On November 5, 2024 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported a business update and provided its cash position and revenue as of September 30, 2024 (Press release, MaaT Pharma, NOV 5, 2024, View Source [SID1234647689]).

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Pipeline highlights

MaaT013

In hemato-oncology:
In September 2024, MaaT Pharma announced that MaaT013 batches were ready for distribution for clinical supply in the US and that MaaT Pharma advanced the readiness phase for the initiation of clinical activities.
In October 2024, MaaT Pharma announced the completion of patient recruitment for ARES, its European Phase 3 clinical trial designed to evaluate the efficacy and safety of its lead asset MaaT013 in the treatment of steroid refractory and ruxolitinib refractory or intolerant acute Graft-versus-Host Disease (aGvHD) (NCT04769895).
MaaT033

In hemato-oncology:
In July 2024, the Company announced that the DSMB completed its first safety assessment of the Phase 2b trial PHOEBUS (NCT05762211) aiming at assessing MaaT033 impact in improving overall survival in patients receiving hematopoietic stem cell transplantation, and recommended continuation of the trial without modification. The trial is a European, multi-center, randomized, double-blind study, testing MaaT033, an oral freeze-dried formulation against placebo, set to be conducted in up to 56 clinical investigation sites and expected to enroll 387 patients.
Cash position1

As of September 30, 2024, total cash and cash equivalents were EUR 27 million, as compared to EUR 31.2 million as of June 30, 2024, and EUR 24.3 million as of December 31, 2023. The net decrease in cash position of EUR 4.2 million during the third quarter 2024 reflects continued investments in R&D activities across the pipeline, offset in part by the payment of a repayable advance of EUR 3.2 million in public funding the development of MaaT033 and the balance of the 2023 R&D tax credit of EUR 1.0 million.
The Company believes it has sufficient cash to cover its current operating needs and development programs into the second quarter of 2025.
Revenues in Q3 20241

MaaT Pharma reported revenues from its Early Access Program of EUR 0.6 million for the quarter ended September 30, 2024, an increase of EUR 0.2 million over the third quarter of 2023. Total revenues for the first nine months of 2024 amounted to EUR 2.3 million compared with EUR 1.8 million for the same period of 20231. This trend is a direct reflection of the continued demand from the medical community for MaaT Pharma’s drug candidate MaaT013.
Management Changes

Eric Soyer was appointed Chief Financial Officer of MaaT Pharma as of November 2024. As CFO, Eric will supervise all corporate functions, investor relations, legal affairs and human resources. Eric is a senior financial executive with extensive experience in the biotech industry with companies listed on Euronext Paris and on the U.S. Nasdaq market. Prior to joining MaaT Pharma, Eric successfully conducted as CFO & COO of Erytech Pharma, a French late-stage drug development company, its Nasdaq IPO and subsequent financial rounds amounting to over EUR 300 million and supervised the scale-up of the company’s operations from a single site based in France to a multi-site company of 240 employees in US and Europe and most recently its strategic merger. Throughout his 30-year career, Eric has developed a solid international experience across a variety of operational settings and will now leverage this experience to assist MaaT Pharma in its next stage of development.
Eric Soyer, CFO of MaaT Pharma stated: "I am thrilled to join the team of MaaT Pharma at this transformational moment for the Company. MaaT Pharma has developed a robust foundation and is now one of the oncology worldwide leaders for microbiome-based therapeutic innovations, as evidenced by the recent announcement of the completion of patient enrollment in its Phase 3 ARES European trial in aGvHD – a significant milestone in the goal of bringing life-saving therapeutic options for patients within this severe indication. I look forward to contributing to MaaT Pharma’s continued success and to fully realize the potential of its therapeutic platform globally."
After 8 years as CFO, Siân Crouzet will become Chief of Staff to fully support a smooth transition of functions to Eric over the coming months. In her tenure, Siân has successfully structured the financial and administrative functions of the Company, building a competent team across the various functions overseen and with strict discipline to support the corporate goals and progressive growth in a highly challenging economic context. Siân was instrumental in the numerous financing operations accomplished since 2016, totaling more than EUR 115 million, including the Company’s successful IPO on Euronext Paris in November 2021.
Siân Crouzet, CoStaff of MaaT Pharma stated: "I am proud of the Company we have built and developed as a team and the numerous accomplishments, not least the recent completion of enrolment of our Phase 3 clinical trial of MaaT013. It has been a real pleasure and honor to serve as CFO over the last 8 years. At this juncture, it is the right time from a professional and personal perspective for me to hand over the reins to take MaaT Pharma to the next stage of its development."
Hervé Affagard, CEO and co-founder of MaaT Pharma added: "I am delighted to welcome Eric Soyer as our new Chief Financial Officer. His extensive experience and strategic financial acumen will be invaluable as we continue to advance our mission to improve survival in patients with few therapeutic options. MaaT Pharma is at a turning point with the preparation of the filing of MaaT013, in Europe, for severe GvHD patients together with our goal to expand in the US. I am confident that Eric will bring the necessary skills to the team to navigate the next chapter of our growth. I also want to take a moment to extend my heartfelt thanks to Siân Crouzet for her invaluable contributions over the past 8 years. She has been instrumental in strengthening our financial foundation and steering us through important phases of development."
Upcoming investor and medical conferences participation

