On August 1, 2019 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer,reported financial results for the second quarter ended June 30, 2019 and provided a business update (Press release, Adaptimmune, AUG 1, 2019, View Source [SID1234537996]).

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James Noble, Adaptimmune CEO said: "I have spent twenty years in the field of T-cell receptors and am convinced that they will have an important role in treating patients. We have already seen compelling data in sarcoma with ADP-A2M4 and have recently started a trial with a next-generation SPEAR T-cell and a combination trial with low-dose radiation to improve the depth and durability of responses. It is my final pleasure as CEO to be handing over to Adrian – I know that he will do an excellent job in taking the Company through its next evolution towards commercialization. The changes that he is already introducing and the plans for the future will bring energy and focus to the tasks ahead, accelerating execution across the Company."

Adrian Rawcliffe, incoming Adaptimmune CEO said: "Looking forward, I intend to further focus the Company on increasing the pace of execution, which has already enabled us to initiate our first late-stage trial: SPEARHEAD-1, our first next-gen trial: SURPASS, and the low-dose radiation sub-study in recent weeks. In addition, we will strengthen our capabilities to be ready to launch our first product in 2022. Finally, we intend to leverage the state-of-the-art integrated capabilities that we have built in research, development and supply, to apply them in cell therapy more broadly."

Clinical programs

Initiated Phase 2 SPEARHEAD-1 trial with ADP-A2M4 in synovial sarcoma and myxoid/ round cell liposarcoma patients

Initiated SURPASS trial with ADP-A2M4CD8 — the first next-generation trial with SPEAR T-cells

Preclinical data show that these next-generation SPEAR T-cells may improve long term T-cell function as well as antitumor activity

Initiated low-dose radiation sub-study of ADP-A2M4 Phase 1 trial in collaboration with the MD Anderson Cancer Center

There is emerging data showing that low-dose radiation could enhance T-cell tumor trafficking and responses

Focusing efforts on ADP-A2M4, ADP-A2M4CD8, and ADP-A2AFP, with enrollment in the ADP-A2M10 trials closing by the end of 2019

Will provide clinical updates on all ongoing clinical trials at medical conferences

At ESMO (Free ESMO Whitepaper) 2019, Brian Van Tine, MD, PhD of Washington University in St. Louis will present an oral presentation titled "ADP-A2M4 (MAGE-A4) in patients with Synovial Sarcoma" on September 30, 2019

Since our last clinical update, the Company has reported serious adverse events (SAEs) in three patients

All three patients received the highest lymphodepletion regimen: fludarabine (30mg/m2/day for 4 days) and cyclophosphamide (1800 mg/m2/day for 2 days)

One patient in each of the ADP-A2M10 and ADP-A2M4 trials had reports of severe prolonged pancytopenia. Both patients subsequently died of complications.

In the third patient (in the ADP-A2M4 trial) there was one report of Grade 3 neurotoxicity. The patient subsequently died of a stroke that the Company believes was unrelated to SPEAR T-cell therapy.

Subsequently, the protocols for all ADP-A2M4 and ADP-A2M10 trials have been amended to include changes in eligibility criteria and reversion to the previously used lower dose of cyclophosphamide

These protocol changes have been communicated to the FDA

Commercial readiness

Working with Vineti as part of Adaptimmune’s strategy to deliver products through clinical trials and into scaled-up production post approval. Vineti is the first cloud-based software platform to efficiently take T-cell therapies through clinical trials and into mainstream medicine at commercial scale.

Pipeline

· Initiated collaboration with Alpine Immune Sciences to develop next-generation products

· Progress with allogeneic program continues with updates to be presented at future conferences

Other corporate news

Today’s changes to the Executive Team follow the Company’s recent news that Adrian Rawcliffe will assume the role of Chief Executive Officer on September 1, 2019, succeeding James Noble who will transition to a non-executive director role

John Lunger, previously SVP Manufacturing and Supply Chain, is promoted to the Executive Team as Chief Patient Supply Officer from August 1, 2019, as Adaptimmune gears up for commercial delivery of products to patients

Rafael Amado, President of R&D, will leave the Company on August 12, 2019

The new Executive Team will comprise Adrian Rawcliffe (Chief Executive Officer), Bill Bertrand (Chief Operating Officer), Helen Tayton-Martin (Chief Business Officer and co-Founder), and John Lunger (Chief Patient Supply Officer)

The Company is recruiting a Chief Medical Officer and a Chief Financial Officer

Financial Results for the three-month period ended June 30, 2019

Cash / liquidity position: As of June 30, 2019, Adaptimmune had cash and cash equivalents of $34.6 million and Total Liquidity(1) of $133.4 million.

