On March 29, 2022 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) ("Aeterna" or the "Company"), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products, reported its financial and operating results for the year ended December 31, 2021 (Press release, AEterna Zentaris, MAR 29, 2022, View Source [SID1234611162]). The Company also provided an update on progress in its pre-clinical and clinical development programs.

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"With the in-licensing of six new pre-clinical development programs, 2021 was a transformational year for Aeterna Zentaris. As we work to make a positive impact across multiple therapeutic areas with unmet needs, our team is executing on all fronts to advance these pre-clinical assets toward in-human clinical studies," commented Dr. Klaus Paulini, Chief Executive Officer of Aeterna. "Since the start of 2022, we have continued to build momentum and position ourselves to unlock the full potential of our pipeline and value for all stakeholders. Bolstered by our cash position, which provides us with the funding for operations through 2023, we are laser focused on our development and strategic priorities."

Recent Highlights

●Expanded research program with Julius-Maximilians-University Wuerzburg aimed to accelerate development of vaccine programs on COVID-19 and Chlamydia
●Announced Notice of Allowance for U.S. patent covering AEZS-150 for the potential treatment of chronic hypoparathyroidism.
●Appointed Giuliano La Fratta as Chief Financial Officer.

Pre-Clinical and Clinical Programs Update:

Therapeutics Development Pipeline

AIM Biologicals: Targeted, highly specific autoimmunity modifying therapeutics for the potential treatment of neuromyelitis optica spectrum disorder ("NMOSD") and Parkinson’s disease (PD)

In January 2021, Aeterna entered into an exclusive patent license and research agreement with the University of Wuerzburg, Germany, for worldwide rights to develop, manufacture, and commercialize AIM Biologicals for the potential treatment of NMOSD. Additionally, the Company has engaged Prof. Dr. Joerg Wischhusen from the University Hospital in Wuerzburg as well as neuro-immunologist Dr. Michael Levy from the Massachusetts General Hospital in Boston as consultants for scientific support and advice in the field of inflammatory CNS disorders, autoimmune diseases of the nervous system, and NMOSD. In September 2021, the Company entered into an additional exclusive license with the University of Wuerzburg for early pre-clinical development towards the potential treatment of Parkinson’s disease.

AIM Biologicals (Auto-Immunity Modifying Biologicals) is based on a natural process during pregnancy, which induces immunogenic tolerance of the maternal immune system to the partially foreign fetal antigens. Fetal proteins are processed and presented on certain immunosuppressive major histocompatibility complex class I molecules to induce this tolerance. In an autoimmune disease is the immune system misdirected and targeting the body’s own protein. With AIM Biologicals, the Company aims to restore the tolerance against such proteins to treat autoimmune diseases.

For the development of AIM Biologicals as potential PD therapeutics, Aeterna plans to utilize, among others, an innovative animal model on neurodegeneration by α-synuclein-specific T cells in AAV-A53T-α-synuclein Parkinson’s disease mice, which has recently been published by University of Wuerzburg researchers.

Next Steps – NMOSD

Conduct in-vitro and in-vivo assessments to select an AIM Biologicals-based development candidate.
●Manufacturing process development for selected candidate.

Next Steps – Parkinson’s Disease

●Design and produce antigen-specific AIM Biologics molecules for the potential treatment of Parkinson’s disease.
●Conduct in-vitro and in-vivo assessments in relevant Parkinson’s disease models.

Delayed Clearance Parathyroid Hormone ("DC-PTH") Fusion Polypeptides: Potential treatment for primary hypoparathyroidism

In March 2021, Aeterna entered into an exclusive patent and know-how license agreement and research agreement with The University of Sheffield, United Kingdom, for the intellectual property relating to DC-PTH fusion polypeptides with delayed clearance for all human uses. In consultation with The University of Sheffield, Aeterna has selected AEZS-150 as the lead candidate in its DC-PTH program. AEZS-150 is being developed with the goal of providing a potential new treatment option of primary hypoparathyroidism in adults.

Next Steps

●Work with The University of Sheffield to conduct in depth characterization of development candidate (in-vitro and in-vivo).
●Develop manufacturing process.
●Formalize pre-clinical development of AEZS-150 in preparation for a potential IND filing for conducting the first in-human clinical study.

