On November 9, 2020 Adaptimmune Therapeutics plc (Nasdaq: ADAP), a leader in cell therapy to treat cancer, reported data from the dose escalation cohorts of its Phase 1 SURPASS trial using ADP-A2M4CD8 in a poster at the Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) ("SITC") Conference (Press release, Adaptimmune, NOV 9, 2020, View Source [SID1234570434]).

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In these cohorts of heavily pre-treated patients with advanced cancers (n=6), three were treated with target doses of 1 billion SPEAR T-cells, and three with target doses of 5 billion. Most adverse events were consistent with those typically experienced by cancer patients undergoing cytotoxic chemotherapy or cancer immunotherapy.

"We have seen responses in two out of six patients treated in the safety cohorts of the SURPASS trial as well as antitumor activity in five of them. The responses and antitumor activity we have seen with our next-generation ADP-A2M4CD8 SPEAR T-cells, across a range of solid tumors, support our belief that this is a highly active product," said Ad Rawcliffe, Adaptimmune’s Chief Executive Officer. "Based on these data, we will initiate the Phase 2 trial in gastroesophageal cancers in the first half of 2021 and look forward to identifying additional indications to take into late-stage development."

There were two confirmed partial responses (PRs): one in a patient with esophagogastric junction (EGJ) cancer, previously reported, and one in a patient with head and neck cancer, reported as unconfirmed in May. The four other patients had best overall responses of stable disease (SD). Overall, five out of six patients treated had initial tumor shrinkage.

In vitro translational data using the manufactured products from patients in the SURPASS trial indicate that co-expression of the CD8α co-receptor on CD4+ ADP-A2M4 SPEAR T-cells enables them to kill MAGE-A4 expressing target cells with equal potency as CD8+ SPEAR T-cells targeting MAGE-A4. These data, combined with the responses and antitumor activity observed at low doses, indicate that ADP-A2M4D8 may be a more potent product than the first-generation ADP-A2M4 SPEAR T-cells.

Best Overall Response (BOR) and maximum changes from baseline in target lesions in Cohorts 1 and 2

Indication Dose x 109 BOR Tumor reduction
Head and neck 4.6 PR -63.16%
EGJ 1.2 PR -51.52%
EGJ 1.0 SD -34.07%
Ovarian 1.1 SD -16.13%
Esophageal 6.0 SD -13.37%
MRCLS 5.7 SD +1.35%

As of data cut-off: October 1, 2020

At SITC (Free SITC Whitepaper), Adaptimmune also presented a poster entitled "Inhibition of AKT signaling during expansion of TCR-engineered T-cells from patient leukocyte material generates SPEAR T-cells with enhanced functional potential in vitro." These preclinical data indicate that AKT inhibition during the manufacture of SPEAR T-cells results in a more consistent expansion and phenotype of the final product. This process is currently being used for manufacture of ADP-A2M4CD8 for the SURPASS trial.

The Company also presented two posters summarizing data for the two completed Phase 1 trials with ADP-A2M10 (a previously terminated program)

On November 9, 2020 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported financial results for the third quarter 2020 and recent business progress (Press release, Abeona Therapeutics, NOV 9, 2020, View Source [SID1234570433]).

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"Abeona remains committed to pursuing the development of our portfolio of advanced and early stage programs toward providing our novel gene and cell therapies to patients who currently have no approved treatment options," said Michael Amoroso, Chief Operating Officer of Abeona. "In particular, we are continuing to propel our clinical programs in recessive dystrophic epidermolysis bullosa (RDEB) and Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB) to drive Abeona’s long-term growth and unlock shareholder value."

Third Quarter and Recent Highlights

Corporate and Business Development Updates

In August 2020, Abeona entered into definitive agreements with Taysha Gene Therapies to license its rights to ABO-202, an AAV-based gene therapy for CLN1 disease (also known as infantile Batten disease). As part of the transaction, Abeona received initial cash proceeds of $7.0 million in October 2020. Abeona is also eligible to receive up to $56.0 million upon the achievement of certain clinical, regulatory and sales milestones, plus royalty-based payments based on net sales.
In October 2020, Abeona entered into a license agreement with Taysha Gene Therapies with respect to certain intellectual property rights, materials, and know-how relating to a potential AAV-based gene therapy for Rett syndrome. In connection with the agreement, Abeona received a one-time upfront payment of $3.0 million and is eligible to receive up to $56.5 million upon the achievement of certain clinical, regulatory and sales milestones, plus royalty-based payments based on net sales.
In October 2020, Michael Amoroso, Senior Vice President and Chief Commercial Officer at Abeona, was promoted to Chief Operating Officer. In this newly created role, Mr. Amoroso’s responsibilities are broadened to include oversight and leadership of all operations.
Abeona continues to work with Jefferies LLC as its financial advisor in the review of strategic options focused on advancing the Company’s mission and maximizing stakeholder value.
EB-101 (Autologous, Gene-Corrected Cell Therapy)

