On March 24, 2022 Aptevo Therapeutics Inc. (NASDAQ:APVO), a clinical-stage biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported its financial results for the year ended December 31, 2021 and provided 2021 business highlights (Press release, Aptevo Therapeutics, MAR 24, 2022, View Source [SID1234610925]).

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"2021 was an eventful year for Aptevo Therapeutics. A year of progress against a backdrop of macro challenges at both the industry and global levels. Among our achievements was completing and announcing positive results for our Phase 1b dose escalation trial for lead drug candidate APVO436 in the treatment of acute myeloid leukemia, or AML, and myelodysplastic syndrome, or MDS. Those results showed a positive safety and tolerability profile and encouraging signs of clinical activity. More specifically, of 40 evaluable patients, promising clinical activity was observed in 11 of 40 or 27.5%, including two complete remissions among AML patients and three complete marrow responses among MDS patients," said Marvin L. White, President and Chief Executive Officer.

Mr. White continued, "Also on the APVO436 front, we initiated the second part of that trial — a dose expansion phase — structured as a multi-center, multi cohort study that will evaluate up to 90 adult AML patients in both monotherapy and in combination with standard of care chemotherapies. Most exciting, we announced a complete remission in the fourth quarter and have since shared that this patient is proceeding to transplant. Our pipeline also continues to progress, as we plan to file an Investigational New Drug Application (IND) for ALG.APV-527 for the treatment of solid tumors, later this year. We are excited about the opportunity to expand our clinical programs and view our progress as continued validation of our technology platforms."

"From a financial perspective we have sufficient cash for at least the next 12 months. Importantly, completion of enrollment of the APVO436 expansion trial and initiation of the ALG.APV-527 clinical program are expected inside this cash window," he concluded.

2021 Highlights

Announced results from the Phase 1b dose escalation trial evaluating lead drug candidate, APVO436, for the treatment of AML and MDS. Results showed:
APVO436 exhibited a favorable safety profile with acceptable tolerability and generally manageable drug-related adverse events.
Promising clinical activity was observed in 11 of 40 patients (27.5%) evaluable for efficacy. This included two complete remissions in patients with AML and three complete marrow responses in patients with MDS.
Initiated the dose expansion part of the APVO436 Phase 1b trial, evaluating adult patients with acute myeloid leukemia (AML) in a multi-center, multi-cohort study of up to 90 patients who will receive APVO436 in combination and monotherapy.
Published three articles in two peer-reviewed publications, Cancers and Frontiers in Medicine, discussing APVO436 data. Results were also presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in November 2021.
Announced that a high-risk AML patient treated with a combination of chemotherapy plus APVO436 achieved a complete remission after one cycle of therapy. The chemotherapy regimen included the standard leukemia drugs Mitoxantrone, Etoposide, and Cytarabine. The patient tolerated treatment without evidence of overt toxicity.
More recently, Aptevo reported that this patient will proceed to transplant.
Solidified plans with Alligator Bioscience to submit an IND for ALG.APV-527 in the second half of 2022 to evaluate the compound for the treatment of multiple solid tumor types.
Aptevo will investigate the initial differentiating benefits of ALG.APV-527 to induce stronger and more tumor-directed T cell activity with the potential for improved safety and efficacy than existing 4-1BB monoclonal antibody treatments
Received $35 million from our sale of the right to royalty payments made by Pfizer Inc. ("Pfizer") with respect to net sales of RUXIENCE to an entity managed by HealthCare Royalty Management, LLC ("HCR").
Earned a $10 million milestone payment related to 2021 sales of RUXIENCE under the terms of its royalty purchase agreement with HCR. Received the proceeds from the milestone payment in March 2022, which will be used to pay down our MidCap Financial term loan to $5 million, further strengthening the company’s balance sheet.
2021 Summary Financial Results

Cash Position: Aptevo had cash and cash equivalents as of December 31, 2021 totaling $46.3 million, including restricted cash of $1.3 million. The restricted cash is expected to be released over the next twelve months.

Royalty Revenue: Royalty revenue increased by $8.0 million from $4.3 million for the year ended December 31, 2020 to $12.3 million for the year ended December 31, 2021. The increase is related to royalty revenue from Pfizer on global net sales of RUXIENCE, a biosimilar to the drug RITUXAN, launched by Pfizer in early 2020. Due to our continuing involvement under the Definitive Agreement originally between Trubion and Wyeth, we continue to recognize royalty revenue on net sales of RUXIENCE and record the royalty payments to HCR as a reduction of the liability when paid. As such payments are made to HCR, the balance of the liability will be effectively repaid over the life of the Royalty Purchase Agreement. RUXIENCE is a trademark of Pfizer; RITUXAN is a trademark of Biogen.

