On August 9, 2021 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, reported operating results for the second quarter ended June 30, 2021 and provided pipeline updates (Press release, AnaptysBio, AUG 9, 2021, View Source [SID1234586124]).

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"We continue to make progress in advancing our wholly-owned pipeline and look forward to multiple clinical data readouts over the upcoming 18 months," said Hamza Suria, president and chief executive officer of AnaptysBio. "The recent approval of JEMPERLI, which is the first AnaptysBio-generated antibody to be approved in the US and EU, validates AnaptysBio’s antibody discovery platform and provides additional revenues to support our capital-efficient business model."

Imsidolimab (Anti-IL-36 Receptor) Program

Following an end-of-Phase 2 meeting with the FDA in Q2, we publicly disclosed trial designs for our imsidolimab generalized pustular psoriasis (GPP) Phase 3 trials, called GEMINI-1 and GEMINI-2. We anticipate initiating GEMINI-1 in Q3 2021. The primary endpoint of our Phase 3 program is the proportion of patients achieving clear or almost clear skin as determined by a Generalized Pustular Psoriasis Physician’s Global Assessment (GPPPGA) score of zero or 1 at week 4 of GEMINI-1, while GEMINI-2 is designed for 6 months of safety follow-up assessment.
We continue to enroll GPP patients in our worldwide registry of GPP patients, called RADIANCE, which is designed to improve our understanding of GPP patient journeys and support enrollment of our GEMINI Phase 3 trials. Medical claims analyses recently conducted by IQVIA indicate approximately 37,000 unique patients were diagnosed with GPP at least once, and approximately 15,000 unique patients were diagnosed with GPP at least twice, in the United States by a physician between 2017 and 2019 using the International Classification of Diseases 10th Revision (ICD-10) billing code pertaining to GPP (L40.1).
Full data from our completed Phase 2 GALLOP trial of imsidolimab in GPP, including efficacy and safety of imsidolimab treatment through week 16, will be disclosed in an oral presentation at the European Academy of Dermatology and Venereology (EADV) Congress on October 2nd, 2021.
We are continuing to advance imsidolimab through Phase 2 clinical trials in multiple additional indications associated with IL-36 signaling dysfunction. Our 120-patient placebo-controlled ACORN Phase 2 trial of imsidolimab in moderate-to-severe acne is anticipated to read out top-line data in the first half of 2022. Imsidolimab is also being tested versus placebo in hidradenitis suppurativa, where our 120-patient HARP trial is anticipated to generate top-line data in the second half of 2022. We continue to enroll our 45-patient placebo-controlled EMERGE Phase 2 trial of imsidolimab in EGFR/MEK-mediated skin toxicities, where we anticipate an interim analysis by the end of 2021. We also continue to enroll patients in our INSPIRE Phase 2 trial in ichthyosis where top-line data is anticipated during 2022.
Rosnilimab (Anti-PD-1 Agonist) Program

We anticipate top-line data in Q4 2021 from our ongoing Phase 1 healthy volunteer clinical trial of rosnilimab, our wholly-owned PD-1 agonist antibody, designed to assess the safety, pharmacokinetics and pharmacodynamics of rosnilimab in single and multiple ascending dose cohorts.
We plan to initiate a placebo-controlled Phase 2 clinical trial of rosnilimab in alopecia areata in Q4 2021.
ANB032 (Anti-BTLA Modulator) Program

We are advancing ANB032, our wholly-owned BTLA modulator antibody, in a healthy volunteer Phase 1 single and multiple ascending dose clinical trial where top-line data is anticipated during the first half of 2022.
GSK Partnered Programs