Patient enrollment is ongoing for Abeona’s EB-101 pivotal Phase 3 VIITAL study for RDEB. The Company currently anticipates completing enrollment in the VIITAL study in the first half of 2021, depending upon the impact from the COVID-19 pandemic, including travel restrictions and safety concerns.
ABO-102 and ABO-101 (AAV-based Gene Therapies)

Target enrollment in the ABO-102 Transpher A study for MPS IIIA (15 to 22 patients) has been achieved. To date, 18 patients have been dosed in the Transpher A study, including 12 patients dosed in cohort 3. Abeona intends to continue enrolling patients into the study through the first quarter of 2021 given the lack of treatment options for MPS IIIA and encouraging efficacy and safety data from cohort 3.
In the ABO-101 Transpher B study for MPS IIIB, 11 patients have been dosed to date, including 4 patients dosed in cohort 3. The Company anticipates completing target enrollment in the Transpher B study (15 to 20 patients) in the first quarter of 2021.
In the third quarter, Abeona presented its plan toward registration of ABO-102 for MPS IIIA (Sanfilippo syndrome type A) during a kick-off meeting under the European Medicines Agency’s (EMA) PRIority MEdicines (PRIME) program that offers a path for accelerated assessment of promising therapies targeting unmet medical needs. Based on the meeting, along with previous input from the Committee for Medicinal Products for Human Use and the Pediatric Committee of the EMA, Abeona anticipates submitting a marketing authorization application for EU conditional approval of ABO-102 for MPS IIIA after completion of two-year follow-up of the last patient treated in the Transpher A study. Regarding the U.S. regulatory path for ABO-102 in MPS IIIA, Abeona plans to request a meeting with the FDA to take place in the first quarter of 2021, depending on FDA’s availability.
Manufacturing Activities

Process development at the Company’s GMP manufacturing facility in Cleveland, Ohio is ongoing that will enable production of the retrovirus used for EB-101 manufactured at the facility, allowing for increased control of the supply chain and product quality, as well as reduced costs. In addition, Abeona continues process development activities to enable in-house manufacturing of commercial supply of ABO-102 and ABO-101.
Third Quarter Financial Results

Cash, cash equivalents, receivables and short-term investments totaled $103.9 million as of September 30, 2020, compared to $129.3 million as of December 31, 2019. Accounts receivable were $7.0 million at September 30, 2020, which was paid by the customer in October 2020. Net cash used in operating activities was $10.7 million for the third quarter of 2020.

License and other revenues for the third quarter of 2020 were $7.0 million, comprised of initial proceeds from the ABO-202 transaction, compared to zero revenues in the year-ago quarter.

Research and development (R&D) expenses were $8.0 million for the third quarter of 2020 and $20.9 million for the nine months ended September 30, 2020, compared to $10.9 million and $39.0 million in the comparable periods in 2019. The decrease in R&D expenses was primarily due to decreased manufacturing, clinical and non-clinical development activities arising from the effects of the COVID-19 pandemic, and cost savings associated with the decision to internally manufacture retrovirus for the EB-101 program.

General and administrative (G&A) expenses were $4.4 million for the third quarter of 2020 and $16.4 million for the nine months ended September 30, 2020, compared to $4.7 million and $16.0 million in the comparable periods in 2019. The decrease in G&A expenses in the third quarter of 2020 was primarily due to decreased salary and related costs. The increase in G&A expenses in the nine months ended September 30, 2020 was primarily from severance costs associated with management changes, partially offset by decreased professional fees.

Net loss was $7.2 million for the third quarter of 2020 and $68.4 million for the nine months ended September 30, 2020, compared to net loss of $17.4 million and $59.9 million for the comparable periods in 2019. The increase in net loss for the nine months ended September 30, 2020 includes the non-cash impairment charge of $32.9 million related to the termination of the license agreement with REGENXBIO.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast tomorrow, Tuesday, November 10, 2020 at 8:30 a.m. ET, to discuss its third quarter 2020 financial results and business update. To access the call, dial 844-369-8770 (U.S. toll-free) or 862-298-0840 (international) five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

On November 9, 2020 Affimed N.V. (Nasdaq: AFMD), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, and Roivant Sciences, a global biopharmaceutical company, reported that they have entered into a licensing and strategic collaboration agreement to develop and commercialize novel ICE molecules in oncology (Press release, Affimed, NOV 9, 2020, View Source [SID1234570420]).

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The collaboration grants Roivant a license to the preclinical molecule AFM32. The collaboration will also leverage Affimed’s proprietary Redirected Optimized Cell Killing (ROCK) platform to generate ICE molecules against targets not included in Affimed’s current pipeline.

