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: 

• Compugen (CGEN), 16,232% surge in interest
• Argenx (ARGX), 1,386% surge in interest
• Liquidia (LQDA), 637% surge in interest
• Aldeyra (ALDX), 483% surge in interest
• TFF Pharmaceuticals (TFFP), 415% surge in interest
• Crinetics Pharmaceuticals (CRNX), 393% surge in interest
• Tracon Pharmaceuticals (TCON), 360% surge in interest
• MacroGenics (MGNX), 269% surge in interest
• Syros Pharmaceuticals (SYRS), 213% surge in interest
• Nanostring Technologies (NSTG), 209% surge in interest

Pipeline and key clinical candidates for these companies:

Compugen is a clinical-stage therapeutic discovery and development company utilizing its broadly applicable predictive computational discovery capabilities to identify new drug targets and biological pathways for developing cancer immunotherapies. Compugen has developed two proprietary product candidates: COM701, a potential first-in-class anti-PVRIG antibody and COM902, a potential best-in-class antibody targeting TIGIT for the treatment of solid tumors. Compugen also has a clinical stage partnered program, rilvegostomig (previously AZD2936), a PD-1/TIGIT bispecific antibody where the TIGIT component is derived from Compugen’s clinical stage anti-TIGIT antibody, COM902, in Phase 3 development by AstraZeneca through a license agreement for the development of bispecific and multispecific antibodies. In addition, the company’s therapeutic pipeline of early-stage immuno-oncology programs consists of programs aiming to address various mechanisms of immune resistance, of which the most advanced program, COM503, is in IND enabling studies. COM503 is a potential first-in-class, high affinity antibody which blocks the interaction between IL-18 binding protein and IL-18, thereby freeing natural IL-18 in the tumor microenvironment to inhibit cancer growth.

Argenx is an immunology company that says it is “committed to improving the lives of people suffering from severe autoimmune diseases” and “aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines.” Argenx developed and is commercializing what it calls “the first-and-only approved neonatal Fc receptor blocker in the U.S., the EU and UK, and Japan.” The company is evaluating efgartigimod in multiple autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises.

Liquidia Corporation is a biopharmaceutical company focused on the development and commercialization of products in pulmonary hypertension and other applications of its PRINT Technology. Liquidia Technologies has developed Yutrepia inhalation powder for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease. Liquidia Technologies is also developing L606, an investigational liposomal formulation of treprostinil administered twice-daily with a short-duration next-generation nebulizer, for use in North America. Liquidia PAH provides the commercialization for pharmaceutical products to treat pulmonary disease, such as generic Treprostinil Injection.

Aldeyra is a clinical-stage biotechnology company whose pre-commercial product candidates are reproxalap, a potential treatment for dry eye disease and allergic conjunctivitis, and ADX-2191, a potential treatment for primary vitreoretinal lymphoma, proliferative vitreoretinopathy, and other rare sight-threatening retinal diseases. In addition, Aldeyra is developing other product candidates, including ADX-629 and chemically related molecules, for the potential treatment of systemic and retinal immune-mediated diseases.

TFF Pharmaceuticals is a clinical-stage biopharmaceutical company engaging patented rapid freezing technology to develop and transform medicines into potent dry powder formulations for better efficacy, safety, and stability. The company’s TFF technology platform has broad applicability to convert most any drug, including vaccines, small and large molecules, and biologics, into an elegant dry powder highly advantageous for inhalation, or for topical delivery to the eyes, nose and the skin. TFF Pharmaceuticals has two lead drug candidates in the clinic: TFF VORI and TFF TAC. 

Crinetics Pharmaceuticals is focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. Paltusotine, an investigational, oral somatostatin receptor type 2 agonist, is in Phase 3 clinical development for acromegaly and Phase 2 clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics has demonstrated pharmacologic proof-of-concept in Phase 1 clinical studies for CRN04777, an investigational, oral somatostatin receptor type 5 agonist in development for congenital hyperinsulinism, and for CRN04894, an investigational, oral ACTH antagonist in development for the treatment of Cushing’s disease, congenital adrenal hyperplasia, and other diseases of excess ACTH.

Tracon is advancing a pipeline of novel targeted cancer therapeutics and seeks to partner with other life science companies. The company’s clinical-stage pipeline includes: Envafolimab, a PD-L1 single-domain antibody given by rapid subcutaneous injection that is being studied in the pivotal ENVASARC trial for sarcoma; YH001, a potential best-in-class CTLA-4 antibody in Phase 1 development; TRC102, a Phase 2 small molecule drug candidate for the treatment of lung cancer; and TJ004309, a CD73 antibody in Phase 1 development for the treatment of advanced solid tumors. Tracon has said it believes it “can serve as a solution for companies without clinical and commercial capabilities in the United States or who wish to become CRO-independent.”

