On January 16, 2023 Bavarian Nordic A/S (OMX: BAVA) reported preliminary, unaudited financial results for 2022, exceeding the financial guidance for the year (Press release, Bavarian Nordic, JAN 16, 2023, View Source [SID1234626266]). During 2022, the Company upgraded its financial guidance seven times. The results reported today are compared to the latest guidance, which was issued on September 7, 2022. For comparison with original guidance, see table below.

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Revenue for the full year was approximately DKK 3,151 million, compared to guided revenue in the interval of DKK 2,800 to 3,000 million. Revenue was comprised of:
DKK 1,730 million from sale of JYNNEOS/IMVANEX/IMVAMUNE mpox/smallpox vaccine
DKK 879 million from sale of Rabipur/RabAvert
DKK 299 million from sale Encepur
DKK 79 million from sale of third-party products
DKK 164 million in other income
The operating result (EBITDA) was a profit of approximately DKK 329 million, compared to a guided loss in the interval DKK -200 to 0 million.

Cash and cash equivalents at year-end was DKK 1,742 million, compared to a guided amount of DKK >1,700 million. The guidance assumed a bank debt level at year-end of DKK 600 million, however remaining bank debt was repaid during the fourth quarter.

The improved revenue is largely resulting from shipment of more mpox vaccines than anticipated in the latest guidance, which reflected some uncertainty related to the timing of deliveries and invoicing towards the end of 2022. Also, a continued strong performance in the rabies business has contributed to the improvement. Combined with a general cost-conscious approach, the operating result has been significantly improved, and Bavarian Nordic will now deliver profitability on EBITDA for 2022.

"Compared to our original guidance for 2022, we have nearly tripled our revenues and have turned profitable in a year that was otherwise expected to draw on our cash resources as we embarked on two large Phase 3 trials which have driven significant R&D costs. It has been a remarkable year and we are very pleased to announce these results, which primarily have been made possible due to the significant order flow for our mpox vaccine during the year, and it is highly encouraging that we have been able to advance our promising late-stage pipeline and still deliver such a strong financial result for the year," said Paul Chaplin, President and CEO of Bavarian Nordic.

The consolidated, audited financial results for 2022 will become available on March 2, 2023, in connection with publication of the Company’s annual report for 2022.

DKK million

Numbers are approximate

FY 2022 guidance
original, 04-Mar-2022

FY 2022 guidance

latest, 07-Sep-2022

FY 2022 preliminary

unaudited

Revenue

1,100 – 1,400

2,800 – 3,000

3,151

EBITDA

(1,300) – (1,000)

(200) – 0

329

Cash and cash equivalents

1,000 – 1,200*

> 1,700*

1,742*

* Guidance assumed a bank debt level of DKK 600 million whereas actual year-end cash position includes no bank debt, as this was repaid during fourth quarter.

On January 16, 2023 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported that the first patient has been treated in a Phase 1 first-in-human study evaluating the safety, tolerability, and efficacy of MP0533, the company’s candidate for acute myeloid leukemia (AML) (Press release, Molecular Partners, JAN 16, 2023, View Source [SID1234626265]). MP0533 is designed to focus an immune attack against AML in a new way that preferentially spares healthy cells, which has been a historic challenge for CD3-targeting therapeutics.

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"AML is a notoriously difficult cancer to treat, largely due to the overlapping targets expressed on both healthy and leukemic cells. Our team has worked relentlessly over the past three years to develop a molecule intended to target these cancerous cells while avoiding healthy cells. MP0533’s mechanism represents a new level of precision targeting in complex cancers that may permit greater use of the cytotoxic power of engaging CD3," said Nicolas Leupin, M.D., Ph.D., Chief Medical Officer of Molecular Partners. "We are grateful to our team and our collaborators for reaching this milestone and look forward to learning more about the potential of MP0533 to help these patients."

MP0533 simultaneously targets three surface proteins, CD33, CD123, and CD70, that are vastly more likely to be expressed together on AML blast cells and leukemic stem cells over healthy cells. It also targets CD3 on cytotoxic T cells, which will preferentially activate when at least two of the surface proteins are bound. This novel mechanism is intended to greatly favor CD3 activation in leukemic stem cells rather than the systemic activation seen in previous CD3-based T cell engagers.

The Phase 1 open-label dose escalation study will enroll patients with relapsed/refractory AML and higher-risk myelodysplastic syndromes (MDS). It is designed to assess the safety, tolerability, and efficacy of MP0533 in addition to a range of secondary endpoints, such as the effect on LSCs, pharmacokinetics, T-cell activation, and cytokine release. Between 20-45 patients are expected to be enrolled across five sites in Switzerland and the Netherlands in collaboration with select sites within the HOVON cooperative group. Additional clinical sites are planned as well.

