On May 1, 2024 Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") and Poseida Therapeutics, Inc. (NASDAQ: PSTX, President and CEO: Kristin Yarema, "Poseida") reported that Xyphos Biosciences, Inc., (a wholly owned subsidiary of Astellas, "Xyphos") and Poseida have entered into a research collaboration and license agreement to develop novel convertibleCAR programs by combining the innovative cell therapy platforms from each of the companies (Press release, Astellas, MAY 1, 2024, View Source [SID1234642506]).

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Poseida is advancing differentiated cell and gene therapies with the capacity to cure certain cancers and rare diseases. In oncology, its pipeline includes allogeneic CAR-T cell therapy product candidates for both solid and liquid tumors that address patient populations with high unmet medical need. Xyphos utilizes a novel and proprietary ACCELTM technology*1 platform that uses its convertibleCAR (convertible Chimeric Antigen Receptor)*1 in combination with proprietary MicAbodies*1 to target tumor cells.

Under the terms of the agreement, the companies plan to combine Poseida’s proprietary allogeneic CAR-T platform with Xyphos’ ACCELTM technology to create one Poseida-developed CAR-T construct to form the basis of two convertibleCAR product candidates targeting solid tumors. Xyphos will reimburse Poseida for costs incurred as part of the research agreement and will be responsible for the development and future commercialization of products generated from the collaboration. Poseida will receive US $50 million upfront plus potential development and sales milestones and contingency payments of up to US $550 million in total. Additionally, Poseida is eligible for up to low double digit tiered royalties as a percentage of net sales.

Kristin Yarema, Ph.D., President and CEO of Poseida
"We are excited to expand our relationship with Astellas, where we share a vision that cutting edge, off the shelf cell therapies can address significant unmet needs of patients with solid tumor malignancies. Today’s agreement further reinforces the economic value of Poseida’s highly differentiated non-viral technologies and enables development in areas beyond our core pipeline focus. It also highlights Poseida’s role as the partner of choice in allogeneic CAR-T."

Adam Pearson, Chief Strategy Officer (CStO) of Astellas
"At Astellas, we have a strong commitment to developing novel treatments for patients with cancer and have positioned Immuno-Oncology as a Primary Focus of our R&D strategy*2. By leveraging our extensive expertise, experience in cancer biology and unique technologies, we are focused on reinvigorating the immune system’s ability to discover, disarm and destroy cancers in more patients. By combining the ACCELTM platform with Poseida’s elegant and cutting-edge genetic editing platforms, we believe the collaboration will bring synergies between the two companies’ breakthrough research and will ultimately lead to expansion of Astellas’ portfolio and to delivery of innovative CAR-T cell therapies to cancer patients."

In August 2023, Astellas and Poseida announced a strategic investment by Astellas to support Poseida’s commitment to redefining cancer cell therapy.

*1 ACCELTM technology and convertibleCAR: ACCELTM technology is based on a synthetic biology approach that utilizes the binding of an engineered protein ligand to an orthogonal engineered receptor which forms the extracellular domain of a convertible CAR (chimeric antigen receptor). The convertibleCAR is targeted to tumor cells with a tumor-associated antigen-specific engineered antibody-like molecule (MicAbody) containing the engineered ligand. For more information, please visit View Source

*2: Astellas has established a Focus Area Approach for its research and development strategy. For more information, please visit our website at Areas of Interest | Astellas Pharma Inc.

On May 1, 2024 Alkermes plc (Nasdaq: ALKS) reported financial results for the first quarter of 2024 (Press release, Alkermes, MAY 1, 2024, View Source [SID1234642504]).

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"The first quarter of 2024 marks our first full quarter as a profitable, pure-play neuroscience company. During the quarter, we continued to advance our strategic priorities across the business, highlighted by solid underlying prescription growth for LYBALVI and advancement of ALKS 2680, our novel, investigational, oral orexin 2 receptor (OX2R) agonist in development as a once-daily treatment for narcolepsy," said Richard Pops, Chief Executive Officer of Alkermes. "For ALKS 2680, we recently initiated our Vibrance-1 phase 2 study in narcolepsy type 1 and announced positive topline phase 1b results in narcolepsy type 2. With these new data now in hand, we plan to initiate a phase 2 study in narcolepsy type 2 in the second half of 2024. In an area where there remains significant unmet patient need, orexin 2 biology represents an important new potential approach to treating disorders characterized by excessive daytime sleepiness. ALKS 2680 is the first candidate from our orexin portfolio to advance in the clinic and we plan to share details regarding our other orexin development programs later this year."