November 6-8, 2024 – 39th SITC (Free SITC Whitepaper) annual meeting in Houston, USA
November 20-22, 2024 – SFGM-TC annual meeting in Toulouse, France
November 25-27, 2024 – Deutsches Eigenkapitalforum annual meeting​​ in Frankfurt, Germany
November 26, 2024 – Investir Day event in Paris, France.
December 5, 2024 – CF&B Midcap Events in Geneva, Switzerland
December 7-10, 2024 – 66th ASH (Free ASH Whitepaper) annual meeting in San Diego, USA, followed by a webinar on the updated dataset from the EAP of MaaT013 in aGvHD.

On November 5, 2024 PharmaMar Group (MSE: PHM) has received a $10 million payment from Janssen Products LP (Janssen), a subsidiary of Johnson & Johnson, upon reaching a commercial milestone established in the licensing agreement for Yondelis (trabectedin) in the United States (Press release, PharmaMar, NOV 5, 2024, View Source [SID1234647674]).

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In August 2019, PharmaMar signed a new licensing agreement with Johnson & Johnson that replaced the 2001 agreement under which Johnson & Johnson reserved the right to exclusively sell and distribute trabectedin in the United States.

Currently, trabectedin is approved in more than 70 countries for the treatment of soft tissue sarcoma and also in some of these countries for ovarian cancer.

On November 5, 2024 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, and ProBioGen, a leading CDMO in biologics, vaccines and viral vectors, reported that the companies have entered into a license agreement for ProBioGen’s AGE1.CR.pIX suspension cell line (Press release, Transgene, NOV 5, 2024, View Source [SID1234647673]). This partnership aims to add value by combining ProBioGen’s specific production technology with the manufacturing capabilities of Transgene’s individualized cancer vaccine program and its myvac platform.

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ProBioGen’s AGE1.CR.pIX suspension cell line showcases innovation and reliability in bioprocessing with a proven track record and is currently in use for multiple late-stage clinical trials. Renowned for its high yield and scalability, this platform enables efficient industrial manufacturing processes, translating into cost-effectiveness and increased productivity in manufacturing. Additionally, the AGE1.CR.pIX cell line offers robust growth and excellent genetic stability to ensure consistent and reliable production.

"We are delighted to embark on this collaborative journey with ProBioGen," said Dr. Alessandro Riva, CEO of Transgene. "The AGE1.CR.pIX suspension cell line represents a significant addition to further expand the manufacturing processes for our individualized cancer vaccines while we work towards providing patients with tailored patient-specific therapies against cancer on a larger scale."

The partnership between Transgene and ProBioGen underscores a shared commitment to advancing the field of individualized medicine and to support manufacturing scalability and optimization.

"We are thrilled to partner with Transgene in advancing the frontier of personalized cancer vaccines," said Dr. Volker Sandig, CSO of ProBioGen. "Our collaboration holds immense potential to impact the personalized vaccine space and to offer new treatments and hope to cancer patients."

On Nov. 04, 2024 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported financial results for the third quarter ended September 30, 2024, and provided a corporate update (Press release, BioCryst Pharmaceuticals, NOV 4, 2024, View Source [SID1234648574]).

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"Our third quarter performance continues to build on the outstanding year we are having, with significant revenue growth, strong patient demand, pipeline advancement and operating profitability in the quarter. As we look ahead, we see robust and durable revenue growth, new opportunities for younger children to benefit from ORLADEYO, and data readouts from BCX17725 in Netherton syndrome and avoralstat in DME, all while moving closer to sustainable profitability," said Jon Stonehouse, president and chief executive officer of BioCryst.

