On March 30, 2022 KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system (CNS) diseases, reported its financial results for the fourth quarter and year ended December 31, 2021 (Press release, KemPharm, MAR 30, 2022, View Source [SID1234611307]).

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"KemPharm advanced on multiple fronts during the fourth quarter of 2021 and into early 2022, cementing the past twelve months as the most substantial in KemPharm’s history," stated Travis Mickle, Ph.D., President and Chief Executive Officer of KemPharm. "In January, we announced our strategic focus on developing and commercializing therapeutics targeting rare CNS and neurodegenerative conditions starting with KP1077. The data released earlier this month from the Phase 1 clinical trial exploring the safety and PK of ‘higher dose SDX,’ affirms the opportunity to develop multiple SDX-based drug candidates, led by KP1077 for the treatment of IH, a rare sleep disorder with limited treatment options. Our recent interactions with the FDA have confirmed that we may proceed with the submission of an IND application for KP1077, which we expect to file as early as the second quarter of 2022. Upon clearance of the IND, we plan to initiate a Phase 2 clinical trial of KP1077 in IH later this year with a second trial in narcolepsy targeted to begin as early as the second half of 2022. In addition, we remain active on the business development front with the goal of acquiring or licensing complimentary clinical stage assets in rare CNS and neurodegenerative diseases."

"As KemPharm focuses on advancing KP1077 and expanding our development pipeline, we remain bullish on the potential for the commercial success of AZSTARYS, which is now being commercialized nationally by Corium. There have been significant gains in payor access, and prescription volume is beginning to grow. If this growth continues along the same trajectory observed since the beginning of 2022, the potential to achieve the initial sales milestones provided in the licensing agreement with an affiliate of Gurnet Point Capital becomes more tangible."

"Supporting these strategic and product development efforts is our strong financial position. With $127.8 million in cash, cash equivalents and long-term investments as of December 31, 2021, our current capital resources enable us to advance our internal pipeline while also potentially seeking external opportunities. The strength of our capital position sets us apart from many other development-stage biopharmaceutical companies, particularly in this challenging capital market environment. Our existing $50 million share repurchase program, which extends through 2023, also provides a mechanism to return value to shareholders as we achieve success."

Q4 and Full-Year 2021 Financial Results:

KemPharm’s revenue for Q4 2021 was $2.6 million, as compared to Q4 2020 revenue of $2.4 million. Q4 2021 revenue was derived primarily from $2.0 million in service fee revenue, and approximately $0.6 million of various royalty payments under the license agreement which covers AZSTARYS. The contracts under which service fee revenue is derived will end on March 31, 2022, although some amount of service fee revenue is expected to continue beyond that date.

KemPharm’s net loss for Q4 2021 was ($2.7) million, or ($0.08) per basic and diluted share, compared to a net loss of ($4.9) million, or a loss of ($1.07) per basic and diluted share for the same period in 2020. Net loss for Q4 2021 was driven primarily by a loss from operations of ($2.8) million, partially offset by net interest and other income of $0.1 million. The net operating loss of ($2.8) million for Q4 2021 was a decrease of ($0.4) million compared to net operating loss of ($3.2) million in the same period in 2020.

For FY 2021, KemPharm reported revenue of $28.7 million, which was primarily driven by $20.6 million in milestone and royalty revenue received under the license agreement which covers AZSTARYS, and approximately $8.1 million derived under service fee arrangements and related reimbursements. FY 2020 revenue was $13.3 million.

KemPharm’s net loss attributable to common stockholders for FY 2021 was ($62.9) million, or ($2.11) per basic and diluted share, compared to net loss attributable to common stockholders of ($12.8) million, or ($3.21) per basic and diluted share for FY 2020. Net loss attributable to common stockholders for FY 2021 was driven primarily by aggregate non-cash deemed dividends of ($54.3) million, or ($1.83) per basic and diluted share, which were recognized as a result of the warrant inducement transactions completed in the first half of 2021, a non-cash net loss on extinguishment of debt of ($16.1) million, or ($0.54) per basic and diluted share related to the debt extinguishment in Q1 2021, partially offset by net income from operations of $7.7 million.

