On May 4, 2021 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, reported operating results for the first quarter ended March 31, 2021 and provided pipeline updates (Press release, AnaptysBio, MAY 4, 2021, View Source [SID1234579061]).

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"We look forward to advancing imsidolimab into a Phase 3 trial for GPP following our recent end-of-Phase 2 meeting with the FDA," said Hamza Suria, president and chief executive officer of AnaptysBio. "AnaptysBio continues to pursue a capital-efficient business model where partnership revenues continue to support advancement of our wholly-owned pipeline poised to generate multiple clinical data catalysts through 2021 and 2022."

Imsidolimab (Anti-IL-36 Receptor) Program

We held an end-of-Phase 2 meeting with the FDA during Q2 2021 to review an orphan disease Phase 3 development plan for imsidolimab for the treatment of GPP and anticipate announcing key aspects of our Phase 3 trial design upon its initiation in mid-2021. Our worldwide registry of GPP patients, named RADIANCE, is ongoing, and we expect that this study will improve our understanding of the patient journey and support enrollment of our Phase 3 clinical trial. While initial GPP epidemiology studies suggested at least 3,000 GPP patients in the United States, medical claims analyses conducted by IQVIA indicate approximately 37,000 unique patients were diagnosed with GPP at least once, and approximately 15,000 unique patients were diagnosed with GPP at least twice, by a physician between 2017 and 2019 using the International Classification of Diseases 10th Revision (ICD-10) billing code pertaining to GPP (L40.1).
We initiated a Phase 2 clinical trial of imsidolimab in hidradenitis suppurativa, named HARP, where 120-patients are randomized equally between two dose levels of imsidolimab and placebo, and top-line data is anticipated in H2 2022. We also commenced an imsidolimab Phase 2 trial in moderate-to-severe acne, named ACORN, where 120-patients are randomized equally between two dose levels of imsidolimab and placebo, and top-line data is anticipated in H1 2022. We continue to anticipate top-line data at the end of 2021 from our EMERGE Phase 2 trial of imsidolimab in EGFR/MEK-mediated skin toxicities and top-line data from our Phase 2 INSPIRE trial in ichthyosis during 2022.
We announced in Q1 2021 top-line data from our POPLAR phase 2 clinical trial of imsidolimab monotherapy in PPP, which failed to meet the trial’s primary endpoint. While we continue to review secondary endpoints to further understand the activity of imsidolimab in various PPP patient subsets, we do not currently plan to pursue further clinical development of imsidolimab in PPP.
ANB030 (Anti-PD-1 Agonist) Program

We anticipate top-line data in H2 2021 from our ongoing Phase 1 healthy volunteer clinical trial of ANB030, our wholly-owned PD-1 agonist antibody, designed to assess the safety, pharmacokinetics and pharmacodynamics of ANB030 in single and multiple ascending dose cohorts.
We plan to initiate Phase 2 clinical trials of ANB030 in alopecia areata and vitiligo in Q4 2021.
Preclinical translational data using ANB030 was presented in March 2020 at the Festival of Biologics Meeting.
ANB032 (Anti-BTLA Modulator) Program

We achieved first-in-human dosing of ANB032, our wholly-owned BTLA modulator antibody, upon initiation of a healthy volunteer Phase 1 trial in the first quarter of 2021, under an Australian Clinical Trial Notification (CTN), and anticipate top-line data from this trial during the first half of 2022.
We presented preclinical data regarding ANB032 at the 2020 Federation of Clinical Immunology Societies (FOCIS) Virtual Annual Meeting in October 2020.
GSK Partnered Programs

