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), 11,950% surge in interest
• Cara Therapeutics (CARA), 705% surge in interest
• TFF Pharmaceuticals (TFFP), 508% surge in interest
• Aldeyra (ALDX), 435% surge in interest
• Crinetics Pharmaceuticals (CRNX), 358% surge in interest
• Mirium Pharmaceuticals (MIRM), 323% surge in interest
• IGM Biosciences (IGMS), 275% surge in interest
• Cyclacel Pharmaceuticals (CYCC), 254% surge in interest
• Checkpoint Therapeutics (CKPT), 234% surge in interest
• Prothena (PRTA), 188% 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, 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 (AZN) through a license agreement for the development of bispecific and multispecific antibodies. 

Cara Therapeutics is a commercial-stage biopharmaceutical company that notes that its Kapruvia injection is “the first and only” MHRA-approved treatment for moderate-to-severe pruritus associated with chronic kidney disease in adults undergoing hemodialysis. The company is developing an oral formulation of difelikefalin and has Phase 3 programs ongoing for the treatment of pruritus in patients with non-dialysis dependent advanced chronic kidney disease and atopic dermatitis. In addition, the company has initiated a Phase 2/3 program of oral difelikefalin for the treatment of moderate-to-severe pruritus in patients with notalgia paresthetica.

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. The company continues collaborations with a broad array of pharmaceutical companies, academic institutions, and government partners to “revolutionize healthcare around the globe,” TFF states.

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.

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.

Mirum Pharmaceuticals is focused on the treatment of rare liver diseases. Mirum’s approved medication is Livmarli , or maralixibat oral solution, which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older. In Europe, the European Committee for Medicinal Products for Human Use has issued a positive opinion for Livmarli for the treatment of cholestatic pruritus in patients with Alagille syndrome two months of age and older. A decision by the European Commission is expected by year-end 2022. Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults.

IGM Biosciences is a clinical-stage biotechnology company committed to developing and delivering a new class of medicines to treat patients with cancer, autoimmune and inflammatory diseases and infectious diseases. IGM’s pipeline of clinical and preclinical assets is based on the IgM antibody, which has 10 binding sites compared to conventional IgG antibodies with only 2 binding sites. IGM also has an exclusive worldwide collaboration agreement with Sanofi to create, develop, manufacture, and commercialize IgM antibody agonists against oncology and immunology and inflammation targets.

Cyclacel is a clinical-stage, biopharmaceutical company developing innovative cancer medicines based on cell cycle, transcriptional regulation, epigenetics and mitosis biology. The transcriptional regulation program is evaluating fadraciclib, a CDK2/9 inhibitor, and the epigenetic/anti-mitotic program plogosertib, a PLK1 inhibitor, in patients with both solid tumors and hematological malignancies. Cyclacel’s strategy is to build a diversified biopharmaceutical business based on a pipeline of novel drug candidates addressing oncology and hematology indications.

Checkpoint Therapeutics is focused on the acquisition, development and commercialization of novel treatments for patients with solid tumor cancers. Checkpoint is evaluating its lead antibody product candidate, cosibelimab in an ongoing global, open-label, multicohort Phase 1 clinical trial in checkpoint therapy-naive patients with selected recurrent or metastatic cancers, including ongoing cohorts in locally advanced and metastatic cutaneous squamous cell carcinoma, or “cSCC,” intended to support one or more applications for marketing approval. Checkpoint is evaluating its lead small-molecule, targeted anti-cancer agent, olafertinib, formerly CK-101, as a potential new treatment for patients with EGFR mutation-positive non-small cell lung cancer.

Prothena Corporation is a late-stage clinical biotechnology company with expertise in protein dysregulation and a pipeline of investigational therapeutics with the potential to change the course of devastating neurodegenerative and rare peripheral amyloid diseases. Prothena is advancing a pipeline of therapeutic candidates for a number of indications and novel targets. Prothena’s pipeline includes both wholly-owned and partnered programs being developed for the potential treatment of diseases including AL amyloidosis, ATTR amyloidosis, Alzheimer’s disease, Parkinson’s disease and a number of other neurodegenerative diseases.

Recent news on these stocks:

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.”

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.

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.