· Revenue: Revenue for both the three and six months ended June 30, 2019 was $0.2 million, compared to $9.0 million and $17.2 million for the same periods in 2018. The revenue in the three and six months ended 2019 is due to the commencement of development on the third target nominated by GSK under the Collaboration and License Agreement, whereas the revenue for the same periods in 2018 was recognized due to the performance under the NY-ESO transition program and the PRAME development plan, which were completed in 2018.

·Research and development (R&D) expenses: R&D expenses for the three and six months ended June 30, 2019 were $25.5 million and $47.5 million, respectively, compared to $26.6 million and $52.0 million for the same periods of 2018. The decreases in both periods were primarily due to a reduction in expenditure associated with the NY-ESO program, which was transferred to GSK on July 23, 2018.

·General and administrative (G&A) expenses: G&A expenses for the three and six months ended June 30, 2019 were $10.1 million and $21.9 million respectively, compared to $11.3 million and $22.5 million for the same periods of 2018. The decreases in both periods are due to a reduction in general corporate costs including travel and IT expenditure.

·Other expense, net: Other expense, net for the three and six months ended June 30, 2019 was $6.3 million and $0.9 million respectively, compared to an expense of $15.4 million and $8.3 million for the same periods of 2018. Other expense, net primarily comprises unrealized foreign exchange losses, which fluctuate depending on exchange rate movements and the amount of foreign currency assets and liabilities.

·Net loss: Net loss attributable to holders of the Company’s ordinary shares for the three and six month periods ended June 30, 2019 was a loss of $41.1 million and $68.5 million respectively, and $(0.07) and $(0.11) per ordinary share respectively, compared to a loss of $43.8 million and $64.6 million respectively and $(0.08) and $(0.11) per ordinary share respectively in the same periods of 2018.

Financial guidance

The Company believes that its existing cash, cash equivalents and marketable securities will fund the Company’s current operations through the third quarter of 2020.

Conference Call and Webcast Information

The Company will host a live teleconference at 8:00 a.m. EDT (1:00 p.m. BST) today, August 1, 2019. The live webcast of the conference call will be available in the investor section of Adaptimmune’s corporate website at www.adaptimmune.com. An archive will be available after the call at the same address. To participate in the live conference call, please dial (833) 652-5917 (U.S. or Canada) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (8299695).

On August 1, 2019 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a physician-led biopharmaceutical company focused on immuno-inflammatory and dermatological diseases, reported it will report financial results for the second quarter 2019, Thursday, August 8th, after U.S. financial markets close (Press release, Aclaris Therapeutics, AUG 1, 2019, View Source [SID1234537994]).

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Management will conduct a conference call at 5:00 PM ET to discuss Aclaris’ financial results and provide a general business update. The conference call will be webcast live over the Internet and can be accessed by logging on to the "Investors" page of the Aclaris Therapeutics website, www.aclaristx.com, prior to the event. A replay of the webcast will be archived on the Aclaris Therapeutics website for 30 days following the call.

To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 3391498 prior to the start of the call.

On August 1, 2019 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the second quarter ended June 30, 2019 and provided a business update (Press release, Ophthotech, AUG 1, 2019, View Source [SID1234537986]).