Research and Development Expenses: Research and development expenses increased by $1.1 million, to $19.0 million for the year ended December 31, 2021 from $17.9 million for the year ended December 31, 2020. The increase is due to higher spending on consulting services for our APVO436 clinical trial as we continue to advance that trial and increased spending on analytical analysis for our preclinical programs, including ALG.APV-527, APVO603, and APVO442. Further, we have now started dosing in our Phase 1b expansion program for our APVO436 clinical trial.

General and Administrative Expenses: For the year ended December 31, 2021, general and administrative expenses increased by $0.7 million, or 5%, to $14.7 million from $14.0 million for the year ended December 31, 2020. This increase was primarily due to higher costs related to responding to stockholder activism matters and higher employee costs.

Other Expense: Other expense consists primarily of costs related to debt extinguishment, accrued exit fees on debt, non-cash interest on financing agreements, and interest on debt. Other expense, net was $8.0 million for the year ended December 31, 2021 and $3.4 million for the year ended December 31, 2020. This increase is primarily due to interest expense and accrued exit fees for the MidCap Credit Agreement, as well as non-cash interest expense for the Royalty Purchase Agreement.

Discontinued Operations: Income from discontinued operations was $1.0 million for the year ended December 31, 2021 and $13.2 million for the year ended December 31, 2020. For the year ended December 31, 2021, we collected $0.5 million related to the sale of hyperimmune business to Saol as a result of the collection of certain accounts receivable and deferred payment of $0.5 million received from Medexus related to IXINITY sales. For the year ended December 31, 2020, we recognized net income from discontinued operations totaling $13.2 million. This included the gain on the sale of Aptevo BioTherapeutics of $14.3 million, net operating losses from Aptevo BioTherapeutics of $1.6 million related to the period prior to the sale on February 28, 2020, and $0.4 million deferred payment from Medexus related to IXINITY sales.

Net Loss: Aptevo’s net loss for the year ended December 31, 2021 was $28.5 million or $6.07 per share, compared to a net loss of $17.8 million or $5.23 per share for the corresponding period in 2020.

On March 24, 2022 KemPharm, Inc. (NasdaqGS: KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, reported that Travis C. Mickle, Ph.D., President and CEO of KemPharm, will deliver a virtual presentation at Maxim’s 2022 Virtual Growth Conference being held March 28-30, 2022 (Press release, KemPharm, MAR 24, 2022, View Source [SID1234610924]).

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Registered investors will be able to view the pre-recorded presentation by Dr. Mickle highlighting KemPharm’s business and recent corporate achievements, as well as anticipated milestones. Members of the company’s management team will be available for one-on-one virtual meetings.

On March 24, 2022 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported a global collaboration and license agreement with Chugai Pharmaceutical Co., Ltd. that gives exclusive access to Halozyme’s ENHANZE drug delivery technology, recombinant human hyaluronidase PH20 enzyme (rHuPH20), for an undisclosed target (Press release, Halozyme, MAR 24, 2022, View Source [SID1234610923]). Chugai intends to explore the potential use of ENHANZE for a Chugai drug candidate.

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Under the terms of the agreement, Chugai will make an upfront payment of $25 million to Halozyme and is obligated to make potential future payments of up to $160 million in the aggregate, subject to achievement of specified development, regulatory and sales-based milestones. Halozyme will also be entitled to receive royalties on sales of commercialized medicines using the ENHANZE technology.

"We are thrilled to announce our latest collaboration and license agreement for ENHANZE and look forward to working closely with Chugai to advance this program," said Dr. Helen Torley, president and chief executive officer. "Our twelfth collaboration demonstrates the continued strong interest in providing patients with the option to receive treatment through a subcutaneous injection. This new collaboration further expands the number of Wave 5 potential launch programs, which we project will extend the revenue duration of our ENHANZE franchise."

Chugai represents Halozyme’s twelfth global collaboration and license partner for the ENHANZE technology. These collaborations cover more than 60 therapeutic targets and include five commercialized products to date.

About ENHANZE Technology

Halozyme’s proprietary ENHANZE drug-delivery technology is based on its patented recombinant human hyaluronidase enzyme (rHuPH20). rHuPH20 has been shown to remove traditional limitations on the volume of biologics that can be delivered subcutaneously (just under the skin). By using rHuPH20, some biologics and compounds that are administered intravenously may instead be delivered subcutaneously. ENHANZE may also benefit subcutaneous biologics by reducing the need for multiple injections. This delivery has been shown in studies to reduce health care practitioner time required for administration and shorten time for drug administration.