As of December 31, 2021, total cash, cash equivalents and long-term investments was $127.8 million, which was a decrease of $3.7 million compared to $131.5 million as of September 30, 2021, driven in part by share repurchases of $2.4 million which were settled during Q4 2021. Based on the Company’s current operating forecast, existing cash, cash equivalents and long-term investments are expected to be sufficient to continue operations through and beyond 2025.

Conference Call Information:

KemPharm will host a conference call and live audio webcast with slide presentation today at 5:00 p.m. ET, to discuss its corporate and financial results for the fourth quarter of 2021 and fiscal year of 2021.

Telephone Access: To access the conference call telephonically, interested participants and investors will be required to register via the following online form: View Source

Once registered, all individuals will be provided with participant dial-in numbers, a passcode, and a registrant ID, which can then be used to access the conference call.

Participants may register at any time. It is recommended that the registration process be completed at least 15 minutes prior to the start of the call.
Webcast Access: The live audio webcast with slide presentation will be accessible via the Investor Relations section of KemPharm’s website, View Source An archive of the webcast and presentation will be available for 90 days beginning at approximately 6:00 p.m. ET, on Wednesday, March 30, 2022.

On March 30, 2022 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a biotechnology company developing novel LAG-3-related immunotherapy treatments for cancer and autoimmune disease, reported that new interim data in 2nd line metastatic NSCLC from its Phase II TACTI-002 trial (Press release, Immutep, MAR 30, 2022, View Source [SID1234611242]). The data was published in a poster presentation today at ESMO (Free ESMO Whitepaper)’s European Lung Cancer Congress (ELCC) 2022 in Prague, Czech Republic and is also available on the Company’s website:

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This part of TACTI-002, known as Part B, evaluates Immutep’s lead product candidate, eftilagimod alpha ("efti" or "IMP321") in combination with MSD’s KEYTRUDA (pembrolizumab) in a total of 36 patients with PD-L1 unselected 2nd line PD-X refractory metastatic NSCLC. The new data reflects the first interim results combining Stages 1 (23 patients) and 2 (13 patients) in 2nd line NSCLC.

Immutep CSO and CMO, Dr Frederic Triebel, noted: "It is very encouraging to see efti, in combination with pembrolizumab, showing an encouraging early overall survival rate of 73% at the six-month landmark, and promising interim disease control and tumour growth kinetics. The early signs are supportive that efti may boost the patient’s immune system to enable pembrolizumab to work more effectively in these patients with advanced lung cancer, while being safe and well tolerated."

TACTI-002 Principal Investigator, Dr Matthew G. Krebs of The University of Manchester and The Christie NHS Foundation Trust, said: "These interim results show an encouraging disease control rate of 36.1%, with 26% of patients being progression free at the 6-month landmark. These patients are a challenging population to treat, having progressed after previous lines of immunotherapy or chemo-immunotherapy and have limited options available for further treatment. So, it is pleasing to see the potential that efti in combination with pembrolizumab has to provide meaningful benefit in this patient group."

Condition of the patients as they entered the trial

A total of 36 patients were enrolled and treated. Patients were advanced in their disease. They had progressed after prior standard of care treatment with either anti-PD-(L)1 mono therapy (28%) or a combination of chemotherapy and anti-PD-(L)1 therapy (72%) and are referred to as PD-X refractory. Disease progression was confirmed by two consecutive CT-scans at least four weeks apart, eliminating the possibility of pseudo-progressions.1 The majority of patients (69%) had a PD-L1 tumour proportion score (TPS) of less than 50% at baseline, making them generally less likely to respond to anti-PD-(L)1 therapy.

Key Findings – data cut-off date 21 January 2022

73.7% of evaluable patients (14/19) had tumour shrinkage or tumour growth deceleration, according to tumour growth kinetics analysis

73% of patients alive at 6 months landmark in this difficult-to-treat patient population

DCR of 36.1% (13/36), with 26% being progression free at the 6-month landmark

ORR of 5.6% (2/36) with two patients reporting confirmed and durable partial responses, participating in the study for over 9 months and 23 months, respectively

6 patients still under therapy in the trial

Median OS has not yet been reached, which is encouraging given the advanced nature of the disease in this patient population

Table 1 – TACTI-002 Interim Results for Part B of TACTI-002

Tumour Response
Part B

2nd line NSCLC2

Best Overall Response as per iRECIST Stage 1 & 2
N (%)
Total N=36
Complete Response (CR)

0 (0)
Partial Response (PR)

