Biotech Alert: Searches spiking for these stocks today

These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include:

• Krystal Biotech (KRYS), 1,664% surge in interest
• Milestone Pharmaceuticals (MIST), 515% surge in interest
• Neu Base Therapeutics (NBSE), 449% surge in interest
• iBio (IBIO), 386% surge in interest
• Celldex Therapeutics (CLDX), 204% surge in interest
• Altimmune (ALT), 172% surge in interest
• Actinium Pharmaceuticals (ATNM), 110% surge in interest
• BioLineRx (BLRX), 62% surge in interest
• Apellis Pharmaceuticals (APLS), 61% surge in interest
• Cytokinetics (CYTK), 49% surge in interest

Pipeline and key clinical candidates for these companies:

Krystal Biotech is a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases. The company’s pipeline is based on its proprietary redosable HSV vector. In May, Vyjuvek was approved by the FDA for the treatment of dystrophic epidermolysis bullosa. Vyjuvek is a non-invasive, topical, redosable gene therapy designed to treat DEB at the molecular level.

Milestone Pharmaceuticals is a biopharmaceutical company developing and commercializing cardiovascular solutions for people living with complex and life-altering heart conditions. Milestone’s lead investigational product is etripamil, a novel calcium channel blocker nasal spray that is being studied for patients to self-administer without medical supervision to treat highly symptomatic episodic attacks associated with PSVT and AFib-RVR.

NeuBase Therapeutics is a biotechnology company developing Stealth Editors to perform in vivo gene editing without triggering the immune system.

iBio develops next-generation biopharmaceuticals using computational biology and 3D-modeling of subdominant and conformational epitopes, prospectively enabling the discovery of new antibody treatments for hard-to-target cancers and other diseases. iBio’s mission is to “decrease drug failures, shorten drug development timelines, and open up new frontiers against the most promising targets,” the company stated.

Celldex is a clinical stage biotechnology company leading the science at the intersection of mast cell biology and the development of transformative therapeutics for patients. The company’s pipeline includes antibody-based therapeutics which have the ability to engage the human immune system and/or directly affect critical pathways to improve the lives of patients with severe inflammatory, allergic and autoimmune and other devastating diseases.

Altimmune is a clinical-stage biopharmaceutical company that says it is focused on developing “innovative next-generation therapeutics for the treatment of patients with liver diseases and obesity.” The company’s lead product candidate, pemvidutide, is a GLP-1/glucagon dual receptor agonist that is being developed for the treatment of obesity and NASH. In addition, Altimmune is developing HepTcell, an immunotherapeutic designed to achieve a functional cure for chronic hepatitis B.

Actinium develops targeted radiotherapies to meaningfully improve survival for people who have failed existing oncology therapies. Advanced pipeline candidates Iomab-B, an induction and conditioning agent prior to bone marrow transplant, and Actimab-A, a therapeutic agent, have demonstrated potential to extend survival outcomes for people with relapsed and refractory acute myeloid leukemia. Actinium plans to advance Iomab-B for other blood cancers and next generation conditioning candidate Iomab-ACT to improve cell and gene therapy outcomes.

BioLineRx is a pre-commercial-stage biopharmaceutical company focused on oncology. The company’s lead development program, motixafortide, a novel selective inhibitor of the CXCR4 chemokine receptor, may support diverse therapeutic approaches in oncology and other diseases. APHEXDA was successfully evaluated in a Phase 3 study in stem cell mobilization for autologous transplantation for multiple myeloma patients, has reported positive results from a pre-planned pharmacoeconomic study in the U.S., and has had its NDA submission accepted by the FDA with an assigned PDUFA date of September 9, 2023.

Apellis Pharmaceuticals says the company “ushered in the first new class of complement medicine in 15 years” with the approval of the first and only targeted C3 therapy. Apellis is advancing this science to “continually develop transformative medicines for people living with rare, retinal, and neurological diseases,” it stated.

Cytokinetics is a late-stage, specialty cardiovascular biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators and next-in-class muscle inhibitors as potential treatments for debilitating diseases in which cardiac muscle performance is compromised. As a leader in muscle biology and the mechanics of muscle performance, the company is developing small molecule drug candidates specifically engineered to impact myocardial muscle function and contractility.

