Adaptimmune Reports Fourth Quarter / Full Year 2020 Financial Results and Business Update

On February 25, 2021 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported financial results for the fourth quarter and year ended December 31, 2020, and provided a business update (Press release, Adaptimmune, FEB 25, 2021, View Source [SID1234575726]).

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"We are building the cell therapy company of the future for people with cancer. With our ‘2-2-5-2’ by 2025 strategic plan, we will deliver value with marketed SPEAR T-cell products starting with ADP-A2M4 for people with synovial sarcoma," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "We completed enrollment in our SPEARHEAD-1 trial in approximately 12 months to support our first BLA, which is strong evidence of our ability to execute rapidly. We are focusing the SURPASS trial, using our next-generation ADP-A2M4CD8 product, on lung, gastroesophageal, head and neck, and bladder cancers to identify new indications to take forward to late-stage development."

Planned 2021 data updates1

SPEARHEAD-1 trial with ADP-A2M4 for people with synovial sarcoma
June: preliminary data at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)
November: full update at Connective Tissue Oncology Society (CTOS)
SURPASS Phase 1 trial with ADP-A2M4CD8 (next-generation product targeting MAGE-A4)
September: update at European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper)
Additional clinical updates
September: update at International Liver Cancer Association (ILCA) conference for ADP-A2AFP Phase 1 trial for people with liver cancer
October: update at American Society for Radiation Oncology (ASTRO) for radiation sub-study of the ADP-A2M4 Phase 1 trial2
November: ADP-A2M4 translational data update at Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)
"2-2-5-2" by 2025 strategic plan
At an Investor Day held in November 2020, the Company outlined its "2-2-5-2" by 2025 strategic plan encompassing:

"2" – Two marketed products targeting MAGE-A4
Estimated potential addressable population in tumor types with significant MAGE-A4 expression, factored for HLA-A23, is ~39,000 patients per year in the US and EU

Durable responses in synovial sarcoma – on track to file a Biologics License Application (BLA) for ADP-A2M4 in 2022

The first commercial opportunity for SPEAR T-cells targeting MAGE-A4 will be in synovial sarcoma with plans to file a BLA in the US in 2022
In 2020, the Company received positive endorsements from regulatory authorities with Regenerative Medicine Advanced Therapy (RMAT) designation and Orphan Drug Designation (ODD) in the US, and access to PRIority MEdicines (PRIME) regulatory support and ODD in the EU
Data from the Phase 1 trial with ADP-A2M4 (presented at CTOS 2020)
In 16 patients with synovial sarcoma, there was an Overall Response Rate of 44% and a Disease Control Rate of 94%
Responses were durable with a median duration of response of 28 weeks with ongoing responses beyond 72 weeks in two patients (median overall survival had not been reached)
These data are considerably superior4 to response rates observed with available second line therapies in synovial sarcoma
SURPASS-2 in esophageal and esophagogastric junction (EGJ) cancers in 1H 2021

The Company will initiate a Phase 2 trial, SURPASS-2, with ADP-A2M4CD8 (next-generation SPEAR T-cells targeting MAGE-A4 that co-express CD8α intended to increase potency) for patients with esophageal or EGJ cancers in 1H 2021
SURPASS-2 is supported by encouraging data from the Phase 1 SURPASS trial (presented at SITC (Free SITC Whitepaper) 2020) with one confirmed partial response (PR) in a patient with EGJ cancer and tumor reductions in two additional patients (1 with esophageal and 1 with EGJ cancer)
The trial will be conducted at multiple centers in North America and the EU, and is intended to enroll 45 people with esophageal or EGJ cancers to be treated with doses up to 10 billion SPEAR T-cells
The Company also presented preclinical data at SITC (Free SITC Whitepaper) 2020 indicating 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 the Phase 1 SURPASS trial.
"2" – Two additional BLAs for SPEAR T-cell products

Adaptimmune’s Phase 1 SURPASS trial with ADP-A2M4CD8 continues to enroll patients, focusing on lung, gastroesophageal, head and neck, and bladder cancers – indications for which the Company has reported responses or signs of efficacy with its MAGE-A4 targeted products
In 2020, Adaptimmune initiated SPEARHEAD-2 with its first-generation SPEAR T-cells targeting MAGE-A4 in combination with pembrolizumab for people with head and neck cancers
The Phase 1 trial with ADP-A2AFP for people with liver cancer is ongoing. As presented at ILC 2020, nine patients were treated as of the data cut-off and best responses were:
One patient with a complete response, one with stable disease (SD), and two with progressive disease (PD) among the four patients who received ~5 billion or more SPEAR T-cells
Five patients with SD who received doses of 100 million and 1 billion SPEAR T-cells in the first two dose cohorts
"5" – Five new autologous products in the clinic