2 (5.6)
Stable Disease (SD)

11 (30.6)
Progressive Disease (PD)

22 (61.1)
Not Evaluable

1 (2.8)
Overall Response Rate (ITT)

2/36 (5.6)
Disease Control Rate (ITT)

13/36 (36.1)
Overall Response Rate (evaluable patients)

2/35 (5.7)
Disease Control Rate (evaluable patients)

13/35 (37.1)

A pseudo-progression refers to an increase in the apparent size of a tumour or number of metastases on an imaging test, that can falsely create the appearance of disease progression. This may be caused by the infiltration of immune cells into the tumour site or a delay in the development of an adaptive immune response following immunotherapy.

Safety

The combination of efti plus pembrolizumab is safe and well-tolerated, continuing efti’s good safety profile to date and compares favourably to standard of care chemotherapy options.

Conclusion

The interim data from the TACTI-002 study shows that efti in combination with pembrolizumab is demonstrating encouraging early signs of antitumour activity in 2nd line confirmed PD-X refractory, NSCLC patients.

Next results

More mature data from the 2nd line NSCLC cohort is planned to be presented later this year, along with other data from the TACTI-002 trial.

About the TACTI-002 Trial

TACTI-002 (Two ACTive Immunotherapies) is being conducted in collaboration with Merck & Co., Inc., Kenilworth, NJ, USA (known as "MSD" outside the United States and Canada). The study is evaluating the combination of eftilagimod alpha (efti) with MSD’s KEYTRUDA (pembrolizumab) in patients with second line head and neck squamous cell carcinoma or non-small cell lung cancer in first and second line.

The trial is a Phase II, Simon’s two-stage, non-comparative, open-label, single-arm, multicentre clinical study that is taking place in study centres across Australia, Europe, and the US.

Patients participate in one of the following:

Part A – first line Non-Small Cell Lung Cancer (NSCLC), PD-X naïve – given the promising results of the first two stages of Part A, an expansion stage with 74 additional patients was commenced in November 2020 to assist with trial design in subsequent late-stage settings

Part B – second line NSCLC, PD-X refractory

Part C – second line Head and Neck Squamous Cell Carcinoma (HNSCC), PD-X naïve

TACTI-002 is an all-comer study in terms of PD-L1 status, a well-known predictive marker for response to pembrolizumab monotherapy especially in NSCLC and HNSCC.

On March 30, 2022 Zealand Pharma A/S (Nasdaq: ZEAL) (CVR-no. 20045078,) a biotechnology company focused on the discovery and development of innovative peptide-based medicines, reported a corporate restructuring intended to leverage its peptide platform by prioritizing investment in its research and development pipeline programs and streamline its commercial operations (Press release, Zealand Pharmaceuticals, MAR 30, 2022, View Source [SID1234611227]).

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The changes will refocus the Zealand’s resources reducing expenses while investing in strategic development and commercialization partnerships of pipeline assets. Dr Adam Steensberg will replace Emmanuel Dulac as Chief Executive Officer of Zealand as of 30 March 2022.

"The Board is focused on ensuring that the company continues to play to its strengths and believes that Dr. Adam Steensberg, its current Executive Vice President of Research & Development and Chief Medical Officer, has the right skill set and experience having developed the company’s rich and deep pipeline under his tenure," said Martin Nicklasson, Chairman of the Board of Zealand Pharma. "I want to thank Emmanuel for his stewardship of the company over the last three years."

Following a review of all business operations, Zealand plans to maximize the value of V-Go and Zegalogue through strategic partnerships and will restructure its commercial organization in the United States while ensuring continuity of services by maintaining patient, physician, and payor support for V-Go and Zegalogue. Zealand will also seek commercial partnership opportunities for its late-stage clinical pipeline programs as it looks to further leverage its peptide platform though strategic collaborations. With the restructuring, the US workforce will be reduced 90% by Q3 of this year with additional cost reductions implemented in Denmark.

"I realize that these changes impact employees in our organization and we are grateful to all our colleagues for their dedication and all they have done to improve the lives of patients," said Adam Steensberg, President and Chief Executive Officer of Zealand Pharma. "We have made the decision to restructure because we believe that seeking commercial partnerships will generate more value for the company and shareholders as we transform the company into a more focused and cost-effective organization. By improving our operational efficiency and targeting business development efforts, we will be in position to fully leverage the value of our most advanced assets and develop new peptide-based therapies. We have a strong R&D pipeline with Phase III readouts this year for dasiglucagon in CHI and, glepaglutide in SBS in the second and third quarters respectively, and Phase I data for our Amylin analogue targeting obesity later this year."