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"We are pleased with the Company’s progress as we achieved several milestones to advance and expand our gene therapy pipeline for orphan inherited retinal diseases, or IRDs," stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. "We entered into exclusive global license agreements for rights to gene therapy product candidates for the treatment of BEST1-related IRDs and Leber congenital amaurosis type 10, or LCA10. We are encouraged by new research data from our miniCEP290 collaboration that support our plans to move this program forward in LCA10, and we expanded our gene therapy portfolio with the addition of a minigene research program for USH2A related IRDs, including Usher syndrome type 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa. Manufacturing activities for IC-100 and IC-200, our two lead gene therapy product candidates, are currently ongoing using state-of-the-art manufacturing capabilities of Paragon Gene Therapy, part of Catalent Biologics."

Kourous A. Rezaei, MD, Chief Medical Officer of IVERIC bio added, "With the addition of the USH2A program to our portfolio, IVERIC bio is seeking to treat vision loss associated with what are reportedly the most common form of autosomal dominant retinitis pigmentosa (rhodopsin-mediated) and the most common form of autosomal recessive retinitis pigmentosa (USH2A-related). Retinitis pigmentosa is the most common orphan IRD. We look forward to presenting our gene therapy pipeline and discussing our programs in greater detail with highly-recognized gene therapy scientists and key opinion leaders in retinal diseases at our upcoming Gene Therapy R&D Investor Day."

Second Quarter 2019 and Recent 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 ongoing. The Company expects to initiate a Phase 1/2 clinical trial for IC-100 in the second half of 2020.

IC-200: BEST1-Related IRDs
In April 2019, the Company entered into an exclusive global license agreement with the University of Pennsylvania and the University of Florida Research Foundation for rights to develop and commercialize novel adeno-associated virus (AAV) gene therapy product candidates for the treatment of BEST1-related IRDs, including Best vitelliform macular dystrophy, also known as Best disease. The Company expects to initiate a Phase 1/2 clinical trial for IC-200 in 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.

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.

Gene Therapy Manufacturing
In June 2019, the Company announced that it had entered into a strategic manufacturing relationship with Paragon Gene Therapy, part of Catalent Biologics. This agreement is for production and manufacturing of AAV vectors for pre-clinical activities and planned Phase 1/2 clinical trials for IC-100 and IC-200.

Gene Therapy R&D Investor Day Scheduled
The Company will host its first "Gene Therapy R&D Investor Day" in New York, NY on Friday, September 13, 2019. The agenda includes updates from IVERIC bio’s management on its gene therapy portfolio in orphan IRDs, and scientific discussions from gene therapy scientists and key opinion leaders in orphan inherited retinal diseases including;

Guangping Gao, PhD, University of Massachusetts Medical School

William A. Beltran, DVM, PhD, University of Pennsylvania

Charles A. Gersbach, Duke University

Hemant Khanna, PhD, University of Massachusetts Medical School

Gustavo D. Aguirre, VMD, PhD, University of Pennsylvania School of Veterinary Medicine

Andreas K. Lauer, MD, Oregon Health & Science University

Bart P. Leroy, MD, PhD, Ghent University and Children’s Hospital of Philadelphia

Alfred S. Lewin, PhD, University of Florida

Therapeutic Programs
The Company expects initial top-line data for its ongoing Phase 2b clinical trial of Zimura (avacincaptad pegol), a C5 complement inhibitor, for the treatment of geographic atrophy secondary to dry age-related macular degeneration to be available in the fourth quarter of 2019. The Company expects initial top-line data for its Phase 2b clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease to be available in the second half of 2020.

Second Quarter 2019 Financial Results

Operational Update
As of June 30, 2019, the Company had $106.9 million in cash and cash equivalents. After taking into account the Company’s recent in-licensing transaction for its miniCEP290 program and sponsored research agreement for its miniUSH2A program, the Company reaffirms its estimate that year-end 2019 cash and cash equivalents will range between $80 million and $85 million. This estimate is based on its current 2019 business plan, including the continuation of its current research and development programs. This estimate does not reflect any additional expenditures, including associated development

costs, in the event the Company in-licenses or acquires any new product candidates or commences any new sponsored research programs.

R&D Expenses: Research and development expenses were $10.0 million for the quarter ended June 30, 2019, compared to $8.5 million for the same period in 2018. For the six months ended June 30, 2019, research and development expenses were $17.7 million compared to $16.2 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, personnel expenses and professional and consulting fees.