Adaptimmune has a deep preclinical pipeline from which it expects to bring five new products into the clinic.
These include multiple possibilities for next-generation autologous SPEAR T-cells such as:
ADP-A2AFP SPEAR T-cells co-expressing CD8α
ADP-A2M4 SPEAR T-cells co-expressing IL-7, IL-15, dnTGFβ, and/ or PDE7
Enhancing SPEAR T-cells with IL-7 for proliferation and survival and CCL19 for migration into tumor in collaboration with Noile-Immune Biotech, Inc.
Enhancing SPEAR T-cells using transmembrane and surface immunoregulatory mechanisms with Alpine Immune Sciences, Inc.
The Company is also developing new products, including:
Expanding into HLAs beyond HLA-A2 to increase the addressable patient population
HLA-independent TCR (HiT) candidates for multiple targets including GPC3
A new program for next-generation TILs co-expressing IL-7 in melanoma in collaboration with leading TIL therapy center (CCIT, Denmark)
"2" – Two allogeneic products in the clinic

In January 2020, Adaptimmune announced it had entered into an agreement to co-develop and co-commercialize stem-cell derived allogeneic cell therapies with Astellas
The first target nominated is a HiT targeting mesothelin
At ASGCT (Free ASGCT Whitepaper) 2020, Adaptimmune presented data with evidence of its allogeneic platform demonstrating differentiation of functional T-cells from human-induced pluripotent stem cells (hiPSCs) that can kill MAGE-A4 expressing target cells in vitro –targeted to become the Company’s first allogeneic product in the clinic
Corporate updates from 2020

Despite the impact of the COVID-19 pandemic on the biotechnology industry, Adaptimmune continued to see improved enrollment in its clinical trials
In Q1, the Company received an upfront payment of $50 million from Astellas. The Company is also entitled to receive research funding of up to $7.5 million per collaboration target per year
Underwritten public offering in Q1 generated net proceeds of approximately $90 million
Underwritten public offering in Q2 generated net proceeds of approximately $244 million
Financial Results for the fourth quarter and year ended December 31, 2020

Cash / liquidity position: As of December 31, 2020, Adaptimmune had cash and cash equivalents of $56.9 million and Total Liquidity5 of $368.2 million.

Revenue: Revenue for the fourth quarter and year ended December 31, 2020 was $1.5 million and $4.0 million, respectively, compared to $0.7 million and $1.1 million for the same periods in 2019. The increase was due to revenue arising under the collaboration agreement with Astellas, which was entered into in January 2020.

Research and development (R&D) expenses: R&D expenses for the fourth quarter and year ended December 31, 2020 were $25.8 million and $91.6 million, respectively, compared to $20.4 million and $97.5 million for the same periods in 2019. R&D expenses in the year ended December 31, 2019 included the accrual of a purchase commitment and higher costs for in-process research and development; excluding the impact of these, research and development expenses have increased as the Company progresses development of its cell therapies.

General and administrative (G&A) expenses: G&A expenses for the fourth quarter and year ended December 31, 2020 were $13.2 million and $45.8 million, respectively, compared to $10.7 million and $43.4 million for the same periods in 2019. The increase in G&A expenses was due to an increase in general corporate costs, including professional fees and insurance.

Net loss: Net loss attributable to holders of the Company’s ordinary shares for the fourth quarter and year ended December 31, 2020 was $36.6 million and $130.1 million ($(0.15 per ordinary share), compared to $29.4 million and $137.2 million ($(0.22) per ordinary share) for the same periods in 2019.
Financial Guidance
The Company believes that its existing cash, cash equivalents and marketable securities will fund the Company’s current operations into early 2023, as further detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, to be filed with the Securities and Exchange Commission following this earnings release.

Conference Call Information
The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EST (1:00 p.m. GMT) today, February 25, 2021. The live webcast of the conference call will be available via the Events page 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, if preferred, please dial (833) 652-5917 (US 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 (2099860).