With the restructuring, the company is updating its financial guidance for 2022.

Net operating expenses in 2022 are now expected to be DKK 1,000 million +/-10%. This is a decrease of DKK 200 million from the prior guidance issued on March 10, 2022, and due to the change in commercial strategy, net product revenue from the sales of commercial products is now expected to be DKK 115 million +/- 10%. This is a decrease of DKK 125 million from the prior guidance and does not include any revenue from existing license agreements or from any potential partnerships. If such partnerships occur, Zealand will update the financial guidance accordingly.

On March 30, 2022 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported that financial results and a business update for the full fiscal year ended December 31, 2021 (Press release, VolitionRX, MAR 30, 2022, View Source [SID1234611226]). Volition management will host a conference call tomorrow, March 31 at 8:00 a.m. U.S. Eastern Time to discuss these results. Conference call details may be found below.

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"I could not be prouder of the team’s achievement in securing a global licensing and supply agreement for our Nu.Q Vet Cancer Screening Test with Heska Corporation, one of the industry’s leading companies," commented Cameron Reynolds, President and Group Chief Executive Officer of Volition. "Volition Veterinary is an exciting, fast-moving part of our business with clear potential to generate significant revenue for the company, initially under our agreements with Heska and SAGE Healthcare, through both milestone payments and ongoing sales of kits and key components. We have also made good progress in other key areas including our Nu.Q NETs and Nu.Q Capture programs as we shift gears towards our goal of becoming a commercial company with products."

An interview with Cameron Reynolds, President and Group Chief Executive Officer of Volition, Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development LLC, and Terig Hughes, Group Chief Financial Officer of Volition: View Source

Company Highlights

Financial

Cash and cash equivalents as of December 31, 2021, totaled approximately $20.6 million compared with $19.4 million as of December 31, 2020.
Subsequent to year end, Volition is due to receive a $10 million upfront payment as a result of the execution of the agreement with Heska Corporation.
Cash burn rate averaged approximately $2 million per month.
Net loss for the full year of 2021 was $27 million versus $20.4 million for 2020 with this increase primarily due to non-cash items in addition to new hire staffing costs.
Signed our first Nu.Q Discover agreement in 2021 and subsequent to year end have signed an additional five agreements worth an aggregate of approximately $200,000 on a full-year basis.
Made strong progress in improving internal controls and processes and anticipate the remaining weakness regarding segregation of duties in finance will be resolved in 2022.
Commercial Opportunity

We estimate our annual total addressable market ("TAM") to be approximately $70 billion worldwide including Nu.Q Vet, Nu.Q Discover, Nu.Q NETs and Nu.Q Cancer.
All estimates are global and on an annualized basis using key assumptions as follows:
We estimate the Nu.Q Vet TAM to be approximately $11 billion based on global canine and feline populations that are eligible for screening and monitoring and a price assumption of $50 per test to the consumer.
For Nu.Q Discover we estimate the TAM to be over $200 million using drug pipeline data of registered clinical trial programs for relevant epigenetic targets.
We estimate the Nu.Q NETs TAM to be approximately $22 billion calculated using estimated hospital admissions and discharges for sepsis and the associated average length of stay.
We estimate our Nu.Q Cancer TAM to be approximately $37 billion calculated based on eligible populations for annual screening, target participation rates and incidence/prevalence of specific cancers, and risk stratification use cases.
Price per test assumptions for Nu.Q and Nu.Q NETs range from $120 for the U.S., $50 for Europe and $25 for the rest of the world.
Personnel/ Operational

To support our commercial expansion, we welcomed Terig Hughes as our Chief Financial Officer, Gael Forterre as our Chief Commercial Officer, Dr. Tom Butera as the Chief Executive Officer of our veterinary subsidiary, and Mr. Nick Plummer as Group General Counsel.
In addition, Gaetan Michel was promoted to Chief Operating Officer and has relocated to the U.S. to accelerate the development of Volition America.
Dr. Terry Kelly was promoted to Chief Innovation Officer and is building a team focused on cutting-edge, discovery research at our new innovation hub which opened in California in March 2022.
Volition Veterinary