Recent news on these stocks:

February 28

BioLineRx announced that the first patient has been dosed in the randomized CheMo4METPANC Phase 2 combination clinical trial evaluating the company’s CXCR4 inhibitor motixafortide, the PD-1 inhibitor cemiplimab, and standard of care chemotherapies gemcitabine and nab-paclitaxel, versus gemcitabine and nab-paclitaxel alone, in first-line pancreatic cancer, or PDAC. The investigator-initiated trial is being conducted in collaboration with Columbia University and is the first large, multi-center, randomized study evaluating motixafortide with a PD-1 inhibitor and first-line PDAC chemotherapies. Findings from the single-arm pilot phase of the CheMo4METPANC trial will be shared by Dr. Manji at the 10th Annual Immuno-Oncology (IO) 360 degrees Summit in Brooklyn, New York on Thursday, February 29, 2024. The findings were previously presented during an oral presentation at the American Association of Cancer Research, or AACR, Special Conference on Pancreatic Cancer in Boston, Massachusetts, September 28, 2023. As of July 2023, 7 of the 11 patients in the pilot phase experienced a partial response of which 5 were confirmed PRs at the time of the data cut; one patient experienced resolution of the hepatic metastatic lesion; and three patients experienced stable disease, resulting in a disease control rate of 91%. Motixafortide, BioLineRx’s lead therapeutic candidate, was approved by the FDA in September 2023, in combination with filgrastim, to mobilize hematopoietic stem cells for collection and subsequent autologous transplantation in patients with multiple myeloma, under the brand name APHEXDA. Motixafortide is also being evaluated in a Phase 1 clinical trial evaluating motixafortide as a monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell mobilization for gene therapies in sickle cell disease.

February 27

Apellis reported Q4 EPS (73c) against a consensus of (69c), and reported Q4 revenue of $146.4M against a consensus of $143.59M. “I am extremely proud of our team, their accomplishments and resilience in a year faced with many highs and some unexpected challenges. The SYFOVRE launch exceeded even our own expectations, with demand growth continuing into the first quarter of 2024, and we remain encouraged by the uptake and high compliance rates for EMPAVELI in PNH,” said Cedric Francois, M.D., Ph.D., CEO of Apellis. “We are well-positioned to continue our strong execution in 2024, with a focus on bringing SYFOVRE to even more patients in the U.S. and globally, maximizing EMPAVELI in PNH and C3G/IC-MPGN, advancing our earlier-stage pipeline, and delivering on our mission for patients now and in the future.” As of December 31, 2023, Apellis had $351.2M in cash and cash equivalents, compared to $551.8M in cash and cash equivalents as of December 31, 2022. Apellis anticipates its cash balance, combined with cash anticipated to be generated from the unwind of the capped call transactions and from the sales of EMPAVELI and SYFOVRE, will be sufficient to fund its projected operating expenses and capital expenditures for the foreseeable future.

Cytokinetics reported Q4 EPS ($1.38) against a consensus of (97c), and reported Q4 revenue of $1.67M against a consensus of $10.77M. “We ended 2023 strong with positive results from SEQUOIA-HCM which now propel our company forward to the next stages of planning towards our specialty cardiology business model,” said Robert I. Blum, Cytokinetics’ CEO. “As we prepare regulatory submissions for aficamten, we are executing on commercial readiness activities while also conducting Phase 3 clinical trials in patients with oHCM and nHCM which we believe may further generate evidence in support of our next-in-class objectives to reach a broader array of patients struggling with hypertrophic cardiomyopathy. With a strong balance sheet enabling ample cash runway and multiple levers to access capital, we are pleased to be turning the page onto the next chapter for Cytokinetics and all stakeholders.”

February 26

Krystal Biotech reported Q4 EPS 30c against a consensus of (38c), and reported Q4 revenue $42.1M against a consensus of $28.61M. “2023 was an inflection point for Krystal with the approval and launch of VYJUVEK, the first-ever FDA approved therapy for dystrophic epidermolysis bullosa and only medicine that delivers the corrective power of gene therapy in an easy-to-apply, redosable, topical gel formulation that can be administered at home,” said Krish Krishnan, chairman and CEO of Krystal Biotech. “Our U.S. commercial launch trajectory is tracking closely to that of the best recent rare disease launches with $50.7 million in net product revenue only six months since approval, supported by high patient and physician demand, broad access, and high compliance. We look forward to expanding VYJUVEK access in the U.S. and globally in the years to come, while advancing our clinical pipeline and reinforcing our leadership in redosable gene therapy.”