Milestone Pharmaceuticals to Present at Upcoming Investor Conferences

On February 25, 2021 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, reported that Joseph Oliveto, President and Chief Executive Officer, will present at the following investor conferences (Press release, Milestone Pharmaceuticals, FEB 25, 2021, View Source [SID1234575722]):

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The Cowen 41st Annual Health Care Conference’s panel on Cardiovascular Disease and Hospital Products on Thursday, March 4, 2021 at 1:30 p.m. Eastern Time.
The H.C. Wainwright Global Life Sciences Conference on Tuesday, March 9, 2021 on demand beginning at 7:00 a.m. Eastern Time.
The Oppenheimer 31st Annual Healthcare Conference on Tuesday, March 16, 2021 at 10:00 a.m. Eastern Time.
A replay of the pre-recorded H.C. Wainwright presentation and live webcast Oppenheimer presentation can be accessed in the News & Events section of Milestone’s website at www.milestonepharma.com. An archived replay of the webcasts will be available on the same website for approximately 90 days following the presentations.

ESSA Pharma Announces Clinical Collaboration with Astellas to Evaluate the Combination of EPI-7386 and Enzalutamide for Patients with Metastatic Castration-Resistant Prostate Cancer

On February 25, 2021 ESSA Pharma Inc. (Nasdaq: EPIX) ("ESSA" or the "Company"), a clinical-stage pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, reported that the Company has entered into a clinical collaboration and supply agreement with Astellas Pharma Inc. ("Astellas") to evaluate ESSA’s lead product candidate, EPI-7386, a first-in-class N-terminal domain androgen receptor inhibitor, in combination with Astellas and Pfizer Inc.’s androgen receptor inhibitor, enzalutamide, in patients with metastatic castration-resistant prostate cancer ("mCRPC") (Press release, ESSA, FEB 25, 2021, View Source [SID1234575721]).

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Under the terms of the agreement, ESSA will sponsor and conduct a Phase 1/2 study to evaluate the safety, tolerability and preliminary efficacy of the combination of EPI-7386 and enzalutamide in mCRPC patients who have not yet been treated with second-generation antiandrogen therapies. Astellas will supply enzalutamide for the trial. ESSA will retain all rights to EPI-7386. The clinical study is expected to start in 2021.

"We are delighted to collaborate with Astellas to explore the potential clinical role of EPI-7386 in combination with Astellas’ enzalutamide in patients with metastatic castration-resistant prostate cancer, who have progressed on androgen deprivation therapy," said Dr. David R. Parkinson, Chief Executive Officer, ESSA Pharma Inc. "Combining our two therapies will simultaneously target both ends of the androgen receptor. In preclinical models, we have seen that combining EPI-7386 with current antiandrogens can lead to deeper and broader inhibition of androgen biology. We look forward to investigating the combination of these therapies and their potential role together in the treatment of prostate cancer."

About EPI-7386
EPI-7386 is an investigational, highly-selective, oral, small molecule inhibitor of the N-terminal domain of the androgen receptor. EPI-7386 is currently being studied in a Phase 1 clinical trial (NCT04421222) in men with mCRPC whose tumors have progressed on current standard-of-care therapies. The Phase I clinical trial of EPI-7386 began in Q3 of 2020 following FDA allowance of the IND and Health Canada acceptance. The U.S. FDA has granted Fast Track designation to EPI-7386 for the treatment of adult male patients with mCRPC resistant to standard-of-care treatment. ESSA retains all rights to EPI-7386 worldwide.

Synthetic Biologics to Report 2020 Year End Operational Highlights and Financial Results on March 4, 2021

On February 25, 2021 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need, reported operational highlights and financial results for the year ended December 31, 2020 on Thursday, March 4, 2021, and will host a conference call the same day at 4:30 p.m. ET (Press release, Synthetic Biologics, FEB 25, 2021, View Source [SID1234575720]). The dial-in information for the call is as follows:

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Participants are asked to dial in 15 minutes before the start of the call to register. The call will also be webcast over the Internet at View Source . An archived replay of the call will be available for approximately ninety (90) days at the same URL, View Source beginning approximately one hour after the call’s conclusion.

