On November 12, 2019 Precision BioSciences, Inc. (Nasdaq: DTIL) ("Precision"), a genome editing company dedicated to improving life through the application of its pioneering, proprietary ARCUS platform, reported financial results for the third quarter ended September 30, 2019 and provided a corporate update (Press release, Precision Biosciences, NOV 12, 2019, View Source [SID1234551044]).

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Key Highlights

After quarter end, announced publication of an abstract accepted for presentation at the upcoming 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Orlando, Florida, December 7-10, 2019, supporting the safety and clinical activity of Precision BioSciences’s lead CD19-targeted off-the-shelf (allogeneic) chimeric antigen receptor (CAR) T product candidate, PBCAR0191. Abstract reports initial data from first three patients treated at Dose Level 1 as of August 1, 2019. Trial is ongoing and updated data, including from patients treated at Dose Level 2, will be presented during the ASH (Free ASH Whitepaper) annual meeting on December 9, 2019 at 6:00 pm ET.

Announced an investigator update event during ASH (Free ASH Whitepaper) meeting to discuss the PBCAR0191 data presented, starting at 8:15pm ET on December 9, 2019, with accompanying live webcast.

Received acceptance from US Food and Drug Administration (FDA) of investigational new drug (IND) application for second and wholly-owned allogeneic CAR T therapy candidate PBCAR20A, targeting CD20. Phase 1/2a clinical trial to begin in the fourth quarter of 2019.

Further enhanced senior leadership team with appointment of David Thomson, PhD, as Chief Operating Officer, and Nicholas Riddle, MD, PhD, as Vice President, Financial Strategy and Investor Relations.

Ended the quarter with $206.3 million in cash and cash equivalents, which is expected to fund operating expenses and capital expenditure requirements into 2021.

"We reached a transformative moment for Precision BioSciences this quarter; it is very exciting to report initial data from our first clinical trial with a product candidate that leverages our unique approach to allogeneic CAR T therapy," commented Matt Kane, Chief Executive Officer and Co-Founder of Precision BioSciences. "These data bring the reality of a true off-the-shelf CAR T therapy a step closer for patients in need of new and improved treatment options. While preliminary and from a limited number of patients, the safety profile, in vivo cell expansion and early evidence of clinical activity we have demonstrated with PBCAR0191 in the absence of biologic lymphodepletion is very encouraging and gives us confidence in the approach we have taken to allogeneic CAR T. We are looking forward to sharing updated results from patients treated at both Dose Level 1 and Dose Level 2 at ASH (Free ASH Whitepaper). The team at Precision is committed to advancing our pipeline of differentiated CAR T product candidates as rapidly as possible to bring these potentially transformative therapies to patients."

Recent Developments and Upcoming Milestones

Program updates

On November 6, 2019, Precision announced that initial results from the ongoing Phase 1/2a trial of its lead investigational allogeneic CAR T cell therapy candidate, PBCAR0191, will be presented during the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Orlando, Florida, December 7-10, 2019. PBCAR0191, which is being developed in collaboration with Servier, is Precision’s first allogeneic CAR T therapy candidate in clinical trials and targets the well characterized cancer cell surface protein CD19. The Phase 1/2a trial includes adult patients with relapsed or refractory (R/R) non-Hodgkin’s lymphoma (NHL) or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL). The abstract outlining initial data from patients treated with PBCAR0191 at Dose Level 1 can be accessed on the ASH (Free ASH Whitepaper) conference website. Data in the abstract include results as of the cutoff date of August 1, 2019 for three patients with advanced NHL treated at Dose Level 1. No significant toxicities were observed, including no serious adverse events and no dose-limiting toxicities. All patients had a minimum follow-up of 28 days (median 60 days). Two of the three patients experienced an objective tumor response by Lugano criteria, at day 14 and day 28, respectively. The third patient, who had previously progressed following treatment with axicabtagene ciloleucel (Yescarta), an approved anti-CD19 autologous CAR T therapy, had not met the definition of response, but demonstrated evidence of central necrosis, decreased tumor size, and decreased PET-avidity at day 28, in the context of post-infusion tumor site pain and mild CRS symptoms. Peripheral blood analysis for CAR T cell expansion has identified preliminary evidence of cell expansion.

