On May 8, 2023 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported financial results for the first quarter ended March 31, 2023 and provided business highlights (Press release, Akebia, MAY 8, 2023, View Source [SID1234631126]).

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"Approval of Vafseo (vadadustat) by the European Commission is a significant milestone for us and for dialysis patients in Europe with anemia due to chronic kidney disease," said John P. Butler, Chief Executive Officer of Akebia. "This is an important step as we continue to deliver on our commitment to better the lives of people with kidney disease. Now we are working to complete a partnership to launch Vafseo in Europe, and we are eager to conclude our appeal process with the FDA."

In April, the European Commission granted marketing authorization for Vafseo (vadadustat), for the treatment of symptomatic anemia associated with chronic kidney disease (CKD) in adults on chronic maintenance dialysis. Akebia plans to select a partner to commercialize Vafseo in Europe. Vadadustat is now approved in 32 countries. Akebia also expects a regulatory opinion on vadadustat in the United Kingdom, Switzerland and Australia over the course of this year.

In the U.S., Akebia has continued to engage with the Office of New Drugs on its Formal Dispute Resolution regarding vadadustat. Dr. Stein, the deciding authority on the appeal, indicated he has completed his internal discussions, and the company expects a response within the next 30 days.

Akebia also recently reported positive top-line results from FO2CUS, a study evaluating the efficacy and safety of vadadustat in hemodialysis patients who were converted from a long-acting erythropoiesis-stimulating agent (ESA) to three times weekly oral vadadustat dosing for the maintenance treatment of anemia. The data demonstrated that vadadustat met the primary and secondary efficacy endpoints and was non-inferior to an ESA in the treatment of anemia due to chronic kidney disease in patients on hemodialysis when used three times a week at the time of dialysis and with a comparable safety profile to the current standard of care.

"Our team has been diligent in efforts to manage spend and sustain a reduction in operating costs," said David A. Spellman, Chief Financial Officer of Akebia. "Auryxia revenue was impacted by a $5 million reduction in the volume of channel inventory from year end. We believe quarterly fluctuations in our revenue will continue, which do not impact revenue guidance of $175-$180 million. Our team is delivering operating expense savings, with an almost 50% reduction versus the first quarter of 2022, and almost 30% reduction from the fourth quarter of 2022. We continue to execute strategic decisions to enable us to operate with cash on hand and Auryxia revenue for at least the next twelve months."

Financial Results

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Revenues: Total revenue was $40.1 million in the first quarter of 2023 compared to $61.7 million for the first quarter of 2022.

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Net product revenue was $34.8 million in the first quarter of 2023 compared to $41.4 million in the first quarter of 2022, a 15.9% decrease; and compared with $49.7 million in the fourth quarter of 2022, a 30.0% decrease. The decrease compared to the first quarter of 2022 was primarily due to a reduction in inventory of Auryxia by certain customers, as well as a decline in volume, partially offset by higher net price per tablet. The decrease compared to the fourth quarter of 2022 is primarily due to the previously discussed year-end inventory build up by a customer at the end of 2022, which has now been reduced significantly.

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License, collaboration, and other revenue was $5.3 million for the first quarter of 2023 compared to $20.3 million for the first quarter of 2022. The decrease was primarily related to a reduction in revenue from the termination of the U.S. and international collaboration agreements between Akebia and Otsuka in the second quarter of 2022.

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COGS: Cost of goods sold was $19.5 million for the first quarter of 2023 compared to $31.3 million for the first quarter of 2022. The decrease was primarily due to lower excess and obsolescence reserves associated with Auryxia, lower manufacturing costs associated with the supply of Vafseo to Mitsubishi Tanabe Pharma Corporation for commercial sale in Japan, and lower freight costs as a result of a lower volume of shipments. The company continues to carry a non-cash intangible amortization charge of $9.0 million per quarter through the fourth quarter of 2024.