Macimorelin Therapeutic: Ghrelin agonist in development for the treatment of ALS (Lou Gehrig’s disease)

In January 2021, the Company entered into a material transfer agreement with the University of Queensland, Australia, to provide macimorelin for the conduct of pre-clinical and subsequent clinical studies evaluating macimorelin as a potential therapeutic for the treatment of ALS (Lou Gehrig’s disease). The University of Queensland researchers have filed for supportive grants and aim to conduct pre-clinical studies in multiple pre-clinical models to demonstrate the therapeutic potential of macimorelin to slow disease progression and disease-specific pathology.

Macimorelin, a potent ghrelin agonist, is an orally active small molecule that stimulates the secretion of growth hormone from the pituitary gland. Acting via this mechanism, which was established during the development as a diagnostic test for growth hormone deficiency, it is believed that macimorelin may slow the progression of certain neurodegenerative diseases like ALS.

Apart from already available pre-clinical and clinical data on macimorelin for the development as a diagnostic, Aeterna may utilize the established supply chain to support this development. Alternative formulations are currently also under development, as a further option in addition to the existing oral solution already approved for the diagnostic use in adult growth hormone deficiency (AGHD).

Next Steps

●Work with The University of Queensland to conduct proof-of-concept studies with macimorelin in disease-specific animal models.
●Assess alternative formulations.
●Formalize pre-clinical development plan.

Diagnostics Development Pipeline

Macimorelin Diagnostic: Ghrelin agonist in development for diagnostic use in childhood-onset growth hormone deficiency ("CGHD")

Aeterna is currently conducting its pivotal Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of CGHD.

Children and adolescents from two to less than 18 years of age with suspected growth hormone deficiency are to be included. The study is expected to include approximately 100 subjects in Europe and North America, with at least 40 subjects in pre-pubertal and 40 subjects in pubertal status. Macimorelin growth hormone stimulation test ("GHST") will be performed twice for repeatability data and two standard GHSTs will be used as controls: arginine (i.v.) and clonidine (p.o.).

On April 22, 2021, the U.S. FDA Investigational New Drug Application associated with this clinical trial became active.

The first clinical sites in the U.S. and in Europe are open for patient recruitment. In Europe, national clinical trial approval procedures and site initiation activities are ongoing. Site activation and patient enrollment continues to be impacted by the ongoing COVID-19 pandemic. The Company is actively monitoring delays to mitigate potential impact of COVID-19 on estimated trial completion dates. Additionally, clinical trial sites originally planned in the Ukraine and Russia are being halted due to the conflict in Ukraine intensifying following the Russian invasion. As a result, further delays with enrollment are expected as the DETECT-trial planned to recruit at least 25% (25 subjects) within those countries. Due to these circumstances and the resulting feasibility data from the Company’s CRO on potential options, Aeterna believes recruitment for the DETECT-trial may now continue until later into 2023.

The Company continues to advance its ongoing business development discussions to secure commercialization partners for macimorelin in additional markets. In addition to its previously established agreements, Aeterna recently entered into a license agreement with NK Meditech Ltd., for the development and commercialization of macimorelin in the Republic of Korea, and a distribution agreement with Er-Kim Pharmaceuticals Bulgaria EOOD for the commercialization of macimorelin in Turkey and some Balkan countries.

Vaccine Development Pipeline

Bacterial Vaccine Platform: Orally active, live-attenuated bacterial vaccine platform with potential application against viruses and bacteria, such as coronavirus types, including COVID-19 (SARS-CoV-2) and Chlamydia

In February 2021, Aeterna entered into an exclusive option agreement with the University of Wuerzburg to evaluate a pre-clinical, potential COVID-19 vaccine developed at the University of Wuerzburg. In March 2021, the Company exercised its option and entered into a license agreement where the Company was granted an exclusive, world-wide, license to certain patent applications and know-how owned by the University of Wuerzburg to research and develop, manufacture, and sell a potential COVID-19 vaccine. The Company’s vaccine platform is currently undergoing pre-clinical studies for the prevention of coronavirus diseases, including COVID-19 (SARS-CoV-2) with the planned start of clinical development targeted for H1 2023.

