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

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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

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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."

On November 12, 2019 Molecular Templates, Inc. (Nasdaq: MTEM, "Molecular," "Molecular Templates" or "MTEM"), a clinical-stage biopharmaceutical company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer, reported financial results for the third quarter of 2019 (Press release, Molecular Templates, NOV 12, 2019, View Source [SID1234551035]). As of September 30, 2019, MTEM’s cash and investments totaled $51.4 million, and is expected to fund operations through 2020.

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"So far in 2019, we have reported Phase I/Ib data for MT-3724, started three Phase II studies for MT-3724, initiated a Phase I study for MT-5111, and our partner Takeda is initiating a Phase I study for TAK-169," said Eric Poma, Ph.D., Molecular Templates’ Chief Executive and Scientific Officer. "We expect to provide study updates on the three ongoing MT-3724 Phase II studies and the MT-5111 Phase I study by the end of the year and continue to advance our pipeline of both clinical and preclinical stage programs into 2020."

Company Highlights and Upcoming Milestones

TAK-169 (CD38-targted ETB)

Takeda and MTEM announced the acceptance of the IND for TAK-169 in 2Q19. Dosing in the Phase I trial is expected to start in 4Q19.
MT-3724 (CD20-targeted ETB)

Results of the Phase I/Ib study are scheduled to be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, December 7-10, 2019 in Orlando, Florida.
MTEM is conducting a Phase II monotherapy study of MT-3724 in relapsed/refractory diffuse large B-cell lymphoma (DLBCL). This study has the potential to serve as a registration study. MTEM expects to provide an update on this study in 4Q19.
MTEM is also conducting two Phase II studies in earlier lines of DLBCL; one with MT-3724 in combination chemotherapy (gemcitabine and oxaliplatin, or GemOx) and the other with MT-3724 in combination with Revlimid. The Company expects to report an update on both MT-3724 combination studies in 4Q19.
MT-5111 (HER2-targeted ETB)

Dosing began in 4Q19 for the ongoing Phase I study of MT-5111 in patients with HER2 positive solid tumors. MTEM expects to report an update on this study in 4Q19.
A presentation titled "MT-5111, a novel HER2 targeting engineered toxin body, under clinical development to overcome mechanisms of resistance to existing HER2 targeted therapies" will be delivered on December 11, 2019, at the San Antonio Breast Cancer Symposium (SABCS) in San Antonio, Texas.
Research

MTEM expects to start a Phase I study for MT-6035, its ETB targeting PD-L1 (with antigen seeding), in 2020.
A presentation titled "In vivo efficacy of a PD-L1 targeted Engineered Toxin Body (ETB) comprised of direct cytotoxicity and T-cell mediated tumor targeting" was delivered on November 9, 2019 at The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in National Harbor, Maryland.
Several other ETB candidates are in preclinical development, targeting both solid and hematological cancers.
Takeda Multi-Target Collaboration

Takeda and MTEM are conducting lead optimization for ETBs against two undisclosed targets selected by Takeda under the collaboration. Should Takeda exercise its option to license ETBs for both targets, MTEM would receive $25.0 million and would be eligible to receive up to $547.0 million in milestone payments and tiered royalties on sales.
Investor Activities

MTEM will be hosting an Analyst & Investor Breakfast on Friday November 15, 2019, from 8:00am to 10:00am ET in New York City. A webcast will be available on the MTEM website.
Financial Results

The net loss attributable to common shareholders for the third quarter of 2019 was $38.2 million, or $1.03 per basic and diluted share. This compares with a net loss attributable to common shareholders of $5.2 million, or $0.19 per basic and diluted share, for the same period in 2018.

Revenues for the third quarter of 2019 were $3.6 million, compared to $6.8 million for the same period in 2018. Revenues for the third quarter of 2019 were comprised of revenues from collaborative research and development agreements with Takeda, and grant revenue from CPRIT. Total research and development expenses for the third quarter of 2019 were $15.2 million, compared with $8.3 million for the same period in 2018. Total general and administrative expenses for the third quarter of 2019 were $4.5 million, compared with $3.5 million for the same period in 2018. In the third quarter, there was also a non-cash impairment charge of $22.1 million relating to the Company’s legacy program, Evofosfamide. Specifically, loss on impairment of in-process research and development for the third quarter of 2019 was $22.1 million, compared to no loss on impairment of in-process research and development for the same period in 2018. The impairment charge related entirely to this legacy program, which the Company acquired from Threshold Pharmaceuticals in 2017, and is unrelated to the Company’s current ETB technology and related pipeline programs.