Compugen Reports Fourth Quarter and Full Year 2020 Results

On February 25, 2021 Compugen Ltd. (NASDAQ: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, Compugen, FEB 25, 2021, View Source [SID1234575719]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Our encouraging new data reinforces our conviction in our clinical development strategy to comprehensively evaluate dual and triple blockade of DNAM axis members, PVRIG and TIGIT, along with the intersecting PD-1 pathway, across our clinical studies," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "We have now observed durable responses in patients treated with COM701 as a monotherapy and in combination with Opdivo, including a patient with a confirmed complete response we reported earlier today. Most notably, the durable responses and signals of anti-tumor activity reported across our COM701 studies were achieved in highly refractory patients in indications usually unresponsive to available checkpoint inhibitors. These encouraging signals demonstrate that COM701 is clinically active and strengthen our hypothesis that PVRIG is an important new checkpoint target for immunotherapy and served as the basis for expanding our collaboration with Bristol Myers Squibb."

Dr. Cohen-Dayag added, "With these data in hand, we are now focused on executing across our expanded clinical programs. Most importantly, as the only company with wholly owned clinical assets targeting PVRIG and TIGIT, we are uniquely positioned to evaluate PVRIG in monotherapy and in dual blockade with PD-1 or TIGIT, as well as the triple blockade of PVRIG with PD-1 and TIGIT. We expect to initiate our combination study for COM701 with COM902 in the second half of this year and to share initial data from our COM902 monotherapy and triple combination dose escalation studies in the fourth quarter of this year. We look forward to further revealing the potential of DNAM axis blockade to expand the reach of immunotherapy to patients unresponsive or refractory to current treatment options."

Recent and Fourth Quarter 2020 Corporate Highlights

Announced updated data from COM701 monotherapy and combination with Opdivo (nivolumab) studies
Announced expansion of clinical collaboration agreement with Bristol Myers Squibb with planned Phase 1b cohort expansion study evaluating COM701 with Opdivo in patients with ovarian, breast, endometrial and microsatellite-stable colorectal cancers. The study is on track to initiate in the second quarter of 2021
Announced first development milestone of $2 million received under the license agreement with AstraZeneca for the development of bispecific and multi-specific antibody products
Presented preclinical data at the 2020 TIGIT Therapies Digital Summit demonstrating the potential of PVRIG inhibition to enhance T cell priming and infiltration into both inflamed and less inflamed tumors, as well as providing further support for the therapeutic combination of TIGIT and PD-1 inhibitors to address patient populations who do not benefit from available immune checkpoint inhibitors
Expanded IP portfolio for COM902 with composition of matter patent in China, in addition to composition of matter and use patents for COM902 previously issued in the United States and Europe
Financial Results

Revenues for the fourth quarter ended December 31, 2020, were $2.0 million, related to the milestone payment in the license agreement with AstraZeneca for the development of bispecific and multi-specific antibody products.

R&D expenses for the fourth quarter and year ended December 31, 2020, were $8.1 million, and $22.8 million, respectively, compared with $4.3 million and $19.8 million for the comparable periods in 2019. The increase in both cases is attributed mostly to increase in expenses associated with our various Phase 1 clinical studies, COM701 and COM902 manufacturing and other chemistry, manufacturing and controls activities.

General and administrative expenses for the fourth quarter and year ended December 31, 2020, were $2.7 million, and $9.8 million, respectively, compared with $2.2 million and $8.4 million for the comparable periods in 2019. The increase in both the quarterly and annual periods is attributed mainly to headcount-related expenses and increased corporate-related expenses.

Net loss for the fourth quarter of 2020 was $8.6 million, or $0.10 per basic and diluted share, compared with a net loss of $6.5 million, or $0.10 per basic and diluted share, in the comparable period of 2019. Net loss for the year ended December 31, 2020 was $29.7 million, or $0.37 per basic and diluted share, compared with a net loss of $27.3 million, or $0.43 per basic and diluted share, for the year ended December 31, 2019.

As of December 31, 2020, cash, cash related accounts, short-term and long-term bank deposits totaled approximately $124.4 million, compared with approximately $43.9 million on December 31, 2019. The Company has no debt.

Opdivo is a registered trademark of Bristol Myers Squibb.

Conference Call and Webcast Information

The Company will hold a conference call today, February 25, 2021, at 8:30 AM ET to review its fourth quarter and full year 2020 results. To access the conference call by telephone, please dial 1-866-744-5399 from the United States, or +972-3-918-0610 internationally. The call will also be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website.