In September 2021, the Company exercised its option under the agreement with the University of Wuerzburg on a then undisclosed field, now known to be Chlamydia. Chlamydia trachomatis is a sexually transmitted bacterium infecting over 130 million subjects annually. Asymptomatic disease can spread to the reproductive tract eventually inducing infertility, miscarriage, or ectopic pregnancy, which is a life-threatening condition. Ocular infections can lead to inclusion conjunctivitis or trachoma, which is the primary source of visual impairment or infectious blindness. Additionally, Prof. Dr. Thomas Rudel of the University of Wuerzburg was engaged by the Company in September 2021 as a scientific consultant to support development of the salmonella-based vaccine platform for the coronavirus and Chlamydia vaccines.

Recently, the Company expanded its research agreement with the University of Wuerzburg to conduct supplementary research activities and pre-clinical development studies on the potential vaccines, the results of which are covered within the scope of the license agreements. Under the expanded research program, the University of Wuerzburg will validate and utilize innovative human 3D intestinal tissue models to study the infection biology of Salmonella strains towards clinical development.

Next Steps – Coronavirus Vaccine

●Evaluate administration route, dose and immunization scheme.
●Initiate in-vivo immunology experiments with antigen variant candidates in relevant mice models.
●Conduct virus challenge experiments in immunized transgenic animals.
●Start manufacturing process assessment / development.
●Conduct pre-clinical safety and toxicology assessment.

Next Steps – Chlamydia Vaccine

●Design and prepare candidate vaccine strains.
Evaluate administration route, dose and immunization scheme.
●Conduct In-vivo immunology experiments with candidate strains in relevant mouse models.

Summary of Fourth Quarter and Full Year 2021 Financial Results

All amounts are in U.S. dollars

Cash and cash equivalents

The Company had $65.3 million in cash and cash equivalents at December 31, 2021.

Results of operations for the three-month period ended December 31, 2021

For the three-month period ended December 31, 2021, we reported a consolidated net loss of $2.9 million, or $0.02 loss per common share (basic and diluted), as compared with a consolidated net loss of $1.3 million, or $0.02 loss per common share (basic and diluted) for the three-month period ended December 31, 2020. The $1.6 million increase in net loss is primarily from a $0.5 million increase in total operating expenses and a $1.4 million decrease in revenues, offset by a $0.4 million reduction in income tax expense.

Revenues

●Our total revenue for the three-month period ended December 31, 2021 was $1.0 million as compared with $2.4 million for the same period in 2020, representing a decrease of $1.4 million, primarily due to $1.4 million decline in product sales of macimorelin to its licensees, $0.5 million decline in license fees offset by $0.5 million increase in development services.

Operating Expenses

●Our total operating expenses for the three-month period ended December 31, 2021 was $4.1 million as compared with $3.6 million for the same period in 2020, representing an increase of $0.5 million. This increase arises primarily from a $1.3 million increase in research and development expenses, a $0.5 million increase in general and administrative expenses and $0.1 million in costs incurred in 2020 and not incurred in 2021 (comprised of $0.1 million in reversal of impairment of other asset), offset by a decline of $1.4 million in cost of sales.

Net Finance Income

●For the three-month period ended December 31, 2021, our net finance income was $0.3 million as compared to $0.3 million for the three-month period ended December 31, 2020.

Results of operations for the year ended December 31, 2021

For the twelve-month period ended December 31, 2021, we reported a consolidated net loss of $8.4 million, or $0.07 loss per common share (basic and diluted), as compared with a consolidated net loss of $5.1 million, or $0.12 loss per common share (basic and diluted), for the year ended December 31, 2020. The $3.3 million increase in net loss is primarily from a $4.5 million increase in operating expenses and a $0.8 million decline in net finance income, partially offset by an increase of $1.6 million in total revenues and a change in income tax recovery of $0.5 million

Revenues

●Our total revenue for the twelve-month period ended December 31, 2021 was $5.3 million as compared with $3.7 million for the same period in 2020, representing an increase of $1.6 million, primarily due to $3.3 million increase in development services with Novo and $0.8 million increase in license fees related to the partial recognition of the €5 million up front payment received from Novo in 2020. Offset by a decrease in product sales by $2.5 million.

Operating Expenses

●Our total operating expenses for the twelve-month period ended December 31, 2021 was $13.9 million as compared with $9.4 million for the same period in 2020, representing an increase of $4.5 million. This increase arises primarily from a $5.1 million increase in research and development expenses, $1.1 million increase in general and administration expenses, $0.3 million in increase in selling expenses and $0.3 million in costs incurred in 2020 and not incurred in 2021 (comprised of $0.2 million in gain on modification of building lease and $0.1 million in reversal of impairment of other asset), offset by a $2.2 million decrease in cost of sales.