Key Financial Highlights

Revenues

(In millions)

Three Months Ended March 31,

2024

2023

Total Revenues

$

350.4

$

287.6

Total Proprietary Net Sales

$

233.5

$

214.7

VIVITROL

$

97.7

$

96.7

ARISTADA[i]

$

78.9

$

80.1

LYBALVI

$

57.0

$

38.0

Profitability

(In millions)

Three Months Ended March 31,

2024

2023

GAAP Net Income (Loss) From Continuing Operations

$

38.9

$

(12.1)

GAAP Net Loss From Discontinued Operations

$

(2.1)

$

(29.8)

GAAP Net Income (Loss)

$

36.8

$

(41.8)

Non-GAAP Net Income From Continuing Operations

$

76.2

$

30.1

Non-GAAP Net Loss From Discontinued Operations

$

(2.1)

$

(27.6)

Non-GAAP Net Income

$

74.1

$

2.4

EBITDA From Continuing Operations

$

51.5

$

7.2

EBITDA From Discontinued Operations

$

(2.5)

$

(36.0)

EBITDA

$

49.0

$

(28.8)

1

Revenue Highlights

LYBALVI

•
Revenues for the quarter were $57.0 million.
•
Revenues and total prescriptions for the quarter grew 50% and 56%, respectively, compared to the first quarter of 2023.
•
Inventory in the channel decreased by approximately $2.3 million during the quarter.
ARISTADAi

•
Revenues for the quarter were $78.9 million.
•
Inventory in the channel decreased by approximately $3.6 million during the quarter.
VIVITROL

•
Revenues for the quarter were $97.7 million.
•
Inventory in the channel decreased by approximately $4.3 million during the quarter.
Manufacturing & Royalty Revenues

•
Royalty revenues from INVEGA SUSTENNA/XEPLION, INVEGA TRINZA/TREVICTA and INVEGA HAFYERA/BYANNLI for the quarter were $62.7 million.
•
VUMERITY manufacturing and royalty revenues for the quarter were $31.3 million.

Key Operating Expenses

Please see Note 1 below for details regarding discontinued operations.

(In millions)

Three Months Ended March 31,

2024

2023

R&D Expense – Continuing Operations

$

67.6

$

63.8

R&D Expense – Discontinued Operations

$

2.5

$

29.9

SG&A Expense – Continuing Operations

$

179.7

$

167.8

SG&A Expense – Discontinued Operations

$

—

$

6.6

•
Year-over-year increase in R&D expense related to continuing operations was driven primarily by investment in the ALKS 2680 development program and approximately $3.2 million of non-recurring share-based compensation expenses.
•
Year-over-year increase in SG&A expense related to continuing operations was driven primarily by investment in the LYBALVI direct-to-consumer advertising campaign and approximately $6.2 million of non-recurring share-based compensation expenses.

Balance Sheet

At March 31, 2024, the company recorded cash, cash equivalents and total investments of $807.8 million, compared to $813.4 million at Dec. 31, 2023. The company’s total debt outstanding as of March 31, 2024 was $290.1 million.

Financial Expectations for 2024

Alkermes reiterates its financial expectations for 2024, as set forth in its press release dated Feb. 15, 2024.

2

Recent Events

•
In March 2024, the company announced the appointment of new independent director Nancy S. Lurker to the company’s board of directors.
•
In April 2024, the company presented data from its long-term safety study of LYBALVI (olanzapine and samidorphan) at the 2024 Congress of the Schizophrenia International Research Society (SIRS).
•
In April 2024, the company announced positive topline results from the narcolepsy type 2 and idiopathic hypersomnia cohorts in its phase 1b study of ALKS 2680, the company’s novel, investigational orexin 2 receptor (OX2R) agonist in development as a once-daily treatment for narcolepsy.
•
In April 2024, the company announced initiation of the Vibrance-1 phase 2 study of ALKS 2680 in patients with narcolepsy type 1.

Notes and Explanations

1.
The company determined that upon the separation of its oncology business, completed on Nov. 15, 2023, the oncology business met the criteria for discontinued operations in accordance with Financial Accounting Standards Board Accounting Standards Codification 205, Discontinued Operations. Accordingly, the accompanying selected financial information has been updated to present the results of the oncology business as discontinued operations for the three months ended March 31, 2023.