MacroGenics is a biopharmaceutical company focused on developing, manufacturing and commercializing innovative monoclonal antibody-based therapeutics for the treatment of cancer. The company generates its pipeline of product candidates primarily from its proprietary suite of next-generation antibody-based technology platforms, which have applicability across broad therapeutic domains. “The combination of MacroGenics’ technology platforms and protein engineering expertise has allowed the company to generate promising product candidates and enter into several strategic collaborations with global pharmaceutical and biotechnology companies,” MacroGenics states.

Syros is committed to developing new standards of care for the frontline treatment of patients with hematologic malignancies. Driven by the motivation to help patients with blood disorders that have largely eluded other targeted approaches, Syros is developing tamibarotene, an oral selective RARalpha agonist in frontline patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia with RARA gene overexpression.

NanoString Technologies says it offers “an ecosystem of innovative discovery and translational research solutions” to map the universe of biology – including the GeoMx Digital Spatial Profiler, the CosMx Spatial Molecular Imager and the AtoMx Spatial Informatics Platform. The CosMx SMI and AtoMx SIP platforms are expected to launch in 2022.

Recent news on these stocks:

December 20

Argenx announced topline results from the ADDRESS study evaluating efgartigimod subcutaneous, or SC, in adults with pemphigus vulgaris, or PV, and pemphigus foliaceus, or PF. The ADDRESS results show the proportion of PV patients achieving the primary endpoint of complete remission on a minimal dose of steroids was not significantly different between efgartigimod SC and placebo. Argenx will not pursue additional development in pemphigus and plans to prioritize clinical development of efgartigimod in its ongoing severe autoimmune indications. The Phase 3 ADDRESS study enrolled 222 adult patients with newly diagnosed or relapsing moderate-to-severe PV or PF. Patients were randomized to efgartigimod SC or placebo with both treatment groups receiving concomitant steroids at a starting dose of 0.5mg/kg, which is a lower dose than recommended by current treatment guidelines and was tapered according to protocol upon achievement of complete remission. Consistent pharmacodynamic, or PD, effect of efgartigimod SC: Treatment with efgartigimod SC led to total immunoglobulin G and desmoglein autoantibody reductions up to 75%. The observed PD effect was consistent with previous clinical trials of efgartigimod. Unexpected PD effect of corticosteroids: There was a higher than expected response to background treatment with corticosteroids, which showed a reduction of DSG-1 and DSG-3 levels of up to 70% in the placebo arm and correlated to sustained clinical benefit. The level of autoantibody reduction driven by corticosteroids in both treatment arms was sufficient for patients to achieve CRmin. The significant PD effect of corticosteroids was specific to DSG-1 and DSG-3 autoantibodies, while the observed effect on total IgG reduction was in line with the literature. Treatment with efgartigimod SC led to CRmin in 35.5% of patients compared to 30.3% with placebo. Secondary endpoints were also not met, including CRmin in the overall pemphigus population, cumulative dose of corticosteroids and time to disease control or complete remission. Efgartigimod SC was well-tolerated in ADDRESS. The observed safety and tolerability profile was consistent with other clinical trials and the confirmed safety profile of Vyvgart and Vyvgart Hytrulo. Argenx is reviewing the BALLAD study in light of the ADDRESS results and the comparable biology between pemphigus and bullous pemphigoid, and has decided not to make a GO/NO GO decision at this time but rather wait for learnings from all currently enrolled patients and consider a new trial design for the path forward.

Liquidia Corporation announced that the U.S. Court of Appeals for the Federal Circuit affirmed the earlier decision by the Patent Trial and Appeal Board, or PTAB, which found all claims of U.S. Patent No. 10,716,793 – ‘793 Patent – to be unpatentable due to the existence of prior art cited by Liquidia in inter partes review proceedings. As a result of the decision, Liquidia will immediately seek to set aside the injunction issued by the U.S. District Court for the District of Delaware in the lawsuit filed by United Therapeutics (UTHR) under the Drug Price Competition and Patent Term Restoration Act and pursue final regulatory approval of YUTREPIA by the FDA.

TRACON Pharmaceuticals announced that the ongoing pivotal Phase 2 ENVASARC trial has enrolled more than 70 of the 80 planned patients in Cohort C of single agent envafolimab treatment at a dose of 600 mg subQ every three weeks. Additional safety and efficacy data were reviewed for 46 patients enrolled into cohort C who were the subject of the September independent data monitoring committee review. At that time, patients had completed a minimum of 12 weeks of efficacy evaluations and the objective response rate was 13% by investigator review and 8.7% by blinded independent central review. Since then, an additional patient has achieved an objective response by investigator review, which increased the ORR by investigator review to 15%. The most recent objective response has not yet been confirmed by BICR and the patient remains on treatment. Median duration of response by BICR remains greater than six months. In addition, envafolimab remains well tolerated and grade greater than 3 related toxicity has not been reported to date. “We continue to believe that these data position envafolimab to become a potentially compelling treatment option for patients with the refractory sarcoma subtypes of UPS and MFS based on the ORR and tolerability data to date,” said Charles Theuer, M.D., Ph.D., TRACON’s Chief Executive Officer. “ENVASARC enrollment continues to be brisk, reflecting the high unmet need that exists for these patients.”