Program Updates

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

"The real-world efficacy and convenience patients and physicians are experiencing with ORLADEYO are driving accelerated commercial momentum that translates to nearly 36 percent revenue growth in our fourth year on the market. Based on this established sales trajectory, and the continued, durable strength of patient demand we see for ORLADEYO, we are more confident than ever of achieving peak sales of $1 billion for ORLADEYO," said Charlie Gayer, chief commercial officer of BioCryst.

ORLADEYO net revenue in the third quarter of 2024 was $116.3 million (+35.7 percent year-over-year (y-o-y)).

Start forms in the U.S. are up 14.2 percent over the past 12 months compared to the prior 12 months; and 67 new U.S. prescribers were added during the third quarter, one of the highest totals over the most recent eight quarters.

The reimbursed product rate in the U.S. increased 0.4 percent in the third quarter to 74.8 percent.

Sales from outside the U.S. contributed 11.4 percent of global ORLADEYO net revenues in the third quarter.

Since launch, approximately half of patients who have started ORLADEYO have switched from another prophylactic therapy. The company has begun the observational Phase 4 APeX-T study, designed to generate real-world data to inform physicians on the best individual approaches to support transition to ORLADEYO.
Rare Disease Pipeline

"The goal with our pipeline is to build on our success with ORLADEYO by bringing the next highly differentiated product to patients living with rare disease. We are making excellent progress, with BCX17725 now in the clinic, avoralstat nearing the clinic and initial patient data from both programs expected next year. These programs would address significant unmet needs for patients with Netherton syndrome and DME, respectively," said Dr. Helen Thackray, chief research and development officer at BioCryst.

The company has advanced BCX17725, its KLK5 inhibitor for the treatment of Netherton syndrome, into clinical trials and expects initial data from the program in 2025.

Netherton syndrome is a serious, rare, lifelong genetic disorder affecting the skin, hair, and immune system, caused by lack of normal function of a natural inhibitor of KLK5. People with Netherton syndrome often have red, scaly, inflamed skin, fragile hair, and are more likely to develop skin infections, allergies, asthma and eczema. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there are no approved treatments for Netherton syndrome.

In 2025, the company plans to advance avoralstat, a plasma kallikrein inhibitor, into a clinical trial of patients with diabetic macular edema (DME).

DME is an important cause of vision loss in diabetes and is due to leakage from the blood vessels in the retina. While current treatments focus on VEGF inhibition, DME can develop from other mechanisms, such as the kallikrein-bradykinin pathway. This is supported by observations that many DME patients have an incomplete response to intravitreal anti-VEGF therapies that are administered every four to eight weeks. Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space as a depot formulation, is designed to provide high dose levels to the retinal vessels with long-lasting exposure, which could result in less frequent injections and a reduced burden on patients and the healthcare system.
Third Quarter 2024 Financial Results

"It is exciting to see continued ORLADEYO growth alongside the significant progress advancing our pipeline, and another quarter of operating profit. BioCryst is in the strongest financial position in its history," said Anthony Doyle, chief financial officer at BioCryst.

For the three months ended September 30, 2024, total revenues were $117.1 million, compared to $86.7 million in the third quarter of 2023 (+35.1 percent y-o-y). The increase was primarily due to $116.3 million in ORLADEYO net revenue in the third quarter of 2024, compared to $85.7 million in ORLADEYO net revenue in the third quarter of 2023 (+35.7 percent y-o-y).

Research and development expenses for the third quarter of 2024 decreased to $41.1 million from $46.9 million in the third quarter of 2023 (-12.4 percent y-o-y), primarily due to decreased investment following the discontinuation of the BCX10013 program, partially offset by investments to advance our early clinical and discovery programs and an increased investment in the APeX-P program in preparation for regulatory filings next year.

Selling, general and administrative expenses for the third quarter of 2024 increased to $65.1 million, compared to $50.6 million in the third quarter of 2023 (+28.7 percent y-o-y). The increase was primarily due to increased commercial investment to support our growing revenue, newly launched regions and expanded international operations. There was also an increase in general and administrative expenses to support commercial growth.