On March 24, 2022 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of refractive and retinal eye disorders, reported financial results for the fourth quarter and year ended December 31, 2021 and provided a corporate update (Press release, Ocuphire Pharma, MAR 24, 2022, View Source [SID1234610922]).

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"2021 proved to be a highly productive year for Ocuphire with 2022 setting up to be a more transformative year given our series of late-stage data read-outs in MIRA, LYNX and ZETA trials throughout the year, ending with our first planned NDA filing. We have an ambitious vision in ophthalmology targeting highly prevalent refractive and diabetic retinal diseases with our 2 lead small molecule drug candidates," said Mina Sooch, MBA, Founder and CEO of Ocuphire Pharma. "We are pleased to rapidly exceed enrollment in and complete 4 clinical trials across Nyxol and APX3330 in the first months of 2022. At our R&D Day in January, we reported for the first time positive Phase 2 results in presbyopia for Nyxol as a single-agent. With this new chronic opportunity for Nyxol alone as a pupil modulation agent, we can potentially realize synergies in presbyopia and NVD patients. We recently held a FDA Type-C meeting and gained clear guidance for the VEGA Phase 3 presbyopia program, for which we plan to initiate in mid-2022. With the successful enrollment of the 24-week study for our retinal candidate APX3330 and the continued favorable systemic and ocular safety profile that we shared at our recent R&D Day, we are also excited to lead the retinal landscape with an oral option for diabetic retinopathy patients and report our topline data from our placebo-controlled, double-masked, Phase 2b ZETA-1 trial in the second half of 2022."

Jay Pepose, MD, PhD, Ocuphire’s Medical Advisor and Board Member stated, "Ocuphire’s product candidates, if approved, would give eye care practitioners the ability to enhance their patients’ vision and overall experience. As a refractive surgeon, I am particularly excited about Nyxol for RM because there is currently no FDA approved commercially available product to treat this major clinical need and patient complaint. For presbyopia, I am impressed by Nyxol’s favorable tolerability profile and durable near vision improvements for at least 12 hours across a broad age of patients (40 to 64 years old) in the recent VEGA-1 Phase 2 trial. Nyxol is differentiated from the other miotics in the presbyopia landscape by its mechanism of action inhibiting the iris dilator muscle to achieve an optimal pupil size. Since Nyxol does not engage the iris sphincter or ciliary muscle, as a single drop, this may become a viable treatment option for presbyopia patients with high myopia, for whom miotics are contraindicated because of the risk of retinal detachment."

Cam Gallagher, Chairman of the Board for Ocuphire added, "In the past year, Ocuphire has elevated its profile within the ophthalmology and optometry medical community and we are delighted to have expanded our prestigious Medical Advisory Board to over 15 refractive and retinal KOLs. The team led by Mina has executed and delivered on several key clinical development milestones and set the momentum for a catalyst-rich 2022 that has the potential to build significant value for our company and shareholders."

Key Anticipated Future Milestones

Reversal of Mydriasis (RM):
MIRA-3: Report topline results from the Phase 3 MIRA-3 registration trial at the end of 1Q 2022
MIRA-4: Report topline results from pediatric safety trial in 2Q 2022
New Drug Application (NDA): If the results are positive from the ongoing MIRA trials, expect to file an NDA with the FDA for Nyxol in RM indication in late 2022 with potential launch as first dilation reversal drop in 2H 2023
Presbyopia: Initiate VEGA Phase 3 program in mid-2022 investigating Nyxol alone and Nyxol with 0.4% LDP as adjunctive treatment; and, if successful, expect to file an NDA in 2023
Night Vision Disturbances (NVD): Report top-line results in 2Q 2022 from the Nyxol Phase 3 LYNX-1 trial
Diabetic Retinopathy (DR) and Diabetic Macular Edema (DME): Report top-line results from the APX3330 Phase 2b ZETA-1 trial in 2H 2022
Fourth Quarter and Recent Business Highlights

Corporate

In January 2022, the Company held an Investor R&D Day webinar that featured six ophthalmic Key Opinion Leaders (KOLs): Jay Pepose, MD, PhD, James Katz, MD and Mitchell Jackson, MD from refractive surgery, Paul Karpecki, OD from optometry, and David Boyer, MD and Peter Kaiser from retina practice areas who discussed the unmet needs in RM, presbyopia and DR being addressed by Ocuphire’s two late-stage clinical drug assets, Nyxol and APX3300. A replay of the event can be found on the company’s corporate website here.
In December 2021, the Company strengthened its Medical Advisory Board with the addition of six world-class KOLs: David Brown, MD, FACS; David Lally, MD; Y. Ralph Chu, MD; James Katz, MD; Mitchell Jackson, MD; and Douglas Devries, OD.
Clinical Development

