Palvella Therapeutics (PVLA): what its 10-K says it depends on

Suppliers

Nemera Le Tréport SAS

depends onnamed as a risk

Nemera is a sole source supplier of these pumps, and we are required under a supply agreement to purchase from Nemera.Business (Item 1)

Partners and platforms

Ligand Pharmaceuticals, Inc.

tiered royalties ranging from 8.0% to 9.8%named as a risk

In addition, we are currently obligated to pay to Ligand tiered royalties ranging from 8.0% to 9.8% of any aggregate annual worldwide net product sales of any products based on QTORIN rapamycin.Business (Item 1)

Ligand Pharmaceuticals, Inc.

up to $5.0 millionnamed as a risk

Accordingly, we may be required to make payments in an aggregate amount of up to $5.0 million prior to the time at which we are able to generate revenue, if any, from sales of QTORIN rapamycin for any indication, if approved.Risk factors (Item 1A)

Yale University

depends onnamed as a risk

We exclusively license from Yale University one allowed U.S. Application directed to the topical administration of an effective amount of a HMG CoA reductase inhibitor, including pitavastatin, to treat porokeratosis.Business (Item 1)

Also named by QBTS

Other companies

RAPAMUNE

depends onnamed as a risk

To support our Section 505(b)(2) NDA, we plan to bridge QTORIN rapamycin and RAPAMUNE based on a cross-study comparison between pharmacokinetic data of QTORIN rapamycin from the Phase 3 trial and the prescribing information for RAPAMUNE; the FDA recommends that bridging to support an NDA for the treatment of microcystic LM be done in a relative bioavailability study comparing the pharmacokinetics of a topical product applied under maximal use conditions and the approved oral drug.Business (Item 1)

Competitors

Hyftor

competes withnamed as a risk

For example, upon approval in March 2022 of a Section 505(b)(2) NDA for the treatment of facial angiofibroma associated with tuberous sclerosis, Hyftor, a topical gel product containing sirolimus (also known as rapamycin), received three years of new product exclusivity.Risk factors (Item 1A)

Compounding pharmacies (FDCA Section 503A)

competes withnamed as a risk

In addition, some of the market demand for topical rapamycin or pitavastatin may be satisfied by state-licensed compounding pharmacies lawfully operating under Section 503A of the FDCA.Risk factors (Item 1A)

Kaken Pharmaceutical Co., Ltd.

competes withnamed as a risk

To the best of our knowledge, potential competitors with product candidates in development for serious, rare skin diseases and vascular malformations include Kaken Pharmaceutical Co., Ltd., Nobelpharma Co., Ltd., Novartis Pharmaceuticals, Protara Therapeutics, Inc., Relay Therapeutics, Inc., Vaderis Therapeutics AG, and Quoin Pharmaceuticals.Business (Item 1)

Nobelpharma Co., Ltd.

competes withnamed as a risk

To the best of our knowledge, potential competitors with product candidates in development for serious, rare skin diseases and vascular malformations include Kaken Pharmaceutical Co., Ltd., Nobelpharma Co., Ltd., Novartis Pharmaceuticals, Protara Therapeutics, Inc., Relay Therapeutics, Inc., Vaderis Therapeutics AG, and Quoin Pharmaceuticals.Business (Item 1)

Novartis Pharmaceuticals

competes withnamed as a risk

To the best of our knowledge, potential competitors with product candidates in development for serious, rare skin diseases and vascular malformations include Kaken Pharmaceutical Co., Ltd., Nobelpharma Co., Ltd., Novartis Pharmaceuticals, Protara Therapeutics, Inc., Relay Therapeutics, Inc., Vaderis Therapeutics AG, and Quoin Pharmaceuticals.Business (Item 1)

Protara Therapeutics, Inc.

competes withnamed as a risk

To the best of our knowledge, potential competitors with product candidates in development for serious, rare skin diseases and vascular malformations include Kaken Pharmaceutical Co., Ltd., Nobelpharma Co., Ltd., Novartis Pharmaceuticals, Protara Therapeutics, Inc., Relay Therapeutics, Inc., Vaderis Therapeutics AG, and Quoin Pharmaceuticals.Business (Item 1)

Relay Therapeutics, Inc.

competes withnamed as a risk

To the best of our knowledge, potential competitors with product candidates in development for serious, rare skin diseases and vascular malformations include Kaken Pharmaceutical Co., Ltd., Nobelpharma Co., Ltd., Novartis Pharmaceuticals, Protara Therapeutics, Inc., Relay Therapeutics, Inc., Vaderis Therapeutics AG, and Quoin Pharmaceuticals.Business (Item 1)

Vaderis Therapeutics AG

competes withnamed as a risk

To the best of our knowledge, potential competitors with product candidates in development for serious, rare skin diseases and vascular malformations include Kaken Pharmaceutical Co., Ltd., Nobelpharma Co., Ltd., Novartis Pharmaceuticals, Protara Therapeutics, Inc., Relay Therapeutics, Inc., Vaderis Therapeutics AG, and Quoin Pharmaceuticals.Business (Item 1)

Quoin Pharmaceuticals

competes withnamed as a risk

To the best of our knowledge, potential competitors with product candidates in development for serious, rare skin diseases and vascular malformations include Kaken Pharmaceutical Co., Ltd., Nobelpharma Co., Ltd., Novartis Pharmaceuticals, Protara Therapeutics, Inc., Relay Therapeutics, Inc., Vaderis Therapeutics AG, and Quoin Pharmaceuticals.Business (Item 1)