Under the terms of the agreement, Affimed will receive $60 million in upfront consideration, comprised of $40 million in cash and pre-paid R&D funding, and $20 million of newly issued shares in Roivant. Affimed could receive further short-term proceeds in the form of option fees contingent on the commencement of additional programs contemplated under the agreement. The company is eligible to receive up to an additional $2 billion in milestones over time upon achievement of specified development, regulatory and commercial milestones, as well as tiered royalties on net sales.

Pursuant to the agreement, Affimed will be primarily responsible for driving the discovery and research phases of molecule development through filing of the IND. Roivant will be responsible for clinical development and commercialization worldwide, and Affimed retains an option for co-promotion.

"This partnership represents an important milestone as it further validates our platform and scientific expertise in the selection of promising targets to develop ICE molecules in oncology indications where patients are underserved by existing therapies," said Dr. Adi Hoess, Affimed’s Chief Executive Officer. "Partnering with Roivant, an innovative trailblazer in biopharmaceutical development, is another step towards accelerating the growth of our current and future pipeline."

"We are extremely pleased to have entered into this agreement with Affimed given their leadership position in the science of innate immunity and extensive expertise in the preclinical development of bispecifics," commented Dr. Roger Sidhu, Chief Medical Officer and Head of R&D at Roivant. "We look forward to working together to deliver meaningful therapies to patients."

About the ROCK Platform

Affimed’s proprietary, fit-for-purpose ROCK platform technology generates diverse, tetravalent, bispecific antibodies known as innate cell engagers (ICE) which can be customized to target specific binding domains on hematologic and solid tumor cells. Affimed’s ROCK -generated ICE use a distinct, dual mechanism of action that activates CD16A on natural killer cells and macrophages and binds to specific antigens on tumor cells, restoring the body’s innate ability to overcome tumor invasion and destroy tumor cells.

On November 9, 2020 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported financial results and business highlights for the third quarter of 2020 (Press release, CASI Pharmaceuticals, NOV 9, 2020, View Source [SID1234570419]).

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Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "We are pleased to report that EVOMELA revenues for Q3 were $4.2 million. For the full year 2020 revenue, we expect to exceed $14 million, performing better than we had previously forecasted. We are thrilled with the progress we are seeing across our hematology oncology product portfolio. With our recently announced partnership with BioInvent, we gained exclusive Greater China development and commercialization rights to BI-1206, a first-in-class anti-FcyRIIB monoclonal antibody. BI-1206 has broad potential clinical applications across multiple tumor types in many first line indications and in refractory settings, which we look forward to exploring."

Dr. He continued, "With respect to our commercial asset CNCT-19 (CD19 CAR-T), our partner Juventas is making good progress with their current Phase 1 trials in B-NHL and B-ALL, and is expecting to initiate registration trials by the end of 2020. We expect to initiate our Phase 1 study for CID-103 (anti-CD38 monoclonal antibody) in the EU during the first quarter of 2021. We will continue to execute on a number of key milestones across our broad portfolio in the quarters ahead. In parallel, our team will continue tactically evaluating additional strategic opportunities that complement our growing portfolio."

Third Quarter 2020 Financial Results

Revenues consisted primarily of product sales of EVOMELA that launched in August of 2019. Revenues were $4.2 million for the three months ended September 30, 2020 compared to $2.7 million for the three months ended September 30, 2019.
Costs of revenues were $1.8 million for the three months ended September 30, 2020 compared to $2.6 million for the three months ended September 30, 2019. The decrease in cost of revenues is a result of the transfer to a new manufacturer, resulting in a considerable decrease in the unit cost of inventories of EVOMELA.
Research and development expenses for the three months ended September 30, 2020 were $2.8 million, compared with $1.8 million for the three months ended September 30, 2019. The increases in R&D expenses are primarily due to increases in 2020 R&D expenses incurred related to the development of CID-103, and costs associated with the EVOMELA post marketing study.
General and administrative expenses for the three months ended September 30, 2020 were $5.3 million, compared with $8.0 million for the three months ended September 30, 2019. The decrease in general and administrative expenses was primarily because the 2019 period included costs related to sales and marketing efforts to prepare for the August 2019 launch of EVOMELA, as well as lower professional fees and travel costs incurred during the 2020 period.
Selling and marketing expenses for the three months ended September 30, 2020 were $2.1 million, compared with $975,000 for the three months ended September 30, 2019. The increase is due to selling costs related to commercial sales of EVOMELA that began in August of 2019.
Acquired in-process R&D expenses for the three months ended September 30, 2020 was $10.9 million, compared to $0 million for the three months ended September 30, 2019. Expense of $0.6 million relates to 2020 milestone fees paid to Pharmathen due to the first submission to the National Medical Products Administration in China for Octreotide which was achieved during 2020 and $10.3 million relates to milestone fees paid to Juventas.
Net loss for the third quarter of 2020 was $16.8 million compared to $9.7 million for the same period in 2019.
As of September 30, 2020, the Company had cash and cash equivalents of $74.6 million compared to $44.9 million as of June 30, 2020. As reported, the Company consummated an underwritten public offering in July 2020 generating gross proceeds of approximately $43.7 million.
Further information regarding the Company, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, can be found at www.casipharmaceuticals.com.

