On April 29, 2019 Cassava Sciences, Inc. (Nasdaq: SAVA), a biopharmaceutical company, reported financial results for the first quarter ended March 31, 2019 (Press release, Pain Therapeutics, APR 29, 2019, View Source [SID1234535562]). . Net loss was $1.4 million, or $0.08 per share . This compared to a net loss of $2.2 million, or $0.33 per share, for the same period in the prior year. Cash and cash equivalents were $19.1 million as of March 31, 2019. The Company has no debt. Cassava Sciences utilized $0.7 million of cash during the first quarter of 2019 and expects cash use to be $5.0 – $6.0 million for full year 2019.

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"As we enter 2019, our financial expectations reflect a thoughtful balance between maintaining fiscal discipline and advancing our series of product candidates aimed at Alzheimer’s disease", said Remi Barbier, President & CEO. "One thing that won’t change is our focus on developing potential breakthrough innovations and an unwavering dedication to improve people’s lives. This emphasis has characterized our history and remains core to our strategy for 2019 and beyond."

Cassava Sciences is conducting a Phase 2 clinical program with its investigational drug, PTI-125, in patients with Alzheimer’s disease. PTI-125 is designed to exert anti-neuroinflammatory effects and to restore the function of three key receptors in the brain. The National Institutes of Health (NIH) is providing substantial scientific and financial support for the Company’s clinical program.

Cassava Sciences expects to announce results of its Phase 2a study in the second half of 2019, after study participants complete drug treatment and their data are analyzed.

Financial Highlights for First Quarter 2019

At March 31, 2019, cash and cash equivalents were $19.1 million, compared to $19.8 million at December 31, 2018. The company has no debt. Net cash utilized during the first quarter 2019 was $0.7 million.
Net loss was $1.4 million compared to $2.2 million for the same period in the prior year, representing a 37% decrease. Net loss per share was $0.08 compared to $0.33 for the same period in the prior year.
We received research grant funding reimbursements of $0.8 million from NIH and recorded this as a reduction in research and development expenses ("R&D"). This compared to $0.4 million of NIH grant receipts received for the same period in the prior year.
R&D expenses were $0.6 million. This compared to $1.1 million for the same period in the prior year, representing a 46% decrease. The decrease was due primarily to the increase in NIH grant funding in 2019 compared to the prior year combined with a decrease in non-cash stock-based compensation expense. R&D expenses included non-cash stock related compensation costs of $0.1 million compared to $0.4 million for same period in the prior year.
General and administrative ("G&A") expenses were $0.9 million. This compared to $1.1 million for the same period in the prior year, representing a 20% decrease. G&A expenses included non-cash stock-based compensation costs of $0.2 million compared to $0.5 million for the same period in the prior year.
Our Scientific Approach
The target of PTI-125 is an altered form of filamin A (FLNA). FLNA is a scaffolding protein found throughout the body. The function of a scaffolding protein is to bring multiple proteins together and to ensure they interact properly. However, an altered and highly toxic form of FLNA is found in the Alzheimer’s brain. Altered FLNA disrupts the normal function of neurons, leading to neurodegeneration and brain inflammation. Our investigational drug candidate, PTI-125, is designed to restore the normal shape & function of FLNA in the brain. This drug effect exerts powerful anti-neuroinflammatory effects and improves the function of multiple brain receptors.

In animal models of disease, treatment with PTI-125 resulted in dramatic improvements in brain health, such as improved learning and memory; improved insulin receptor signaling; and significant reductions in levels of inflammatory cytokines in the brain.

We are also developing a biomarker/diagnostic to detect Alzheimer’s disease with a simple blood test. This program, called PTI-125Dx, also receives scientific and financial support from NIH.

The underlying science for our programs in neurodegeneration is published in several prestigious peer-reviewed technical journals, including Journal of Neuroscience, Neurobiology of Aging, and Journal of Biological Chemistry. As previously announced, NIH awarded us two research grants in 2018 following an in-depth, confidential review of our science and technology. These two NIH grants represent up to $6.7 million of non-dilutive financing.

About Alzheimer’s Disease
Alzheimer’s disease is a progressive brain disorder that destroys memory and thinking skills. Eventually, a person with Alzheimer’s disease may be unable to carry out even simple tasks. Currently, there are no drug therapies to halt Alzheimer’s disease, much less reverse its course.
An estimated 5.8 million Americans of all ages are living with Alzheimer’s disease in 2019.

On April 29, 2019 G1 Therapeutics, Inc. (Nasdaq: GTHX), a clinical-stage oncology company, reported a regulatory update on trilaciclib, a first-in-class myelopreservation agent designed to protect the bone marrow from damage by chemotherapy and improve patient outcomes (Press release, G1 Therapeutics, APR 29, 2019, View Source [SID1234535553]).