The PBCAR0191 Phase 1/2a trial is ongoing and updated results from patients treated at Dose Level 1 and Dose Level 2 will be presented at the ASH (Free ASH Whitepaper) annual meeting on December 9, 2019 starting at 6:00 p.m. ET. Precision will host a live webcast of an investigator update event during the ASH (Free ASH Whitepaper) meeting to discuss the presented data, beginning at 8:15 p.m. ET on December 9, 2019. The webcast will be accessible from the "Events & Presentations" page within the Investors & Media section of the Precision website and a replay will be available for 30 days following the call.

On September 16, 2019, Precision announced that the FDA accepted its IND application for PBCAR20A. Wholly-owned by Precision, PBCAR20A is an allogeneic anti-CD20 CAR T therapy candidate in development for the treatment of patients with NHL, chronic lymphocytic leukemia (CLL), and small lymphocytic lymphoma (SLL). Precision plans to initiate a Phase 1/2a clinical trial in the fourth quarter of 2019, with initial data expected in 2020.

Senior leadership appointments

On September 30, 2019, Precision announced that Nicholas Riddle, MD, PhD, joined as Vice President, Financial Strategy and Investor Relations. Dr. Riddle joined Precision from J.P. Morgan where he was an Executive Director in the global healthcare investment banking group.

On September 23, 2019, Precision appointed David Thomson, PhD, to the position of Chief Operating Officer. Dr. Thomson previously served as Precision’s Chief Development Officer since 2017.

Upcoming Corporate Presentations

Precision’s senior management team will be presenting and meeting with investors at the following upcoming conferences:

Barclays Gene Editing and Gene Therapy Summit, New York, NY, November 13, 2019

Stifel Healthcare Conference, New York, NY, November 20, 2019

Jefferies London Healthcare Conference, London, UK, November 20, 2019

Piper Jaffray Annual Health Care Conference, New York, NY, December 3–5, 2019

J.P. Morgan Healthcare Conference, San Francisco, CA, January 13-16, 2020

Third Quarter 2019 Financial Results

Cash and Cash Equivalents: As of September 30, 2019, Precision had approximately $206.3 million in cash and cash equivalents. We expect that existing cash and cash equivalents will be sufficient to fund operating expenses and capital expenditure requirements into 2021.

Revenues: Total revenues for the quarter ended September 30, 2019 were $4.9 million, compared to $2.5 million for the quarter ended September 30, 2018. This increase was primarily due to research funding from a collaboration partner, offset by a decrease in license fees.

Research and Development Expenses: Research and development expenses were $19.8 million for the quarter ended September 30, 2019, as compared to $9.7 million for the same period in 2018. This increase of $10.1 million was primarily due to platform development and early-stage research expenses.

General and Administrative Expenses: General and administrative expenses were $7.1 million for the quarter ended September 30, 2019, as compared to $3.3 million for the same period in 2018. The increase of $3.8 million was primarily due to increased employee-related costs for additional personnel and facility costs associated with our growing infrastructure needs.

Net Loss: Net loss was $20.7 million, or $(0.41) per share, for the quarter ended September 30, 2019, compared to a net loss of $9.8 million, or $(0.62) per share, for the same period in 2018.

On November 12, 2019 IVERIC bio, Inc. (Nasdaq: ISEE) reported further details of the clinical trial results for the Company’s Zimura (avacincaptad pegol) program in geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD), reviewed the Company’s financial results for the third quarter 2019 and provided a general business update (Press release, Ophthotech, NOV 12, 2019, View Source [SID1234551043]).

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Zimura (avacincaptad pegol) Highlights

On October 28, 2019, IVERIC bio provided topline data confirming that Zimura (avacincaptad pegol), the Company’s complement factor C5 inhibitor, met its prespecified primary endpoint in reducing the mean rate of geographic atrophy (GA) growth in patients with dry age-related macular degeneration (AMD). Today, the Company provided further clinical details and the development strategy for Zimura in GA secondary to AMD. These announcements will be discussed during today’s conference call/webcast (also see the second press release issued earlier today and the press release issued on October 28, 2019.)

"We are excited to have achieved a major milestone with our recent Zimura clinical trial results in geographic atrophy secondary to dry AMD and we look forward to start enrolling patients in a second pivotal clinical trial in the first quarter of 2020. We believe these events have the potential to be a catalyst for our company," stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. "IVERIC bio is now in a strong position, with a diversified, retina-focused portfolio, including both a late stage clinical program for a large market retinal disease and early stage gene therapy programs in inherited retinal diseases. We are committed to efficiently progressing these programs with the goal of continuing to create value for our shareholders."