Net Finance Income

●Our net finance income for the twelve-month period ended December 31, 2021, was $0.2 million as compared with $1.0 million for the same period in 2020, representing a decrease of $0.8 million. This is primarily due to a $1.1 million change in fair value of warrant liability, a $0.4 million decline in gain due to change in foreign currency, offset by a $0.7 million decline in other finance costs. During the prior year, the Company registered the common shares underlying certain warrants which allowed the Company to reclassify such warrants from liability to shareholders’ equity in the condensed interim consolidated statements of financial position. As such the change in fair value of such warrants liabilities was classified as a finance cost in the consolidated statements of loss in 2020; there was no such change in fair value in 2021.

Consolidated Financial Statements and Management’s Discussion and Analysis

For reference, the Management’s Discussion and Analysis of Financial Condition and Results of Operations for the fourth quarter and full year 2021, as well as the Company’s unaudited consolidated interim financial statements as of December 31, 2021, will be available on the Company’s website (www.zentaris.com) in the Investors section or at the Company’s profile at www.sedar.com and www.sec.gov.

On March 29, 2022 Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases reported 2021 annual results including its major achievements and progress (Press release, Innovent Biologics, MAR 29, 2022, View Source [SID1234611483]).

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Dr. Michael Yu, Founder, Chairman and CEO of Innovent, stated: "2021 is the 10th year since our company’s inception. Over the past decade, bearing the mission to develop and commercialize high-quality biopharmaceutical products that are affordable to ordinary people, Innovent has developed into a leading Chinese biopharmaceutical company with fully-integrated platform capability, strong execution with track record and a healthy financial position. For the past year, we are determined with the global innovation strategy and have made tremendous progress in all aspects including R&D, clinical development, CMC, commercialization and business collaboration. The company has rolled out development for a pipeline of 32 valuable assets in various clinic stages. We plan to expand commercial portfolio to over ten products in two years with continuous strong revenue growth, supported by our validated commercial platform capability and competitive cost advantage under large scale and high quality manufacturing facilities. Besides, we further fortified R&D structure and platform to expedite the development for over a dozens of clinical stage candidates with global potential and more preclinical projects with global innovation. For the year of 2022, as the inflection point for next decade of development, we will adhere to the company strategy of global innovation, strive to be a global premier biopharmaceutical company with steady growth prospects, and create sustainable value for patients, employees, shareholders and the society."

Business Highlight Overview

For the year of 2021, achieved RMB4,261million total revenue and RMB4,001million product revenue with an increase of 69.0% compared to the prior year.
For the year of 2021, commercial portfolio increased to six products with two innovative anti-cancer drugs approved.
Expanded strategic partnership with Eli Lilly in March 2022 added the seventh newly approved product and a ready-to-launch product at New Drug Application (NDA) stage.
TYVYT received three additional first-line indication approvals for major types of cancer and successfully included in the updated National Reimbursement Drug List (NRDL), also as the only PD-1 inhibitor globally with positive Phase 3 data in the first-line treatment for 5 major types of cancer.
6 molecules at NDA or late clinical stage.
7 molecules achieved positive Proof of Concept (PoC) data readout.
Acceleration of Global R&D and clinical development, to explore first-in-class (FIC) targets with full-functional global development platform and global talents.
About 10 strategic collaborations with global and regional partners in research, development and commercialization, unleashing the platform potential and explore synergic value of pipeline portfolio.
Commercial – Strong Track Record

Product revenue RMB4,001 million in 2021: an increase of 69.0% compared with the prior year.
Expansion of commercial portfolio into six approved products, including: TYVYT, BYVASDA, SULINNO, HALPRYZA, PEMAZYRE and NAILIKE by the end of 2021.
In March 2022, expansion of strategic collaboration with Eli Lilly, which showed further recognition by the global MNC: for Innovent to obtain sole commercialization right of the 7th approved product Cyramza (ramucirumab) and NDA-stage asset Retsevmo (selpercatinib), and the right of first negotiation for future potential commercialization of late-stage asset Pirtobrutinib (BTK inhibitor) in mainland China.
Broad coverage in commercial channels and networks with an experienced and professional sales and marketing team expanded to cover over 5,100 hospitals and 1,100 pharmacies across more than 320 cities, and a well-structured commercial team with nearly 3,000 people.
Pipeline – Expedited Speed to Launch

