax’s anti-CSF-1R monoclonal antibody, in patients with chronic graft versus host disease (cGVHD). The Company now anticipates results from this trial in the second half of 2020. The objectives of this trial are to evaluate the safety and preliminary efficacy of SNDX-6352 in cGVHD and to identify a recommended Phase 2 dose and schedule.

Corporate Updates

Syndax announced the appointment of Michael A. Metzger to its Board of Directors. Mr. Metzger has served as Syndax’s President and Chief Operating Officer since May 2015.

Second Quarter 2019 Financial Results

As of June 30, 2019, Syndax had cash, cash equivalents and short-term investments of $80.5 million and 31.6 million shares and share equivalents issued and outstanding.

Second quarter 2019 research and development expenses decreased to $12.3 million from $14.9 million. The second quarter decrease was primarily due to decreased development activities primarily in the ENCORE programs, and decreased CMC activities associated with SNDX-6352, partially offset by accrued milestone expenses associated with the development of SNDX-5613.

General and administrative expenses for the second quarter 2019 decreased to $3.5 million from $4.5 million. The decrease was primarily due to decreased professional fees and employee compensation expenses.

For the three months ended June 30, 2019, Syndax reported a net loss attributable to common stockholders of $14.9 million or $0.47 per share compared to $18.4 million or $0.74 per share for the prior year period.

Financial Guidance

Today the Company provided operating expense guidance for the third quarter and full year 2019. For the third quarter and full year 2019, research and development expenses are expected to be $11 to $12 million and $45 to $46 million, respectively, and total operating expenses are expected to be $15 to $16 million and $60 to $63 million, respectively.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Wednesday, August 7, 2019.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 8959885
Domestic Dial-in Number: 855-251-6663
International Dial-in Number: 281-542-4259
Live Webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

On August 7, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that a conference call will be held, Friday, August 9, 2019 at 8:30 AM ET to discuss results for the second quarter of 2019 and provide a business outlook for the remainder of the year (Press release, TG Therapeutics, AUG 7, 2019, http://ir.tgtherapeutics.com/news-releases/news-release-details/tg-therapeutics-host-conference-call-second-quarter-2019 [SID1234538297]). Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer will host the call.

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In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Second Quarter 2019 Business Update Call. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for replay on the Company’s website, for a period of 30 days after the call.

TG Therapeutics will announce its financial results for this period in a press release to be issued prior to the call.

On August 7, 2019 Constellation Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported its second-quarter and six-month 2019 financial results (Press release, Constellation Pharmaceuticals, AUG 7, 2019, View Source [SID1234538296]).

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"2019 is a year of data for Constellation," said Jigar Raythatha, president and chief executive officer of Constellation Pharmaceuticals. "We made multiple important data presentations in the second quarter that help to advance our vision of becoming a late-stage oncology development company, with an exciting pipeline of development and discovery programs.

"We look forward to providing further data updates in the second half of 2019 across our pipeline," Mr. Raythatha continued. "We remain deeply committed to delivering important new medicines to cancer patients around the world in order to reduce their suffering and improve their lives."

Program Updates

CPI-0610

Data presented at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) from the MANIFEST study suggest that CPI-0610 may have disease-modifying effects.

In addition to improvements in spleen volume and constitutional symptoms, the interim data suggest improvements in anemia, transfusion dependence, and bone marrow fibrosis.

Our vision for CPI-0610 is to create a differentiated treatment for MF in ruxolitinib-resistant patients and to transform standard of care as a first-line therapy. We have begun planning for pivotal trials.
EZH2 Franchise

Enrollment for the ProSTAR clinical trial for CPI-1205 continues on track.

We are enrolling patients in three cohorts:
— CPI-1205 + abiraterone in second-line mCRPC;
— CPI-1205 + enzalutamide in second-line mCRPC, randomized against enzalutamide alone; and
— CPI-1205 + enzalutamide in heavily pre-treated patients who have progressed after treatment with each of enzalutamide, abiraterone, and chemotherapy.

We plan to provide an update for ProSTAR in the fourth quarter and additional data in early 2020.

The IND for CPI-0209, our second-generation and potentially best-in-class EZH2 inhibitor, was filed and cleared by the FDA.

CPI-0209 could address additional patient populations beyond those targeted by first-generation EZH2 inhibitors.
Milestones

The Company anticipates achieving the following milestones during the second half of 2019:

CPI-0610 MANIFEST Study

Update spleen volume, symptom, and anemia data from about 40 ruxolitinib-resistant patients and bone-marrow-fibrosis changes from a subset of patients.

Update status of conversion from transfusion dependence to transfusion independence from about 16 ruxolitinib-resistant patients.

Disclose spleen volume and symptom data for 10-15 JAK-inhibitor-naïve (first-line) patients.
EZH2 Franchise

Dose the first patients in a Phase 1 clinical trial of CPI-0209.

Provide an update from the ProSTAR clinical trial of CPI-1205 across various patient contexts.
Second Quarter 2019 Financial Results

Cash, cash equivalents, and marketable securities as of June 30, 2019, were $98.1 million, a decline of 14.4% compared to December 31, 2018, primarily due to operating expenses.

Research and development (R&D) expenses increased 67.3% year over year to $16.0 million in the second quarter of 2019 mainly due to increased clinical trial expenses.

General and administrative (G&A) expenses grew 96.5% year over year to $4.9 million in the second quarter of 2019, primarily due to building out the organization of the company.

The net loss increased 73.9% year over year to $20.8 million for the second quarter of 2019, mainly due to increased R&D and G&A expenses. The net loss per share attributable to common shareholders decreased 92.0% to $0.80 per share due to an increase in shares outstanding as a result of the initial public offering in 2018 and conversion of preferred stock to common stock.
First Half 2019 Financial Results

Research and development (R&D) expenses increased 63.0% year over year to $31.6 million in the first half of 2019, mainly due to increased clinical trial expenses.

General and administrative (G&A) expenses grew 94.5% year over year to $9.3 million in the first half of 2019, primarily due to building out the organization of the company.

The net loss increased 67.2% year over year to $40.2 million for the first half of 2019, mainly due to increased R&D and G&A expenses. The net loss per share attributable to common shareholders decreased 92.9% to $1.56 per share due to an increase in shares outstanding as a result of the initial public offering in 2018 and conversion of preferred stock to common stock.
Financial Guidance

Constellation expects that cash, cash equivalents, and marketable securities as of June 30, 2019, will enable the Company to fund planned operating expenses and capital expenditure requirements until late third-quarter 2020.