Executed a global licensing and supply agreement with one of the industry’s leading companies, Heska Corporation.
In exchange for granting Heska exclusive worldwide rights to sell the Nu.Q Vet Cancer Screening Test for companion animals at the Point of Care, Volition will receive:
a $10 million upfront payment on signing,
up to $18 million based upon the achievement of near/mid-term milestones, and
ongoing additional revenue for key components.
In addition, Volition has granted Heska non-exclusive rights to sell the Nu.Q Vet Cancer Screening Test in kit format for companion animals, through Heska’s network of central reference laboratories for which Volition will receive ongoing additional revenue for such kit sales.
Appointed SAGE Healthcare as a non-exclusive licensee and distributor of our Nu.Q Vet Cancer Screening Test for the Asian market.
We remain in advanced negotiations with other potential licensing partners in our efforts to make Nu.Q Vet as accessible as possible worldwide and anticipate further announcements in 2022.
Published two peer-reviewed clinical papers and two abstracts at the Veterinary Cancer Society Annual Conference including the first data demonstrating the use of Nu.Q Vet as a disease monitoring tool.
Clinical – NETosis including COVID-19

Volition believes the Nu.Q NETs assay will have wide applicability for monitoring diseases with a NETs component (such as COVID-19, influenza, sepsis, autoimmune diseases, and cancer) and potentially to risk-stratify patients for treatment selection.
Volition expects to register CE marks on its first NETs product, across multiple platforms including ELISA plate, automated beads, and a proof of concept on a very high throughput platform by the summer of 2022.
Volition intends to register the Nu.Q NETs product with a broad almost C-reactive protein (CRP) style claim "for the detection and evaluation of infection, tissue injury, inflammatory disorders and diseases associated with NETosis."
Posters published during 2021 (at the International Society on Thrombosis and Haemostasis Congress) showed that results on admission using the same Nu.Q NETs assay could predict future COVID-19 disease severity and that serial results correlate with disease progression including 28-day mortality.
Further larger studies have been completed in COVID-19 and sepsis which are now awaiting publication, as well as studies in other diseases in progress, with results expected in the coming quarters.
Clinical – Cancer

Published data at ASCO (Free ASCO Whitepaper) GI 2022 for two colorectal cancer studies (asymptomatic and symptomatic populations) conducted with the National Taiwan University.
Results demonstrated that using Nu.Q assays in conjunction with FIT could potentially reduce unnecessary colonoscopies by up to 28% in patients with gastrointestinal symptoms and that this dual approach could also improve the effectiveness of FIT-based screening programs for asymptomatic patients.
The Lung Cancer team at the National Taiwan University has completed collection and preliminary analysis and submitted abstracts to upcoming conferences.
Volition has also undertaken active and continuing negotiations in Asia, in addition to platform development on its first human cancer launch in China.
Collection for the U.S. Early Detection Research Network ("EDRN") study restarted in June 2021 and enrollment has been slow but steady. The EDRN has diverted some efforts to drive recruitment and it still anticipates study completion in the fourth quarter of 2022.
With regards to Volition’s U.S. blood cancer studies, the timing of expected completion for each has been impacted by the COVID-19 pandemic due to sample collection and protocol issues.
Given the pandemic, and resultant delays in the larger Non-Hodgkin Lymphoma ("NHL") study (of 1500 subjects), the Company has taken the opportunity to upgrade the platform to a high-throughput platform which will help facilitate an FDA compliant product. Consequently, Volition now expects this study to initiate recruitment in the second quarter of 2022 with study completion anticipated in 2023.
A smaller, proof of concept NHL monitoring study is ongoing. However, due to missing serial samples as a result of pandemic collection issues, we are not in a position to publish yet. Collection continues and we anticipate analyzing a more complete data set in 2022.
Nu.Q Capture

The Nu.Q Capture program now has several strands of technology which:
essentially remove background noise, thereby amplifying the signal,
look to identify the signal in a novel way including through mass spectrometry, or
isolate various chromatin fragments, including nucleosomes and transcription factors.
Nu.Q Capture, when used in combination with either sequencing, mass spectrometry and/or Volition’s Nu.Q assays could potentially aid diagnosis, treatment selection, and both treatment and disease monitoring in addition to aiding biomarker discovery.
Further to the publication in 2021 of a novel method utilizing Nu.Q Capture and mass spectrometry, Volition is developing a large 1000-plus patient study in lung cancer and colorectal cancer with further announcements expected in 2022.
Intellectual Property

