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: 

• Seelos Therapeutics (SEEL), 970% surge in interest
• Applied Therapeutics (APLT), 834% surge in interest
• Marinus Pharma (MRNS), 575% surge in interest
• Revance Therapeutics (RVNC), 544% surge in interest
• Galera Therapeutics (GRTX), 470% surge in interest
• Atara Biotheraputics (ATRA), 319% surge in interest
• Vir Biotechnology (VIR), 291% surge in interest
• Orchard Therapeutics (ORTX), 289% surge in interest
• Apellis Pharmaceuticals (APLS), 157% surge in interest
• Navidea Biopharmaceuticals (NAVB), 66% surge in interest

Pipeline and key clinical candidates for these companies:

Seelos Therapeutics is focused on the development and advancement of novel therapeutics to address unmet medical needs for the benefit of patients with central nervous system, or CNS, disorders and other rare diseases. The company’s portfolio includes several late-stage clinical assets targeting indications including Acute Suicidal Ideation and Behavior in Major Depressive Disorder, amyotrophic lateral sclerosis, or ALS, and spinocerebellar ataxia, or SCA, as well as early-stage programs in Huntington’s disease, Alzheimer’s disease, and Parkinson’s disease.

Applied Therapeutics says it is “developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need.” The company’s lead drug candidate, AT-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG. The company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart.

Marinus is a commercial stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. The company’s commercial product, Ztalmy oral suspension CV, has been approved by the FDA for the treatment of seizures associated with CDKL5 deficiency disorder in patients two years of age and older. The potential of ganaxolone is also being studied in other rare seizure disorders, including in Phase 3 trials in tuberous sclerosis complex and refractory status epilepticus. Ganaxolone is a neuroactive steroid GABAA receptor modulator that acts on a well-characterized target in the brain known to have anti-seizure effects. It is being developed in IV and oral formulations to maximize therapeutic reach for adult and pediatric patients in acute and chronic care settings.

Revance is a biotechnology company whose aesthetics portfolio includes Daxxify for injection, the RHA Collection of dermal fillers, and OPUL, the “first-of-its-kind Relational Commerce platform for aesthetic practices.” Revance has also partnered with Viatris to develop a biosimilar to Botox, which will compete in the existing short-acting neuromodulator marketplace. Revance’s therapeutics pipeline is currently focused on muscle movement disorders including evaluating Daxxify in two debilitating conditions, cervical dystonia and upper limb spasticity.

Galera Therapeutics is focused on developing and commercializing a pipeline of therapeutic candidates that have the potential to transform radiotherapy in cancer. Galera’s selective dismutase mimetic product candidate avasopasem manganese, avasopasem, or GC4419, is being evaluated for radiotherapy-induced toxicities. The company’s second product candidate, rucosopasem manganese, rucosopasem, or GC4711, is in clinical-stage development to augment the anti-cancer efficacy of stereotactic body radiation therapy in patients with non-small cell lung cancer and locally advanced pancreatic cancer.

Atara is “harnessing the natural power of the immune system to develop off-the-shelf cell therapies for difficult-to-treat cancers and autoimmune conditions, including multiple sclerosis, that can be rapidly delivered to patients within days. With cutting-edge science and differentiated approach, Atara is the first company in the world to receive regulatory approval of an allogeneic T-cell immunotherapy,” the company states.

Vir Biotechnology is an immunology company focused on combining cutting-edge technologies to treat and prevent infectious diseases and other serious conditions. Vir has assembled two technology platforms that are designed to stimulate and enhance the immune system by exploiting critical observations of natural immune processes. Its current clinical development pipeline consists of product candidates targeting hepatitis B and hepatitis delta viruses, influenza A and B, human immunodeficiency virus and COVID-19. Vir has several preclinical candidates in its pipeline, including RSV/MPV and HPV.

Orchard Therapeutics discovers, develops and commercializes new treatments that tap into the curative potential of hematopoietic stem cell, or HSC, gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment. In 2018, the company acquired GlaxoSmithKline’s (GSK) rare disease gene therapy portfolio, which originated from a collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies.

