On August 1, 2019 Kaleido Biosciences, Inc. (Nasdaq: KLDO), a clinical-stage healthcare company with a chemistry-driven approach to leveraging the potential of the microbiome organ to treat disease and improve human health, reported financial results for the second quarter ended June 30, 2019, and provided a corporate update (Press release, Kaleido Biosciences, AUG 1, 2019, View Source [SID1234540067]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In the second quarter we made important progress with our pipeline and now have five clinical studies underway with our Microbiome Metabolic Therapy (MMT) candidates," said Alison Lawton, President and Chief Executive Officer of Kaleido. "We initiated Kaleido’s first Phase 2 clinical trial with the advancement of KB195 for urea cycle disorders (UCD) under an IND. For our KB174 program, we completed enrollment in a dosing study in healthy subjects and continued to progress our clinical study in patients with cirrhosis. We also advanced a third MMT, KB109, into a clinical study in patients colonized with multi-drug resistant pathogens. We look forward to a number of key milestones expected during the second half of the year, including data from three clinical studies in two different patient populations."

Recent Highlights

Received approval from regulatory authorities in the United Kingdom and Belgium for the Company’s clinical trial application (CTA) for its Phase 2 clinical trial of KB195 in UCD

Initiated the Phase 2 clinical trial evaluating KB195 in patients with UCD

Initiated a non-IND clinical study of KB109 in patients colonized with multi-drug resistant pathogens

Expanded Kaleido’s Scientific Advisory Board to include six leading experts in microbiology, chemistry and microbiome research across a variety of therapeutic areas:

Laurie H. Glimcher, M.D., President and CEO, Dana-Farber Cancer Institute, Director, Dana-Farber/Harvard Cancer Center, and Richard and Susan Smith Professor of Medicine, Harvard Medical School;

Robert Jenq, M.D., Assistant Professor in the Department of Genomic Medicine, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center;

C. Ronald Kahn, M.D., Head of the Section on Integrative Physiology and Metabolism, Past President & CEO, Joslin Diabetes Center, and Mary K. Iacocca Professor of Medicine, Harvard Medical School;

Laura L. Kiessling, Ph.D., Novartis Professor of Chemistry, Massachusetts Institute of Technology;

Max Nieuwdorp, M.D., Professor of Internal Medicine and endocrinologist, University of Amsterdam’s Faculty of Medicine (AMC-UvA); and

Peter J. Turnbaugh, Ph.D., Associate Professor, Department of Microbiology and Immunology, G.W. Hooper Research Foundation, and Investigator, Chan Zuckerberg Biohub, University of California, San Francisco.

Appointed Jerald Korn as General Counsel and Corporate Secretary

Members of Kaleido’s leadership team were featured speakers at the 4th Microbiome Movement – Drug Development Summit held June 26-28, 2019 in Boston

Anticipated Key Milestones
Safety and tolerability data from a non-IND clinical study of KB195 in patients with UCD accepted for presentation at the annual Society for the Study of Inborn Errors of Metabolism Symposium, Sept 3-6, 2019

Data from a non-IND clinical study of KB174 in healthy subjects to explore dosing expected in Q4 2019

Data from a non-IND clinical study of KB174 in patients with well-compensated cirrhosis expected in Q4 2019
Second Quarter 2019 Financial Results
For the second quarter ended June 30, 2019, Kaleido reported a net loss of approximately $24.6 million, or $0.83 per share, basic and diluted, compared to a net loss for the quarter ended June 30, 2018 of $12.3 million, or $2.57 per share, basic and diluted.
Total operating expenses for the quarter ended June 30, 2019 were $25.0 million compared to $12.4 million for the quarter ended June 30, 2018.
Research and development expenses for the quarter ended June 30, 2019 were $18.8 million, compared to $9.1 million for the quarter ended June 30, 2018. The increase in expense for the quarter was driven by an increase in external manufacturing and research costs to support the production of study material used in preclinical studies, human clinical studies, and clinical trials as well as clinical research organizations costs associated with MMT candidates for 2019.
General and administrative expenses were $6.2 million for the quarter ended June 30, 2019, compared to $3.2 million for the quarter ended June 30, 2018. The increase in general and administrative expenses compared to the same quarter last year was driven by increased professional services and employee-related costs as the Company continues to expand its infrastructure to support its growth.
Total operating expenses for the quarter ended June 30, 2019 include non-cash stock-based compensation expense of $2.4 million compared to $0.5 million of non-cash stock-based compensation expense for the quarter ended June 30, 2018.
Cash and cash equivalents were $100.0 million as of June 30, 2019.

