On May 1, 2024 Defence Therapeutics Inc. ("Defence" or the "Company"), (CSE: DTC, OTCQB: DTCFF, FSE: DTC), a Canadian biopharmaceutical company developing novel immune-oncology therapeutics and drug delivery technologies, reported the successful completion of a pre-clinical vaccination trial using its ARM-002TM vaccine against pancreatic cancer (Press release, Defence Therapeutics, MAY 1, 2024, View Source;utm_medium=rss&utm_campaign=pancreactic-cancer-succesful-trial-against-pancreatic-cancer-with-defences-arm-002-anti-cancer-vaccine [SID1234642510]). The vaccine was shown to be therapeutically effective against pre-established pancreatic cancer especially when combined with the anti-PD-1 immune-checkpoint inhibitor.

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In the context of an in vivo pre-clinical study, Defence tested its ARM-002TM vaccine pulsed with a pancreatic cancer lysate in combination with the anti-PD-1 immune-checkpoint in animals with pre-established Pan02 tumors. Animals’ follow-up revealed that the vaccine is indeed potent as all treated animals remained alive for over 40 days (equivalent to almost 5 years in the human scale) with tumor growth heavily impaired/blocked compared to other treatments/controls. Since Defence is interested in targeting "hard-to-treat" cancers, these results represent an important infliction point and an incentive to redirect its Phase I trial using the ARM-002TM anti-cancer vaccine to target pancreatic cancer.

Pancreatic cancer begins when uncontrolled cellular growth occurs in the pancreas. It is rarely diagnosed at early stages when the chance of curing the disease is the greatest as it often doesn’t cause symptoms until it metastasises to other organs. This classifies this type of cancer as a "hard-to-treat" cancer as patients at that point face limited treatment options.

Treatment for pancreatic cancer depends on the cancer stage as well as its location. Although the first goal of pancreatic cancer treatment is to get rid of the cancer, this unrealistic option often shifts to improving quality of life and keeping the cancer from growing or causing more harm. As such, treatments available for pancreatic cancer often involve surgery, radiation, chemotherapy or a combination, and are associated with limited clinical benefits or life-related complications. For instance, even if surgery to remove the whole pancreas is performed, patients would then rely on taking medicine their entire life to replace the hormones and enzymes made by the pancreas. Alternatively, chemotherapy (often combined with radiation therapy) can shrink the cancer, but it is often associated with resistance and/or relapse. As these standards of care fail in large set of patients, the next available option relies on immunotherapy, which uses the body’s immune system to kill cancer cells.

"Defence’s goal is to bring our proprietary and innovative immune therapies to the clinical stage for the benefit of the cancer patients. We often say that our Accum platform is highly versatile as it can lead to the development of verticals promoting different products for multiple indications. This also applies to a single product as it can be adapted to various diseases as demonstrated with our ARM-002TM vaccine in vivo pre-clinical studies, which was shown to impair the growth of solid T-cell lymphoma, melanoma and now pancreatic cancer" says Mr. Plouffe, Chief Executive Officer of Defence Therapeutics.

Pancreatic Cancer Market size is estimated to surpass USD 36 Billion by the end of 2036, growing at a CAGR of 18% during the forecast period of 2024-2036. In 2023, the industry size of the pancreatic cancer was over USD 6 Billion. The growth of the market can be attributed to the rising prevalence of cancer cases among people across the world.

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On May 01, 2024 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of masked, conditionally activated biologic therapeutics, reported that it will report first quarter 2024 results and provide an initial CX-904 Phase 1a dose escalation update on Wednesday, May 8, 2024, after the close of U.S. markets. Following the announcement, the Company will host a conference call and webcast at 5:00 p.m. ET / 2:00 p.m. PT (Press release, CytomX Therapeutics, MAY 1, 2024, View Source [SID1234642509]).

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Participants may access the live webcast of the conference call from the Events and Presentations page of CytomX’s website at View Source Participants may register for the conference call here and are advised to do so at least 10 minutes prior to joining the call. An archived replay of the webcast will be available on the Company’s website.

On May 1, 2024 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, reported its results for the quarter ended March 31, 2024 (Press release, Corcept Therapeutics, MAY 1, 2024, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-first-quarter-financial-results-1 [SID1234642507]).

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Financial Results

Revenue of $146.8 million, a 39 percent increase over the same period in 2023
Increase in 2024 revenue guidance to $620 – $650 million, from $600 – $630 million
Net income per common share of $0.25 (diluted), compared to $0.14 in first quarter 2023
Cash and investments of $451.0 million as of March 31, 2024
"There were a record number of new Korlym prescribers in the first quarter and a record number of patients receiving Korlym. Physicians are increasingly aware that hypercortisolism is much more prevalent than was previously assumed, so they are screening more patients for the disorder," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "When Korlym is prescribed, we use the expertise and infrastructure that we have developed and refined over many years to support physicians and patients. This additional care helps create a life-changing impact for patients who receive Korlym treatment."