JEMPERLI (dostarlimab), our proprietary anti-PD-1 antagonist antibody, was approved by the FDA and the European Medicines Agency (EMA) during April 2021 for treatment of advanced or recurrent mismatch repair deficient endometrial cancer. This is the first AnaptysBio-generated antibody, of eight currently under clinical development, to obtain regulatory approval. GSK has recently disclosed peak annual sales estimates of £1-£2 billion for JEMPERLI, and AnaptysBio will earn 8-25% royalties on global net sales of JEMPERLI. We received $20 million and $10 million milestone payments upon FDA and EMA approval of JEMPERLI, respectively. We anticipate earning an additional $20 million milestone payment upon a second FDA BLA approval for JEMPERLI in pan-deficient mismatch repair tumors during the second half of 2021. AnaptysBio is due an additional $15 million and $165 million upon certain JEMPERLI regulatory and commercial milestones, respectively.
Second Quarter Financial Results

Cash, cash equivalents and investments totaled $396.3 million as of June 30, 2021, compared to $411.2 million as of December 31, 2020, for a decrease of $14.9 million. The decrease relates primarily to cash used for operating activities.
Collaboration revenue was $30 million and $41.3 million for the three and six months ended June 30, 2021. The $30 million earned during the second quarter relates to milestone revenue for the US and EU approval of JEMPERLI (dostarlimab), compared to zero and $15 million of milestone revenue for the three and six months ended June 30, 2020.
Research and development expenses were $25.3 million and $49.5 million for the three and six months ended June 30, 2021, compared to $17.9 million and $38.9 million for the three and six months ended June 30, 2020. The increase was due primarily to continued advancement of the Company’s clinical programs.
General and administrative expenses were $5.2 million and $10.7 million for the three and six months ended June 30, 2021, compared to $4.7 million and $9 million for the three and six months ended June 30, 2020. The increase was due primarily to personnel-related expenses, including share-based compensation.
Net loss was $0.4 million and $18.6 million for the three and six months ended June 30, 2021, or a net loss per share of $0.02, and $0.68, compared to a net loss of $21.5 million and $29.8 million for the three and six months ended June 30, 2020, or a net loss per share of $0.79 and $1.09.
Financial Guidance

AnaptysBio expects its net cash burn in 2021 will be less than $100 million. We anticipate that our cash, cash equivalents and anticipated revenues will fund our current operating plan at least into 2024.

On August 9, 2021 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the second quarter and six months ended June 30, 2021 and highlighted recent corporate progress (Press release, Avidity Biosciences, AUG 9, 2021, View Source [SID1234586123]).

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"The FDA clearance to proceed with our AOC 1001 Phase 1/2 MARINA trial in adults with myotonic dystrophy (DM1) is a huge milestone for Avidity and our AOC platform. AOC 1001 will be the first program based on our novel technology to enter clinical development. This also marks an important step forward for the DM1 community who have no approved therapies and so desperately needs therapeutic options," said Sarah Boyce, president and chief executive officer. "In addition, over the past quarter we made significant advances in our pipeline including nominating AOC 1044 as the clinical development candidate for our lead DMD program. We remain on track for both AOC 1044 and our AOC FSHD program to advance into the clinic in 2022."

"We are well funded with $280 million at the end of Q2’21, along with an additional $155 million in estimated net proceeds from our successful financing in August 2021. Our strong financial position allows us to further progress our late stage programs while continuing to invest in our skeletal muscle pipeline and our AOC platform," said Mike MacLean, chief financial officer.

AOC Platform and Pipeline Highlights

Received FDA clearance to proceed with clinical studies for AOC 1001 in adults with DM1.

Avidity recently received clearance to proceed with the Phase 1/2 MARINA trial of AOC 1001 in adults with DM1, under its Investigational New Drug application (IND). Avidity continues to be on track to initiate the Phase 1/2 MARINA clinical trial this year. In the second half of 2022, the Company plans to conduct a preliminary assessment of safety, tolerability and key biomarkers.