Conference Call

The Company will host a conference call reviewing the third quarter highlights today at 4:30 p.m. ET. The conference call can be accessed by dialing (833) 647-4459 (U.S.), (800) 870-0181 (China), (400) 682-8629 (China, domestic), (580) 86567 (Hong Kong) to listen to the live conference call. The conference ID number for the live call is 8835514.

This call will be recorded and available for replay by dialing (855) 589-2056 (U.S.) or (404)-537-3406 (international) and enter 8835514 to access the replay.

On November 9, 2020 Pulse Biosciences, Inc. (Nasdaq: PLSE), a novel bioelectric medicine company developing its Nano-Pulse Stimulation (NPS) technology, reported financial results for the third quarter ended September 30, 2020 (Press release, Pulse Biosciences, NOV 9, 2020, View Source [SID1234570418]).

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Recent Highlights

Submitted a 510(k) Premarket Notification application to the U.S. Food and Drug Administration (FDA) for the CellFX System seeking to obtain initial clearance for a general dermatologic indication. Potential clearance remains on track to be received as early as the first quarter of 2021.

Received FDA Investigational Device Exemption (IDE) approval and completed enrollment ahead of schedule in a pivotal comparison study to evaluate the treatment of sebaceous hyperplasia (SH) lesions using the CellFX System, accelerating the planned 510(k) submission into the first quarter of 2021 versus the previous estimate of the second quarter of 2021.

Submitted a Medical Device License application to Health Canada for the CellFX System after receiving the Medical Device Single Audit Program certification, now on track for a potential Health Canada license as early as the first quarter of 2021.

Advanced the interactive review process with the notified body and on track for potential controlled launch in the European Union as early as the first quarter of 2021.

Clinical results from four studies of the CellFX System were presented at the American Society for Dermatologic Surgery virtual annual meeting on October 9-11, 2020.

"We made excellent progress over the last several months on our clinical and regulatory objectives. We remain on track to potentially receive marketing clearances for our CellFX System in our three top geographies, the United States, Canada, and the European Union by the end of the first quarter of 2021. Additionally, completing enrollment in our sebaceous hyperplasia pivotal study in just over five weeks, two months ahead of schedule, is a testament to the interest physicians and patients have regarding the CellFX procedure for this application," said Darrin Uecker, President and CEO of Pulse Biosciences. "Importantly, I would like to thank our team for their hard work and recognize their accomplishments during this last quarter."

Financial Update

Cash, cash equivalents and investments totaled $29.6 million as of September 30, 2020, compared to $37.8 million as of June 30, 2020. Cash used in the third quarter of 2020 totaled $8.2 million. This compares with $7.9 million used in the second quarter of 2020 net of the rights offering proceeds.

Operating expenses for the three months ended September 30, 2020 were $12.9 million, compared to $12.0 million for the prior year period. Third quarter 2020 operating expenses included stock-based compensation expense of $2.6 million, compared to $2.7 million in the third quarter of 2019. The increase in operating expenses was primarily driven by general and administrative increases for facilities expansion, and research and development increases for headcount growth.

Operating expenses for the nine months ended September 30, 2020 were $36.2 million, compared to $34.0 million for the prior year period. Stock-based compensation expense for the nine months ended September 30, 2020 was $7.7 million, consistent with the prior year period. The increase in operating expenses was primarily driven by the expansion of operational infrastructure and increased headcount to support commercial preparations.

Net loss for the three months ended September 30, 2020 was ($12.9) million compared to ($11.7) million for the three months ended September 30, 2019. Net loss for the nine months ended September 30, 2020 was ($36.1) million compared to ($33.2) million for the nine months ended September 30, 2019.

Impact of COVID-19

Operations in the third quarter of 2020 experienced minimal impacts as a result of the COVID-19 pandemic. Product development and regulatory timelines have not been materially affected at this time but due to the uncertain scope and duration of the pandemic, future impact to our operations and financial results cannot be reasonably estimated.

Webcast and Conference Call Information

Pulse Biosciences’ management will host a conference call today, November 9, 2020 beginning at 1:30pm PT. Investors interested in listening to the conference call may do so by dialing 1-877-705-6003 for domestic callers or 1-201-493-6725 for international callers. A live and recorded webcast of the event will be available at View Source