Gene Therapy Highlights

Orphan IRD Gene Therapy Programs

IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Natural history studies and IND-enabling activities for IC-100 are on track. The Company expects to initiate a Phase 1/2 clinical trial for IC-100 in patients with rhodopsin mediated adRP in the second half of 2020.

IC-200: BEST1-Related IRDs
Natural history studies and IND-enabling activities for IC-200 are on track.

The Company expects to initiate a Phase 1/2 clinical trial for IC-200 in patients with BEST1 related retinal diseases during the first half of 2021.

miniCEP290: Leber Congenital Amaurosis Type 10 (LCA10)
Encouraging results from the Company’s collaboration with the University of Massachusetts Medical School (UMass Medical School) in its miniCEP290 program led the Company to exercise its option and, in July 2019, the Company entered into an exclusive global license agreement with the University of Massachusetts for rights to develop and commercialize mutation independent novel AAV minigene therapy product candidates for the treatment of LCA10, which is due to mutations in the CEP290 gene and is the most common type of LCA. The Company plans to provide an update on the lead minigene construct in early 2020.

miniABCA4 Program for Stargardt Disease (STGD1)
The Company, through its collaborative sponsored research agreement with UMass Medical School, is evaluating several ABCA4 minigene constructs in both in vitro and in vivo experiments. The Company expects to have results from the miniABCA4 program in 2020.

miniUSH2A: USH2A-Related IRDs Including Usher Syndrome Type 2A and USH2A-Associated Nonsyndromatic Autosomal Recessive Retinitis Pigmentosa
In July 2019, the Company entered into a sponsored research agreement with UMass Medical School and an exclusive option agreement with the University of Massachusetts for rights to develop and commercialize novel AAV gene therapy product candidates utilizing a mutation independent minigene therapy approach for the treatment of USH2A-related IRDs. This group of orphan IRDs include Usher syndrome Type 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa.

On October 29, 2019, Abraham Scaria, PhD was appointed to the position of Chief Scientific Officer. Dr. Scaria will lead the Company’s research and pre-clinical gene therapy activities. Dr. Scaria’s extensive experience includes positions at Genzyme, Sanofi and most recently at Casebia Therapeutics, leading multiple ocular gene therapy programs.

Second Quarter 2019 Financial Results
Operational Update
As of September 30, 2019, the Company had $94.9 million in cash and cash equivalents. The Company reaffirms its estimate that year-end 2019 cash and cash equivalents will range between $80 million and $85 million. With the initiation of enrollment for the Company’s second pivotal clinical trial for Zimura in GA planned for the first quarter of 2020, the Company estimates that its cash and cash equivalents will be sufficient to fund its operations and capital expenditure requirements as currently planned through the first half of 2021. These estimates are based on the Company’s current business plan, including the continuation of its current research and development programs. This estimate does not reflect any additional expenditures in the event the Company were to in-license or acquire any new product candidates or commences any new sponsored research programs.

R&D Expenses: Research and development expenses were $10.4 million for the quarter ended September 30, 2019, compared to $9.4 million for the same period in 2018. For the nine months ended September 30, 2019, research and development expenses were $28.1 million compared to $25.6 million for the same period in 2018. Research and development expenses increased primarily due to increases in costs associated with the Company’s gene therapy programs and HtrA1 inhibitor program, offset by decreases in costs associated with the Company’s Zimura programs.

G&A Expenses: General and administrative expenses were $4.7 million for the quarter ended September 30, 2019, compared to $6.0 million for the same period in 2018. For the nine months ended September 30, 2019, general and administrative expenses were $15.4 million compared to $17.9 million for the same period in 2018. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s operations and infrastructure.

Net Income: The Company reported a net loss for the quarter ended September 30, 2019 of $14.4 million, or ($0.35) per diluted share, compared to a net loss of $14.7 million, or ($0.41) per diluted share, for the same period in 2018. For the nine months ended September 30, 2019, the Company reported a net loss of $41.4 million or ($1.00) per diluted share, compared to a net loss of $41.0 million or ($1.13) for the same period in 2018.

Conference Call/Web Cast Information
IVERIC bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for November 12, 2019 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-208-1711 (USA) or 323-994-2082 (International), passcode 5526863. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the IVERIC bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 5526863.