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.

Navidea Biopharmaceuticals is a biopharmaceutical company focused on the development of precision immunodiagnostic agents and immunotherapeutics. Navidea is developing multiple precision-targeted products based on its Manocept platform to enhance patient care by identifying the sites and pathways of disease and enable better diagnostic accuracy, clinical decision-making, and targeted treatment. The Manocept platform serves as the molecular backbone of Tc99m tilmanocept, the first product developed and commercialized by Navidea based on the platform.

Recent news on these stocks:

September 20

Seelos Therapeutics announced top line data demonstrating clinically meaningful treatment effects across multiple endpoints and a well-tolerated safety profile from the double-blind, placebo-controlled cohort, Part 2, of its Phase II study of SLS-002, or intranasal racemic ketamine, for Acute Suicidal Ideation and Behavior, or ASIB, in adults with Major Depressive Disorder, or MDD. SLS-002 versus placebo demonstrated early and persistent reductions in symptoms of depression as assessed by the Montgomery-Asberg Depression Rating Scale, or MADRS. The graph presents results from the mixed model for repeated measures analysis of change from baseline in MADRS total score. Target enrollment of this study was 220 patients, however, due to financial constraints, only 147 patients diagnosed with MDD requiring psychiatric hospitalization due to significant risk of suicide were randomized. MADRS results at 4 hours after dosing demonstrated a statistically significant change relative to placebo; MADRS results at 24 hours after dosing utilizing 2-way ANCOVA3 with baseline MADRS as a covariate demonstrated clinically meaningful results, but did not achieve statistical significance under the methodology used; MADRS results at 24 hours after dosing utilizing an exploratory ANOVA analysis demonstrated statistically significant change relative to placebo; MADRS results at Day 16 demonstrated a statistically significant change relative to placebo- demonstrating persistence of effect:Meaningful results further supported by: MADRS Response Rate, defined as greater than or equal to 50% reduction from baseline, at Day 16; MADRS Remission Rate, defined as a total score less than or equal to 12, at Day 16; MADRS at end of 2-week safety follow up (Day 29/30) revealed continued improvement, demonstrating no evidence of return of symptoms.

RBC Capital raised the firm’s price target on Marinus Pharmaceuticals to $21 from $20 and kept an Outperform rating on the shares following the company’s’ virtual analyst event. The company highlighted key pipeline updates, which included affirmation of refractory status epilepticus interim data timing of Q1 2024, favorable pre-reported Q3 Ztalmy sales, and reduced target enrollment for tuberous sclerosis given low dropouts, the analyst tells investors in a research note. The firm continues to like the setup into the RSE readout, especially after the recent share weakness.

Needham lowered the firm’s price target on Revance to $35 from $40 but kept a Buy rating on the shares. The company’ is well-funded and expected to generate positive adjusted EBITDA in 2025, and the stock’s downward move – including the 17% decline on the discontinuation of the Opul business – is likely “overdone”, the analyst told investors in a research note. The new pricing strategy also removes a key barrier for Daxxify uptake, the firm added.

Goldman Sachs lowered the firm’s price target on Revance to $33 from $38 but keeps a Buy rating on the shares. The company delivered a “sustainable” Investor Day presentation, outlining several noteworthy strategic adjustments which have the potential to ultimately strengthen its longer-term outlook, the analyst tells investors in a research note. The decision to back away from their premium-pricing approach is likely triggering a net negative impact on revenues with accompanying market dynamic uncertainties, but it is net positives for Daxxify and Revance’s outlook in the longer-term as it directly addresses product cost/practice economics factors which have been the key concern, the firm added.

