On March 31, 2022 TILT Biotherapeutics, a clinical-stage biotechnology company developing oncolytic immunotherapy for enabling therapies based on T cells, such as immune checkpoint inhibitors and adoptive cell therapies, reported that positive interim progress and safety data from its phase 1 clinical trials in metastatic melanoma (T215 – the ‘TUNINTIL’ trial) and solid tumors (T115 – the ‘TUNIMO’ trial) (Press release, TILT Biotherapeutics, MAR 31, 2022, View Source [SID1234611203]). These two trials, with patients in Denmark, France, and Finland, are of the company’s oncolytic immunotherapy asset TILT-123, designed to stimulate T-cells to better fight cancer.

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TILT Biotherapeutics’ CEO, Akseli Hemminki, a biotech entrepreneur and cancer clinician who has personally treated almost 300 patients with ten different oncolytic viruses, said, "We are delighted that our phase 1 trial in solid tumors (T115), which consists of a total of 11 patients being treated to date, is progressing at pace. Our first-in-human melanoma (T215) trial is showing equally positive results, with 10 patients being treated and some now being treated under an extension protocol. I am thankful for the excellent work by the clinical teams. We are showing that the treatment is well tolerated, has prominent biological effects on tumors, and has the potential to increase the efficacy of immunotherapies, and deliver the anti-tumor benefits of armed oncolytic viruses."

The ‘TUNINTIL’ clinical trial (1) is a phase 1, open-label, dose-escalation study of the company’s oncolytic adenovirus coding for Tumor Necrosis Factor Alpha (TNF alpha) and Interleukin 2 (IL-2), known as TILT-123. A total of about 15 metastatic melanoma patients receive TILT-123 as an initial monotherapy over one month, followed by up to two administrations of tumor infiltrating lymphocytes (TILs) in the second month as well as ongoing consecutive doses of TILT-123, for up to 24 months.

The ‘TUNIMO’ clinical trial (2) is also a phase 1 trial of TILT-123. A total of 15-20 patients with solid tumors receive TILT-123 as a monotherapy over a three-month period, and, as with the TUNINTIL protocol, followed up by a 24- month extension period.

The primary objective of both trials is to evaluate the safety of TILT-123 and to deliver insights about the behavior of TILT-123 in humans, such as systemic tumor transduction following intravenous delivery and virus replication in the tumor, as well as immunological responses.

To date, most frequent adverse events across both trials have been fever, chills, and fatigue, consistent with the administration of an oncolytic adenoviral immunotherapy, with no treatment related serious adverse events.

The heart of TILT’s innovative approach revolves around the use of armed oncolytic adenoviruses, using cytokines and other molecules to boost the patient’s immune response to better enable it to find and destroy cancer cells. The company is advancing its pipeline of other assets and programs with TILT-123 towards further clinical trials, including in combination with the immune checkpoint inhibitors (3) (4).

On March 30, 2022 Oxilio, the pioneering oncology company, repurposing existing drugs to address unmet needs in cancer therapy, reported that it has acquired LSHS Consulting Limited in a share transaction (Press release, Oxilio, MAR 30, 2022, View Source [SID1234621607]).

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LSHS is a specialist life science consultancy firm established by Dr Dan Gooding, comprising intellectual property assets relevant to Oxilio’s OXL001 programme.

Dr Gooding was CEO of Nuformix plc until June 2020 and has significant experience in the pre-clinical and clinical development of formulations relevant to OXL001. Through him, Oxilio commenced discussions for the licensing of Nuformix’s NXP-001 cocrystal aprepitant formulation for the treatment of cancer.

Dr Gooding has been instrumental in helping Oxilio make rapid scientific and commercial progress with both our co-crystal and lipid-based formulation routes, in addition to the exploration of further formulation technologies which remain under evaluation. Dr Gooding also introduced Oxilio to TRx Biosciences and facilitated the negotiation of our exclusive licence to use TRX’s lipid platform technology with our aprepitant formulation, again for the treatment of cancer. Since that time, Dr Gooding has become a Director of TRx Biosciences.

As a result of the transaction, Oxilio secures continuing access to Dr Gooding’s expertise and know-how in both our lipid and co-crystal product development routes.

Dr Gooding has undertaken much of our formulation development work with the TRx scientists and provided the additional capability and expertise Oxilio requires to transfer this technology to Quotient Sciences for clinical trials.

We are looking forward to continuing close co-operation with Dr Gooding across a range of scientific developments at Oxilio.

On March 30, 2022 Iktos reported a collaboration agreement with Ono Pharmaceuticals Co., Ltd., a leading pharmaceutical company based in Japan with a focus on innovative medicines dedicated to the fight against disease and pain (Press release, Iktos, MAR 30, 2022, View Source [SID1234613366]). Under the agreement, Iktos will apply its de novo ligand and structure-based generative modelling technologies and software Makya, its AI-based retrosynthesis analysis and planning tool Spaya, and know-how complementing Ono’s drug discovery capabilities to expedite the identification of potential pre-clinical candidates and to identify additional novel chemical matter with suitable properties for Ono’s drug discovery programmes.