Detailed information on the MARINA study was presented during Volume 2 of Avidity’s virtual investor and analyst event series. Volume 2 featured presentations on AOC 1001, the MARINA study and a presentation on the clinical impact of DM1 by Nicholas E. Johnson, MD, MSCI, FAAN, an associate professor, division chief of neuromuscular, and vice chair of research in the department of neurology at Virginia Commonwealth University. The event also featured a live Q&A session with Avidity’s management team and Dr. Johnson. Volume 2 in the series follows Volume 1 which was focused on the years of engineering underpinning Avidity’s AOC platform and AOC 1001. Replays of Volume 1 and Volume 2 can be found on the events page in the investors section of the Avidity website.

In July, the FDA granted Orphan Drug Designation for AOC 1001 for the treatment of DM1. The FDA grants Orphan Drug Designation to novel drugs that seek to treat a rare disease or condition and, if the drug is approved for the designated orphan indication, provides 7 years of market exclusivity, along with certain financial incentives, including tax credits, opportunities for grant funding towards clinical trial costs and FDA user-fee waivers.

In May, Avidity reported results from its IND-enabling toxicology study of AOC 1001. Results from the study showed the highest dose tested was the maximum feasible dose and was the no-observed adverse effect level (NOAEL). The Company did not observe any treatment-related histopathologic toxicity or any changes in safety pharmacology parameters (cardiac, respiratory and neurological). All dose levels in the study produced a greater than 80% reduction in the expression of dystrophy myotonic protein kinase (DMPK) across multiple skeletal muscles, demonstrating that pharmacology was essentially saturated even at the lowest dose tested.
Progressed Skeletal Muscle Pipeline including nominating AOC 1044 to move into IND-enabling studies.

AOC 1044 was recently nominated as the clinical development candidate for the DMD program targeting Exon 44. AOC 1044 is entering into IND-enabling studies and is on track with plans to advance into the clinic in 2022.

Data from the AOC FSHD program targeting facioscapulohumeral muscular dystrophy (FSHD) was presented at the 28th Annual FSHD Society International Research Congress (IRC) in June. The data demonstrated promising preclinical activity in both in vitro and in vivo experiments in the cells of patients with FSHD.
Second Quarter 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $279.5 million as of June 30, 2021, compared to $328.1 million as of December 31, 2020.
Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $2.6 million for the second quarter of 2021 compared with $1.5 million for the second quarter of 2020, and $5.3 million for the first six months of 2021 compared with $2.9 million for the first six months of 2020.
Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $22.7 million for the second quarter of 2021 compared with $9.0 million for the second quarter of 2020, and $43.4 million for the first six months of 2021 compared with $14.5 million for the first six months of 2020. The increases were primarily driven by the advancement of AOC 1001, AOC 1044 and our AOC FSHD program, as well as costs related to the expansion of our overall research capabilities.
General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs, and patent filing and maintenance fees. These expenses were $6.3 million for the second quarter of 2021 compared with $2.9 million for the second quarter of 2020, and $12.2 million for the first six months of 2021 compared with $4.9 million for the first six months of 2020. The increases were primarily due to higher personnel costs (including noncash stock-based compensation), professional fees and insurance costs related to being a public company.

On August 9, 2021 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported that financial results for the quarter ending June 30, 2021 and updates on the Company’s key pipeline developments, business operations, and upcoming milestones (Press release, Rocket Pharmaceuticals, AUG 9, 2021, View Source [SID1234586122]).

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"We are grateful to the FDA for its support and for working with us on our Danon program toward resuming our trial, which we believe will occur in the third quarter," said Gaurav Shah, M.D., chief executive officer of Rocket Pharma. "Further, we have observed durable clinical benefit in the low dose adult cohort, which we believe is supportive of its potential as a viable Phase 2 dose. As of July 2021, we see improvement in two of three low dose patients in NYHA class. In these two patients, we also observed substantial improvement of a key marker of heart failure, BNP, which decreased from a pretreatment baseline by 75 percent in one patient and 79 percent in the other as well as improvement in cardiac output by 35 percent in one patient and 62 percent in the other as measured by invasive hemodynamics. The third patient has demonstrated stabilization of NYHA class and BNP. Given the positive benefit/risk profile in the low dose, and additionally to mitigate safety concerns observed at the higher dose, in agreement with FDA we will no longer treat Danon patients with the higher dose (1.1e14). With our full focus on the low dose, we look forward to progressing our trial on behalf of Danon patients devastated by this disease."