About Microbiome Metabolic Therapies (MMT)
Kaleido’s Microbiome Metabolic Therapies, or MMTs, are designed to drive the function and distribution of the microbiome organ’s existing microbes in order to decrease or increase the production of metabolites, or to advantage or disadvantage certain bacteria in the microbiome community. The Company’s initial MMT candidates are targeted glycans that are orally administered, have limited systemic exposure, and are selectively-metabolized by enzymes in the microbiome. Kaleido utilizes its human-centric discovery and development platform to study MMTs in microbiome samples from healthy subjects and patients in an ex vivo setting, followed by advancing MMT candidates rapidly into non-Investigational New Drug (non-IND) clinical studies in healthy subjects and patients. These non-IND human clinical studies are conducted under regulations supporting research with food, measuring safety, tolerability and potential markers of effect. For MMT candidates that are further developed as therapeutics, the Company conducts clinical trials under an IND or regulatory equivalent outside the U.S., and in Phase 2 or later development.

On August 1, 2019 Pfenex Inc. (NYSE American: PFNX) and Arcellx, Inc. reported a Development, Evaluation and License agreement under which Arcellx gains access to the proprietary Pfenex Expression Technology platform to advance multiple proprietary sparX proteins that activate, silence and reprogram Antigen- Receptor Complex T cell based therapies (Press release, Pfenex, AUG 1, 2019, View Source [SID1234538434]). Pfenex has successfully completed expression screening and process development activities for the first sparX program. Technology transfer of the program to a cGMP manufacturing facility is underway. The success of the first program has encouraged both parties to initiate a second sparX program, both programs are focused on the treatment of hematologic malignancies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Under the terms of the agreement, Pfenex is eligible to receive development funding in addition to development, regulatory and commercial milestones ranging from $2.6M up to $18M for each product incorporating a SparX protein expressed using the Pfenex Expression Technology as well as royalties on worldwide sales of any such products.

"Our collaboration with Arcellx fits our strategy of leveraging the Pfenex protein production platform to advance our products and those of our collaborators. Pfenex’s success with the first sparX program further validates the versatility of our proprietary protein expression platform and the quality of our development capabilities. We look forward to collaborating with Arcellx through the combination of our respective platforms in support of developing new therapies for patients in need." said Eef Schimmelpennink, chief executive officer of Pfenex.

"The uniquely efficient protein expression technologies and process expertise developed by Pfenex have been critical in accelerating development of our first sparX program," said David Hilbert, chief executive officer of Arcellx. "Our continued development of new sparX programs provides the basis for a forward looking Pfenex collaboration involving multiple sparX proteins with the potential to revolutionize immune cell therapies."

On August 1, 2019 La Jolla Pharmaceutical Company (Nasdaq: LJPC), a leader in the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, reported financial results for the three and six months ended June 30, 2019 and highlighted recent corporate progress (Press release, La Jolla Pharmaceutical, AUG 1, 2019, View Source [SID1234538315]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Recent Corporate Progress

GIAPREZATM (angiotensin II)

Net Sales: For the three months ended June 30, 2019, GIAPREZA net sales were $5.7 million, up 258% from the same period in 2018, and up 30% from the three months ended March 31, 2019. For the six months ended June 30, 2019, GIAPREZA net sales were $10.1 million, up 320% from the same period in 2018.

Positive CHMP Opinion: In June 2019, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for La Jolla’s Marketing Authorisation Application (MAA) for GIAPREZA for the treatment of refractory hypotension in adults with septic or other distributive shock. The CHMP’s positive opinion was sent to the European Commission (EC), which has the authority to approve medicines for the 28 European Union member countries. Approval would also be recognized in Iceland, Norway and Liechtenstein. We expect a final approval decision on the GIAPREZA MAA by the EC in the third quarter of 2019.

Investigational Products

Breakthrough Therapy Designation and Orphan Drug Designation Received from the FDA for LJPC-0118 (artesunate): The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation and Orphan Drug designation for LJPC-0118 for the treatment of malaria in April 2019 and July 2019, respectively. The active pharmaceutical ingredient in LJPC-0118, artesunate, was demonstrated to be superior to quinine in reducing mortality in patients with severe falciparum malaria infection in two randomized, controlled, clinical studies. We plan to file a New Drug Application (NDA) for LJPC-0118 with the FDA in the fourth quarter of 2019.