Conference Call

Alkermes will host a conference call and webcast presentation with accompanying slides at 8:00 a.m. ET (1:00 p.m. BST) on Wednesday, May 1, 2024, to discuss these financial results and provide an update on the company. The webcast may be accessed on the Investors section of Alkermes’ website at www.alkermes.com. The conference call may be accessed by dialing +1 877 407 2988 for U.S. callers and +1 201 389 0923 for international callers. In addition, a replay of the conference call may be accessed by visiting Alkermes’ website.

On May 1, 2024 Alkermes plc (Nasdaq: ALKS) reported financial results for the first quarter of 2024 (Press release, Alkermes, MAY 1, 2024, View Source [SID1234642503]).

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"The first quarter of 2024 marks our first full quarter as a profitable, pure-play neuroscience company. During the quarter, we continued to advance our strategic priorities across the business, highlighted by solid underlying prescription growth for LYBALVI and advancement of ALKS 2680, our novel, investigational, oral orexin 2 receptor (OX2R) agonist in development as a once-daily treatment for narcolepsy," said Richard Pops, Chief Executive Officer of Alkermes. "For ALKS 2680, we recently initiated our Vibrance-1 phase 2 study in narcolepsy type 1 and announced positive topline phase 1b results in narcolepsy type 2. With these new data now in hand, we plan to initiate a phase 2 study in narcolepsy type 2 in the second half of 2024. In an area where there remains significant unmet patient need, orexin 2 biology represents an important new potential approach to treating disorders characterized by excessive daytime sleepiness. ALKS 2680 is the first candidate from our orexin portfolio to advance in the clinic and we plan to share details regarding our other orexin development programs later this year."

Key Financial Highlights

Revenues

(In millions)

Three Months Ended
March 31,

2024

2023

Total Revenues

$

350.4

$

287.6

Total Proprietary Net Sales

$

233.5

$

214.7

VIVITROL

$

97.7

$

96.7

ARISTADAi

$

78.9

$

80.1

LYBALVI

$

57.0

$

38.0

Profitability

(In millions)

Three Months Ended
March 31,

2024

2023

GAAP Net Income (Loss) From Continuing Operations

$

38.9

$

(12.1)

GAAP Net Loss From Discontinued Operations

$

(2.1)

$

(29.8)

GAAP Net Income (Loss)

$

36.8

$

(41.8)

Non-GAAP Net Income From Continuing Operations

$

76.2

$

30.1

Non-GAAP Net Loss From Discontinued Operations

$

(2.1)

$

(27.6)

Non-GAAP Net Income

$

74.1

$

2.4

EBITDA From Continuing Operations

$

51.5

$

7.2

EBITDA From Discontinued Operations

$

(2.5)

$

(36.0)

EBITDA

$

49.0

$

(28.8)

Revenue Highlights

LYBALVI

Revenues for the quarter were $57.0 million.
Revenues and total prescriptions for the quarter grew 50% and 56%, respectively, compared to the first quarter of 2023.
Inventory in the channel decreased by approximately $2.3 million during the quarter.
ARISTADAi

Revenues for the quarter were $78.9 million.
Inventory in the channel decreased by approximately $3.6 million during the quarter.
VIVITROL

Revenues for the quarter were $97.7 million.
Inventory in the channel decreased by approximately $4.3 million during the quarter.
Manufacturing & Royalty Revenues

Royalty revenues from INVEGA SUSTENNA/XEPLION, INVEGA TRINZA/TREVICTA and INVEGA HAFYERA/BYANNLI for the quarter were $62.7 million.
VUMERITY manufacturing and royalty revenues for the quarter were $31.3 million.
Key Operating Expenses

Please see Note 1 below for details regarding discontinued operations.

(In millions)

Three Months Ended
March 31,

2024

2023

R&D Expense – Continuing Operations

$

67.6

$

63.8

R&D Expense – Discontinued Operations

$

2.5

$

29.9

SG&A Expense – Continuing Operations

$

179.7

$

167.8

SG&A Expense – Discontinued Operations

$

–

$

6.6

Year-over-year increase in R&D expense related to continuing operations was driven primarily by investment in the ALKS 2680 development program and approximately $3.2 million of non-recurring share-based compensation expenses.
Year-over-year increase in SG&A expense related to continuing operations was driven primarily by investment in the LYBALVI direct-to-consumer advertising campaign and approximately $6.2 million of non-recurring share-based compensation expenses.
Balance Sheet

At March 31, 2024, the company recorded cash, cash equivalents and total investments of $807.8 million, compared to $813.4 million at Dec. 31, 2023. The company’s total debt outstanding as of March 31, 2024 was $290.1 million.

