On July 29, 2021 HanAll Biopharma (KRX: 009420.KS) reported that the company had achieved sales of 23.4 billion won, operating profit of 1.6 billion won, and net profit of 3.3 billion won in the second quarter of 2021 (Press release, HanAll Biopharma, JUL 29, 2021, View Source [SID1234585417]).

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In the first half of this year, cumulative sales were reported as 51.2 billion won, a 15% increase compared to the same period last year, and operating profit increased by 56% to 7 billion. In particular, with increased sales in major products such as Normix, an antibiotic for gastrointesinal infection; Eligard, a prostate cancer treatment; and Biotop, a probiotic product; operating margin was at 14%, indicating an increase of 362 basis-point in profitability compared to the first half of last year.

As of the end of the first half, HanAll Biopharma maintained a stable financial status with a debt ratio of 25% and assets of 201.9 billion won, equity of 161.2 billion won, and liabilities of 40.6 billion won.

[R&D Pipeline]

– HL161 (a novel, fully human, subcutaneous anti-FcRn antibody therapeutic for autoimmune diseases)

HL161 (INN: batoclimab), an antibody-drug for autoimmune diseases caused by pathogenic antibodies, is currently undergoing clinical trials in the US, and China through its partners Immunovant and Harbor BioMed.

In early June, global partner Immunovant announced plans to resume clinical trials, which were put on hold in February this year due to an issue related to increased cholesterol. As a result of a detailed analysis of patients participating in the clinical trials, it said lipid elevations are predictable and manageable. It plans to return to the clinic and initiate a pivotal MG trial in late 2021 or early 2022 as well as resume its trial in WAIHA on a similar timeframe. In addition, the company has a plan to initiate at least two additional clinical studies over the next 12 months, including another pivotal trial in 2022.

Meanwhile, HanAll Biopharma is preparing a Phase 3 clinical trial for the treatment of myasthenia gravis in Japan in collaboration with Immunovant.

In China, Harbour BioMed is conducting clinical trials in myasthenia gravis, thrombocytopenia, and neuromyelitis optica. In July, it announced positive topline results from the Phase 2 clinical trial of batoclimab in myasthenia gravis.

Batoclimab demonstrated a statistically significant improvement (p=0.043) compared to placebo in patients with myasthenia gravis on MG-ADL (Myasthenia Gravis Activities of Daily Living), the primary endpoint of the study. In addition, the drug induced rapid, substantial and persistent clinical improvement over placebo as measured by all four predefined clinical efficacy scales – MG-ADL, QMG (Quantitative Myasthenia Gravis), MGC (Myasthenia Gravis Composite) and MG-QoL (Myasthenia Gravis Quality of Life).

Furthermore, batoclimab has shown to be overall safe and well-tolerated, with incidence of adverse events (AE) comparable to placebo, majority of AEs characterized as mild, no serious adverse events (SAE) and no discontinuation due to AEs. Harbor BioMed plans to enter Phase 3 clinical trial of HL161 in myasthenia gravis in China in the second half of this year.

– HL036 (a novel, topical anti-TNF biologic therapy for dry eye disease)

HL036 (INN: tanfanercept), jointly developed by HanAll Biopharma and Daewoong Pharmaceutical, is a novel biologic treatment under development to treat inflammatory eye diseases by inhibiting TNF, which could cause inflammation in the eye. To target the dry eye disease market in the US, clinical trials are in progress in the US since Phase 2 and the first Phase 3 clinical trial for dry eye disease was completed in the first half of last year.

HanAll Biopharma submitted the IND for the second Phase 3 clinical trial (study name: VELOS-3) to the U.S. FDA on July 22, and is expected to begin clinical trials in the third quarter of this year and have topline results by the first half of next year.

The VELOS-3 study is a Phase 3, multicenter, randomized, double-masked and placebo-controlled study evaluating the efficacy and safety of tanfanercept (HL036) ophthalmic solution 0.25% compared to placebo in subjects with dry eye disease. Following two week screening period, 300 patients with dry eye disease will be randomized into treatment group and placebo. Each group will benefit administration of tanfanercept or placebo twice per day respectively for eight weeks.

Harbor BioMed, a license holder for the Chinese market, started a pivotal Phase 3 trial in dry eye disease in the first half of the year, and is still in progress. The first patient administration was started in March, and the efficacy and safety of tanfanercept will be evaluated in patients with dry eye disease throughout the study.