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Based on written feedback from its end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) and discussions with European regulatory authorities, the company plans to submit marketing applications in the U.S. and Europe for trilaciclib for myelopreservation in small cell lung cancer (SCLC). These submissions will be based on currently available data from three randomized, double-blind, placebo-controlled SCLC clinical trials, as well as safety data collected across all completed and ongoing clinical trials.

"We are pleased with the feedback from our recent meetings with regulatory authorities. We look forward to continuing a collaborative dialogue regarding the marketing applications, as well as discussing further clinical development of trilaciclib," said Raj Malik, M.D., Chief Medical Officer. "We believe trilaciclib represents an important advance in the care of SCLC patients. We will also move forward with a robust development program to evaluate trilaciclib in multiple tumor types and chemotherapy regimens."

G1 will request a pre-New Drug Application (NDA) meeting with the FDA and anticipates it will be scheduled later this year. The company will provide further details regarding the NDA submission and timeline following that meeting. The company plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) subsequent to an NDA filing.

Chemotherapy is an effective and important weapon against cancer. However, chemotherapy does not differentiate between healthy cells and cancer cells and kills both, including important stem cells in the bone marrow that produce white blood cells, red blood cells and platelets. This chemotherapy-induced bone marrow damage is known as myelosuppression. When white blood cells, red blood cells and platelets become depleted, chemotherapy patients are at increased risk of infection, experience anemia and fatigue, and are at increased risk of bleeding. Myelosuppression often requires the administration of rescue interventions such as growth factors and blood or platelet transfusions, and may also result in chemotherapy dose delays and reductions.

"In clinical trials, trilaciclib demonstrated the ability to protect bone marrow from chemotherapy damage and meaningfully reduced the need for supportive care interventions, such as G-CSF and transfusions," said Jared Weiss, M.D., Associate Professor, University of North Carolina Lineberger Comprehensive Cancer Center, and trilaciclib clinical trial investigator. "By providing a proactive approach to reduce myelosuppression, trilaciclib improves the patient experience during chemotherapy treatment, reducing side effects and the need for associated interventions commonly given to treat them."

About Trilaciclib

Trilaciclib is a first-in-class myelopreservation agent designed to protect the bone marrow from damage by chemotherapy and improve patient outcomes. Trilaciclib is being evaluated in four randomized Phase 2 clinical trials; G1 reported positive results from all these trials in 2018.

On April 29, 2019 Eagle Pharmaceuticals, Inc. ("Eagle" or the "Company") (Nasdaq: EGRX) reported that the Company will release its 2019 first quarter financial results on Tuesday, May 7, 2019, before the market opens (Press release, Eagle Pharmaceuticals, APR 29, 2019, View Source [SID1234535549]).

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Scott Tarriff, Chief Executive Officer, and Pete Meyers, Chief Financial Officer, will host a conference call to discuss the results as follows:

Date Tuesday, May 7, 2019
Time
8:30 a.m. EDT

Toll free (U.S.) 877-876-9176
International 785-424-1670
Webcast (live and replay)
www.eagleus.com, under the "Investor Relations" section

A replay of the conference call will be available for one week after the call’s completion by dialing 800-839-5493 (US) or 402-220-2552 (International) and entering conference call ID EGRXQ119. The webcast will be archived for 30 days at the aforementioned URL.

On April 29, 2019 Intellia Therapeutics, Inc. (NASDAQ:NTLA), reported that it will present new data, including the first demonstration of targeted gene insertion with CRISPR/Cas9 in the liver of non-human primates (NHPs), at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper), taking place April 29-May 2, 2019, in Washington, D.C (Press release, Intellia Therapeutics, APR 29, 2019, View Source [SID1234535545]). Researchers also will present today new in vitro data from Intellia’s lead engineered cell therapy program in acute myeloid leukemia (AML). Later this week at the 2019 ASGCT (Free ASGCT Whitepaper) meeting, Intellia will present new data from its primary hyperoxaluria (PH1) program.

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"The data we are presenting at ASGCT (Free ASGCT Whitepaper) reflects our rapid progress with Intellia’s modular CRISPR/Cas9 platform across a variety of in vivo and engineered cell therapeutic applications," said Intellia President and Chief Executive Officer John Leonard, M.D. "Today’s presentation of our most recent targeted gene insertion data depicts Intellia’s second successful demonstration of CRISPR-mediated gene editing in non-human primates, both in collaboration with Regeneron Pharmaceuticals, Inc. The first was through gene knockout in our transthyretin amyloidosis program and, now, we have used our targeted insertion approach with Factor 9 as a model gene. We also continue to make strides with our collaborators at IRCCS Ospedale San Raffaele toward developing engineered cell therapies for a variety of intractable cancers, such as acute myeloid leukemia."