29 patent families (plus three in-licensed families) covering both human and animal use of Volition’s Nucleosomics platform.
84 granted patents (12 in the U.S., 14 in Europe, and 58 rest of world).
93 patents pending worldwide.
Continued focus on filings and expect portfolio to grow in the quarters and years ahead.
Publications

In 2021, data for the Nu.Q platform was presented at five international conferences and Volition has collaborated on four clinical papers.
In 2022, to date, two posters were presented at the ASCO (Free ASCO Whitepaper) GI 2022 conference.
These publications are another very important step forward for the Company.
Upcoming Milestones

Drive near term revenue in the following key areas:
Licensing of its technology, with a particular but not exclusive focus on Nu.Q Vet.
Complete Heska Corporation agreement milestones in order to receive further milestone payments.
Sales of key components of Point of Care test with Heska.
Sales of kits from non-exclusive agreements for the use of Nu.Q Vet via central reference labs.
Ongoing and new Nu.Q Discover agreements.
Sales of its disease monitoring tests (e.g. COVID-19, sepsis).
Continue to progress the research program for the use of Nu.Q in NETosis, in monitoring disease progression of COVID-19, sepsis, and potentially other diseases and as a possible companion diagnostic for a treatment for sepsis.
Continue to advance its previously announced large-scale blood, lung, and colorectal cancer trials in Europe, Asia, and the U.S.
Publish several abstracts and peer-reviewed scientific papers with clinical results showing the robustness and utility of its Nu.Q platform.
Advance the development of Nu.Q Capture.
Continue to file patents to expand and extend its intellectual property portfolio.
VolitionRx Limited Full Fiscal 2021 Financial Results and Business Update

Cameron Reynolds, President and Group Chief Executive Officer of Volition, will host the call along with Terig Hughes, Group Chief Financial Officer of Volition, Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development LLC, and Scott Powell, Executive Vice President, Investor Relations of Volition.

A live audio webcast of the conference call will also be available on the investor relations page of Volition’s corporate website at View Source

In addition, a telephone replay of the call will be available until April 14, 2022. The replay dial-in numbers are 1-844-512-2921 (toll-free) in the U.S. and Canada and 1-412-317-6671 (toll) internationally. Please use replay pin number 13728406.

On March 30, 2022 LianBio (Nasdaq: LIAN), a biotechnology company dedicated to bringing innovative medicines to patients in China and other major Asian markets, reported financial results for the fourth quarter and year ended December 31, 2021 (Press release, LianBio, MAR 30, 2022, View Source [SID1234611224]).

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"We are proud of our progress over the past year, highlighted by the achievement of key clinical and corporate milestones, including the initiation of the Phase 3 EXPLORER-CN trial of mavacamten in Chinese patients with symptomatic oHCM, and the approval of infigratinib for the treatment of cholangiocarcinoma patients in the Hainan Province in China," said Yizhe Wang, Ph.D., Chief Executive Officer of LianBio. "LianBio continues to advance our pipeline of clinically validated therapeutic candidates and we plan to initiate pivotal studies of three additional programs, TP-03, NBTXR3 and infigratinib, in China by year-end. We remain focused on our cross-border development strategy and are committed to building our pipeline of novel medicines for patients in Greater China and other Asian markets."
Recent Business Highlights and Clinical Development Updates
Phase 3 EXPLORER-CN trial of mavacamten in symptomatic oHCM patients initiated and PK trial dosing completed

•In November 2021, LianBio initiated and completed enrollment and dosing in a pharmacokinetic (PK) study of mavacamten in healthy Chinese volunteers.

•In January 2022, LianBio initiated the Phase 3 EXPLORER-CN clinical trial of mavacamten in Chinese patients with symptomatic oHCM.

•In February 2022, LianBio’s partner Bristol Myers Squibb (BMS) announced positive topline results from the Phase 3 VALOR-HCM trial, evaluating mavacamten in patients with oHCM who are eligible for septal reduction therapy.

image_0a.jpg

•In February 2022, LianBio announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation in China for mavacamten for the treatment of patients with oHCM.