G&A Expenses: General and administrative expenses were $5.2 million for the quarter ended June 30, 2019, compared to $6.3 million for the same period in 2018. For the six months ended June 30, 2019, general and administrative expenses were $10.7 million compared to $12.0 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 June 30, 2019 of $14.4 million, or ($0.35) per diluted share, compared to a net loss of $13.2 million, or ($0.37) per diluted share, for the same period in 2018. For the six months ended June 30, 2019, the Company reported a net loss of $26.9 million or ($0.65) per diluted share, compared to a net loss of $26.3 million or ($0.73) 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 August 1, 2019 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 800-458-4121 (USA) or 323-794-2597 (International), passcode 3528260. 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 3528260.

On August 1, 2019 Intrexon Corporation (NASDAQ: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported it will release second quarter and first half 2019 financial results after the market closes on Thursday, August 8th, 2019 (Press release, Intrexon, AUG 1, 2019, View Source [SID1234537985]). The Company will host a conference call that day at 5:30 PM ET to discuss the results and provide a general business update.

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The conference call may be accessed by dialing 1-888-317-6003 (Domestic US), 1-866-284-3684 (Canada), and 1‑412-317-6061 (International) and providing the number 4443860 to join the Intrexon Corporation Call. Participants may also access the live webcast through Intrexon’s website in the Investors section at View Source

On August 1, 2019 (the "Effective Date"), AVEO Pharmaceuticals, Inc. ("AVEO") reported that it has entered into an amendment (the "Amendment") to the license agreement dated December 21, 2006 (the "KKC Agreement") with Kyowa Kirin Co., Ltd. (formerly Kirin Brewery Co., Ltd.) ("KKC") (Filing, 8-K, AVEO, AUG 1, 2019, View Source [SID1234537983]). Under the KKC Agreement, KKC granted AVEO an exclusive license to research, develop, manufacture and commercialize tivozanib in all human diseases and conditions in the territory licensed to AVEO, which covers all territories in the world except for Asia and the Middle East (the "AVEO Territory"). Pursuant to the Amendment, KKC repurchased the non-oncology rights to tivozanib in the AVEO Territory, excluding the rights AVEO sublicensed to EUSA Pharma (UK) Limited ("EUSA") under the license agreement between AVEO and EUSA dated December 18, 2015.

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In consideration for KKC’s repurchase of the non-oncology rights to tivozanib in the AVEO Territory, KKC has upfront, milestone and royalty payment obligations to AVEO under the Amendment. The Amendment provides that KKC (a) will make an upfront payment of $25.0 million within thirty (30) days after the Effective Date, (b) waives a one-time milestone payment of $18.0 million otherwise payable by AVEO upon AVEO obtaining marketing approval for tivozanib in the U.S., (c) will make milestone payments to AVEO of up to an aggregate of $390.7 million upon the successful achievement of certain development and sales milestones of tivozanib in non-oncology indications, and (d) will make tiered royalty payments to AVEO on net sales of tivozanib in non-oncology indications in the AVEO Territory, which range from high single digit to low double digits as a percentage of net sales. The royalty rate escalates within this range based on increasing tivozanib sales, subject to certain adjustments. KKC’s royalty payment obligations in a particular country in the AVEO Territory begin on the date of the first commercial sale of tivozanib in that country, and end on the later of the expiration date of the last valid claim of a patent application or patent owned by KKC covering tivozanib or 10 years after the date of first commercial sale of tivozanib in non-oncology indications in that country. If KKC sublicenses any of its non-oncology rights to tivozanib to a third party, KKC is required to pay AVEO a percentage of amounts KKC receives from its sublicensees related to the AVEO Territory, including upfront license fees, milestone payments and royalties, but excluding amounts KKC receives in respect of research and development reimbursement payments or equity investments, subject to certain limitations.

The foregoing summary of the Amendment does not purport to be complete and is qualified in its entirety by the full text of the Amendment, a copy of which is attached hereto as Exhibit 10.1 and is incorporated herein by reference.