Positive Results Announced from Pre-Specified Interim Analysis of Phase 2 Study of LJPC-401 in Patients with Hereditary Hemochromatosis: In June 2019, we announced positive results from the pre-specified interim analysis of our Phase 2 study of LJPC-401 (synthetic human hepcidin) in patients with hereditary hemochromatosis (HH). The interim analysis of efficacy included 26 patients who had reached the end of the 16-week treatment period, and the interim analysis of safety included 60 randomized patients. Treatment with LJPC-401 resulted in a statistically significant reduction in transferrin saturation (TSAT) from baseline to the end of treatment (16 weeks), the primary efficacy endpoint of the study: LJPC‑401‑treated patients had a mean reduction in TSAT of 42% compared to placebo-treated patients who had a mean reduction of 6% (p<0.0001). The requirement for and frequency of phlebotomy procedures, a key secondary endpoint of the study, also was statistically significant: LJPC-401-treated patients had 0.06 phlebotomies per month compared to placebo-treated patients who had 0.41 phlebotomies per month (p=0.003). There were 3 phlebotomies in 2 LJPC-401-treated patients and 24 phlebotomies in 9 placebo-treated patients. LJPC-401 was well tolerated. The most frequent treatment-emergent adverse events (TEAEs) were injection site reactions (ISRs). The ISRs were all mild or moderate in severity, and no ISRs resulted in treatment discontinuation. As of the interim analysis, there were no serious TEAEs reported. We expect to announce top-line results of LJ401-HH01 in the fourth quarter of 2019.

"We are pleased with the progress made in the first half of 2019, which included the achievement of significant milestones for each of GIAPREZA, LJPC-0118 and LJPC-401," said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. "In the second half of 2019, we look forward to continued growth in GIAPREZA net sales, the final approval decision

on the GIAPREZA MAA by the EC, our filing of an NDA for LJPC-0118 with the FDA and top-line results of our Phase 2 study of LJPC-401 in patients with HH."

Financial Results

For the three and six months ended June 30, 2019, GIAPREZA net sales were $5.7 million and $10.1 million, respectively, compared to $1.6 million and $2.4 million, respectively, for the same periods in 2018. La Jolla’s net loss for the three and six months ended June 30, 2019 was $30.4 million and $62.1 million, or $1.12 per share and $2.29 per share, respectively, compared to $52.8 million and $103.3 million, or $2.02 per share and $4.22 per share, respectively, for the same periods in 2018. La Jolla continues to expect full-year 2019 GIAPREZA net sales of $24 million to $28 million.

As of June 30, 2019, La Jolla had $123.4 million in cash, compared to $172.6 million as of December 31, 2018. Net cash used in operating activities for the three and six months ended June 30, 2019 was $16.5 million and $49.2 million, respectively, compared to $37.5 million and $83.4 million, respectively, for the same periods in 2018. La Jolla has no debt. La Jolla continues to expect that its net cash used in operating activities in 2019 will be $89 million to $94 million.

About GIAPREZA

In December 2017, GIAPREZA (angiotensin II) was approved by the U.S. Food and Drug Administration (FDA) as a vasoconstrictor indicated to increase blood pressure in adults with septic or other distributive shock. GIAPREZA mimics the body’s endogenous regulatory peptide that is central to the renin-angiotensin-aldosterone system to increase blood pressure. Prescribing information for GIAPREZA is available at www.giapreza.com. GIAPREZA is marketed by La Jolla Pharmaceutical Company on behalf of La Jolla Pharma, LLC, its wholly owned subsidiary.

IMPORTANT SAFETY INFORMATION

Contraindications

None

Warnings and Precautions

There is a potential for venous and arterial thrombotic and thromboembolic events in patients who receive GIAPREZA. Use concurrent venous thromboembolism (VTE) prophylaxis.

Adverse Reactions

The most common adverse reactions that were reported in greater than 10% of GIAPREZA-treated patients were thromboembolic events.

Drug Interactions

Angiotensin converting enzyme (ACE) inhibitors may increase response to GIAPREZA. Angiotensin II receptor blockers (ARB) may reduce response to GIAPREZA.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

For additional information, please see Full Prescribing Information for the United States.