On November 12, 2019 Forty Seven, Inc. (Nasdaq:FTSV), a clinical-stage, immuno-oncology company focused on developing therapies to activate macrophages in the fight against cancer, reported financial results for the third quarter ended September 30, 2019 and provided a business update (Press release, Forty Seven, NOV 12, 2019, View Source [SID1234551042]).

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"In the third quarter, we continued to enroll patients in our Phase 1b clinical trial of magrolimab in myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), while preparing to initiate potential registration-enabling trials in MDS and diffuse large B cell lymphoma (DLBCL) in the first quarter of 2020," said Mark McCamish, M.D., Ph.D., President and Chief Executive Officer of Forty Seven. "We have plans in place for both programs that we believe could enable us to pursue accelerated paths to approval and to address the unmet needs of substantial patient populations in need of safe, well-tolerated and effective new options."

Dr. McCamish continued, "We also made important progress with our preclinical candidates, FSI-174 and FSI-189, and remain on track to advance both into clinical testing next year. This morning, we announced a new collaboration with bluebird bio to evaluate our antibody-based conditioning regimen, comprised of magrolimab and FSI-174, in combination with LentiGlobin. We believe this partnership will allow us to accelerate and expand our efforts to provide an alternative, antibody-only conditioning regimen that avoids chemotherapy/radiation exposure for patients undergoing hematopoietic stem cell (HSC) transplantation. We are excited to work with the bluebird team as we continue our efforts to fully exploit the CD47 pathway as a novel therapeutic target."

Third Quarter and Recent Business Highlights:

Magrolimab Clinical Programs:

Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)

Forty Seven continues to enroll patients in its ongoing, single-arm trial evaluating magrolimab in combination with azacitidine in approximately 90 patients with untreated, intermediate to very high risk MDS, treated with weekly dosing. The primary endpoint of the trial is overall response rate (ORR) with durability of response. The company expects to complete enrollment in the third quarter of 2020. In parallel, Forty Seven continues to engage with the U.S. Food and Drug Administration (FDA) under its Special Protocol Assessment (SPA) to finalize key parameters of a second clinical trial, evaluating every two week dosing, which may support a potential accelerated approval. The company is moving forward in parallel with chemistry, manufacturing and controls (CMC) activities to support an expected filing of a biologics license application (BLA) in the fourth quarter of 2021, consistent with prior guidance.

In September 2019, Forty Seven announced that the FDA granted Fast Track designation to magrolimab for the treatment of MDS and AML.

Non-Hodgkin Lymphoma (NHL)

Forty Seven has finalized the patient eligibility criteria for its planned registration-enabling trial evaluating magrolimab in combination with rituximab in approximately 100 patients with diffuse large B-cell lymphoma (DLBCL). The company plans to enroll patients who have failed at least two prior lines of therapy. The primary endpoint of the trial will evaluate ORR and durability of response. Forty Seven expects to initiate the study in the first quarter of 2020, and to report interim efficacy data by the fourth quarter of 2020. In parallel, the company continues to evaluate biomarkers for potential predictive value, which could enable advancement into earlier lines of treatment.

O: 650-352-4150 F: 650-618-2308 W: fortyseveninc.com A: 1490 O’Brien Drive, Suite A, Menlo Park, CA 94025, United States

Helping Patients Defeat Their Cancer

Solid Tumors

Forty Seven recently submitted abstracts including data from its ongoing Phase 1b trial of magrolimab in combination with avelumab in patients with ovarian cancer, and its Phase 1b trial of magrolimab in combination with cetuximab in patients with colorectal cancer, to major medical meetings which will occur in the first quarter of 2020. The company plans to announce initial data from both studies at the time of those presentations.

FSI-174:

In November 2019, Forty Seven entered into a collaboration with bluebird bio to evaluate Forty Seven’s antibody-based conditioning regimen, which is comprised of FSI-174 and magrolimab, with bluebird’s LentiGlobin gene therapy platform for the treatment of beta thalassemia and sickle cell disease. The companies expect to initiate a Phase 1b trial in 2020.
oming Milestones:

Forty Seven will present expanded efficacy and durability data from the Phase 1b trial of magrolimab in combination with azacitidine in patients with MDS and AML in an oral presentation at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which will be held December 7-10, 2019 in Orlando, Florida. Also at ASH (Free ASH Whitepaper), Forty Seven will present a poster detailing preclinical data for FSI-174.