Financial Expectations for 2024

Alkermes reiterates its financial expectations for 2024, as set forth in its press release dated Feb. 15, 2024.

Recent Events

In March 2024, the company announced the appointment of new independent director Nancy S. Lurker to the company’s board of directors.
In April 2024, the company presented data from its long-term safety study of LYBALVI (olanzapine and samidorphan) at the 2024 Congress of the Schizophrenia International Research Society (SIRS).
In April 2024, the company announced positive topline results from the narcolepsy type 2 and idiopathic hypersomnia cohorts in its phase 1b study of ALKS 2680, the company’s novel, investigational orexin 2 receptor (OX2R) agonist in development as a once-daily treatment for narcolepsy.
In April 2024, the company announced initiation of the Vibrance-1 phase 2 study of ALKS 2680 in patients with narcolepsy type 1.
Notes and Explanations

1. The company determined that upon the separation of its oncology business, completed on Nov. 15, 2023, the oncology business met the criteria for discontinued operations in accordance with Financial Accounting Standards Board Accounting Standards Codification 205, Discontinued Operations. Accordingly, the accompanying selected financial information has been updated to present the results of the oncology business as discontinued operations for the three months ended March 31, 2023.

Conference Call
Alkermes will host a conference call and webcast presentation with accompanying slides at 8:00 a.m. ET (1:00 p.m. BST) on Wednesday, May 1, 2024, to discuss these financial results and provide an update on the company. The webcast may be accessed on the Investors section of Alkermes’ website at www.alkermes.com. The conference call may be accessed by dialing +1 877 407 2988 for U.S. callers and +1 201 389 0923 for international callers. In addition, a replay of the conference call may be accessed by visiting Alkermes’ website.

On May 1, 2024 Akari Therapeutics, Plc (Nasdaq: AKTX, "Akari") and Peak Bio Inc. (OTC: PKBO, "Peak") reported completion of a joint portfolio prioritization review pursuant to which the combined entity, following completion of the previously announced merger of Akari and Peak, will focus on Peak’s ADC (antibody drug conjugate) platform technology and Akari’s PAS-nomacopan Geographic Atrophy (GA) program (Press release, Akari Therapeutics, MAY 1, 2024, View Source [SID1234642502]). Peak’s ADC platform technology is a proprietary technology using antibody plus linker plus Peak Bio toxin with immune modulation and includes a novel pre-clinical ADC candidate targeting TROP-2. Akari’s PAS-nomacopan is a bispecific complement and leukotriene B4 inhibitor with prolonged duration of action being developed for GA.

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"As part of the merger process, the boards of each company collaboratively decided on this portfolio prioritization plan. The boards believe by prioritizing the ADC and PAS-nomacopan GA programs the post-closing combined company will be in the best position to secure business development opportunities and funding while continuing to advance these important programs," said Hoyoung Huh, M.D., Ph.D. Chairman, Peak Bio, and Ray Prudo, M.D. Chairman, Akari Therapeutics.

Key Elements of Program Prioritization

ADC toolkit (proprietary technology using antibody plus linker plus Peak Bio toxin with immune modulation)

•
Oncology platform includes a novel pre-clinical ADC candidate targeting TROP-2
•
Strategy includes continued IND enabling development

•
Strategy includes developing and progressing our novel toxin(s) including our lead toxin, PH-1 (Thailanstatin) which can be used in multiple important cancer targets including our TROP-2 candidate
•
Ongoing and continued business development activities with significant market interest in ADC candidates
PAS-nomacopan for Geographic Atrophy

•
Complement only inhibitors recently approved for treatment of GA
•
PAS-nomacopan has been shown in animal models to have prolonged biologic residence in the eye, suggesting ability for q2 to q4 month dosing, a potentially important competitive advantage
•
PAS-nomacopan, with its unique, non-pegylated bispecific inhibition of leukotriene B4 and complement may offer safety and/or efficacy advantages over traditional complement inhibitors
•
Continued IND enabling development including full scale manufacturing of drug for clinical use with a pre-IND meeting expected 1H 2024
Programs for Further Evaluation