A BLA for our most advanced partnered program, which is an anti-PD-1 antagonist antibody called JEMPERLI (dostarlimab), was approved by the FDA in April 2021 for the treatment of advanced or recurrent deficient mismatch repair endometrial cancer (dMMREC). This is the first AnaptysBio-generated antibody, of eight currently under clinical development, to obtain FDA approval. We earned a $20.0 million milestone payment as a result of this FDA approval.
In April 2021 the European Medicines Agency (EMA) granted conditional marketing authorization in the European Union for JEMPERLI for use in women with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) recurrent or advanced endometrial cancer who have progressed on or following prior treatment with a platinum containing regimen, which approval makes JEMPERLI the first anti-PD-1 therapy available for endometrial cancer in Europe. We earned a $10.0 million milestone payment as a result of this approval.
A second BLA submitted by GSK was accepted by the FDA during the first quarter of 2021 for JEMPERLI in pan-deficient mismatch repair tumors (PdMMRT). We received a $10.0 million cash milestone payment upon the FDA acceptance of GSK’s second FDA BLA for JEMPERLI and anticipate an additional $20.0 million cash milestone payment upon FDA approval of this second FDA BLA of JEMPERLI during the second half of 2021. We anticipate an additional $15.0 million and $165.0 million in milestone payments upon achievement of certain JEMPERLI regulatory and commercial milestones, respectively.
During Q1 2021, we recognized $1.2 million in royalty revenue related to GSK’s Zejula product sales, which we estimated based on GSK’s historical sales. In October 2020, we amended our GSK collaboration which resulted in increased royalties on global net sales of JEMPERLI to 8-25%, a 1% royalty rate on GSK’s global net sales of Zejula and a one-time cash payment of $60.0 million.
First Quarter Financial Results

Cash, cash equivalents and investments totaled $387.4 million as of March 31, 2021 compared to $411.2 million as of December 31, 2020, for a decrease of $23.8 million. The decrease relates primarily to cash used for operating activities.
Collaboration revenue was $11.2 million for the three months ended March 31, 2021, $10.0 million related to milestone revenue for the FDA accepted BLA filing of the second dostarlimab indication and $1.2 million related to royalties on GSK’s Zejula product sales, compared to $15.0 million of milestone revenue for the three months ended March 31, 2020.
Research and development expenses were $24.2 million for the three months ended March 31, 2021, compared to $21.0 million for the three months ended March 31, 2020. The increase was due primarily to continued advancement of the Company’s clinical programs.
General and administrative expenses were $5.4 million for the three months ended March 31, 2021, compared to $4.3 million for the three months ended March 31, 2020. The increase was due primarily to personnel-related expenses, including share-based compensation.
Net loss was $18.2 million for the three months ended March 31, 2021, or a net loss per share of $0.66, compared to a net loss of $8.3 million for the three months ended March 31, 2020, or a net loss per share of $0.30.
Financial Guidance

AnaptysBio expects its net cash burn in 2021 will be close to $100 million. We anticipate that our cash, cash equivalents and anticipated revenues will fund our current operating plan at least into 2024.

On May 4, 2021 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that it has entered into a joint research agreement with BioArctic AB, a Swedish research-based biopharma company focusing on disease-modifying treatments and reliable biomarkers and diagnostics for neurodegenerative diseases, such as Alzheimer’s disease and Parkinson’s disease (Press release, Alligator Bioscience, MAY 4, 2021, View Source [SID1234579060]). Under the agreement, Alligator will employ its proprietary antibody generation technologies in collaboration with BioArctic to develop new product candidates.

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"We are very pleased that BioArctic has recognized the power of Alligator’s proprietary phage display libraries which have been successfully used to generate Alligator’s suite of immuno-oncology programs. The application of this technology to the neurodegenerative field demonstrates the broad applicability of our powerful platform," said Malin Carlsson, interim CEO of Alligator Bioscience.

"BioArctic’s focus is to develop new treatments to help patients with neurological diseases. We are looking forward to working together with Alligator, utilizing their phage display platform, in the development of new antibody therapeutic candidates with novel mechanisms of action," said Johanna Fälting, Vice President and Head of Research at BioArctic.