Intellia’s First Demonstration of Targeted Gene Insertion in NHPs

In a follow-up to Intellia’s presentation at the 2018 European Society of Gene and Cell Therapy (ESGCT) meeting of the first robust demonstration of CRISPR-mediated insertion of transgenes in the liver of mice using Factor 9 (F9) as a model gene, the company will present at ASGCT (Free ASGCT Whitepaper) an advancement of its modular hybrid delivery system, which combines Intellia’s lipid nanoparticle (LNP) platform with an adeno-associated virus (AAV). F9 is a gene that encodes FIX, a blood-clotting protein that is missing or defective in hemophilia B patients.

In a collaboration between Intellia and Regeneron, researchers delivered F9 DNA via a proprietary bi-directional insertion template to demonstrate targeted gene insertion in NHPs, resulting in circulating human FIX protein levels within the normal range of human FIX protein levels (3-5 μg/mL; source: Amiral et al., Clin. Chem., 1984). Researchers observed circulating human FIX protein levels of ~3-4 μg/mL at day 14 after a single dose in an ongoing study, with levels sustained through 28 days (~3-5 μg/mL).

Today, researchers additionally will share updated results from an ongoing durability study, first reported in October, that the circulating supratherapeutic human FIX protein levels achieved in mice with Intellia’s hybrid LNP-AAV delivery system have remained stable through 10 months of observation. Further, researchers will show that they can regulate FIX protein levels in mice by varying LNP doses, AAV doses or insertion site.

Today’s presentation, titled "CRISPR/Cas9-Mediated Targeted Insertion of Human F9 Achieves Therapeutic Circulating Protein Levels in Mice and Non-Human Primates," will be made by Hon-Ren Huang, associate director, Vector Biology, Intellia. This presentation will be accessible through the Events and Presentations page of the Investor Relations section of Intellia’s website at www.intelliatx.com.

Data Update from Intellia’s Acute Myeloid Leukemia Program

Intellia and its research collaborator, IRCCS Ospedale San Raffaele, will provide an update on the company’s lead engineered cell therapy program in AML. Researchers will present new in vitro data showing that CRISPR/Cas9 editing resulted in >98% knockout of the endogenous T cell receptor (TCR), while achieving transfer of various Wilms’ Tumor 1 (WT1)-specific TCRs into >95% of isolated T cells. Researchers generated and tested a library of TCRs with different epitope specificities and human leukocyte antigen (HLA) restrictions, building on the data reported at the 2018 ESGCT meeting. Several lead TCRs restricted to the HLA-A*02:01 allele, a frequently expressed allele in the western hemisphere, show the desired T cell characteristics, including high affinity, epitope specificity and killing of a panel of primary leukemic blast cells that expressed the WT1 antigen.

Intellia and OSR are collaborating to develop best-in-class CRISPR-edited T cells directed to a specific epitope of WT1, a tumor-associated antigen overexpressed across a wide range of different tumor types and a known driver of leukocyte blasts in hematological cancers. Intellia’s first cell therapy tumor target is WT1 for the treatment of AML and other potential hematological malignancies, as well as for solid tumors.

Today’s presentation, titled "Exploiting Clonal Tracking of WT1-Specific T Cells to Generate a Library of Tumor-Specific T Cell Receptors (TCR), for TCR Gene Editing of Acute Leukemia," will be made by Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy.

Additional In Vivo Data to be Presented at the 2019 ASGCT (Free ASGCT Whitepaper) Meeting

Intellia’s third oral presentation at the 2019 ASGCT (Free ASGCT Whitepaper) Meeting will take place later this week, on Thur., May 2, 2019. In a presentation titled "CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria," the company will provide information demonstrating successful independent knockout of two targets of interest, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1 (HAO1), to address PH1 in a PH1 mouse model.

The data shows the continued progression of Intellia’s modular platform capability using CRISPR to knock out liver gene targets. The data being presented includes results from an ongoing collaboration with researchers at the University of Alabama at Birmingham.

On April 29, 2019 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the first quarter ended March 31, 2019 (Press release, CRISPR Therapeutics, APR 29, 2019, View Source [SID1234535456]).

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"This past quarter, we began an important new period for CRISPR Therapeutics with the treatment of the first patient in our clinical trial for CTX001 in hemoglobinopathies," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "This is a significant landmark for the Company and we continue to enroll patients in our trials for both beta thalassemia and sickle cell disease. With the acceptance of our IND and CTA for CTX110, we look forward to the initiation of our clinical trials for our allogeneic CAR-T programs in the near-term and hope to bring other CAR-T programs to the clinic in the next six to twelve months."