Piper Sandler lowered the firm’s price target on Revance to $44 from $50 and keeps an Overweight rating on the shares. The firm attended Revance’s investor day at the company’s hub in Nashville, where management notably outlined revised pricing for Daxxify. Though the optics of the “reset” are admittedly messy, Piper nonetheless would view what is essentially the removal of premium pricing as the elimination of a hurdle to wider adoption in the context of a highly competitive neuromodulator space, but one that is vast in terms of the addressable patient population. Put differently, there is strong practitioner receptivity to Daxxify, but in the context of a more measured pricing structure. That is not a bad thing, and does not change the firm’s view that Revance is well-positioned to drive a $1B-plus sales footprint, Piper added.

Vir Biotechnology announced that the first participant has been dosed in a Phase 1 trial evaluating the safety, reactogenicity and immunogenicity of VIR-1388, an investigational novel T cell vaccine for the prevention of human immunodeficiency virus. The Company expects initial data from the Phase 1 trial in the second half of 2024. “HIV continues to be a major global public health challenge with no approved vaccines despite decades of research efforts,” said Carey Hwang, M.D., Ph.D., Vir’s Senior Vice President, Clinical Research, Head of Chronic Infection. “The initiation of our first clinical trial evaluating VIR-1388 is an important clinical milestone in our pursuit of developing an HIV vaccine and we are grateful to all our partners for their support of this Phase 1 trial. We are hopeful that our unique approach will help close the longstanding public health gap in HIV prevention.” The trial is supported by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, and the Bill & Melinda Gates Foundation.

September 19

Atara Biotherapeutics reported important progress related to the regulatory pathway for tabelecleucel, or tab-cel, in the U.S. Following productive discussions between Atara and the FDA, the FDA and Atara are now aligned on analytical comparability between manufacturing process versions. This alignment supports Atara’s ability to pool the pivotal clinical trial data from different process versions in the Biologics License Application submission. Atara expects to submit the tab-cel BLA in Q2 2024, which will enable Atara to incorporate the latest tab-cel pivotal trial data from the ALLELE study into the BLA filing package. Tabelecleucel is an allogeneic, EBV-specific T-cell immunotherapy which targets and eliminates EBV-infected cells in an HLA-restricted manner. Epstein-Barr virus-positive post-transplant lymphoproliferative disease is a rare, acute, and potentially deadly hematologic malignancy that occurs after transplantation when patient T-cell immune responses are compromised by immunosuppression. It can impact patients who have undergone solid organ transplant or allogeneic hematopoietic cell transplant. Poor median survival of 0.7 months and 4.1 months for HCT and SOT, respectively, is reported in EBV+ PTLD patients for whom standard of care failed, underscoring the significant need for new therapeutic options.

September 18

Orchard Therapeutics announced the FDA has accepted the filing of its Biologics License Application, BLA, for OTL-200 in metachromatic leukodystrophy, MLD, under Priority Review. The agency has set a Prescription Drug User Fee Act, PDUFA, goal date of March 18, 2024. “Today is another significant step forward for patients and families in the U.S. impacted by this devastating and cruel disease who for too long have dealt with the unimaginable burden of going through the diagnostic odyssey, being told there were no treatments beyond supportive care, and then having to watch their child slip away,” said Bobby Gaspar, M.D., Ph.D., co-founder and chief executive officer of Orchard Therapeutics. “We look forward to collaborating with the FDA throughout the review and evaluation of our application. Due to the nature of the disease and the urgency to treat children affected by MLD, we are working diligently in parallel to prepare for a potential launch in 2024 and ensure OTL-200 will be available to patients in the U.S. as quickly as possible.”

Galera Therapeutics announced that a Type A Meeting has been scheduled for September 28, 2023 with the FDA regarding the Complete Response Letter received for the company’s New Drug Application for avasopasem manganese. The FDA is expected to issue written meeting minutes approximately 30 days following the meeting. The Company intends to gain an understanding from the FDA of its evaluation of avasopasem for radiotherapy-induced severe oral mucositis in patients with head and neck cancer undergoing standard-of-care treatment and next steps to support an NDA resubmission. The Type A Meeting is the highest priority classification of meeting that the FDA grants to NDA sponsors.

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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.