– TYVYT as leading brand in PD-(L)1 market:

Newly approved 3 additional indications got successfully included in the NRDL (non-squamous Non-Small Cell Lung Cancer [nsqNSCLC], sqNSCLC, hepatocellular carcinoma [HCC]).
The only PD-1 inhibitor with positive Phase 3 data in first-line treatments of five major types of cancer (1L nsqNSCLC, 1L sqNSCLC, 1L HCC, 1L esophageal squamous cell carcinoma [ESCC], 1L gastric cancer [GC]).
Multiple clinical datasets of TYVYT were published in world-renowned medical journals such as EClinicalMedicine, ESMO (Free ESMO Whitepaper) Open, BMJ etc.
– Six assets at late stage incl. NDA/pivotal trials:

Retsevmo (selpercatinib), NDA submitted
IBI-326 (BCMA CAR-T), planned NDA submission in 2022
IBI-306 (PCSK9), planned NDA submission in 2022
IBI-310 (CTLA-4), planned NDA submission in 2022
IBI-344 (ROS1/NTRK), ongoing pivotal Phase 2
IBI-376 (PI3Kδ), ongoing pivotal Phase 2[1]
– Seven innovative molecules achieved positive PoC data readout, plan to advance into the late stage in clinic:

IBI-188 (CD47): achieved preliminary PoC data readout in Phase 1b study for 1L MDS, promising efficacy data (ORR 83.3%) and a good safety profile. Plan to initiate Phase 3 study for 1L MDS in 2022.
IBI-362 (GLP-1/GCGR): showed robust efficacy in weight loss for Phase 1b in obesity, data published in EClinicalMedicine. Robust efficacy in blood glucose lowering effects in diabetics patients. Potentially the best GLP1-/GCGR dual agonist. Plan to initiate Phase 3 studies for obesity and type 2 diabetes in 2022.
IBI-326 (BCMA CAR-T): demonstrated impressive efficacy and safety in Phase 1/2 study, with improved in-vivo persistency and encouraging efficacy in patients with prior murine BCMA CAR-T treatment failure. Plan to submit NDA in 2022.
IBI-344 (ROS1/NTRK): ORR 90.5% in the crizotinib-naïve patient and ORR 43.8% in the crizotinib-treated patient group. Received NMPA BTD for ROS1+ NSCLC. Pivotal Phase 2 study ongoing.
IBI-310 (CTLA-4): achieved positive PoC in combination with sintilimab for CC and HCC. Plan to submit NDA for CC in 2022.
IBI-302 (VEGF/C): showed visual acuity improvement with edema reduction in Phase 1b study, ongoing Phase 2 study for nAMD.
IBI-112 (IL-23p19): observed significant efficacy signal in the Phase 2 study for psoriasis with long-acting potential. Plan to initiate Phase 3 study in 2022.
– Expedite the clinical development of multiple molecule clusters with global potentials, expected PoC data readout roll out in a well-schemed plan:

CD47 cluster: IBI-188 (CD47), IBI-322 (PD-L1/CD47), IBI-397 (SIRPα)
LAG-3 cluster: IBI-110 (LAG-3), IBI-323 (PD-L1/LAG-3)
TIGIT cluster: IBI-939 (TIGIT), IBI-321 (PD-1/TIGIT)
VEGF cluster: IBI-302 (VEGF/C), IBI-324 (VEGF/ANG-2), IBI-333 (VEGF-A/VEGF-C)
R&D: Infrastructure Set for Global Innovation

– Innovent US Lab established: primarily focused on disease mechanism study and technology-platform development, accelerating the translation of scientific discovery into the next-generation drug candidates.

– The Scientific Advisory Board (SAB) established: comprising three World-renowned scientists to provide insights of frontline global innovation in cancer biology and immunology and contribute scientific advice to our research and clinical pipelines.

– The CMC Advisory Board (CAB) established: comprising industry-leading experts to provide professional advice on production structure and process streamline, resources investment and strategic development.