About LJPC-0118

LJPC-0118 is La Jolla’s investigational product for the treatment of severe malaria. The active pharmaceutical ingredient in LJPC-0118, artesunate, was demonstrated to be superior to quinine in reducing mortality in patients with severe falciparum malaria infection in two randomized, controlled, clinical studies. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation and Orphan Drug designation for LJPC-0118 for the treatment of malaria in April 2019 and July 2019, respectively. La Jolla plans to file a New Drug Application (NDA) for LJPC-0118 with the FDA in the fourth quarter of 2019 for the treatment of severe malaria. Severe malaria is a serious and sometimes fatal disease caused by a parasite that

commonly infects a certain type of mosquito, which feeds on humans. Symptoms include but are not limited to: fever, chills, sweating, hypoglycemia and shock. Severe malaria is often complicated by central nervous system infections that may lead to delirium, which may progress to coma. Infections usually occur a few weeks after being bitten. In 2017, an estimated 219 million cases of malaria occurred worldwide, with an estimated 200 million of these cases occurring in the World Health Organization (WHO) African Region, and, in 2013, the global annual incidence of severe malaria was estimated to be 2 million cases. In 2017, an estimated 435,000 people died from malaria worldwide.

About LJPC-401

LJPC-401, a clinical-stage investigational product, is La Jolla’s proprietary formulation of synthetic human hepcidin. Hepcidin, an endogenous peptide hormone, is the body’s naturally occurring regulator of iron absorption and distribution. In healthy individuals, hepcidin prevents excessive iron accumulation in vital organs, such as the liver and heart, where it can cause significant damage and even result in death. La Jolla is developing LJPC-401 for the potential treatment of iron overload, which occurs as a result of primary iron overload diseases such as hereditary hemochromatosis (HH), or secondary iron overload diseases such as beta thalassemia (BT), sickle cell disease (SCD), myelodysplastic syndrome (MDS) and polycythemia vera. The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has designated LJPC‑401 as an orphan medicinal product for the treatment of beta thalassemia intermedia and major and SCD.

On August 1, 2019 Syros Pharmaceuticals (NASDAQ: SYRS), a leader in the development of medicines that control the expression of genes, reported financial results for the quarter ended June 30, 2019, and provided an update on recent accomplishments and upcoming events (Press release, Syros Pharmaceuticals, AUG 1, 2019, View Source [SID1234538098]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This is an exciting time for Syros as we continue to advance our clinical-stage programs toward multiple data readouts this year and next that have the potential to validate our fundamental approach to drug development and catalyze change in the treatment landscape for cancers with high unmet needs," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "We plan to report updated clinical data for SY-1425 and SY-1365 in the fourth quarter, which we expect will provide additional insights into the safety and clinical activity of both drug candidates and further inform our development plans. We are particularly pleased to announce that we recently began enrolling relapsed or refractory AML patients in a new cohort in our Phase 2 trial of SY-1425 in combination with azacitidine. With this accomplishment, we now have three ongoing trial cohorts with potential for proof-of-concept data in 2020 that, if positive, bring us closer to our vision of providing much-needed therapies for patients."

Upcoming Milestones:

SY-1425

Syros plans to complete enrollment in the second half of 2019 in the Phase 2 trial cohort evaluating the safety and efficacy of SY-1425 in combination with azacitidine in RARA and IRF8 biomarker-positive patients with newly diagnosed AML who are not suitable candidates for standard chemotherapy.
Syros plans to report updated data on SY-1425 in combination with azacitidine in the fourth quarter of 2019 in newly diagnosed patients with AML who are not suitable candidates for standard chemotherapy.
Syros plans to report potential proof-of-concept data from the ongoing cohort evaluating SY-1425 in combination with azacitidine in biomarker-positive patients with relapsed or refractory (R/R) AML in 2020.
SY-1365