– HL189 (tanfanercept)

HanAll Biopharma is planning to expand tanfanercept, which is being developed in dry eye disease, with new inflammatory eye disorders including uveitis. The company will make a decision for the asset in the second half to the year.

– HL186/HL187 (novel immune-oncology therapies)

Currently, HanAll Biopharma is jointly developing a novel immune-oncology antibodies that target TIGIT and TIM-3 with Daewoong Pharmaceutical, and will take a step forward to preclinical study.

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On July 29, 2021 Sanofi reported that its half-year financial report for the period ending June 30, 2021 is now available and has been filed with the French market regulator Autorité des marchés financiers (AMF) and submitted to the U.S. Securities and Exchange Commission (SEC) under form 6-K (Press release, Sanofi, JUL 29, 2021, View Source [SID1234585353]).

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This document may be found on the company’s corporate website: www.sanofi.com and downloaded from the "Investors" page, under the heading "Regulated Information in France".

On July 29, 2021 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the second quarter ended June 30, 2021 and raised full-year 2021 guidance for product revenues to $7.2 to $7.4B (Press release, Vertex Pharmaceuticals, JUL 29, 2021, View Source [SID1234585369]).

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"In the second quarter of 2021, we saw continued, significant growth and strong business performance in our cystic fibrosis franchise. We have now secured reimbursement agreements for the triple combination in more than 15 countries outside the U.S. and started expansion into younger age groups with the U.S. approval in patients 6 to 11 years of age last month. With these advancements, we are poised to reach more patients in 2021 than previously forecasted and are therefore raising our 2021 revenue guidance. Looking forward, we continue to see significant growth ahead in CF, with more than 30,000 CF patients who may benefit from the triple combination but who are not yet treated," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.

"We also made important progress with our pipeline programs in the first half of this year. Our pipeline programs are advancing quickly, with five programs in mid- or late-stage clinical trials. We continue to see impressive clinical results with CTX001, our most advanced program outside of CF, in which we have now dosed more than 45 patients. Our pre-commercial efforts for this program are underway, as we prepare to serve patients with sickle cell disease and beta thalassemia and address the significant market opportunity. We look forward to multiple R&D milestones and data readouts in the coming 6 to 9 months," said Dr. Kewalramani.

Product revenues increased 18% compared to the second quarter of 2020, primarily driven by the uptake of KAFTRIO in Europe and continued strong performance of TRIKAFTA in the U.S. Net product revenues in the second quarter of 2021 increased 4% to $1.26 billion in the U.S. and increased 71% to $536 million outside the U.S., compared to the second quarter of 2020.

GAAP net income decreased compared to the second quarter of 2020, primarily due to a $900 million payment in connection with the amendment of Vertex’s collaboration with CRISPR Therapeutics that was recorded as a GAAP R&D expense in the second quarter of 2021.

Non-GAAP net income increased compared to the second quarter of 2020, largely driven by strong growth in product revenues.

Cash, cash equivalents and marketable securities were $6.71 billion, an increase of $49 million compared to $6.66 billion as of December 31, 2020, primarily driven by strong operating cash flow from Vertex’s revenue growth and profitability, and offset by the $900 million payment to CRISPR and repurchases of our common stock authorized under our 2020 share repurchase program.

Combined GAAP R&D and SG&A expenses increased compared to the second quarter of 2020, primarily due to the $900 million payment to CRISPR in the second quarter of 2021.

Combined Non-GAAP R&D and SG&A expenses increased compared to the second quarter of 2020, primarily due to the expansion of Vertex’s pipeline in CF and other disease areas and incremental investment to support the global launches of Vertex’s medicines.

GAAP income taxes reflected an increased benefit compared to the second quarter of 2020, primarily due to the income tax impact of the $900 million payment to CRISPR partially offset by a decrease in discrete tax benefits. Please refer to Note 2 for further details.

Non-GAAP income taxes increased compared to the second quarter of 2020 primarily due to Vertex’s increased operating income.

Full-Year 2021 Financial Guidance

Vertex today increased its full-year 2021 product revenue guidance based on strong year-to-date performance and the expected impact of recent reimbursement agreements. Vertex’s guidance is summarized below:

Cystic Fibrosis (CF) Marketed Products

Vertex anticipates that the number of CF patients treated with our medicines will continue to grow as we enter into additional reimbursement agreements, achieve new approvals for the treatment of younger patients, and expand treatment options for the approximately 10 percent of patients who do not benefit from CFTR modulators, all of which will lead to continued growth of our CF business in the years ahead.