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R&D Expenses: Research and development expenses were $19.7 million for the first quarter of 2023 compared to $43.8 million for the first quarter of 2022. The decrease was primarily due to decreased headcount related costs as a result of the April 2022 reduction in force, decreased outsourced contract services, decreased clinical trial costs, and development expenses related to vadadustat.

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SG&A Expenses: Selling, general and administrative expenses were $25.2 million for the first quarter of 2023 compared to $44.3 million for the first quarter of 2022. The decrease was primarily due to decreased headcount related costs as a result of the 2022 reductions in force and lower marketing expenses following receipt of the complete response letter for vadadustat from the U.S. Food and Drug Administration (FDA).

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Net Loss: Net loss was $26.2 million for the first quarter of 2023 compared to $62.4 million for the first quarter of 2022. The decrease in net loss was due primarily to lower cost of goods sold and lower operating expenses, partially offset by lower revenues.

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Cash Position: Cash and cash equivalents as of March 31, 2023 were approximately $57.0 million. Akebia believes that its cash resources will be sufficient to fund its current operating plan for at least the next twelve months. Akebia’s objective is to fund its current operating plan with existing cash resources and cash from operations for at least the next twelve months. Future decisions by the FDA or other regulatory agencies related to the potential regulatory approval of vadadustat, or Akebia’s ability to generate additional value from vadadustat through partnerships or other transactions may potentially further extend our cash runway, but are not currently reflected in the operating plan. Akebia also plans to continue to work on initiatives to extend its revenues from Auryxia beyond anticipated loss of exclusivity in March 2025.

Conference Call

Akebia will host a conference call on Monday, May 8 at 8:30 a.m. ET to discuss its financial results and recent business highlights. To access the call, please register by clicking on this Registration Link, and then you will be provided with dial in details. To avoid delays, we encourage dialing into the conference call fifteen minutes ahead of the scheduled start time.

A live webcast of the conference call will be available via the Investors section of Akebia’s website at View Source." target="_blank" title="View Source." rel="nofollow">View Source An online archive of the webcast can be accessed via the Investors section of Akebia’s website at View Source approximately two hours after the event.

On May 8, 2023 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the first quarter of 2023 and provided a corporate update (Press release, Aclaris Therapeutics, MAY 8, 2023, View Source [SID1234631125]).

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"The first quarter of 2023 represented another period of continued progress advancing our clinical stage development programs toward important data milestones," stated Doug Manion, M.D., Chief Executive Officer of Aclaris. "As we continue to move toward data catalysts for zunsemetinib in rheumatoid arthritis and psoriatic arthritis as well as ATI-1777 in atopic dermatitis, we also are making positive progress towards bringing our next potentially broadly applicable candidate, ATI-2138, into its first proof of concept trial in ulcerative colitis."

Continued Dr. Manion, "Regarding our proof-of-concept trial of zunsemetinib in hidradenitis suppurativa, which we reported in March, while we did not see positive efficacy results in this particularly challenging disease, we were able to strengthen our safety database and demonstrate mechanistically that our potentially first-in-class MK2 inhibitor performed as expected."

Research and Development Highlights:

Clinical Development Programs:

Zunsemetinib, an investigational oral small molecule MK2 inhibitor:
Currently being developed as a potential treatment for immuno-inflammatory diseases

Rheumatoid Arthritis (ATI-450-RA-202): This Phase 2b dose ranging trial to investigate the efficacy, safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of multiple doses (20 mg and 50 mg twice daily) of zunsemetinib in combination with methotrexate in subjects with moderate to severe rheumatoid arthritis (RA) is ongoing. Based on the continued positive enrollment momentum, Aclaris is narrowing its timing guidance for topline data to the fourth quarter of 2023.