– The global product development team expansion and infrastructure improvement: have built a full-functional global development team and expect to have 100+ employees in near term. Have established an effective global development platform and process which enables to cooperate seamlessly with China clinical development team of 1,000 people to carry out global development for our novel assets.

– Technology platform collaborations for global innovation:

The development of the proprietary ADC technologies with Synaffix.
The development of three new therapeutic immune-stimulating antibody conjugate (ISAC) candidates with Bolt Biotherapeutics.
The development of up to three enzyme specific inhibitors with Amagma Therapeutics.
The license-out of non-exclusive commercial right of fully-human BCMA CAR construct to SANA Biotechnology for certain in vivo gene therapy and ex vivo hypo-immune cell therapy application.
BD: Meaningful Collaboration to Succeed

– Established multiple co-development and co-commercialization collaboration to unlock assets potential:

Expanded strategic collaboration with Eli Lilly for the sole commercialization right of Cyramza (ramucirumab) and Retsevmo (selpercatinib), and right of first negotiation for future commercialization of pirtobrutinib (BTK inhibitor) in mainland China.
Taletrectinib (ROS1/NTRK) – a next-generation TKI designed to effectively target ROS1 and NTRK, co-development and co-commercialization with AnHeart in China.
NAILIKE (olverembatinib) – a novel third-generation BCR-ABL TKI, co-development and co-commercialization with Ascentage Pharma in China.
GFH925 (KRAS G12C inhibitor) – a novel, orally active, potent KRAS G12C inhibitor, co-development and co-commercialization with GenFleet Therapeutics in China and opt-in right for global development and commercialization.
Orismilast (PDE4 inhibitor) – a novel, orally active PDE4 inhibitor for broad target indications, granted sole development and sole commercialization right in China from UNION therapeutics A/S.
Manufacturing Facilities Upgradation

New Commercial Facility: successfully expanded the production capacity from 24,000L to 60,000L, which is one of the largest stainless steel production capacity in China.
Quality compliance to GMP and cost advantage further strengthen market competitiveness.
Financial Highlights (Non-IFRS measure)

Total revenue was RMB 4,261 million, including product revenue 4,001 million with an increase of 69.0% compared to the prior year.
Gross profit margin was 88.6%，an increase of 3.7% compared to the prior year.
R&D expenses were RMB2,116 million an increase of 23.2% compared to the prior year.
Loss for the year was RMB2,243 million.
Cash on hand and short-term financial assets at the end of 2021 was approximately USD1,415million, which enables strategical focus on the long-term development strategies.[2]
Note: [1] In January 2022, Incyte has withdrawn the application of parsaclisib in FL, MZL and MCL in the U.S. as a business decision and is not related to any changes in either the efficacy or safety of parsaclisib. We plan to have communication with China NMPA regarding the potential submission of PI3Kδ.

[2] The financial numbers mentioned above was based on non-IFRS measure which excluded certain non-cash items and non-recurring events. Detailed disclosure can be found at the Company’s annual result announcement.

On March 29, 2022 Pulmatrix, Inc. (NASDAQ: PULM), a clinical-stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary and non-pulmonary disease using its patented iSPERSE technology, reported fourth quarter and year-end financial results for 2021 and provided a corporate update (Press release, Pulmatrix, MAR 29, 2022, View Source [SID1234611105]).

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Ted Raad, Chief Executive Officer of Pulmatrix commented, "I am excited about the future of Pulmatrix. We are well-positioned financially and operationally to advance multiple programs into the clinic as we continue to leverage our iSPERSETM technology to address significant unmet medical needs. With funding into Q1 2024, we have the opportunity to reach meaningful upcoming data milestones and we have strengthened the organization with several key hires, including the recent appointment of a new Chief Medical Offer, Dr. Margaret Wasilewski."