Dr. Shah continued, "We also presented positive data across three of our lentiviral-based gene therapy programs at ASGCT (Free ASGCT Whitepaper) in May, which we believe support the growing potential of these programs to treat Fanconi Anemia (FA), LAD-1 and PKD patients. Based on these results, we continue our momentum toward advancing these programs. In the case of our FA program, two recent publications reinforce the natural history, clinical design and methods being utilized in our Phase 1/2 FA trial of RP-L102. We look forward to providing updates on all five of our programs in the fourth quarter of 2021."

Dr. Shah added, "Finally, and importantly, we are deeply saddened that the first patient dosed in our Phase 1 IMO trial has passed way from pulmonary hemorrhage related to thrombocytopenia following conditioning therapy and also related to underlying osteopetrosis. This event was considered likely not related to RP-L401 gene therapy. Consistent with the trial protocol, enrollment has been temporarily paused pending a comprehensive evaluation in collaboration with the Independent Data Monitoring Committee, which will include a review of the conditioning regimen and other potential safety measures to mitigate the impact of underlying disease on treatment. This outcome underscores the need to find cures for this devastating disease and has furthered our commitment and dedication to patients and our mission to develop curative gene therapies for rare disease."

Key Pipeline and Operational Updates

Danon Disease:

Progressed toward agreement with FDA on changes to the Phase 1 clinical trial protocol in Danon Disease. Rocket anticipates trial may resume this quarter. The U.S. Food and Drug Administration (FDA) had previously requested Rocket pause patient dosing in the Phase 1 clinical trial of RP-A501 and modify the protocol and other supporting documents with revised guidelines for patient selection and management. No new drug-related safety events were observed in the low- or higher-dose adult cohorts; the previously disclosed SAE of thrombotic microangiopathy, which has since resolved, was reclassified as a SUSAR. All follow-up study activities continue. Longer-term results from the low (6.7e13) and higher dose (1.1e14) adult cohorts will be reported in the fourth quarter. In agreement with FDA, Rocket will no longer continue dosing patients at the higher dose (1.1e14).
Fanconi Anemia (FA):

Presented positive clinical updates from RP-L102 Fanconi Anemia (FA) program at ASGCT (Free ASGCT Whitepaper). Preliminary results from the Phase 1 and 2 trials presented in a poster at ASGCT (Free ASGCT Whitepaper) are from nine pediatric patients. For RP-L102, Rocket’s ex vivo lentiviral gene therapy candidate for FA, increasing evidence of engraftment was observed in at least six of the nine patients, including two patients with at least 15-months of follow-up and four patients with at least 6-months of follow-up. A highly favorable tolerability profile was also observed with all subjects being treated without conditioning and with no reports of dysplasia. One patient experienced a Grade 2 transient infusion-related reaction. The full data presented are available here.
Published two peer-reviewed studies supporting the natural history and clinical design of FA clinical trial. "Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients" was published in the American Journal of Hematology. Data strengthen the natural history of Fanconi Anemia and indicate that reverse mosaicism is a good prognostic factor in FA and is associated with more favorable long-term clinical outcomes. FA mosaicism in hematopoietic cells is a biologic and clinical proof-of principle for autologous gene therapy in FA patients and results provide a compelling rationale for continued clinical evaluation of autologous gene therapy. Additionally, "Improved Collection of Hematopoietic Stem Cells and Progenitors from Fanconi Anemia Patients for Gene Therapy Purposes" was published in Molecular Therapy: Methods & Clinical Development. Results demonstrate the safety and efficacy of filgrastim and plerixafor for mobilization of hematopoietic stem and progenitor cells (HSPCs) and collection by leukapheresis in FA patients, offering crucial information for the enrollment of FA patients for gene therapy studies.
Infantile Malignant Osteopetrosis (IMO):

First patient dosed in the RP-L401 Infantile Malignant Osteopetrosis (IMO) Phase 1 clinical trial passed away from likely non-RP-L401 gene therapy related pulmonary complications. IMO is a bone marrow-derived disorder associated with severe bone and hematologic manifestations leading to death in the first decade of life, frequently within the first two years of life, without an allogenic hematopoietic stem cell transplant (HSCT).