Psoriatic Arthritis (ATI-450-PsA-201): This Phase 2a trial to investigate the efficacy, safety, tolerability, PK and PD of zunsemetinib (50 mg twice daily) in subjects with moderate to severe psoriatic arthritis (PsA) is ongoing. Based on a slower than anticipated study start up in Europe, the trial enrollment has taken longer than expected. Based on current enrollment trends and momentum, particularly in Poland, Aclaris now expects topline data in the first half of 2024, rather than year end 2023.
ATI-1777, an investigational topical "soft" Janus kinase (JAK) 1/3 inhibitor:
Currently being developing as a potential treatment for mild to severe atopic dermatitis (AD)

Atopic Dermatitis (ATI-1777-AD-202): This Phase 2b trial to determine the efficacy, safety, tolerability, and PK of multiple doses and application regimens of ATI-1777 in subjects with mild to severe AD is ongoing. In April 2023, Aclaris modified the protocol to expand the inclusion criteria for the trial to enroll patients with milder disease to broaden the drug’s target indication potential and to further aide enrollment which was challenged by an unexpected milder winter season. As a result, Aclaris currently projects topline data in the second half of 2023, rather than mid-year 2023.
ATI-2138, an investigational oral covalent ITK/JAK3 inhibitor:
Currently being developed as a potential treatment for T cell-mediated autoimmune diseases

Aclaris has selected ulcerative colitis as the intended first clinical development target for ATI-2138. Aclaris is also exploring additional indications that are relevant to the mechanism of action.

Healthy Volunteers (ATI-2138-PKPD-102): This Phase 1 MAD (multiple ascending dose) trial to investigate the safety, tolerability, PK and PD of ATI-2138 in healthy volunteers is ongoing. Aclaris continues to expect topline data in the second half of 2023.
Preclinical Development Program

ATI-2231, an investigational oral MK2 inhibitor compound:
Currently being explored as a potential treatment for pancreatic cancer and metastatic breast cancer as well as in preventing bone loss in patients with metastatic breast cancer

Second MK2 inhibitor generated from Aclaris’ proprietary KINect drug discovery platform and designed to have a long plasma half-life.

Aclaris expects clinical development activities to be initiated in 2023, which is expected to advance as a collaboration with an academic third party.
Financial Highlights:

Liquidity and Capital Resources

As of March 31, 2023, Aclaris had aggregate cash, cash equivalents and marketable securities of $204.4 million compared to $229.8 million as of December 31, 2022.

Additionally, in March 2023, Aclaris issued a placement notice to sell approximately 3.4 million shares under its ATM facility for aggregate net proceeds of $26.7 million. This transaction closed in April 2023.

Aclaris continues to anticipate that its cash, cash equivalents and marketable securities as of March 31, 2023 in combination with the $26.7 million in net proceeds from sales under the ATM facility subsequent to quarter end, will be sufficient to fund its operations through the end of 2025, without giving effect to any potential business development transactions or additional financing activities.

Financial Results

First Quarter 2023

Net loss was $28.2 million for the first quarter of 2023 compared to $18.8 million for the first quarter of 2022.
Total revenue was $2.5 million for the first quarter of 2023 compared to $1.5 million for the first quarter of 2022. The increase was driven by higher licensing revenue primarily from royalties earned on out-licensed intellectual property in the first quarter of 2023.
Research and development (R&D) expenses were $22.6 million for the quarter ended March 31, 2023 compared to $14.3 million for the prior year period.
The $8.3 million increase was primarily the result of higher:
Zunsemetinib development expenses related to drug candidate manufacturing and costs associated with clinical activities for a Phase 2b trial for RA.
ATI-1777 development expenses related to costs associated with a Phase 2b clinical trial for AD.
ATI-2138 development expenses, including costs associated with a Phase 1 MAD trial and other preclinical activities.
Compensation-related expenses due to an increase in headcount.
General and administrative (G&A) expenses were $8.8 million for the quarter ended March 31, 2023 compared to $6.1 million for the prior year period. The increase was primarily due to an increase in compensation-related expenses due to an increase in headcount.
Licensing expenses were $1.1 million for the quarter ended March 31, 2023 resulting from separate third-party contractual obligations related to the non-exclusive patent license agreement with Lilly. There were no licensing expenses for the quarter ended March 31, 2022.
Revaluation of contingent consideration resulted in a $0.8 million credit for the quarter ended March 31, 2023 compared to a credit of $1.2 million for the prior year period.
Conference Call and Webcast

As previously disclosed on May 2, 2023, management will host a conference call and webcast, with an accompanying slide presentation, at 8:00 AM ET today to provide a corporate update. To access the live webcast of the call and the accompanying slide presentation, please visit the "Events" page of the "Investors" section of Aclaris’ website, www.aclaristx.com. The webcast will be archived for at least 30 days on the Aclaris website.