Interest expense was $24.8 million in the third quarter of 2024, compared to $27.3 million in the third quarter of 2023 (-9.2 percent y-o-y). The decrease was primarily due to a decrease in the amortization of interest associated with our royalty financing obligations. Due to the strong cash position of the company, we declined to elect the payment-in-kind (PIK) option related to the Pharmakon term loan for the third quarter of 2024, resulting in a cash interest payment of $10.2 million in the third quarter of 2024 compared to $4.9 million in the third quarter of 2023. The PIK option has now expired. Additionally, the company chose not to execute its option, which expired September 30, 2024, to draw the additional $150 million of debt available to it from Pharmakon.

GAAP operating profit for the third quarter of 2024 was $7.7 million, compared to a GAAP operating loss of $11.9 million for the third quarter of 2023. Non-GAAP operating profit, excluding stock-based compensation expense, was $24.9 million for the third quarter of 2024 compared to $0.4 million for the third quarter of 2023.

Net loss for the third quarter of 2024 was $14.0 million, or $0.07 per share, compared to a net loss of $36.1 million, or $0.19 per share, for the third quarter of 2023.

Cash, cash equivalents, restricted cash and investments totaled $351.7 million at September 30, 2024, compared to $399.2 million at September 30, 2023. Operating cash increased by $13.1 million in the third quarter of 2024.

Financial Outlook for 2024

Based on the strength of patient demand for ORLADEYO seen in the third quarter, and expected continued strength in the fourth quarter, the company is adjusting its outlook for full year 2024 global net ORLADEYO revenue to be between $430 million and $435 million (top end of prior range) and introducing full-year 2024 total revenue guidance of between $443 and $448 million based on additional 2024 RAPIVAB revenue.

Directly related to the revenue strength for both ORLADEYO and RAPIVAB, the company is revising its guidance for operating expenses, and now expects full year 2024 operating expenses to be between $380 million and $390 million. The increase is driven primarily by increased COGS related to new RAPIVAB sales, increased variable costs, including incentive compensation and distribution costs related to the continued strong revenue performance for ORLADEYO and increased expenses that are seasonally booked in Q4 related to our support for the HAE community, including charitable donations. This operating expense outlook does not reflect non-cash stock compensation expense, or one-time expenses related to the previously announced workforce reduction implemented in the first quarter of 2024.

Based on the company’s disciplined approach to capital allocation, and the strong performance of ORLADEYO, the company is confident that it will achieve a full-year operating profit in 2024 (not including non-cash stock compensation), be approaching quarterly positive earnings per share (EPS) and positive cash flow in the second half of 2025 (not including non-cash stock compensation) and be profitable on an EPS basis, with positive cash flow, for full year 2026. The company expects it can achieve these financial milestones without raising additional funds.

On November 4, 2024 BioNTech SE (Nasdaq: BNTX, "BioNTech" or "the Company") reported financial results for the three and nine months ended September 30, 2024 and provided an update on its corporate progress (Press release, BioNTech, NOV 4, 2024, View Source [SID1234648550]).

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"BioNTech’s achievements during the period were the successful launch of our variant-adapted COVID-19 vaccines and the progress across our oncology pipeline. In particular, we initiated later-stage trials and shared important updates for our PD-L1 x VEGF-A bispecific antibody candidate BNT327/PM8002 and for our mRNA cancer vaccine portfolio. These successes reinforce the potential of our multi-platform technology approach and inform our strategy to pursue novel proprietary combinations," said Prof. Ugur Sahin, M.D., CEO and Co-Founder of BioNTech. "We remain focused on advancing our late-stage oncology product candidates towards potential registration. We believe our pipeline and capabilities uniquely position us to execute on our vision of becoming a global multiproduct immunotherapy company."

Financial Review for Third Quarter and Nine Months of 2024

in millions €, except per share data Third Quarter 2024 Third Quarter 2023 Nine Months
2024 Nine Months
2023
Revenues 1,244.8 895.3 1,561.1 2,340.0
Net profit / (loss) 198.1 160.6 (924.8) 472.4
Diluted earnings / (loss) per share 0.81 0.66 (3.83) 1.94
Revenues reported were €1,244.8 million for the three months ended September 30, 2024, compared to €895.3 million for the comparative prior year period. For the nine months ended September 30, 2024, revenues were €1,561.1 million, compared to €2,340.0 million for the comparative prior year period. The higher revenues in the third quarter of 2024 as compared to the comparative prior year period can be largely attributed to the earlier approvals received for its variant-adapted COVID-19 vaccines as compared to last year.