Milestone announced plans to resubmit the New Drug Application for etripamil to the FDA for paroxysmal supraventricular tachycardia. Following the previously announced receipt of a Refusal to File letter, Milestone held a Type A Meeting with FDA. FDA indicated that the timing of adverse events in question had minimal impact on the overall characterization of the etripamil safety profile. To align with FDA’s guidance, the company will restructure the data sets that capture timing of reported AEs, reformat certain data files to facilitate FDA’s analyses, and resubmit the NDA. Based on the guidance received during the Type A Meeting, the company expects that this approach will address the Refusal to File letter from FDA. FDA has not requested that the company complete additional clinical efficacy or safety trials prior to resubmitting the NDA. The company expects a standard NDA review period following resubmission of the NDA for etripamil for PSVT, which is planned for 2Q2024. Considering the revised timeline for NDA submission, the company has undertaken certain cash conservation measures to reduce spend through program deferrals and team restructuring and expects that the company’s existing cash resources will fund operations into mid-2025, including the expected Prescription Drug User Fee Act date for the NDA resubmission. If FDA approval is granted, the company expects to receive a $75M payment under an existing royalty agreement, which is intended to fund the potential commercial launch of etripamil for PSVT.

iBio entered into an asset purchase agreement with Otsuka Pharmaceutical (OTSKY), pursuant to which Otsuka acquired iBio’s assets related to its early-stage programmed cell death protein 1 agonist program. The transaction closed on February 25, 2024. Under the terms of the Agreement, iBio will receive an upfront payment of $1M in cash at closing. iBio will also be eligible to receive additional contingent cash payments totaling up to $52.5M upon the achievement of certain pre-specified clinical development and commercial milestones.

Celldex reported Q4 EPS (83c) against a consensus of (74c), and reported Q4 revenue of $4.1M against a consensus of $840K. “In 2023, Celldex made transformational progress across the barzolvolimab development program, reporting multiple positive data sets across mast cell mediated diseases where patients desperately need better treatment options,” said Anthony Marucci, CEO of Celldex Therapeutics. “We carried this momentum into 2024, presenting positive 12 week data from our ongoing Phase 2 CSU study that directly support the planned initiation of registrational studies in CSU this summer, a major milestone for barzolvolimab and Celldex. This year we will continue to build on our leadership position in mast cell biology-reporting data from multiple barzolvolimab studies, expanding barzolvolimab into additional mast cell mediated diseases and introducing our first bispecific for inflammatory diseases. We look forward to an exciting year.” Celldex believes that the cash, cash equivalents and marketable securities at December 31, 2023 are sufficient to meet estimated working capital requirements and fund current planned operations into 2026.

Actinium Pharmaceuticals reported new analyses from the Phase 3 SIERRA trial of Iomab-B in oral presentations at the at the 2024 Tandem Meetings Transplantation & Cellular Therapy, TCT, Meetings of ASTCT. The presentations reported unprecedented 100% access to potentially curative bone marrow transplant and engraftment in evaluable patients with active relapsed or refractory acute myeloid leukemia and improved long-term survival outcomes greater than 2 years in patients age 65 or older. Patients with r/r AML age 65 and older who also have multiple comorbidities and high-risk cytogenetics have a poor prognosis are seldom offered BMT in current practice due to poor tolerance to induction and conditioning regimens and dismal outcomes. The SIERRA results presented at TCT demonstrate Iomab-B’s ability to overcome multiple high-risk features including a TP53 genetic mutation, advanced age and treatment resistant disease. The two oral presentations at this year’s TCT mark a total of ten oral presentations of the SIERRA results at various leading transplant, hematology and nuclear medicine conferences in the USA and Europe.

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About “Biotech Alert”

The Fly will report on a selection of biotech stocks seeing a surge in interest from retail and financial professional investors, based on data from InvestingChannel.

This Fly exclusive recap reveals the biotech stocks that are seeing a spike in searches among the 20-plus million retail and financial professional investors through InvestingChannel’s online financial news media ecosystem.

This increased attention from the investors may be in response to, or advance of, outsized moves for stocks in the biotech sector, which tend to be volatile and prone to sharp swings in share price around binary events such as clinical study results and FDA approvals.