On May 8, 2023 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that it has entered into a joint development agreement with Bliss Biopharmaceutical (Hangzhou) Co., Ltd. (Headquarters: Zhejiang Province, China, "BlissBio"), for BB-1701, an antibody-drug conjugate (ADC) with option rights for a strategic collaboration (Press release, Eisai, MAY 7, 2023, View Source [SID1234631139]).

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BB-1701 is an ADC that is composed of Eisai’s in-house developed anticancer agent eribulin, and anti-HER2 antibody using a linker, and is expected to have anti-tumor effects on breast, lung and other solid tumors that express HER2. The linker-payload, which uses eribulin as a payload, is a proprietary technology platform developed by Eisai’s U.S. research base Exton Site, and Eisai is investigating the possibilities of using this platform to link to various antibodies. Under a license agreement signed by the two companies in 2018, Eisai has granted BlissBio global exclusive development rights for several ADCs to use eribulin as the payload. Based on the status of the Phase I/II clinical trials of BB-1701 currently being conducted by BlissBio, the both companies have decided to co-develop this drug.

Under the terms of the joint development agreement, Eisai will make upfront and development milestone payments to BlissBio, conduct a Phase II clinical trial in breast cancer, and obtain option rights to develop and commercialize BB-1701 globally, excluding Greater China (China, Hong Kong, Macau, Taiwan). If Eisai exercises the option rights, an additional upfront payment will be made to BlissBio, as well as development and regulatory milestone payments, sales milestone payments and a certain amount of royalties on sales revenue of BB-1701 after the launch. If all development, regulatory and sales milestones are achieved, up to a total of $2 billion USD will be paid.

"BB-1701 is characterized by its payload of eribulin, which is a product of our modern synthetic organic chemistry that has already made contributions to patients with breast cancer and soft tissue sarcoma," said Dr. Takashi Owa, Chief Scientific Officer, Senior Vice President, Eisai Co., Ltd. "Our collaboration with BlissBio will accelerate the development of BB-1701 with the goal of bringing a new treatment option to patients globally."

On May 7, 2023 Independent biopharmaceutical company Specialised Therapeutics Asia (ST) reported that a new therapy to treat rare gastrointestinal stromal tumours (GIST) shown to improve survival has been approved for use in Singapore (Press release, Specialised Therapeutics Asia, MAY 7, 2023, View Source [SID1234631114]).

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The therapy, QINLOCK (ripretinib) is now approved by the Health Sciences Authority (HSA) "for the treatment of adult patients with advanced gastrointestinal stromal tumours (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib, sunitinib, and regorafenib".

Singapore-based senior consultant in medical oncology Dr Richard Quek said QINLOCK represented a major treatment advancement for patients with advanced GIST.

"Since 2013, despite multiple attempts and studies, no therapy was shown to be effective for 4th line GIST patients whose cancers have progressed on existing treatment, until the discovery of QINLOCK," Dr Quek said.

In the pivotal INVICTUS study that led to QINLOCK’s approval, QINLOCK was shown to significantly delay cancer progression.

"This approval in Singapore clearly provides an opportunity for us to improve the outcomes of our GIST patients who are refractory to the current existing treatment."