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On March 30, 2022 EDAP TMS SA (Nasdaq: EDAP) (the "Company"), a global leader in robotic energy-based therapies, reported unaudited financial results for the fourth quarter and full year 2021 (Press release, EDAP TMS, MAR 30, 2022, View Source [SID1234611308]).

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Marc Oczachowski, EDAP’s Chairman and Chief Executive Officer, said: "We finished 2021 on a positive note, having reported several U.S. Focal OneTM installations at renowned academic and community hospitals while growing our pipeline opportunities to their highest level since receiving regulatory approval of Focal One in mid-2018. At the same time, US HIFU treatment volumes, our most important leading indicator, were up 65% for the full year, reflecting ongoing growth in adoption of focal therapy by urologists. We continued to make measured investments in our US team, and with growing support for focal therapy as a necessary part of any comprehensive prostate oncology program, we entered 2022 with a right-sized, world class organization capable of capturing this significant untapped opportunity with our state-of-the-art HIFU technology."

Full Year 2021 Results

Total revenue for the full year 2021 was EUR 44.1 million (USD 51.9 million), an increase of 5.8% from total revenue of 41.7 million (USD 47.8 million) for the full year 2020.

Total revenue in the HIFU business for the full year 2021 were EUR 9.9 million (USD 11.7 million), as compared to EUR 11.4 million (USD 13.1 million) for the full year 2020.

Total revenue in the LITHO business for the full year 2021 was EUR 11.0 million (USD 13.0 million), as compared to EUR 12.9 million (USD 14.8 million) for the full year 2020.

Total revenue in the Distribution business for the full year 2021 was EUR 23.1 million (USD 27.3 million), as compared to EUR 17.3 million (USD 19.9 million) for the full year 2020.

Gross profit for the full year 2021 was EUR 18.4 million (USD 21.7 million), compared to EUR 18.4 million (USD 21.1 million) for the full year 2020. Gross profit margin on net sales was 41.8 % for the full year 2021, compared to 44.1% for the comparable period in 2020.

Operating expenses were EUR 20.0 million (USD 23.6 million) for the full year 2021 as compared to EUR 18.1 million (USD 20.8 million) for the full year 2020.

Operating loss for the full year 2021 was EUR 1.6 million (USD 1.9 million), compared to an operating profit of EUR 0.3 million (USD 0.3 million) for the full year 2020.

Net income for the full year 2021 was EUR 0.7 million (USD 0.8 million), or EUR 0.02 per diluted share, as compared to a net loss of EUR 1.7 million (USD 2.0 million), or EUR 0.06 per diluted share for the full year 2020.

Fourth Quarter 2021 Results

Total revenue for the fourth quarter 2021 was EUR 14.0 million (USD 15.9 million), as compared to total revenue of EUR 15.4 million (USD 18.3 million) for the same period in 2020.

Total revenue in the HIFU business for the fourth quarter 2021 was EUR 4.2 million (USD 4.8 million), as compared to EUR 4.4 million (USD 5.2 million) for the fourth quarter of 2020.

Total revenue in the LITHO business for the fourth quarter 2021 was EUR 3.3 million (USD 3.8 million), as compared to EUR 4.6 million (USD 5.5 million) for the fourth quarter of 2020.

Total revenue in the Distribution business for the fourth quarter 2021 was EUR 6.4 million (USD 7.3 million), as compared to EUR 6.4 million (USD 7.6 million) for the fourth quarter of 2020.

Gross profit for the fourth quarter 2021 was EUR 6.2 million (USD 7.1 million), compared to EUR 7.0 million (USD 8.4 million) for the year-ago period. Gross profit margin on net sales was 44.5 % in the fourth quarter of 2021, compared to 45.8% in the year-ago period. The decrease in gross profit year-over-year was due to lower sales effect on fixed costs.

Operating expenses were EUR 5.8 million (USD 6.5 million) for the fourth quarter of 2021, compared to EUR 5.3 million (USD 6.3 million) for the same period in 2020.

Operating profit for the fourth quarter of 2021 was EUR 0.5 million (USD 0.5 million), compared to an operating profit of EUR 1.7 million (USD 2.1 million) in the fourth quarter of 2020.

Net income for the fourth quarter of 2021 was EUR 1.4 million (USD 1.6 million), or EUR 0.04 per diluted share, as compared to net income of EUR 0.8 million (USD 0.9 million), or EUR 0.03 per diluted share in the year-ago period.

As of December 31, 2021, the company held cash and cash equivalents of EUR 47.2M (USD 53.4M) as compared to EUR 24.7 million (USD 30.2 million) as of December 31, 2020.

Conference Call

An accompanying conference call and webcast will be conducted by management to review the results. The call will be held at 8:30am EDT tomorrow, March 31, 2022. Please refer to the information below for conference call dial-in information and webcast registration.

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.