Corcept’s first quarter 2024 revenue was $146.8 million, compared to $105.7 million in the first quarter of 2023. First quarter operating expenses were $117.3 million, compared to $90.8 million in the first quarter of 2023, due to increased clinical trial activity and expenses to support the expansion of our commercial team. Net income was $27.8 million in the first quarter of 2024 compared to $15.9 million in the same period last year. Cash and investments were $451.0 million at March 31, 2024 compared to $425.4 million at December 31, 2023.

The company increased its 2024 revenue guidance to $620 – $650 million.

Clinical Development

"The results of the open-label phase of our GRACE trial, which we released last week, are compelling," said Dr. Belanoff. "We expect to build on these results in the trial’s randomized withdrawal phase. Results from this portion of the study will be available in the coming weeks. We are on track to submit our NDA for relacorilant in Cushing’s syndrome by the end of this quarter.

"In addition, we have completed enrollment in all our other late-stage studies – GRADIENT, CATALYST, ROSELLA and DAZALS. We expect that these studies will provide potent evidence that cortisol modulation is a powerful therapeutic mechanism with activity in many serious disorders," added Dr. Belanoff. "We expect data from these trials by the end of this year."

Cushing’s Syndrome

GRACE – Phase 3 trial of relacorilant in patients with all etiologies of hypercortisolism – open-label phase demonstrated clinically meaningful and statistically significant improvements in hypertension, hyperglycemia, weight, waist circumference, cognitive impairment and quality of life; results from randomized withdrawal phase expected this quarter
Relacorilant New Drug Application (NDA) – NDA submission for Cushing’s syndrome expected this quarter
GRADIENT – Phase 3 trial of relacorilant in patients with Cushing’s syndrome caused by adrenal adenomas – enrollment completed; results expected in the fourth quarter
CATALYST – Phase 4 trial examining the prevalence of hypercortisolism in patients with difficult-to-control type 2 diabetes; patients with hypercortisolism may enter a randomized, double-blind, placebo-controlled study of Korlym – enrollment completed; preliminary results: approximately 25% of patients enrolled were found to have hypercortisolism; full prevalence phase results expected this quarter and treatment phase results expected by year-end
"Relacorilant has demonstrated tremendous promise as a treatment for patients with Cushing’s syndrome. Patients in GRACE’s open-label phase exhibited significant improvements across a broad range of clinically meaningful endpoints, without significant safety burden. All patients have now completed the randomized withdrawal phase of the study. We plan to present data from both the open-label and randomized withdrawal phases of the study at a medical conference in June," said Bill Guyer, PharmD, Corcept’s Chief Development Officer.

"Our CATALYST trial is the largest and most rigorous study ever conducted to establish the prevalence of hypercortisolism in patients with difficult-to-control diabetes. We believe that CATALYST will be a landmark study to guide physicians as they identify and treat patients with hypercortisolism. We will present final results from the prevalence phase at the American Diabetes Association’s 84th Scientific Sessions in June," said Dr. Guyer.

Oncology

ROSELLA – Pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in patients with platinum-resistant ovarian cancer – enrollment completed; results expected by year-end
Open-label, Phase 1b trial of relacorilant plus pembrolizumab in patients with adrenal cancer with cortisol excess – enrollment completed; results expected by mid-year
Randomized, placebo-controlled, Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer in collaboration with the University of Chicago – enrollment continues
"Fully enrolling ROSELLA takes us a big step closer to addressing the unmet medical need of women with platinum-resistant ovarian cancer. Relacorilant has the potential to become the standard of care for patients with this devastating disease. We expect progression-free survival data, ROSELLA’s primary endpoint, by the end of this year," said Dr. Guyer.

Amyotrophic Lateral Sclerosis (ALS)

DAZALS – Randomized, double-blind, placebo-controlled, Phase 2 trial of dazucorilant in patients with ALS – enrollment completed; results expected by year-end
"Better treatments for ALS are urgently needed. Dazucorilant showed great promise in an animal model of ALS – improving motor performance and reducing neuroinflammation and muscular atrophy. Fully enrolling DAZALS is an important step toward understanding dazucorilant’s potential for significantly improving outcomes for people living with this devastating disease. We expect data by the end of this year," said Dr. Guyer.

Non-alcoholic Steatohepatitis (NASH)

MONARCH – Randomized, double-blind, placebo-controlled, Phase 2b trial of miricorilant in patients with biopsy-confirmed NASH – enrollment continues
"In our Phase 1b study, miricorilant reduced liver fat very rapidly, improved liver health and key metabolic and lipid measures, and was well-tolerated. We look forward to building on these promising results in our MONARCH study," said Dr. Guyer. "This drug has the potential to greatly benefit the millions of patients with NASH."

Conference Call

We will hold a conference call on May 1, 2024, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). Participants must register in advance of the conference call by clicking here. Upon registering, each participant will receive a dial-in number and a unique access PIN. Each access PIN will accommodate one caller. Additionally, a listen-only webcast will be available by clicking here. A replay of the call will be available on the Investors / Events tab of Corcept.com.

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.