QINLOCK is an oral medication used to treat GIST in people who have received at least three prior treatments. It belongs to a drug class called tyrosine kinase inhibitors and works by blocking specific tumour proliferation pathways.2

A pivotal Phase 3 clinical trial of QINLOCK – the INVICTUS study – demonstrated that QINLOCK was able to significantly reduce the risk of disease progression by 85% (hazard ratio of 0.15, p<0.0001) with a median progression-free survival of 6.3 months in patients administered QINLOCK, compared to 1.0 month in the placebo arm.1 QINLOCK was associated with clinically meaningful overall survival of 15.1 months vs 6.6 months and reduced the risk of death by 64% (hazard ratio of 0.36). The objective response rate by Blinded Independent Central Review using modified Response Evaluation Criteria in Solid Tumors (RECIST) was 9.4% with QINLOCK vs 0.0% with placebo (p=0.0504).1,3

In addition, in a long-term follow up analysis of the INVICTUS trial, patients in the QINLOCK arm demonstrated a median overall survival of 18.2 months compared to 6.3 months in the placebo arm and reduced the risk of death by 59% (hazard ratio of 0.41).The objective response rate was 11.8% with QINLOCK vs 0.0% with placebo.3

ST Chief Executive Officer Carlo Montagner said the Singapore approval followed the recent approval of QINLOCK in New Zealand, as well as regulatory and reimbursement approval in Australia.

"Achieving these critical regulatory milestones is testament to the dedication of our regulatory teams to make QINLOCK available to all eligible patients in Singapore who are impacted by this rare gastrointestinal cancer."

ST commercialises QINLOCK in Singapore under an exclusive distribution agreement from US based Deciphera Pharmaceuticals.

Further Inquiries can be directed to ST Senior Manager Communications and Corporate Affairs Emma Power on + 65 31589910 [email protected]

About GIST

Gastrointestinal stromal tumour (GIST) is a cancer affecting the digestive tract or nearby structures within the abdomen, most often presenting in the stomach or small intestine. GIST growth usually begins in the connective tissue in the wall of the affected organ and grows outwards. The common location of GIST is in the stomach (50 to 60%) and small intestines (30 to 40%) but can occur in any site in the digestive system. Other possible GIST sites are the oesophagus, rectum, and colon. GIST cases are rare and estimated to cause between 0.1% and 3% of GI cancer. The risk of GIST diagnosis increases with age, with GIST incidence peaking among people in their fifties and sixties.4

About QINLOCK (ripretinib)

QINLOCK is a switch-control tyrosine kinase inhibitor that was engineered to broadly inhibit KIT and PDGFRA mutated kinases by using a dual mechanism of action that regulates the kinase switch pocket and activation loop. QINLOCK inhibits primary and secondary KIT mutations in exons 9, 11, 13, 14, 17, and 18 involved in GIST, as well as the primary exon 17 D816V mutation. QINLOCK also inhibits primary PDGFRA mutations in exons 12, 14, and 18, including the exon 18 D842V mutation, involved in a subset of GIST.

On May 7, 2023 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported that it has entered into an agreement with Bayer AG (Bayer) to supply Illuccix (TLX591-CDx, kit for the preparation of gallium Ga 68 gozetotide injection)[1] for the Phase III ARASTEP study (ClinicalTrials.gov Identifier: NCT05794906) (Press release, Telix Pharmaceuticals, MAY 7, 2023, View Source [SID1234631113]). This global study is investigating the efficacy of Bayer’s androgen receptor inhibitor (ARi) darolutamide plus androgen deprivation therapy (ADT) versus ADT alone in hormone-sensitive prostate cancer, in patients with high-risk biochemical recurrence who have no evidence of metastatic disease by conventional imaging and a positive PSMA-PET/CT[2] at baseline.

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The study will enrol up to 750 patients across various sites such as in Europe, Japan and the United States. The more sensitive PSMA imaging may identify prostate cancer lesions not detectable by conventional imaging such as computed tomography (CT) scans, magnetic resonance imaging (MRI) and bone scans.

Telix Chief Medical Officer, Dr Colin Hayward stated, "We are pleased to supply Bayer and a number of clinical sites in this important study, reflective of Telix’s unique commitment to delivering advanced prostate cancer imaging globally. The use of PSMA-PET/CT in this setting is illustrative of the potential for this imaging modality to move beyond diagnosis to a disease management tool."