The first patient in the Phase 1 study, a six-year-old child with severe IMO-related anemia and bone abnormalities, was infused with RP-L401 without immediate complications. During the initial weeks after therapy, the patient died of pulmonary complications, most likely pulmonary hemorrhage related to thrombocytopenia following conditioning therapy and also related to underlying osteopetrosis. Pulmonary hemorrhage is a rare but documented complication of HSCT, and pulmonary complications, including life-threatening and fatal complications, have been observed to occur with high frequency in osteopetrosis patients undergoing allogeneic HSCT procedures.

The patient death is not considered to be RP-L401-related by study investigators and as corroborated by autopsy findings. In accordance with the trial protocol, enrollment has been temporarily paused pending a comprehensive evaluation in collaboration with the Independent Data Monitoring Committee.
Leukocyte Adhesion Deficiency-I (LAD-I):

Presented positive clinical updates from RP-L201 Leukocyte Adhesion Deficiency-I (LAD-I) program at ASGCT (Free ASGCT Whitepaper). Phase 1/2 data presented in an oral presentation at ASGCT (Free ASGCT Whitepaper) are from four pediatric patients with severe LAD-I. RP-L201, Rocket’s ex-vivo lentiviral gene therapy candidate showed preliminary activity in all four patients, including one patient with 18-months of follow-up and one patient with 9-months of follow-up. CD18 expression substantially exceeded the 4-10% threshold in all four patients, which is associated with survival into adulthood and consistent with the reversal of severe LAD-I phenotype. Most importantly, all four patients were able to leave the hospital in the weeks following RP-L201 therapy. The full data presented are available here.
Pyruvate Kinase Deficiency (PKD):

Presented positive clinical updates from RP-L301 Pyruvate Kinase Deficiency (PKD) program at ASGCT (Free ASGCT Whitepaper). Updated preliminary Phase 1 data presented in an oral presentation at ASGCT (Free ASGCT Whitepaper) are from two patients with significant anemia and transfusion requirements that showed sustained tolerability. Preliminary activity, measured by peripheral blood VCN levels, was observed in both patients during the initial 9-months and 3-months post-treatment, respectively. Durable normalization of hemoglobin levels were observed, from an average baseline of ~7.4 grams (g)/deciliter (dL) to 13.1 g/dL at 9-months post treatment in the first patient and from a baseline of ~7.0 g/dL to 14.4 g/dL at 6-months post treatment in the second patient. The Phase 1 trial continues to enroll patients with longer-term data expected in the fourth quarter. The full data presented are available here.
Anticipated Milestones

Fanconi Anemia (RP-L102)
Updated "Process B" data (Q4 2021)
LAD-I (RP-L201)
Longer-term Phase 2 data (Q4 2021)
Danon Disease (RP-A501)
Longer-term Phase 1 data (Q4 2021)
PKD (RP-L301)
Longer-term Phase 1 data (Q4 2021)
IMO (RP-L401)
Phase 1 clinical update (Q4 2021)
Upcoming Investor Conference