Cost of sales were €178.9 million for the three months ended September 30, 2024, compared to €161.8 million for the comparative prior year period. For the nine months ended September 30, 2024, cost of sales were €297.8 million, compared to €420.7 million for the comparative prior year period.

Research and development ("R&D") expenses were €550.3 million for the three months ended September 30, 2024, compared to €497.9 million for the comparative prior year period. For the nine months ended September 30, 2024, R&D expenses were €1,642.4 million, compared to €1,205.3 million for the comparative prior year period. R&D expenses were mainly influenced by progressing clinical studies for the Company’s late-stage oncology pipeline candidates.

Sales, general and administrative ("SG&A") expenses2, in total, amounted to €150.5 million for the three months ended September 30, 2024, compared to €153.5 million for the comparative prior year period. For the nine months ended September 30, 2024, SG&A expenses were €466.9 million, compared to €415.4 million for the comparative prior year period. SG&A expenses were mainly influenced by personnel expenses.

Other operating result amounted to negative €354.6 million during the three months ended September 30, 2024, compared to negative €9.0 million for the comparative prior year period. For the nine months ended September 30, 2024, other operating result amounted to negative €616.9 million compared to negative €134.4 million for the prior year period. Other operating result was primarily influenced by provisions for contractual disputes.

Income taxes were realized with an amount of €39.4 million in tax income for the three months ended September 30, 2024, compared to €66.8 million in accrued tax expenses for the comparative prior year period. For the nine months ended September 30, 2024, income taxes were realized with an amount of €54.1 million in tax income for the nine months ended September 30, 2024, compared to €50.5 million of accrued tax expenses for the comparative prior year period.

Net profit was €198.1 million for the three months ended September 30, 2024, compared to €160.6 million net profit for the comparative prior year period. For the nine months ended September 30, 2024, net loss was €924.8 million, compared to a net profit of €472.4 million for the comparative prior year period.

Cash and cash equivalents plus security investments as of September 30, 2024, reached €17,839.8 million, comprising €9,624.6 million in cash and cash equivalents, €7,078.0 million in current security investments and €1,137.2 million in non-current security investments.

Diluted earnings per share was €0.81 for the three months ended September 30, 2024, compared to €0.66 for the comparative prior year period. For the nine months ended September 30, 2024, loss per share was €3.83, compared to diluted earnings per share of €1.94 for the comparative prior year period.

Shares outstanding as of September 30, 2024, were 239,739,752, excluding 8,812,448 shares held in treasury.

"We successfully launched our variant-adapted COVID-19 vaccines upon receipt of earlier approvals as compared to last year. This drove our strong revenues in the third quarter," said Jens Holstein, CFO of BioNTech. "Our cost discipline in combination with our financial position allow us to continue to focus on those assets that we believe offer a fast path to market and the highest potential to generate value for patients and shareholders."

2024 Financial Year Guidance3

The Company expects its revenues for the full 2024 financial year to be at the low end of the guidance range provided in its outlook:

Total revenues for the 2024 financial year low end of €2.5 billion – €3.1 billion
The range reflects certain assumptions and expectations, including, but not limited to: COVID-19 vaccine uptake and price levels, including seasonal variations; inventory write-downs and other charges by BioNTech’s collaboration partner Pfizer Inc. ("Pfizer") that negatively influence BioNTech’s revenues; anticipated revenues from a pandemic preparedness contract with the German government; and revenues from the BioNTech Group service businesses, namely InstaDeep Ltd ("InstaDeep"), JPT Peptide Technologies GmbH, and BioNTech Innovative Manufacturing Services GmbH. Generally, the Company continues to remain largely dependent on revenues generated in its collaboration partner’s territories in 2024.

2024 Financial Year Expenses and Capex

The Company has reduced its previous guidance for expected SG&A expenses and capital expenditures for operating activities for the 2024 financial year:

Guidance March 2024 Guidance November 2024
R&D expenses4 €2,400 million – €2,600 million €2,400 million – €2,600 million
SG&A expenses €700 million – €800 million €600 million – €700 million
Capital expenditures for operating activities €400 million – €500 million €300 million – €400 million
The full interim unaudited condensed consolidated financial statements can be found in BioNTech’s Report on Form 6-K for the period ended September 30, 2024, filed today with the United States Securities and Exchange Commission ("SEC") and available at View Source