December 19

Gilead Sciences (GILD) announced an agreement with Compugen to exclusively license its potential first-in-class, pre-clinical antibody program against IL-18 binding protein, including the COM503 drug candidate. “We are very pleased to add COM503 to our pipeline of investigational immuno-oncology therapies that have the potential to transform care for patients with cancer,” said Flavius Martin, M.D., Executive Vice President, Research, Gilead Sciences. “We believe that this collaboration complements our strategy of developing modalities which promote immune-mediated tumor killing and may enable new combination therapies with programs in our growing oncology portfolio.”

Aldeyra announced top-line results from a Phase 2 clinical trial of ADX-629, an investigational RASP modulator, in patients with atopic dermatitis. Relative to baseline, the clinical trial demonstrated statistically significant and clinically relevant improvement in investigator-assessed and patient-reported outcomes across a number of different physiological and psychosocial assessments, including complete resolution of affected body surface area observed in one patient and elimination of itching reported by two patients. All enrolled patients completed the trial per protocol. No patients experienced flare requiring rescue therapy. Only two adverse events deemed to be at least possibly related to ADX-629 were reported, and both events were mild. There were no observed serious adverse events or discontinuations due to adverse events. Aldeyra expects to initiate a multicenter, randomized, placebo-controlled Phase 1/2 clinical trial of ADX-246 in healthy volunteers and patients with atopic dermatitis in the first half of 2024. Topline results from the trial are expected in the second half of 2024.

TFF Pharmaceuticals announced positive initial data from the company’s ongoing Phase 2 trials of Thin Film Freezing – TFF – VORI and TFF TAC, along with clinical data from the ongoing TFF VORI Expanded Access Program – EAP -. The ongoing Phase 2 trial of TFF VORI is evaluating treatment with TFF VORI versus oral voriconazole over a 13-week period in patients with IPA. Of the five patients treated for at least 8 weeks with TFF VORI: all five patients achieved a clinical response, all five patients achieved a mycologic response, three of four patients with pre- and post-treatment chest CT achieved a radiologic response, there was no need for continued anti-fungal use after treatment with TFF VORI in all five patients. In terms of safety, no all-cause mortality, no IPA-related mortality, no TFF VORI discontinuation due to an AE, majority of treatment emergent adverse events were deemed unrelated to TFF VORI, majority of TEAEs were Grade 2 or lower in severity, no hepatic toxicity. The ongoing Phase 2 trial of TFF TAC is an open-label study in lung transplant patients who require reduced tacrolimus blood levels due to kidney toxicity. Trial endpoints include safety/tolerability, kidney function, and acute allograft rejection. The initial data presented below are from the first four patients enrolled in the trial. Of the four patients enrolled in the TFF TAC Phase 2 study: all four patients were successfully transitioned from oral tacrolimus to TFF TAC, successfully lowered tacrolimus blood levels to 1/2 to 2/3 of the levels on oral tacrolimus in all four patients, no clinical evidence of acute rejection, no signs and symptoms suggestive of acute rejection, no deterioration in spirometry. All three patients who completed Part A chose to remain on TFF TAC and proceeded to Part B. As for safety, there was no mortality, no TFF TAC discontinuation due to an AE, majority of TEAEs were Grade 2 or lower in severity, maintenance of kidney function.

Citi upgraded MacroGenics to Buy from Neutral with a price target of $13, up from $7. The stock has performed very well over the past 2 months, gaining about 80% on a combination of continued execution across the pipeline, increasing interest and investment from Pharma in the ADC class, and an improving biotech tape, the analyst tells investors in a research note. The enoblituzumab safety signal that drove the downgrade last November is no longer part of the debate given an increasing focus on the ADC portfolio, the firm states, adding that it sees MacroGenics positioned to continue benefiting from accelerating momentum in the ADC space, especially as the company begins to share more details on its wave of next-gen ADC programs over the next 12 months.

December 18

Crinetics Pharmaceuticals announced positive initial findings from its ongoing open-label Phase 2 carcinoid syndrome study of paltusotine, an oral, once-daily investigational compound being developed for the treatment of acromegaly and CS.”We are very encouraged by these strong initial findings in our Phase 2 study of paltusotine in people with carcinoid syndrome,” said Scott Struthers, Ph.D., founder and chief executive officer of Crinetics. “These initial results show the potential of paltusotine to significantly reduce both frequency and intensity of bowel movements and flushing, the key carcinoid syndrome symptoms. Further, paltusotine was well-tolerated and the overall pharmacokinetic profile was consistent with prior studies. After completing this Phase 2 study next quarter, we anticipate sharing the results with the FDA to align on the design of a Phase 3 program.”

Hear more from InvestingChannel by signing up for The Spill.

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.