Citi’s 16th Annual BioPharma Virtual Conference, Sept. 8-10, 2021
Second Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of June 30, 2021 were $426.8 million.
R&D expenses. Research and development expenses were $24.8 million for the three months ended June 30, 2021, compared to $16.7 million for the three months ended June 30, 2020, due to an increase in compensation and benefits expense resulting from increased R&D headcount, an increase in non-cash stock compensation expense, an increase in manufacturing and development costs, and an increase in clinical trials expense.
G&A expenses. General and administrative expenses were $9.3 million for the three months ended June 30, 2021, compared to $6.8 million for the three months ended June 30, 2020, due to an increase in non-cash stock compensation expense, an increase in compensation and benefits expense due to increased G&A headcount and an increase in office and administrative costs.
Net loss. Net loss was $34.5 million or $0.55 per share (basic and diluted) for the three months ended June 30, 2021, compared to $25.0 million or $0.45 per share (basic and diluted) for the three months ended June 30, 2020.
Shares outstanding. 63,448,069 shares of common stock were outstanding as of June 30, 2021.
Financial Guidance

Rocket expects its balance in cash, cash equivalents and investments of $426.8 million as of June 30, 2021 to fund its operations into the second half of 2023, including the continued buildout and initiation of AAV cGMP manufacturing capabilities at our Cranbury, New Jersey R&D and manufacturing facility and continued development of our five clinical programs.
Conference Call Details

Rocket management will host a conference call today at 4:30 p.m. ET. To access the call and webcast, please visit the events section of the website. The webcast replay will be available on the Rocket website following the completion of the call.

Investors may access the conference call by dialing (866) 939-3921 from locations in the United States or +1 (678) 302-3550 from outside the United States. Please refer to conference ID number 50210581.

On August 9, 2021 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported its financial results for the second quarter ended June 30, 2021 and provided an update on recent developments (Press release, IGM Biosciences, AUG 9, 2021, View Source [SID1234586121]).

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"We continue to make steady progress in the development of our innovative product pipeline, including the successful completion of the initial dose escalation portion of our IGM-2323 Phase 1 clinical trial," said Fred Schwarzer, Chief Executive Officer of IGM Biosciences. "We look forward to announcing a recommended Phase 2 dose for IGM-2323, presenting initial dose escalation data from the Phase 1 trial of IGM-8444, initiating a Phase 1 trial for IGM-6268 and filing an IND for IGM-7354 this year. "

Pipeline Updates

IGM-2323

Phase 1 dose escalation completed; dose expansion continues. IGM continues to advance the clinical development of IGM-2323, the Company’s IgM-based CD20 x CD3 bispecific antibody, for the treatment of non-Hodgkin’s lymphoma (NHL). The Company cleared its highest planned dose escalation cohort, a top dose of 1000 mg, without a dose limiting toxicity, and is currently treating additional patients in four Phase 1 dose cohorts with top doses of 100 mg, 300 mg, 600 mg and 1000 mg, respectively. IGM expects to select a recommended Phase 2 dose in 2021.
IGM-8444

Additional dose cohorts cleared in Phase 1. IGM continues to advance the clinical development of IGM-8444, the Company’s IgM Death Receptor 5 (DR5) agonist, for the treatment of a potentially broad range of solid tumors and hematologic malignancies. IGM has cleared its third dose cohort (3 mg/kg) of the single-agent portion of its Phase 1 clinical study and is currently treating patients in its highest dose escalation cohort (10 mg/kg) with every two-week single agent dosing. IGM has also cleared the first dose cohort of the FOLFIRI combination portion of the Phase 1 study and is currently treating patients in the second of four planned FOLFIRI combination dose escalation cohorts. IGM expects to report initial data from the dose escalation portion of the Phase 1 trial in 2021.
Clinical testing of birinapant in combination with IGM-8444 expected to begin this year. IGM remains on track to begin clinical testing of birinapant in combination with IGM-8444 in 2021.
IGM-6268