Recent Highlights and Outlook

Hemoglobinopathies

Beta thalassemia
In April 2019, CRISPR Therapeutics and its partner, Vertex, announced that the U.S. Food and Drug Administration (FDA) had granted Fast Track Designation for CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT). In February 2019, CRISPR and Vertex announced that the first patient had been treated with CTX001 in a Phase 1/2 clinical study of patients with TDT, marking the first company-sponsored use of a CRISPR/Cas9 therapy in a clinical trial. Enrollment in the Phase 1/2 study in patients with TDT is ongoing.

Sickle Cell Disease
The companies are also evaluating CTX001 for the treatment of sickle cell disease (SCD) and received Fast Track Designation for CTX001 from the FDA in January 2019 for SCD. The companies announced in February 2019 that the first patient had been enrolled in a Phase 1/2 clinical study of CTX001 in severe SCD in the U.S. and is expected to be infused with CTX001 in mid-2019. Enrollment in the Phase 1/2 study in patients with SCD is ongoing.
Immuno-Oncology

CTX110
Earlier this year, the FDA approved CRISPR Therapeutics’ Investigational New Drug (IND) application for CTX110, its wholly-owned allogeneic CAR-T cell therapy targeting CD19+ malignancies. Additionally, the Company has obtained approval from Health Canada for its Clinical Trial Application (CTA). CRISPR Therapeutics remains on track to initiate a Phase 1/2 trial to assess the safety and efficacy of CTX110 in the first half of 2019 and continues to work closely with various clinical sites to begin the trial. The Company’s proprietary CRISPR-based allogeneic CAR-Ts have the potential to create the next-generation of cell therapies that may have a superior product profile compared to current autologous therapies and allow accessibility to broader patient populations.

CRISPR Therapeutics continues to advance additional allogeneic CAR-T candidates including: CTX120, targeting B-cell maturation antigen (BCMA) for the treatment of multiple myeloma; and CTX130, targeting CD70 for the treatment of solid tumors and hematologic malignancies. Earlier this month, the Company presented preclinical data targeting multiple solid tumor types with anti-CD70 allogeneic CAR-T cells, further demonstrating consistent expression, durability, selectivity and potent cell killing. The Company also presented data targeting CD33 that showed potent preclinical activity against acute myeloid leukemia (AML) cells. These data were presented during poster sessions at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

CRISPR Therapeutics will present additional data at the upcoming 2019 American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting demonstrating the potential of CRISPR/Cas9 allogeneic CAR-T cell candidates for multiple oncology targets. CRISPR/Cas9 allogeneic CAR-T cells show consistently high percent CAR expression, while maintaining durable potency, low exhaustion, and lack of alloreactivity (#841). The Company will also present data evaluating homology-independent CRISPR/Cas9 off-target assessments methods (#134).
Corporate Development

The U.S. Patent Office issued four patents to Dr. Emmanuelle Charpentier, together with The Regents of the University of California and University of Vienna (collectively, CVC). Additionally, CVC was recently granted its third patent in Europe. These patents expand CVC’s wide-ranging CRISPR-Cas9 gene-editing patent portfolio.

Earlier this year, CRISPR Therapeutics announced strategic collaborations with StrideBio, Inc. and ProBioGen. The Company continues to examine new technologies and capabilities in support of existing programs.

In February, CRISPR Therapeutics proposed to elect John T. Greene and Katherine A. High, M.D. to its Board of Directors at the Company’s upcoming annual general meeting to be held later this year. Together, they will bring significant strategic and operational experience to CRISPR Therapeutics.
First Quarter 2019 Financial Results

Cash Position: Cash as of March 31, 2019, was $437.5 million, compared to $456.6 million as of December 31, 2018, a decrease of $19.1 million. Cash used in operations drove the decrease, offset by $25.7 million in net cash provided by financing activities during the quarter.

Revenues: Total collaboration revenues were $0.3 million for the first quarter of 2019 compared to $1.4 million for first quarter of 2018. CRISPR’s collaboration revenue is attributable to its research partnerships with Casebia and Vertex. Cost sharing on the Vertex co-development and co-promotion agreement related to hemoglobinopathies is not included in revenue, but instead as an offset to expense in R&D.

R&D Expenses: R&D expenses were $33.8 million for the first quarter of 2019 compared to $19.5 million for the first quarter of 2018. The increase was driven by headcount and services expense supporting the advancement of the hemoglobinopathies program, the broadening of the wholly-owned immuno-oncology portfolio, as well as increased investment in the Company’s CRISPR/Cas9 platform research.

G&A Expenses: General and administrative expenses were $14.9 million for the first quarter of 2019 compared to $8.8 million for the first quarter of 2018. The increase was driven by headcount-related expense and external professional and consulting service expense.

Net Loss: Net loss was $48.4 million for the first quarter of 2019 compared to a loss of $28.3 million for the first quarter of 2018, driven predominantly by increased R&D expense in the quarter.