Infigratinib approved for the treatment of CCA in the Bo’ao pilot zone of Hainan Province
•In December 2021, infigratinib received approval from the Health Commission and Medical Products Administration of Hainan Province, under the special Named Patient Program (NPP), for the treatment of patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or other rearrangement. The approval enables early commercial access to infigratinib in the Bo’ao Lecheng International Medical Tourism Pilot Zone based on the drug’s approval in other global jurisdictions.

Expanded leadership team with key executive hires and named two additions to board of directors
•In October 2021, LianBio named Michael Humphries, MBBS, as Chief Scientific Advisor to guide research and development (R&D) strategy, advance the pipeline, and lead assessment of new in-licensing opportunities.
•In October 2021, LianBio appointed Jesse Wu and Susan Silbermann to the Board of Directors.
Raised $334.5 million of gross proceeds from IPO
•In November 2021, LianBio raised gross proceeds of $334.5 million and aggregate net proceeds of $304.8 million in connection with its initial public offering and subsequent exercise of the underwriters’ option to purchase additional ADSs.

Key Anticipated Milestones Expected in 2022
Mavacamten
BMS-partnered cardiac myosin inhibitor in development for the treatment of hypertrophic cardiomyopathy and certain forms of heart failure
•LianBio’s partner BMS has announced a Prescription Drug User Fee Act (PDUFA) target action date of April 28, 2022 for its New Drug Application to the U.S. Food and Drug Administration (FDA) for mavacamten for the treatment of patients with oHCM.
TP-03
Tarsus Pharmaceuticals-partnered GABA-Cl channel blocker in development for the treatment of Demodex blepharitis (DB) and meibomian gland disease
•LianBio expects to initiate a Phase 3 trial of TP-03 in Chinese patients with DB in the second half of 2022 to support regulatory approval in China.
•LianBio’s partner Tarsus has announced that it expects to report topline data in April 2022 from the ongoing Phase 3 Saturn-2 trial of TP-03 in DB patients.
NBTXR3
Nanobiotix-partnered radioenhancer in development for multiple solid tumor indications
•LianBio expects to begin dosing Chinese patients in Nanobiotix’s ongoing global pivotal Phase 3 trial of NBTXR3 for the treatment of locally advanced head and neck squamous cell carcinoma in elderly patients ineligible for cisplatin in the second half of 2022.

image_0a.jpg

Infigratinib
QED-partnered FGFR1-3 inhibitor in development for the treatment of individuals with FGFR-driven cancers
•LianBio expects to begin dosing Chinese patients in QED’s ongoing global pivotal Phase 3 PROOF-301 trial of infigratinib in first-line CCA patients with FGFR2 gene fusions/translocations in the second half of 2022.

Fourth Quarter and Full-Year 2021 Financial Results:
Research & Development Expenses

R&D Expenses: Research and development expenses were $7.7 million for the fourth quarter of 2021 compared to $2.7 million for the fourth quarter of 2020, and $158.7 million for the year ended December 31, 2021 compared to $120.9 million for the year ended December 31, 2020. The increase was primarily attributable to increased milestone payments and development activities to support clinical trials and personnel-related expenses (including share-based compensation expense) as a result of increased employee headcount, development activities to support our clinical trials and professional fees.

General & Administrative Expenses

G&A Expenses: General and administrative expenses were $14.4 million for the fourth quarter of 2021 compared to $6.5 million for the fourth quarter of 2020, and $36.9 million for the year ended December 31, 2021 compared to $14.0 million for the year ended December 31, 2020. The increase was primarily attributable to increases in payroll and personnel-related expenses (including share-based compensation expense) for increased employee headcount and increases in legal service costs, consulting costs and accounting services.

Net Loss

Net Loss: Net loss was $21.2 million for the fourth quarter of 2021 compared to net loss of $12.7 million for the fourth quarter of 2020, and $196.3 million for the year ended December 31, 2021 compared to $139.6 million for the year ended December 31, 2020.

Cash Balance

Cash balance: Cash, cash equivalents, marketable securities, and restricted cash at December 31, 2021 totaled $403.2 million, reflecting a net increase of $148.9 million from December 31, 2020. LianBio projects its cash position is sufficient to fund current operations through 2023.