In March 2022, the Company completed enrollment of 103 (target of 80-100) diabetic retinopathy patients in the ZETA-1 Phase 2b trial of first-in-class oral APX3330. Masked safety data from the trial, announced during the R&D Day event in January 2022, demonstrated a favorable safety profile, consistent with prior studies with additional exposure data in diabetic patients with retinal disease.
In March 2022, the Company completed enrollment in MIRA-4 Trial for Nyxol in RM by enrolling 23 healthy (target of 20) pediatric subjects ages 3-11 years.
In February 2022, the Company completed enrollment in MIRA-3 Pivotal Phase 3 Trial for Nyxol in RM, surpassing its enrollment target of 330 with 368 patients ages 12 years and over.
In February 2022, Ocuphire held a Type-C meeting with the FDA from which it obtained guidance regarding the design of pivotal studies and clarification of the CMC and other data requirements for filing an NDA to seek approvals of Nyxol for the treatment of presbyopia, both as a single agent and with LDP as adjunct eye drops. This represents our third Type-C or Type-B End of Phase 2 meeting with FDA for the Nyxol program across indications.
In January 2022, the Company completed enrollment of LYNX-1 Phase 3 Trial investigating Nyxol for the treatment of night vision disturbances (NVD) in 145 patients (target of 140).
In January 2022, the Company announced new positive data from the VEGA-1 Phase 2 trial for Nyxol as a single agent in presbyopia, showing that one drop of Nyxol had statistically significant improvement in efficacy and long durability compared to placebo at 12 hours post-dosing. The Company plans to proceed with the Phase 3 VEGA program to potentially support 2 NDAs: Nyxol as a single drop and Nyxol with low-dose pilocarpine (LDP) as adjunctive treatment.
Presentations, Publications, and Conferences

In February 2022, David Boyer, MD, presented at the Angiogenesis, Exudation, and Degeneration Conference, highlighting the favorable safety data from the ongoing ZETA-1 Phase 2 trial of APX3330 in DR.
In February 2022, Inder Paul Singh, MD, presented at the Cataract Surgery: Telling It Like It Is Conference in Orlando. Dr. Singh presented the positive results from the completed VEGA-1 Phase 2 trial of Nyxol in presbyopia as a single agent and in combination with adjunctive LDP.
In January 2022, Mina Sooch, Founder and CEO participated in the panel discussion titled "The Role of Gender Equality in Changing the Life Sciences Investment and Innovation Landscape" at the 11th LifeSci Partners Corporate Access Event.
In November 2021, clinical data on Nyxol and APX3330 were presented at poster sessions at the American Academy of Ophthalmology (AAO) 2021 annual meeting held in New Orleans. In addition, Ocuphire presented new data on improvement in intermediate vision and Snellen equivalent near vision at the Eyecelerator@AAO 2021 conference. Ocuphire was one of two companies presenting clinical data for presbyopia at this meeting.
In October 2021, the Company announced the publication of a review article titled "Inhibition of APE1/Ref-1 for Neovascular Eye Disease: From Biology to Therapy" in the Special Issue "Advances in Molecular Activity of Potential Drugs" of the International Journal of Molecular Sciences. The article underscores the role of the APE1/Ref-1 protein in pro-angiogenic pathways associated with neovascular eye disease including diabetic retinal diseases and age-related macular degeneration.
In October 2021, the Company announced the publication of a review article in Cells titled "Potential Therapeutic Candidates for Age-Related Macular Degeneration" noting the potential of APX3330 (referred to as "E3330"). The authors conclude that APE1/Ref-1 inhibitors such as APX3330 could inhibit the abnormal blood vessel formation seen in AMD by reducing retinal endothelial cell proliferation, migration, and tube formation.
In October 2021, Michael J. Allingham, MD, PhD presented at the 39th Annual Scientific Meeting of the American Society of Retina Specialists (ASRS) (Diabetic Retinopathy 1 Symposium) held in San Antonio, highlighting the favorable safety and tolerability data for APX3330 in over 300 healthy volunteers and cancer/hepatitis patients across 11 Phase 1 and Phase 2 studies. In addition, Mina Sooch, CEO, presented APX3330 history and the design of the ongoing Phase 2 trial in DR at the OIS Retina Innovation Summit@ASRS on October 7, 2021 in San Antonio, TX.
Fourth Quarter and Year Ended December 31, 2021 Financial Highlights

As of December 31, 2021, the Company had cash and cash equivalents of approximately $24.5 million. Based on current projections, management believes the current cash on hand will be sufficient to fund operations into the second quarter of 2023. Net cash used in operating activities for the quarter and year ended December 31, 2021 was $5.6 million and $19.4 million, respectively.