New pipeline candidate for the treatment and prevention of COVID-19 expected to advance into the clinic this year. In June 2021, IGM announced IGM-6268, which represents the expansion of the Company’s IgM platform into infectious diseases. IGM-6268 is an IgM version of an anti-SARS-CoV-2 IgG monoclonal antibody and is being developed as an intranasally administered agent for the treatment and prevention of COVID-19. IGM expects to initiate a Phase 1 clinical trial of IGM-6268 in 2021.
Nature manuscript published. In June 2021, Nature published an article entitled "Nasal delivery of an IgM offers broad protection from SARS-CoV-2 variants". The article describes results from preclinical studies demonstrating significantly greater neutralization of SARS-CoV-2 with an IgM antibody compared to IgG antibodies, the potent neutralization of all evaluated mutant Variants of Concern and Variants of Interest, and the ability to provide effective preventative and therapeutic protection when delivered intranasally in mice. The article was co-authored by researchers at IGM, The University of Texas Medical Branch at Galveston and The University of Texas Health Science Center at Houston.
IGM-7354

Investigational New Drug (IND) application expected to be filed this year. IGM plans to file an IND application with the U.S. Food and Drug Administration (FDA) for IGM-7354, the Company’s IL‑15 x PD‑L1 bispecific IgM antibody, before the end of 2021 in order to begin clinical testing initially in solid tumors, followed by hematologic malignancies.
Corporate Updates

Chris Takimoto appointed Chief Medical Officer. Dr. Takimoto brings 30 years of experience in cancer research and development. Most recently, Dr. Takimoto was Senior Vice President, Oncology at Gilead Sciences, Inc. Prior to Gilead, he served as Chief Medical Officer of Forty Seven, Inc., a biotechnology company formed out of Stanford University and acquired by Gilead Sciences in 2020.
Second Quarter 2021 Financial Results

Cash and Investments: Cash and investments as of June 30, 2021 were $301.8 million, compared to $366.3 million as of December 31, 2020.
Research and Development (R&D) Expenses: For the second quarter of 2021, R&D expenses were $30.1 million, compared to $15.0 million for the same period in 2020.
General and Administrative (G&A) Expenses: For the second quarter of 2021, G&A expenses were $8.6 million, compared to $4.4 million for the same period in 2020.
Net Loss: For the second quarter of 2021, net loss was $38.7 million, or a loss of $1.16 per share, compared to a net loss of $18.8 million, or a loss of $0.62 per share, for the same period in 2020.
2021 Financial Guidance

IGM reiterates its previously issued financial guidance expecting full year GAAP operating expenses to be between $175 million and $185 million including estimated non-cash stock-based compensation expense of approximately $25 million. IGM expects to end 2021 with a balance of over $200 million in cash and investments.

On August 9, 2021 Adaptimmune Therapeutics plc (Nasdaq: ADAP), a leader in cell therapy to treat cancer, reported financial results for the second quarter ended June 30, 2021 and provided a business update (Press release, Adaptimmune, AUG 9, 2021, View Source [SID1234586120]).

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"With the data presented at ASCO (Free ASCO Whitepaper) for afami-cel and our planned BLA filing next year, I am pleased with progress on our ‘2-2-5-2’ strategy," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "We will present data updates from our Phase 1 trials, SURPASS and ADP-A2AFP, at upcoming conferences and I am confident that Adaptimmune is well-placed to maintain its leadership position in cell therapies for cancer."

Upcoming confirmed data updates
ADP-A2AFP Phase 1 Trial at ILCA

On Sunday, September 5, 2021, Dr. Bruno Sangro of Clinica Universidad de Navarra will present data from Cohort 3 and the expansion phase of the Phase 1 trial of ADP-A2AFP in liver cancer during an oral presentation scheduled for 12:25 p.m. CET at the International Liver Cancer Association’s (ILCA) Annual Conference
As of the April 5th data cut-off used for ILCA, 13 patients had received ADP-A2AFP in Cohort 3 and expansion, 11 patients were evaluable with at least one post-baseline scan
SURPASS Phase 1 Trial at ESMO (Free ESMO Whitepaper)