MP0533 preclinical data demonstrates it induces preferential T cell mediated killing of cells expressing two or three of the tumor associated antigens (TAAs) compared to cells expressing a single TAA. MP0533 also demonstrated an ability to induce T cell activation and killing of AML cells in samples from newly diagnosed and previously treated patients. The research also showed that MP0533 was able to directly target and kill LSCs while sparing a variety of healthy cells including hematopoietic stem cells, endothelial cells, and T cells.

On January 16, 2023 Avectas, a cell engineering technology leader, reported the launch of the Solupore platform at the Advanced Therapies Week, Miami Beach Convention Centre, January 17th – 20th, 2023, Booth # 331 (Press release, Avectas, JAN 16, 2023, View Source [SID1234626264]).

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The Solupore non-viral delivery system enables the clinical manufacturing of advanced cell therapies differentiated by unparalleled cell health and superior cell functionality. This novel system expands the possibilities for complex editing and challenging cargo delivery. Solupore technology works by temporarily permeabilizing the target cell membrane so that molecular cargoes such as mRNA, plasmids, RNP or CRISPR/Cas9 can be delivered while retaining
superior cell health and function.

Avectas has developed an extensive data package based on primary T cells that includes performance in complex editing, cell health characterization, in vitro and in vivo functional performance. Avectas is expanding this dataset to include other cell types, including induced
pluripotent stem cells (iPSCs).

Several partners have accessed the technology under an early access program, including Genscript, Inceptor Bio, and CCRM/Omnia bio. Avectas also has ongoing undisclosed partnerships with CDMOs and cell therapy companies.

Michael Maguire, PhD, CEO of Avectas, said: "I am proud that our brilliant team is launching Solupore today after focused development over several years. I believe Solupore is poised to address the limitations of current transfection technologies for modifying therapeutic cells. Our system will seamlessly integrate into GMP processes to manufacture healthy and highly functional cells. Launching our clinical manufacturing system brings us closer to fulfilling our
vision of working with partners to accelerate the future of cell therapies for patients."

The Solupore system is now available for trialing at partner sites through the Avectas Business Development team, [email protected].

On January 16, 2023 AstraZeneca reported that it has completed the acquisition of Neogene Therapeutics Inc. (Neogene), a global clinical-stage biotechnology company pioneering the discovery, development and manufacturing of next-generation T-cell receptor therapies (TCR-Ts) (Press release, Neogene Therapeutics, JAN 16, 2023, View Source [SID1234626263]).

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Neogene will operate as a wholly owned subsidiary of AstraZeneca, with operations in Amsterdam, the Netherlands and California, US.

Financial considerations
AstraZeneca has acquired all outstanding equity of Neogene in exchange for an initial payment of $200m. Under the terms of the agreement, AstraZeneca will pay up to $120m in additional contingent milestone-based and non-contingent consideration.

ON January 15, 2023 Akeso, Inc. (9926. HK) ("Akeso"), a commercial-stage biopharmaceutical company focused on developing and commercializing first-in-class and best-in-class innovative medicines globally, reported that the anti PD-1 monoclonal antibody drug Anniko (penpulimab), co-developed by the Company with Sino Biopharmaceutical Limited (1177.HK) (together with its subsidiaries ”Sino Biopharm”), combined with chemotherapy as the first-line treatment of locally advanced or metastatic squamous non-small cell lung cancer (sq-NSCLC) has been granted marketing approval by the National Medical Products Administration (NMPA) of China (Press release, Akeso Biopharma, JAN 15, 2023, View Source [SID1234626244]).

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The approval of new indication is based on a randomized, double-blind, multi-center phase III clinical trial (AK105-302, NCT03866993), with 350 locally advanced or metastatic sq-NSCLC patients who have not received prior systemic therapy. According to the trial results published on 2022 ESMO (Free ESMO Whitepaper) Immuno-Oncology, penpulimab combined with chemotherapy shows consistent efficacy benefits across all efficacy endpoints and significantly reduces the risk of disease progression and death.

The median progression-free survival (mPFS) of the treatment group (7.6 months) is significantly superior to the placebo+chemo group (4.2 months).

The objective response rate (ORR) of the treatment group (71.4%) is significantly higher than the placebo+chemo group (44%).

In terms of safety, the treatment group shows no difference in the incidence of adverse event (AE) and serious adverse event (SAE), which shows the favourable safety profile of penpulimab.

Lung cancer is a malignant tumor with high incidence and high mortality rate worldwide. The incidences of lung cancer around the world and in China have exceeded 2.2 million and 810 thousand respectively in 2020. NSCLC patients accounts for 85% of total lung cancer patients, and about 30% of NSCLC patients are sq-NSCLC. The NMPA’s approval of Anniko combined with chemotherapy as the first line treatment of locally advanced or metastatic sq-NSCLC will further expand its potential.

In April 2022, this therapy was included as Grade II recommendation in Guidelines for Non-Small Cell Lung Cancer Treatment (2022) of Chinese Society of Clinical Oncology (CSCO) and was included in Guidelines of Chinese Society of Clinical Oncology Immune Checkpoint Inhibitor Clinical Practice (2022).