On May 4, 2021 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage biotechnology company leading the development of novel antibody drug conjugates (ADCs) to treat hematological malignancies and solid tumors, reported that Chris Martin, Chief Executive Officer, will participate in a fireside chat at two virtual investor conferences in May (Press release, ADC Therapeutics, MAY 4, 2021, View Source [SID1234579059]).

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Details of the events are as follows:

·BofA Securities 2021 Virtual Health Care Conference on Tuesday, May 11, 2021, at 8:00 a.m. ET

·2021 RBC Capital Markets Global Healthcare Conference on Wednesday, May 19, 2021, at 9:45 a.m. ET

A live webcast of each fireside chat will be available via the Events & Presentations page in the Investors section of ADC Therapeutics’ website, ir.adctherapeutics.com. A replay of each webcast will be available for approximately 30 days.

On May 4, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that it will release its first quarter 2021 financial results on Tuesday, May 11, after the close of the U.S. financial markets (Press release, Syndax, MAY 4, 2021, View Source [SID1234579045]).

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In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET on Tuesday, May 11, to discuss the Company’s financial results and provide a general business update.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website, www.syndax.com.

On May 4, 2021 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported that Ellen Kim, MD, Medical Director, Dermatology Clinic, Perelman Center for Advanced Medicine, Professor of Dermatology at the Hospital of the University of Pennsylvania, and the Lead Principal Investigator for the Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study, presented confirmatory data at the Society for Investigative Dermatology (SID) Virtual Meeting, held May 3-8, 2021 (Press release, Soligenix, MAY 4, 2021, View Source [SID1234579044]). The presentation was selected to be shown during a Concurrent Mini-Symposium for Patient-Targeted Research. The presented data demonstrated the ability of HyBryte (SGX301) to treat both patch and plaque disease, including generating complete disease responses, while being associated with fewer and less severe adverse events than other currently approved skin-directed therapies for cutaneous T-cell lymphoma (CTCL).

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Oral Presentation:

Topical hypericin ointment photodynamic therapy is effective and safe in CTCL (FLASH study) Dr. Kim’s presentation is archived on the SID Virtual Meeting site and will be accessible via registration until May 31, 2021. The abstract is available here and attendees can register to see the presentation here.

Key Highlights:

HyBryte is activated by visible light at a wavelength of 500-650 nm, which provides deeper dermal penetration than ultraviolet (UV) spectrum light. This resulted in statistically significant clinical responses observed in patches as well as deeper plaque lesions, which are typically more difficult to treat and generally less responsive to UV light therapy.
In addition to its demonstrated, statistically significant efficacy which ultimately led to 49% of patients achieving at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) after 18 weeks of therapy (p<0.0001), complete responses of all treated index lesions were also shown to occur. These complete responses increased in frequency as treatment with HyBryte continued and photographs demonstrating this response were reviewed.
Compared to other, second-line, approved drugs for the treatment of CTCL, HyBryte demonstrated significantly less safety concerns. This was reflected in the low rate of study discontinuation attributed to adverse events which showed only a 5% overall drop-out rate during the treatment phase in HyBryte treated patients, lower than typically observed in other early stage CTCL trials.
"Additional CTCL therapies with fewer and less severe side effects are desperately needed in our field," noted Dr. Kim. "I believe the efficacy and safety we saw HyBryte demonstrate in this trial proves that it can help us fill that need. This study was the largest multicenter, randomized, double-blind, placebo-controlled skin directed therapy study in MF/CTCL (mycosis fungoides/cutaneous T-cell lymphoma) to date and I would like to thank all site investigators, and especially our patients, for their contributions to developing this promising new therapy."

About the Society for Investigative Dermatology Virtual Meeting

The SID meeting is an annual meeting, dedicated to a broad range of dermatology related topics, as described here. SID’s mission is to advance the sciences relevant to skin diseases through education, advocacy and scholarly exchange of scientific information.

About HyBryte

HyBryte (SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light 16 to 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).

The Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions. Follow-up visits were completed in Q4 2020, and the clinical study report to support the NDA is in the process of being finalized.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte’s mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.