Places

Germany

approximately $219.4 million and $215.1 millionnamed as a risk

As of December 31, 2025, we had German corporate income tax and trade tax net operating loss carryforwards of approximately $219.4 million and $215.1 million, respectively.Risk factors (Item 1A)

Also named by APO, AXGN, BF/B, CPRT, DASH, EBAY, GD, GDDY and 12 more

United States

concentrated in · > 30,000 diagnosed patients in US

Based on >30,000 diagnosed patients in the U.S., we believe the estimated TAM opportunity on an annualized basis is greater than $1 billionBusiness (Item 1)

Also named by ABBV, ADP, AJG, AMGN, APA, AXGN, AXP, BALL and 139 more

India

depends onnamed as a risk

Our active pharmaceutical ingredient, rapamycin, is currently sourced from suppliers located in India.Risk factors (Item 1A)

Also named by A, AAPL, ACN, AJG, AKAM, AMZN, AOS, AVGO and 54 more

Commodities and inputs

Rapamycin (sirolimus)

depends on · three sourcesnamed as a risk

We obtain supplies of drug substance for our product candidates on a purchase order basis from three sources.Business (Item 1)

Regulators

U.S. Food and Drug Administration (FDA)

subject tonamed as a risk

However, the FDA may not agree microcystic LM is an appropriate setting for a baseline-controlled Phase 3 study and has commented that a placebo-controlled trial or additional trials assessing different clinical endpoints may be required to assess the efficacy of QTORIN rapamycin for the treatment of microcystic LM.Risk factors (Item 1A)

Also named by A, ABBV, ABT, AMGN, ARE, ARWR, ATR, AXGN and 112 more

National Institutes of Health (NIH)

named as a risk

Moreover, the Trump Administration has proposed action to freeze or reduce the budget of the National Institutes of Health (“NIH”) as related to its funding for medical research, which could decrease the ability of facilities that rely on NIH funding to enroll and conduct clinical trials or increase the costs to us of conducting clinical trials.Risk factors (Item 1A)

Also named by ARE, BDX, DOC, ILMN, VTR, WAT

Laws and programs

Orphan Drug Act

subject to · 7 years in the United States and 10 years in the European Unionnamed as a risk

We have received Orphan Drug Designation for QTORIN rapamycin for the treatment of microcystic LM from the FDA and European Commission.Risk factors (Item 1A)

Also named by ARWR, IONS, KRYS, NBIX, UTHR, VKTX

Internal Revenue Code Section 382

subject to · $129.4 millionnamed as a risk

We believe that as a result of the Merger, our ability to utilize NOLs acquired in this transaction is expected to be severely limited by Section 382 of the Code.Risk factors (Item 1A)

Also named by AIG, BKH, CHRD, EXE, IONQ, VRNS

FDA Orphan Products Clinical Trials Grant

up to $2.6 millionnamed as a risk

Additionally, it is unclear whether the up to $2.6 million in grant funding we were awarded from the Orphan Products Grants Program to support our SELVA program will be adversely impacted or whether grant funding could be disrupted or terminated.Risk factors (Item 1A)

Medicare

earns fromnamed as a risk

Any reduction in reimbursement from Medicare, Medicaid, or other government programs may result in a similar reduction in payments from private payers.Risk factors (Item 1A)

Also named by ABT, ARWR, AXGN, BAX, BDX, BMY, BSX, CAH and 48 more

Inflation Reduction Act of 2022

subject tonamed as a risk

Although a drug or biological product that has one or more orphan drug designations will be excluded from the IRA’s price negotiation requirements, we do not know if additional drug pricing reforms could modify this exemption in the future.Risk factors (Item 1A)

Also named by ABBV, APD, ARE, BKH, DD, DHR, ELV, ENPH and 30 more

One Big Beautiful Bill Act of 2025

subject tonamed as a risk

Additionally, the One Big Beautiful Bill Act of 2025 included reductions in Medicaid funding, work requirements, and reenrollment requirements that could have the effect of decreasing utilization of, and reimbursement for, our products, if approved.Risk factors (Item 1A)

Also named by COO, FSLR, MRVL, NXT, TT, WDC

Section 505(b)(2) NDA pathway

subject tonamed as a risk

If we are unable to obtain approval for our product candidates through the Section 505(b)(2) NDA process, we may be required to pursue the more expensive and time consuming Section 505(b)(1) approval process, which consists of full reports of investigations of safety and effectiveness conducted by or for the applicant.Risk factors (Item 1A)

FDA Breakthrough Therapy Designation

subject tonamed as a risk

We were granted Breakthrough Therapy Designation by the FDA for QTORIN rapamycin for the treatment of microcystic LM and may seek such designation for QTORIN rapamycin for other indications, and for certain other product candidates.Risk factors (Item 1A)

FDA Fast Track Designation

subject tonamed as a risk

We were granted Fast Track Designation by the FDA for QTORIN rapamycin for the treatment of microcystic LM, the treatment of cutaneous VMs and the treatment of angiokeratomas and may seek such designation for QTORIN rapamycin for other indications, and for certain other product candidates.Risk factors (Item 1A)