Additionally, the company expects to complete investigational new drug (IND)-enabling studies for both FSI-174 and FSI-189 before year-end.

Third Quarter 2019 Financial Results:

Cash Position: As of September 30, 2019, cash, cash equivalents and short-term investments were $166.7 million, as compared to $139.0 million as of December 31, 2018. This increase reflects aggregate gross proceeds of approximately $86.3 million from Forty Seven’s underwritten public offering of common stock that closed in July 2019, as well as an approximately $15.7 million upfront license payment from Forty Seven’s entry into its collaboration with Ono Pharmaceutical. The company expects that its cash, cash equivalents and short-term investments will fund operating expenses and capital expenditure requirements through the first quarter of 2021.

Revenue: Revenues were $15.7 million for the third quarter of 2019, due to the license granted under the Ono agreement. Forty Seven did not record revenues in the third quarter of 2018.

R&D Expenses: R&D expenses were $27.1 million for the third quarter of 2019, as compared to $18.0 million for the third quarter of 2018. The increase was primarily due to a $9.2 million increase in advancing Forty Seven’s current clinical programs focused on lead product candidate, magrolimab, and associated contract manufacturing costs for BLA enabling studies, a $3.2 million increase in preclinical program costs, a $1.5 million decrease in funding recognition under the California Institute for Regenerative Medicine (CIRM) and Leukemia and Lymphoma Society (LLS) grants, and a $1.1 million increase in personnel-related costs, partially offset by a $5.9 million decrease in license fees, primarily due to the non-recurring license fees paid under the BliNK asset purchase and the Synthon license agreements in 2018.

G&A Expenses: G&A expenses were $5.0 million for the third quarter of 2019, as compared to $4.4 million for the third quarter of 2018. The increase was primarily due to a $0.4 million increase in personnel and corporate related costs driven by an increase in headcount.

Net Loss: Net loss was $15.1 million for the third quarter of 2019, or a net loss per share of $0.38, as compared to $21.7 million for the third quarter of 2018, or a net loss per share of $0.71.

O: 650-352-4150 F: 650-618-2308 W: fortyseveninc.com A: 1490 O’Brien Drive, Suite A, Menlo Park, CA 94025, United States

Helping Patients Defeat Their Cancer

Conference Call Information:

Forty Seven will host a live conference call and webcast at 8:00 a.m. ET today to discuss third quarter 2019 financial results and recent business activities. The conference call may be accessed by (866) 953-0780 (domestic) or (630) 652-5854 (international), and by referring to conference ID 7667736. A webcast of the conference call will be available in the Investors section of the Forty Seven website at View Source The archived webcast will be available on Forty Seven’s website approximately two hours after the conference call and will be available for 30 days following the call.

On November 12, 2019 Sorrento Therapeutics, Inc. (NASDAQ: SRNE, "Sorrento"), reported that Dr. Henry Ji, Chairman and CEO, will participate in upcoming investor conferences in the 4th quarter 2019 (Press release, Sorrento Therapeutics, NOV 12, 2019, View Source [SID1234551041]). Dr. Ji will provide a corporate overview and business update focusing on the clinical pipeline progress for Sorrento Therapeutics immuno-oncology and pain programs.

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Stifel 2019 Healthcare Conference

Date: Wednesday, November 20, 2019
Presenter: Dr. Henry Ji
Time: 9:45 AM Eastern Time
Location: Lotte New York Palace Hotel, NY

Evercore ISI 2nd Annual HealthCONx Conference

Date: Tuesday, December 3, 2019
Presenter: Dr. Henry Ji
Time: 12:30 PM Eastern Time
Location: Four Seasons Hotel, Boston, MA

On November 12, 2019 Oncolytics Biotech Inc. (NASDAQ:ONCY) (TSX:ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported the exercise of warrants from it’s August 2019 financing (Press release, Oncolytics Biotech, NOV 12, 2019, View Source [SID1234551039]).

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The Company announced the exercise of approximately 1.4 million warrants, at an exercise price of USD $0.90, for net proceeds of approximately USD $1,250,000.

"We are very happy to see these warrants exercised so early," said Dr. Matt Coffey, President and CEO of Oncolytics Biotech. "This speaks not only to our rapid appreciation as of late, but the belief in Oncolytics’ fundamentals that will continue this momentum through the end of the year and well into 2020. This strengthening of the balance sheet is one more piece in our plan to finance the Company as efficiently as possible through all of it’s catalysts and milestones."