In June, the U.S. FDA approved expansion of the indication of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with CF ages 6 through 11 years. Health Canada granted Marketing Authorization for TRIKAFTA for people with CF ages 12 years and older who have at least one F508del mutation. TRIKAFTA/KAFTRIO is now approved and reimbursed or accessible in more than 15 countries outside the U.S., including Italy and France.

R&D pipeline

Vertex continues to progress a broad pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is noted below:

Cystic Fibrosis

Vertex recently announced plans to initiate Phase 3 studies of the next-in-class, once-daily triple combination of VX-121, tezacaftor and VX-561 in the second half of 2021. Clinical and preclinical data suggest that this triple combination has the potential to provide enhanced benefit for people with CF who have the F508del mutation on at least one allele.
The Phase 3 program will consist of two 48-week trials, which will evaluate the safety and efficacy of the new combination relative to TRIKAFTA in a total of 800 patients. Both studies will measure the regulatory-enabling endpoint of absolute change in ppFEV1, a measure of lung function, that will be analyzed for non-inferiority to TRIKAFTA. Both studies will also assess absolute change from baseline in ppFEV1 and sweat chloride for superiority to TRIKAFTA.

Beta Thalassemia and Sickle Cell Disease

Based on the compelling data generated with CTX001, in April, Vertex and CRISPR Therapeutics announced an amendment to their collaboration for CTX001. In connection with the completion of the transaction in June, Vertex made a $900 million upfront payment to CRISPR.
Data from 22 patients with at least three months of follow-up after CTX001 infusion were presented at EHA (Free EHA Whitepaper) in June and continued to build the profile of a one-time functional cure for patients with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), showing consistent and durable benefit with longer term data from a larger population of patients.
Enrollment and dosing are ongoing in the clinical studies for CTX001 and more than 45 patients have been dosed across the program to date. Vertex anticipates achieving target enrollment in both studies in the third quarter of 2021, with regulatory filings possible in the next 18 to 24 months.
APOL1-mediated Kidney Diseases

Vertex is evaluating the potential of inhibitors of APOL1 function to treat people with APOL1-mediated kidney diseases.
Enrollment is ongoing in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated focal segmental glomerulosclerosis (FSGS) following treatment with VX-147.
Data from this study are expected in the second half of 2021.
Pain

NaV1.8 is a genetically and pharmacologically validated novel target for the treatment of pain, and Vertex has previously demonstrated clinical proof-of-concept with a small molecule investigational treatment targeting NaV1.8 in multiple pain indications including acute pain, neuropathic pain and musculoskeletal pain. Vertex’s approach is to selectively inhibit NaV1.8 using small molecules with the objective of creating a new class of medicines that have the potential to provide superior relief of acute pain without the limitations of opioids, including their addictive potential. VX-548 is the most recent molecule to enter clinical development from Vertex’s portfolio of NaV1.8 inhibitors.
Vertex announced in July that the VX-548 Phase 2 acute pain program has been initiated. The proof-of-concept trial for acute pain following bunionectomy surgery is open for enrollment, and the VX-548 trial following abdominoplasty surgery will commence in the coming weeks.
Data from the bunionectomy trial are expected by early 2022.
Type 1 Diabetes (T1D)

Vertex is evaluating a cell therapy designed to replace insulin-producing islet cells in people with T1D. Vertex is pursuing two programs for the transplant of stem cell-derived, fully differentiated, insulin-producing islet cells into patients: 1) transplantation of islet cells alone, using immunosuppression to protect the implanted cells and 2) implantation of the islet cells inside a novel immunoprotective device.
A Phase 1/2 clinical trial for VX-880, the islet cells alone program, is ongoing in people with T1D. The first patient in this study has been dosed, and initial data from this study are expected in 2022.
Alpha-1 Antitrypsin (AAT) Deficiency

Vertex continues to evaluate small molecule correctors of zAAT protein to target the underlying cause of AATD, and thereby address both lung and liver manifestations of the disease.
Vertex plans to advance one or more novel small molecule zAAT correctors into the clinic in 2022.

On July 29, 2021 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, reported business highlights and financial results for the second quarter ended June 30, 2021 (Press release, Agios Pharmaceuticals, JUL 29, 2021, View Source [SID1234585385]).