2021 and Recent Program and Corporate Highlights

Pulmazole

On January 27, 2021, the Company conducted a Type-C meeting with FDA to finalize its plans to commence a Phase 2b study with topline data on registrational endpoints in allergic bronchopulmonary aspergillosis (ABPA). If successful, these data may enable a Phase 3 registration study.
On November 9, 2021, the Company announced the positive resolution of a contractual dispute with Cipla, thereby allowing Pulmatrix and Cipla to progress together toward the Phase 2b study in ABPA that aims to provide data on potential registrational endpoints in ABPA. Pulmatrix expects to initiate patient dosing for the Phase 2b study in Q1 2023 and to deliver top-line data by mid-2024.
PUR3100

On April 23, 2021, the company hosted a Key Opinion Leader event on PUR3100, an orally-inhaled dihydroergotamine (DHE) therapy. Migraine experts highlighted the potential benefits that PUR3100 (iSPERSE-enabled DHE) may provide to improve the standard of care and address the unmet needs of acute migraine sufferers.
On January 25, 2022, the Company conducted a Type-C meeting with the FDA. This meeting along with pre-IND written responses has helped define the overall non-clinical and clinical program. The next trial will be a Phase 1 study in Australia allowing for generation of the most comprehensive dataset in a cost and time-efficient manner so as to have Phase 1 data in 2022. This study will form the basis for a US IND.
The Phase 1 study is intended to assess not only safety, tolerability, and pharmacokinetics of PUR3100 in humans, but also provide preliminary comparative bioavailability data to support the use of the 505(b)(2) pathway for marketing authorization. The Company anticipates initiating patient dosing for the Phase 1 study in Australia in Q3 2022, with top-line data anticipated in Q4 2022. A Phase 2 study to determine efficacy will be conducted in migraine patients evaluating two doses that were selected based on the Phase 1 study results. This study is projected to commence dosing in Q2 2023 with data by year-end 2023.
PUR1800

On August 18, 2021, the Company announced that results from 6-month and 9-month pre-clinical toxicology studies demonstrated no progression of 28-day findings, suggesting potential for chronic dosing of PUR1800 in indications beyond acute exacerbations of chronic obstructive pulmonary disease (AECOPD) including, but not limited to, steroid resistant asthma, chronic obstructive pulmonary disease (COPD) and idiopathic pulmonary fibrosis (IPF).
On March 21, 2021, the company announced that PUR1800 achieved the primary endpoint of safety and tolerability in a Phase 1b clinical study with no serious adverse events observed. Preliminary pharmacokinetic and pharmacodynamic data are supportive of continued PUR1800 development in its primary therapeutic target of AECOPD as well other chronic inflammatory airway disease such as severe asthma, COPD and IPF. The Company plans to release more detailed results of the Phase 1b clinical study results at a relevant medical conference in the future.
Corporate Highlights

On December 17, 2021, the Company announced the closing of a $6.75 million registered direct offering. Combined with $40 million raised in February 2021, the total funds raised in 2021 equaled $46.75 million in gross proceeds. The Company anticipates that its cash position is sufficient to fund operations into Q1 2024 and enable Pulmatrix to pursue clinical data milestones for PUR3100 Phase 1 and Phase 2 and Pulmazole Phase 2b trials.
On February 28, the Company completed a reverse stock-split at a ratio of 1-for-20 (the "Reverse Split") which reduced the number of outstanding shares of the Company’s common stock from approximately 65.9 million shares to 3.3 million shares. The number of authorized shares of the Company’s common stock remains at 200,000,000.
On March 1, the Company announced the hiring of Dr. Margaret Wasilewski as the Company’s Chief Medical Officer. Dr. Wasilewski leverages over 25 years of experience in pharmaceutical drug development.
On March 17, the Company announced that it regained compliance for its listing on Nasdaq, allowing continued access to capital markets and liquidity for its investors.
Fourth Quarter and Year-End Financial Results

Revenue was $5.2 million for the year ended December 31, 2021 as compared to $12.6 million for the year ended December 31, 2020, a decrease of $7.5 million. The decrease is related to $3.4 million fewer reimbursable pass-through expenses under our collaboration agreements and a $0.9 million decrease in license related revenues due to a pause in the development activities under the collaboration with Cipla during 2021, and a $3.2 million decrease in license related revenues under our prior collaboration agreement with Johnson & Johnson Enterprise Innovation.

Research and development expense was $15.4 million for the year ended December 31, 2021, as compared to $15.6 million for the year ended December 31, 2020, a decrease of $0.2 million. The decrease was primarily due to a decrease in spend of $2.5 million related to the Company’s PUR1800 program as well as a decrease in spend of $1.7 million related to the Phase 2 Pulmazole clinical trial due to its COVID-19-related termination in 2020. This amount was then partially offset by increased spend of $3.2 million on pre-clinical and manufacturing costs related to our PUR3100 program, in addition to $0.8 million of operating costs in support of our overall clinical and pre-clinical programs.