No collaboration revenue was recorded in the fourth quarter. Collaborative revenue was $0.6 million for the year ended December 31, 2021. Revenue was derived from the collaboration and license agreements with Processa and Biosense related to certain Rexhan products and technology transfers. There was no collaboration revenue recognized during the comparable prior year periods.

General and administrative expenses for the quarter and year ended December 31, 2021 were $1.4 million and $8.1 million, respectively, compared to $1.3 million and $2.8 million for the quarter and year ended December 31, 2020, respectively. The $5.3 million increase for the year over year periods was primarily attributable to administrative employee headcount, stock-based compensation, insurance, legal and settlement costs, costs associated with operating as a public company subsequent to the reverse merger, and professional services and other operating costs. General and administrative expenses included $0.3 million and $0.2 million in non-cash stock-based compensation expense during the quarters ended December 31, 2021 and 2020, respectively, and $1.1 million and $0.7 million in non-cash stock-based compensation expense during the years ended December 31, 2021 and 2020, respectively.

Research and development expenses for the quarter and year ended December 31, 2021 were $4.7 million and $15.2 million, respectively, compared to $4.3 million and $6.6 million for the quarter and year ended December 31, 2020, respectively. The $8.5 million increase for the year over year periods was primarily attributable to clinical trial expense, manufacturing activities to support clinical advancement of Nyxol and APX3330, consulting services as well as regulatory and other research and development efforts. Research and development expenses also included $0.2 million and $0.3 million in non-cash stock-based compensation expense during the quarters ended December 31, 2021 and 2020, respectively, and $0.8 million in non-cash stock-based compensation expense during each of the years ended December 31, 2021 and 2020.

The loss from operations for the quarter ended December 31, 2021 was $6.2 million, compared to $14.0 million for the quarter ended December 31, 2020. The loss from operations for the year ended December 31, 2021 was $22.7 million, compared to $20.0 million for the year ended December 31, 2020. Net loss for the quarter ended December 31, 2021 was $6.3 million, compared to $18.7 million for the quarter ended December 31, 2020. Net loss for the year ended December 31, 2021 was $56.7 million, compared to $24.6 million for the year ended December 31, 2020. Net loss per share for the quarter ended December 31, 2021 was $0.35, compared to $2.46 for the quarter ended December 31, 2020. Net loss per share for the year ended December 31, 2021 was $3.82, compared to $5.28 for the year ended December 31, 2020.

The fair value change in derivative and warrant liabilities was a non-cash expense of zero for the quarter ended December 31, 2021 compared to a non-cash expense of $1.6 million for the quarter ended December 31, 2020. The fair value change in derivative and warrant liabilities was a non-cash expense of $33.8 million for the year ended December 31, 2021 compared to a non-cash expense of $1.5 million for the year ended December 31, 2020.

For further details on Ocuphire’s financial results, refer to the Company’s Annual Report on Form 10-K for the year ended December 31, 2021 to be filed with the Securities and Exchange Commission.

On March 24, 2022 Adamis Pharmaceuticals Corporation (NASDAQ: ADMP), a biopharmaceutical company developing and commercializing specialty products for allergy, opioid overdose, respiratory and inflammatory disease, reported that it will host an investor conference call on Thursday, March 31, 2022 at 2 p.m. Pacific Time to discuss its financial and operating results for the fourth quarter and full year 2021 as well as provide a business update (Press release, Adamis Pharmaceuticals, MAR 24, 2022, View Source [SID1234610921]). The company’s press release concerning its fourth quarter and 2021 financial results is expected to be available after 1 p.m. Pacific Time on March 31, 2022, and on its website. The company also expects to file its annual report on Form 10-K for the year ended December 31, 2021 on that date.

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Dennis J. Carlo, Ph.D., President and CEO of Adamis, will host the call along with other members of the management team. The call is open to the public and will provide an update on recent developments, events that have taken place during the year, and certain goals for future periods. Forward-looking statements concerning expectations regarding future company performance may be made during the conference call.

A live audio webcast of the conference call will also be available via this link, with a replay available shortly after the live event.