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"Our first quarter as a company solely focused on genetically defined diseases was marked by several significant milestones, most notably our U.S. and EU regulatory submissions for mitapivat for the treatment of adults with PK deficiency, bringing us one step closer to potentially delivering the first disease-modifying treatment for people with this serious and underserved condition," said Jackie Fouse, Ph.D., chief executive officer at Agios. "We continue to unlock the potential of mitapivat for people with other chronic hemolytic anemias and are gaining momentum on our pivotal programs in thalassemia and sickle cell disease. This year, we look forward to initiating our two registrational Phase 3 trials – ENERGIZE and ENERGIZE-T – in not regularly transfused and regularly transfused adults with thalassemia, as well as our pivotal Phase 2/3 trial of mitapivat in sickle cell disease."

SECOND QUARTER 2021 & RECENT HIGHLIGHTS

Completed two regulatory filings for approval of mitapivat in adults with PK deficiency; submitted new drug application (NDA) in the U.S. and marketing authorization application (MAA) in the EU.
Presented full analysis of data from Phase 3 ACTIVATE and ACTIVATE-T studies of mitapivat in adults with PK deficiency at European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress; studies met primary and secondary endpoints, including patient-reported outcome (PRO) measures that address symptom burden and quality-of-life impact in adults.
Presented data from Phase 2, open-label, multicenter study of mitapivat in adults with non-transfusion dependent α- or β-thalassemia at EHA (Free EHA Whitepaper) Virtual Congress; study met its primary endpoint, with 16 of the 20 patients (80%) achieving a hemoglobin increase of ≥1.0 g/dL from baseline at one or more assessments during Weeks 4-12.
Launched myAgios patient support services for people living with PK deficiency and their caregivers, providing tailored support, educational resources and opportunities to connect with other patients and caregivers in the community.
Completed hiring and training of customer-facing and patient support team that will support the U.S. launch of mitapivat in PK deficiency upon product approval.
Announced succession plan for Chris Bowden, M.D., who will transition from his role as chief medical officer to strategic advisor following his retirement on Sept. 1, at which time Sarah Gheuens, M.D., Ph.D., vice president of clinical development, will assume the role.
Repurchased approximately 10.5 million shares of Agios common stock, inclusive of shares acquired from Bristol-Myers Squibb Company (BMS) and its affiliates, at an average price of $50.41 per share. This accounts for $529 million of the up to $1.2 billion of share repurchases authorized by the Board of Directors following the company’s sale of its oncology business to Servier.
KEY UPCOMING MILESTONES

Initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassemia, by year-end.
Initiate Phase 2/3 study of mitapivat in sickle cell disease by year-end.
Host investor day in fourth quarter to share more information about commercial launch planning for mitapivat in PK deficiency and research and development pipeline.
Data Presentations

Submit data from ongoing collaborator-led clinical studies of mitapivat in sickle cell disease for presentation at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, to be held Dec. 11-14.
Submit data from the Phase 1 healthy volunteer study of AG-946, the company’s next-generation PKR activator, for presentation at the 63rd ASH (Free ASH Whitepaper) Annual Meeting & Exposition.
SECOND QUARTER 2021 FINANCIAL RESULTS

The financial results discussion compares Agios’ continuing operations. All periods have been adjusted to exclude discontinued operations related to the divested oncology business.

Research and Development (R&D) Expenses: R&D expenses for continuing operations were $62.0 million for the second quarter of 2021 compared to $54.1 million for the second quarter of 2020. The year-over-year increase in R&D was driven primarily by start-up costs associated with the Phase 3 studies of mitapivat in thalassemia and sickle cell disease, as well as the NDA and MAA filings for mitapivat in PK deficiency and launch preparation activities.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses for continuing operations were $29.2 million for both the second quarter of 2021 and the second quarter of 2020.

Non-Operating Income: Non-operating income included approximately $2.0 million from TIBSOVO (ivosidenib) royalties for the second quarter of 2021.

Net Loss: Net loss was $86.2 million for the second quarter of 2021 compared to a net loss of $90.5 million for the second quarter of 2020.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2021, were $1.7 billion compared to $794.4 million as of June 30, 2020. The company expects that its cash, cash equivalents and marketable securities as of June 30, 2021 will enable the company to execute its operating plan through major catalysts and to cash-flow positivity without the need to raise additional equity.

CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2021 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 2663508. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.