General and administrative expense was $6.4 million for the year ended December 31, 2021, as compared to $6.9 million for the year ended December 31, 2020, a decrease of $0.5 million. The decrease was primarily due to decreased employment costs of $0.5 million.

Our total cash and cash equivalents balance as of December 31, 2021 was $53.8 million.

On March 29, 2022 Philogen reported that kindly invites you to attend a virtual briefing for analysts on 1 April 2022 to discuss the Company’s Full Year 2021 Financial Results (Press release, Philogen, MAR 29, 2022, View Source [SID1234611121]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Prof. Dr. Dario Neri, Chief Executive Officer, Dr. Laura Baldi, Chief Financial Officer, and Dr. Emanuele Puca, Head of Investor Relations, will host a 1-hour live webcast and conference call for analysts on 1 April 2022 at 9:00 ET / 14:00 BST / 15:00 CET. The presentation will be followed by a Q&A session.

To register for the event, please follow the link or Or join by phone. International numbers available here (Webinar ID: 864 8878 4624).

On March 29, 2022 Cytonus Therapeutics Inc. (Cytonus), a biotechnology company developing therapeutic products for targeted delivery in vivo, reported that their CSO, Dr. Richard Klemke Ph.D., will present the preclinical progress made on Cytonus’ lead Cargocyte product candidates at the annual AACR (Free AACR Whitepaper) meeting in New Orleans (Press release, Cytonus Therapeutics, MAR 29, 2022, View Source [SID1234611139]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Cargocytes armed with interleukin 12 (CA-IL-12) can be engineered to actively home, migrate, and penetrate deep into specific disease foci and synthesize biologically active therapeutic within the target tissues, hours after intravenous administration.

Dr. Klemke will share data demonstrating striking infiltration of engineered Cargocytes into solid tumors in in vivo models of metastatic Triple Negative Breast Cancer (TNBC), localizing and synthesizing the company’s proprietary Interleukin twelve (IL-12) construct. Furthermore, Dr. Klemke will share in vivo data demonstrating the efficacy of tumor-localized activity of CA-IL-12 in reducing tumor burden, particularly in combination with immune checkpoint inhibitors.

Abstract #3519: Bioengineering enucleated cell vehicles for targeted delivery of Interleukin 12 to metastatic tumors.

These results highlight the capabilities of Cytonus’ innovative Cargocyte platform in addressing solid tumor penetration challenges. Importantly, Cargocytes offer localized delivery of potent immunotherapies in a controlled manner to enhance their safety profile.

"While immunocytokine therapeutics can modulate the immune system, systemic delivery often results in adverse events and toxicity which limits their clinical utility. What is remarkable about our data is that we demonstrate a historically toxic cytokine (IL-12) can be localized to metastatic sites with exquisite accuracy and without systemic off-target effects. Our unique approach leads to a substantial reduction in metastatic burden and as such has potential to treat patients with late-stage cancers," said Dr. Remo Moomiaie-Qajar, M.D. co-founder and CEO of Cytonus Therapeutics.

"Arguably the most exciting discovery in immune oncology in the past decade has been the development of immune checkpoint inhibitors (ICI). Unfortunately, only 13% of all patients respond to ICI therapy. Our data clearly demonstrates the ability of CA-IL-12 to make recalcitrant tumors (TNBC) responsive to ICI therapy. These promising findings demonstrates the enabling nature of CA-IL-12 to expand the utility of ICI to the large number of cancer patients normally refractory to this approach," said Dr. Moomiaie-Qajar, M.D.

About Cargocyte

Cargocyte products are derived from enucleated cell lines and are uniquely engineered with specific disease targeting molecules to safely transport therapeutic payloads intravenously and deep into difficult-to-reach target tissues. Cargocytes can deliver and actively produce the therapeutic agent of choice on-site in a controlled, predictable, and safe manner. The proprietary Cargocyte technology is a first-in-class therapeutic platform with numerous potential medical applications across unmet therapeutic areas. Cytonus is developing a broad and deep pipeline of bioengineered Cargocytes for targeted delivery of a wide range of therapeutic modalities including Protein, Peptides, RNAs, ASOs, small molecule drugs, viruses, and gene editing agents.