HSCT-TMA

•
Akari’s HSCT-TMA program will be suspended, with enrollment in its currently active pediatric clinical study discontinued due to cost and timeline
•
The post-closing combined company will work closely with FDA to define the best path forward for this technology
•
Based upon cost and timeline. will consider the opportunity for partnership and licensing, specifically as it relates to the Priority Review Voucher (PRV)
PHP 303 (5th generation Neutrophil Elastase Inhibitor)

•
Peak Bio’s Phase II ready PHP 303 program for Alpha 1 anti trypsin deficiency (AATD), a rare orphan condition, will be discontinued

•
The post-closing combined company will continue ongoing business development discussions around this technology
Nomacopan

•
Continue to seek partnership opportunities for licensing, proof of concept studies, and clinical trials
About the Merger

On March 5, 2024, Akari and Peak Bio announced a definitive agreement to merge as equals in an all-stock transaction. The combined entity will operate as Akari Therapeutics, Plc, which is expected to continue to be listed and trade on the Nasdaq Capital Market as AKTX, under the Chairmanship of Hoyoung Huh, MD, PhD. Under the terms of the agreement, Peak stockholders will receive a number of Akari ordinary shares (represented by American Depositary Shares) for each share of Peak stock they own, as determined on the basis of the exchange ratio described in the agreement. The exchange is expected to result in implied equity ownership in the combined company of approximately 50% for Akari shareholders and approximately 50% for Peak stockholders on a fully diluted basis, subject to adjustment under certain circumstances, including based on each party’s relative level of net cash at the closing of the proposed transaction. The transaction is expected to close by the third quarter of this year subject to the satisfaction of customary closing conditions, including approval by the shareholders of both companies.

On May 01, 2024 Aileron Therapeutics, Inc. ("Aileron") (NASDAQ: ALRN), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, reported the pricing of an underwritten registered direct offering priced at-the-market under Nasdaq rules of 4,273,505 shares of its common stock and accompanying warrants to purchase an aggregate of 4,273,505 shares of common stock (Press release, Aileron Therapeutics, MAY 1, 2024, View Source [SID1234642501]). Each share of common stock and accompanying warrant are being sold together at a combined public offering price of $4.68. The aggregate gross proceeds of the offering are expected to be approximately $20 million, before deducting underwriting discounts and commissions and other offering expenses payable by Aileron, and excluding any proceeds that may be received from exercise of the warrants. The offering is expected to close on or about May 3, 2024, subject to the satisfaction of customary closing conditions.

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The accompanying warrants will have an exercise price of $4.68 per share, will be exercisable immediately and will expire three years from the date of issuance. Aileron may call the warrants for cancellation during the ten trading day period after the date that is thirty (30) days following the public announcement by Aileron of the top-line results from the Phase 1b clinical trial of LTI-03 in patients with idiopathic pulmonary fibrosis, which announcement includes a statement that there were no drug-related adverse events that resulted in a discontinuation of the trial; provided that Aileron may only deliver such call notice if the volume-weighted average price of its shares of common stock exceeds the exercise price of the warrants on the trading day immediately prior to the date it delivers the call notice. Any warrant subject to such call for which a notice of exercise is not received will be cancelled ten trading days after the date of the call notice for consideration equal to $0.001 per warrant share.

Titan Partners Group, a division of American Capital Partners, is acting as sole book-running manager for the offering.

The securities were offered pursuant to a shelf registration statement on Form S-3 (File No. 333-265470) that was previously filed with and declared effective by the Securities and Exchange Commission (SEC) on June 16, 2022. The offering was made only by means of a prospectus supplement and the accompanying prospectus that form a part of the registration statement. A final prospectus supplement relating to the offering will be filed with the SEC and may be obtained for free by visiting the SEC’s website at www.sec.gov. When available, copies of the final prospectus supplement and the accompanying prospectus may also be obtained for free by contacting Titan Partners Group, LLC, a division of American Capital Partners, LLC, 4 World Trade Center, 29th Floor, New York, NY 10007, by phone at (929) 833-1246 or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities, in any state or jurisdiction in which such offer, solicitation or sale would be unlawful, prior to registration or qualification under the securities laws of any such state or jurisdiction.