The Company will present an update from its Phase 1 SURPASS trial in an e-poster at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) congress that will be available online September 16th
As of the August 2nd data cut-off, 25 patients had received ADP-A2M4CD8, 23 patients had at least one post-baseline scan
The trial continues to recruit in lung, gastroesophageal, head and neck, and bladder cancers – focus indications, for which there have been early signs of efficacy, including responses, with SPEAR T-cells targeting MAGE-A4
Based on early data from patients with ovarian cancer treated in the trial, the Company is planning to add this as a focus indication to the SURPASS trial
SPEARHEAD-1 and afami-cel BLA

As presented at ASCO (Free ASCO Whitepaper) 2021, the Company reported an overall response rate for patients with at least one scan (evaluated by RECIST 1.1 per investigator assessment) of 39.3% (13/33), 41.4% (12/29) for synovial sarcoma, including two complete responses, and 25.0% (1/4) for MRCLS from its Phase 2 SPEARHEAD-1 trial
Of the 29 patients with synovial sarcoma, the disease control rate (defined as either response or stable disease) was 86.2% (25/29 patients) with 2 complete responses and 10 partial responses
This data set is intended to support planned BLA filing next year
Next data update planned at CTOS 2021
The European Medicines Agency and the FDA have agreed to Adaptimmune’s pediatric investigational plans
Working with key industry leaders to prepare for a successful commercial launch
Adaptimmune has partnered with Agilent for the development, manufacturing, and supply of a companion diagnostic for the MAGE-A4 biomarker
The Company is also working with Miltenyi Biotec for the process validation, manufacture, and supply of the lentiviral vector for use in the product for commercial launch
Further indications for next-gen SPEAR T-cell therapies
SURPASS-2

Planning to initiate a Phase 2 clinical trial with ADP-A2M4CD8, SURPASS-2, in esophageal and esophagogastric junction cancers in the third quarter of 2021
Other early-stage programs

The Company ceased enrollment in the Radiation Sub-Study of the afami-cel Phase 1 trial at the end of July. Five patients were treated in this sub-study and the Company plans to provide a data update at an upcoming congress
Corporate

The Company received a development milestone payment of $4.2 million in the three months ended June 30, 2021
Financial Results for the three and six months ended June 30, 2021

Cash / liquidity position: As of June 30, 2021, Adaptimmune had cash and cash equivalents of $50.5 million and Total Liquidity1 of $285.4 million.
Revenue: Revenue for the three and six months ended June 30, 2021 was $3.1 million and $3.5 million, respectively, compared to $0.5 million and $1.3 million for the same periods in 2020. Revenue has increased primarily due to an increase in development activities under the Astellas Collaboration Agreement.
Research and development (R&D) expenses: R&D expenses for the three and six months ended June 30, 2021 were $28.9 million and $53.4 million, respectively, compared to $20.5 million and $41.7 million for the same periods in 2020. R&D expenses increased due to an increase in the number of employees engaged in research and development, and increases in costs related to the development of a companion diagnostic assay and our Phase 2 clinical trial associated with ADP-A2M4CD8. These increases were partially offset by an increase in reimbursements receivable for research and development tax and expenditure credits.
General and administrative (G&A) expenses: G&A expenses for the three and six months ended June 30, 2021 were $13.5 million and $27.4 million, respectively, compared to $10.3 million and $19.6 million for the same periods in 2020 due to increases in employee-related costs, share-based compensation expense, and professional fees.
Net loss: Net loss attributable to holders of the Company’s ordinary shares for the three and six months ended June 30, 2021 was $39.1 million and $76.8 million respectively ($(0.04) and $(0.08) per ordinary share), compared to $29.9 million and $58.0 million ($(0.04) and $(0.07) per ordinary share) for the same periods in 2020.
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 Quarterly Report on Form 10-Q for the three and six months ended June 30, 2021, 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 4:30 p.m. EDT (9:30 p.m. BST) today. A live webcast of the conference call and replay can be accessed at https://bit.ly/3zFas59. 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 (7867634).