Syros plans to report initial clinical data in the fourth quarter of 2019 from the expansion portion of its Phase 1 trial, including: initial efficacy and safety assessments from the cohort evaluating SY-1365 as a single agent in high-grade serous ovarian cancer patients who have had three or more prior lines of therapy; initial safety and pharmacokinetic data from the cohort evaluating SY-1365 in combination with carboplatin in high-grade serous ovarian cancer patients who have had one or more prior lines of therapy; and initial safety, efficacy and mechanistic data from the cohort evaluating SY-1365 as a single agent in patients with advanced solid tumors accessible for biopsy.
Syros plans to report additional data from these cohorts, including potential proof-of-concept data from the ongoing cohort in high-grade serous ovarian cancer patients who have had three or more prior lines of therapy, in 2020.
Syros also plans to report potential proof-of-concept data from an ongoing cohort evaluating SY-1365 as a single agent in patients with relapsed ovarian clear cell cancer and initial data from an ongoing cohort in hormone receptor (HR)-positive CDK4/6 inhibitor-resistant breast cancer patients in 2020.
SY-5609

Syros plans to present new preclinical data on SY-5609, including pharmacokinetic and pharmacodynamic data and assessments of anti-tumor activity in patient-derived xenograft models of multiple cancers, in the fourth quarter.
Syros plans to complete investigational new drug (IND)-enabling studies of SY-5609 in 2019 to support the initiation of a Phase 1 oncology trial in early 2020.
Recent Pipeline Highlights:

In May 2019, Syros opened for enrollment the new Phase 2 trial cohort evaluating the safety and efficacy of SY-1425 in combination with azacitidine in RARA and IRF8 biomarker-positive patients with R/R AML.
In May 2019, Syros published a new manuscript, Discovery and Characterization of SY-1365, a Selective, Covalent Inhibitor of CDK7, in the American Association for Cancer Research (AACR) (Free AACR Whitepaper)’s journal Cancer Research. The publication highlighted the discovery, mechanism of action and promise of SY-1365 as a new targeted approach to treat a range of difficult-to-treat cancers.
Recent Corporate Highlights:

In June 2019, Syros appointed Alice Shaw, M.D., Ph.D., Director of the Center for Thoracic Cancers at Massachusetts General Hospital and a Professor of Medicine at Harvard Medical School, to its Board of Directors. Dr. Shaw is a highly respected oncologist and recognized leader in translational medicine and the development of targeted cancer therapies.
Second Quarter 2019 Financial Results:

Syros had cash, cash equivalents and marketable securities of $121.7 million as of June 30, 2019, as compared with $99.7 million in December 31, 2018. This increase in cash reflects aggregate net proceeds of approximately $65.0 million from Syros’ two concurrent underwritten public offerings, which closed in April 2019.

For the second quarter of 2019, Syros reported a net loss of $19.5 million, or $0.47 per share, compared to a net loss of $14.0 million, or $0.43 per share, for the same period in 2018.

Revenues were $0.5 million for the second quarter of 2019, as compared to $0.4 million for the second quarter of 2018. Revenues in both the second quarter of 2019 and the second quarter of 2018 were earned under Syros’ collaboration with Incyte Corporation.
Research and development (R&D) expenses were $15.5 million for the second quarter of 2019, as compared to $11.1 million for the same period in 2018. This increase was primarily attributable to continued advancement of the Company’s existing clinical trials and advancement of its preclinical programs, including SY-5609 into IND-enabling studies.
General and administrative (G&A) expenses were $5.2 million for the second quarter of 2019, as compared to $3.8 million for the same period in 2018. This increase was primarily attributable to an increase in employee-related expenses.
Financial Guidance:
Based on its current plans, Syros believes that its existing cash, cash equivalents and marketable securities will be sufficient to fund its planned operating expenses and capital expenditure requirements to the end of the first quarter of 2021.

Conference Call and Webcast:
Syros will host a conference call today at 8:30 a.m. ET to discuss these second quarter 2019 financial results and provide a corporate update.

To access the live conference call, please dial (866) 595-4538 (domestic) or (636) 812-6496 (international), and refer to conference ID 7254685. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the call.

On August 1, 2019 BioTime, Inc. (NYSE American and TASE: BTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, reported that it will report its second quarter 2019 financial and operating results on Thursday, August 8th, 2019, following the close of the U.S. financial markets (Press release, BioTime, AUG 1, 2019, View Source [SID1234538097]). BioTime management will also host a conference call and webcast on Thursday, August 8th, 2019, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its second quarter 2019 financial results and to provide a business update.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the "BioTime Inc. Call". A live webcast of the conference call will be available online in the Investors section of BioTime’s website. A replay of the webcast will be available on BioTime’s website for 30 days and a telephone replay will be available through August 15th, 2019, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 8783397.