December 18

Cara Therapeutics announced the outcome from the dose-finding Part A of the KIND 1 study evaluating the efficacy and safety of oral difelikefalin as adjunct therapy to topical corticosteroids for moderate-to-severe pruritus in adult patients with atopic dermatitis. Oral difelikefalin as adjunct to TCS did not demonstrate a meaningful clinical benefit compared to TCS alone, resulting in the Company’s decision to discontinue its clinical program in pruritus associated with atopic dermatitis. KIND 1 was a Phase 3, two-part, multicenter, randomized, double-blind, controlled study to evaluate the efficacy and safety of oral difelikefalin as adjunct therapy to TCS for moderate-to-severe pruritus in adults with AD. In Part A, patients were randomized to receive oral difelikefalin 0.25 mg tablets twice a day plus TCS, difelikefalin 0.5 mg tablets BID plus TCS, placebo tablets BID plus TCS or placebo tablets BID plus vehicle. The primary endpoint was the proportion of patients with a greater than or equal to4-point improvement at Week 12 from baseline in the worst itch NRS. Oral difelikefalin as adjunct therapy to TCS did not demonstrate a meaningful clinical benefit compared to TCS alone. Oral difelikefalin was generally well tolerated with a safety profile similar to prior trials.

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.”

Mirum Pharmaceuticals announced top-line results of the Phase 2 EMBARK study evaluating Livmarli oral solution versus placebo given as an adjuvant therapy to Kasai surgery in patients with biliary atresia. The study did not meet its primary endpoint of mean change in bilirubin from baseline to Week 26, or the key secondary endpoints. Baseline characteristics were well-balanced between the groups. Livmarli was generally well-tolerated, with no new safety findings. “We are disappointed in the outcome of the study in this post-surgery, high-need disease setting. We are grateful to the patients, families, and clinicians who participated in the study and advanced the science around this rare liver disease,” said Chris Peetz, president and CEO at Mirum.

Cyclacel Pharmaceuticals announced interim results from its Phase 1, dose escalation 065-101 study of fadraciclib in patients with advanced solid tumors and lymphoma. In the ongoing 065-101 study of oral fadra, a CDK2/9 inhibitor, a total of 29 patients have been treated as monotherapy. The study is enrolling unselected, all comer patients with advanced solid tumors and lymphoma. Six patients have been treated on dose level 6A. The sixth patient on dose level 6A with pancreatic cancer and CDKN2A deletion enrolled on the study experienced dose-limiting toxicity of hyperglycemia. The patient, who has a diabetic profile history and was on metformin treatment, remains on study as blood glucose level was managed. A previous patient on dose level 6A with a pre-diabetic profile had DLT of hyperglycemia which also resolved rapidly. The previous dose level 5 on this schedule accrued six patients with no DLT and per protocol is safe for continued development. Dose level 6B continues accrual with two patients treated, which are ongoing at three and five cycles of treatment. To date single agent activity, including CR, PR and SD, has been observed in patients with advanced endometrial, squamous NSCLC lung cancer and T-cell lymphoma. Encouraging signals of activity were observed in patients with advanced cervical, hepatocellular, ovarian and pancreatic cancers. The Company believes that fadra’s inhibition of CDK2 and CDK9 may be superior to inhibiting either CDK2 or CDK9 alone. Fadra tablets can be given orally with repeat dosing which has led to transient suppression of anti-apoptosis proteins with generally good tolerability and no Grade 3 or higher hematological toxicity in the first cycle. The Phase 2 part of the 065-101 study is designed to further evaluate fadra safety and efficacy in up to 8 cohorts defined by histology and/or NGS. The study is powered to demonstrate response in the molecular subtype suggested by the Phase 1 data and others that may be sensitive. CDKN2A gene deletions occur in over 40% of several solid tumors, including glioma, head and neck, pancreatic, esophageal, lung, bladder, melanoma, and others. CDKN2B deletions occur in over 30% of several solid tumors, including bladder, glioma, pancreatic, esophageal, lung, head and neck, melanoma, and others. MTAP deletions occur in over 25% of several solid tumors, including glioma, mesothelioma, pancreatic, bladder, esophageal and others.1 MTAP deletion confers dependency on the PRMT5 enzyme in cancer cells which was identified as a synthetic lethal target for MTAP deleted cancers.

Checkpoint Therapeutics announced that the FDA has issued a complete response letter for the cosibelimab biologic license application for the treatment of patients with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or radiation. The CRL only cites findings that arose during a multi-sponsor inspection of Checkpoint’s third-party contract manufacturing organization as approvability issues to address in a resubmission. The CRL did not state any concerns about the clinical data package, safety, or labeling for the approvability of cosibelimab. “As the only deficiencies relate to the FDA’s inspection of our third-party contract manufacturing organization, we believe we can address the feedback in a resubmission to enable marketing approval in 2024,” said James Oliviero, President and CEO of Checkpoint. “We are committed to working closely with our third-party manufacturer and the FDA on our resubmission in order to make cosibelimab available to patients living with cSCC.”

December 11

Deutsche Bank initiated coverage of Prothena with a Buy rating and $62 price target. With the selloff over the last few months, the analyst thinks the share price is justified by just birtamimab, cash, and projected milestone payments from collaborations. As such, Prothena’s risk/reward into the Phase 1b MAD data for PRX012 is “fundamentally skewed to the upside,” the analyst told investors in a research note.

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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.